MBMH Issue 8 September 2025

My Blood My Health

Leukiemia

Myeloma

Chronic Lymphocyte Luekemia

Chronic Myleoid Luekemia

Myleoproliferative Neoplasms

Welcome to My Blood, My Health Digital Magazine

Welcome to this edition of My Blood My Health, I am honoured to welcome you to this special edition of My Blood My Health, dedicated to Blood Cancer Awareness Month. This issue is more than just a publication—it’s a platform to inform, inspire, and empower everyone impacted by blood cancers.

Each article has been carefully crafted to provide practical resources, share the latest research insights and treatment updates, and feature the perspectives of patients, caregivers, and healthcare professionals From strategies to improve quality of life to updates on innovative therapies, our goal is to make complex information accessible and meaningful, helping you make informed choices on your journey.

At Heal Canada, we believe that awareness leads to action. By sharing stories of resilience, raising critical questions, and amplifying the patient voice, we are building a community where knowledge is power and advocacy drives change As you read through this issue, I invite you to reflect, share, and connect with others in our growing global network. Together, we can shine a brighter light on blood cancers and continue the vital work of education, support, and advocacy

Cheryl Petruk, MBA B.Mgt. CEO & Founder of Heal Canada

Heal Canada's mission is to empower patientstoaccessbetterandequitable services. We're deeply grateful to our readersfortheircontinuedinterestand support. Your engagement drives us to produceinsightfulandvaluablecontent that encourages patient-centricity in healthcare.

Thank you for participating in our journey, and welcome to this enlighteningissue!

With great humility for reading out a digitalmagazine, Cheryl.

MyBloodMyHealth

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Email:admin@healcanadaorg

ExecutivePublisher&EditorinChief

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Disclaimer: The Patient Advocacy Digital Magazine provides general information and resources to promote patient empowerment and awareness. The content is not a substitute for professional medical advice or treatment. Always consult with qualified healthcare professionals for personalized guidance medical condition or situation.

The Power of Awareness: Why Early Detection Matters in Blood Cancers

by Dr. Kemi Okwuegbuna and Cheryl Petruk, MBA

acted on warning signs, provide expert commentary to deepen your understanding, and offer a clear symptom checklist readers can and should use to spark proactive conversations with healthcareproviders.

EarlyDetectionSavesLives:TheWhy

TheAdvantageofCatchingBloodCancersEarly

While blood cancers often progress differently from solid tumours, early detection still offers criticaladvantages:

Treatment flexibility and less toxicity: In early stages, many therapies can be less aggressive while still effective. Early-stage chronic leukemias or indolent lymphoma, for instance, may be managed with watchful waiting or gentle therapies, avoiding the heavier regimens needed for moreadvanceddisease.

Preserving organ function and reducing complications: In diseases like multiple myeloma, early therapycanpreventirreversiblebonedamage,kidneyfailure,oranemia

Accesstocurativeoptions:Somebloodcancers,suchasacuteleukemiasorcertainlymphomas, arepotentiallycurable particularlywhentreatmentbeginsbeforediseaseburdenbecomestoo heavy.

Improvedqualityoflife:Earlydiagnosismeansfewersymptomslikefatigue,infections,orsevere anemia,helpingpatientsremainactiveandfunctional.

Understandingthesestakesmakesawarenessnotjustbeneficial—itcanbetransformational

The Power of Awareness: Why Early Detection Matters in Blood Cancers

ExpertInsights:UnderstandingtheSignals

Todeepenunderstanding,clinicalexpertshavetoldus:

“Bloodcancersoftenbeginsubtly,”“Symptomslikefatigue,nightsweats,weightloss,orenlarged lymph nodes may develop gradually or be mistaken for aging, stress, or infection. But when thesepersist,ordon’tfitatypicalpattern,it’stimetocheck.”

Theimportanceofroutinebloodwork especiallycompletebloodcount(CBC) forearlyclues: Elevatedorlowwhitecellcounts

Anemia(lowredbloodcells/hemoglobin)

Lowplatelets

Presenceofimmatureorabnormalcells

“When such flags appear, even if symptoms seem mild, recommend swift follow-up with a hematologyconsult”

“Akeychallengeisthatearlysignsofbloodcancersoverlapwithmanybenignconditions “Fever, feverishsweats,orswollennodescanbeduetoviralillnesses Butwhatsetsbloodcancersapart ispersistenceorlackofresponsetousualtreatment oracombinationofsymptoms”

Certain red-flag combinations such as fatigue paired with weight loss, or new pain paired with bruising shouldpromptshadowinglabs,imaging,orbiopsy.

“Don’t wait for a dramatic illness. When subtle symptoms persist beyond a few weeks, get a completebloodcounttest evenifit’s‘justtocheck.’Itcouldtrulysavealife.”

WhyAwarenessStillFallsShort—andHowWeCanChangeThat Despiteclearbenefits,earlydetectionstrugglesagainstmultiplebarriers:

Misattribution of symptoms: Many confuse symptoms with aging, stress, depression, or minorinfections—andsodelaymedicalattention

Fear and denial: The thought of “Cancer?” is frightening Some delay seeking care out of anxietyordenial

Access issues: Not everyone has ready access to health care, regular screening, or routine labs barriersthatmaydelaydetection

Awareness gaps: Public recognition of blood cancer symptoms is limited compared to other cancers (like breast or colon). Few remember their blood counts or know to discuss hematologicflags.

The

Power of Awareness: Why Early Detection Matters in Blood Cancers

StrategiestoImproveAwareness

Education campaigns: Organizations, clinics, and communities can spotlight symptom checklists especiallyduringSeptember.

Community outreach: Mobile clinics, health fairs, informational webinars, and posters in communitycentersorpharmacies.

Trainingprimarycareproviders:Makingsurefamilydoctorsandnursepractitionersknowthe subtlesigns andusesimpleCBCtestsgenerously.

Empowering patients: Encouraging readers that asking “Could this be blood cancer?” is a powerfulphrase onethateverypatienthastherighttoask.

YourSymptomChecklist:WhentoTalktoaHealthcareProfessional Belowisahandychecklisttohelppeoplerecognizepotentialearlysignsofbloodcancers Ifyou or someone you know checks more than one box below consistently, consider asking for a medical evaluation. Note: This is not a replacement for medical advice. If you experience any symptoms,pleaseseekmedicaladvicefromalicensedmedicalpractitioner.

Persistent fatigue or weakness

Unusual or excessive bruising or bleeding (gums, nose, skin)

Unexplained bone or joint pain

Elevated blood counts (red cells, white cells, platelets) on lab work

Unexplained weight loss (>5% in weeks –months)

Persistent night sweats, chills

Itching after warm showers or with no rash (aquagenic pruritus)

New or worsening anemia

Frequent or severe infections

Painless lumps or swollen lymph nodes (neck, groin, armpit)

Feeling “full” quickly or achy under ribs

Abnormal protein levels or elevated calcium (from routine labs

Note: None of these symptoms alone confirms cancer—but when paired with lab abnormalities or persistence over weeks, they merit evaluation.

The Power of Awareness: Why Early Detection Matters in Blood Cancers

TheRippleEffect:BeyondDiagnosistoOutcomes

Earlydetectiondoesn’tjustmatterforanindividual’sprognosis—ithasbroaderimpacts:

Reducing treatment intensity: Starting care sooner often spares patients from highly toxic, prolongedtherapies.

Lowering healthcare costs: Early, controlled disease tends to require fewer interventions and hospitalizations.

Protecting families and systems: Timely treatment reduces complications, urgent admissions, andlong-termburdensonfamiliesandhealthcaresystems

Hope and empowerment: Early diagnosis often allows greater control over one’s schedule, energy,andlifeplans andinjectsemotionalandpsychologicalhope.

CalltoAction:WhatYouCanDoThisSeptember—andBeyond

Forindividuals

Familiarizeyourselfwiththesymptomchecklistabove Ifsomethingunusualpersistsmorethanafewweeks,askforbloodtests. Printorsavethechecklist,andsharewithfriends,family,orcaregivers.

Forcaregiversandlovedones

Encourage action—even gentle nudges (e.g. “Hey, maybe get some blood work just in case?”) can sparkcrucialearlyevaluation.

Forcommunitygroupsandworkplaces

Distributesymptomcards,hostawarenesstalks,orinvitehematologistsforQ&Asessions

Forhealthcareproviders

KeepthethresholdfororderingCBCslowwhensymptomspersist.

Empowerstafftoflagsuspiciousresultsswiftly.

ClosingReflections

Blood cancers may lurk quietly, often camouflaged as benign symptoms—but their early warning signs hold immense power As Sarah, David, and Maria remind us: listening to our bodies and pursuing answers can change trajectories. Through collaborative efforts encompassing education, storytelling, and accessible tools like our symptom checklist early detection becomesarealisticandattainablegoalformany.

This September and every month—let’s honour the power of awareness. Let's empower ourselves and our communities with knowledge, drive early conversations with healthcare professionals,andchampionpathstowardtimelydiagnosisandbetteroutcomes

Stayinformed.Stayvigilant.Knowwhattoask.Itmightjustsavealife

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Understanding the Causes of Blood Cancer by Dr. Kemi Okwuegbuna and Cheryl Petruk, MBA

Currentmedicalandacademictheories,withkeyliterature

Bloodcancers(leukemias,lymphomas,andmyelomas)arisewhenapopulationofblood-forming cellsacquiresadvantagesthatletitexpandabnormally,evadenormalcontrols,andoutcompete healthy cells. No single cause explains all cases; rather, converging factors genetic alterations, agingbiology,environmentalexposures,infections,immuneandmicroenvironmentalinfluences, and inherited predisposition cooperate over time (a “multi-hit,” evolutionary process). Below is acrisptourofwhatweknow(anddon’t),anchoredtohigh-qualityacademicsources.

1)Somaticgenetics:theimmediate“engine”ofdisease

Most blood cancers are driven by acquired (non-inherited) DNA changes in hematopoietic stem/progenitorcells.

Chromosomal translocations create fusion oncogenes that act like stuck accelerators. Classic examples include BCR-ABL1 in chronic myeloid leukemia and PML-RARA in acute promyelocytic leukemia (APL). These aren’t just markers they’re causal drivers and therapeutic targets (e.g., ATRA/arsenicinAPL).

Recurrent point mutations activate growth pathways or derail differentiation In the myeloproliferative neoplasms (MPNs), JAK2 V617F (2005), MPL W515 (2006), and CALR (2013) mutationsareprototypicinitiatinglesionsthatdysregulateJAK–STATsignalling

Hematologic malignancies often reflect cooperating classes of mutations some promote proliferation; others block maturation or alter epigenetic programs fitting long-standing “multistep” models of leukemogenesis. Modern genomic studies extend this to a Darwinian, somatic evolutionframework.(Seesectionsonclonalhematopoiesisandaging.)

Understanding the Causes of Blood Cancer

2)Agingbiology&clonalhematopoiesis:fertilegroundforcancer

With aging, hematopoietic stem cells (HSCs) accumulate “clocklike”mutations(e.g.,SBS1/SBS5signatures)andfunctionaldecline (“inflammaging,” DNA repair stress), creating a background where mutantclonescanexpand.

A central concept is clonal hematopoiesis of indeterminate potential (CHIP/CH) age-related, leukemia-associated mutations (DNMT3A, TET2, ASXL1, etc.) in blood cells without overt cancer. Large cohorts show CH confers a markedly elevated future risk of hematologic malignancy (eg, ~10- to 13-fold in some analyses), thoughmostindividualswithCHneverdevelopleukemia

Why does it matter? CH reframes causation: many blood cancers start years earlier as expanding “pre-leukemic” clones that later acquire cooperating hits under selective pressures (age, inflammation,cytotoxictherapy).

3)Environmentalcarcinogens:radiationandchemicals Someexposureshaverobustcausallinks:

Ionizing radiation (e.g., Hiroshima/Nagasaki Life Span Study) produces a strong, dose-related increase in leukemia risk one ofthefirstandclearestlateeffectsdetected.

Benzene is a Group 1 carcinogen (IARC) that causes myeloid malignancies; risk relates to cumulative exposure in occupationalandenvironmentalsettings

Formaldehyde is classified as carcinogenic to humans; evidenceforleukemiariskhasstrengthenedovertime,buthas been debated current summaries note sufficient evidence overall,withhistoricalqualifiersregardingleukemia.

Cigarettesmokingincreasestheriskofacutemyeloidleukemia (AML); authoritative reports (e.g., the 2014 U.S. Surgeon General) and public-health agencies list AML among smokingcausedcancers.

Understanding the Causes of Blood Cancer

4)Infectionsandimmunestimulation

Certain pathogens causally contribute to distinct blood cancers, often via chronic antigenic drive or direct oncogenic programs.

Helicobacter pylori infection can cause gastric MALT lymphoma;antibioticeradicationfrequentlyinduceslymphoma regression, a rare example where removing a microbial driver treatsacancer.

Epstein–Barr virus (EBV) is etiologically linked to several lymphomas (e.g., endemic Burkitt, a subset of Hodgkin lymphoma, post-transplant lymphoproliferative disease) and someepithelialcancers.

Hepatitis C virus (HCV) is associated with increased nonHodgkin lymphoma risk; in indolent B-cell lymphomas, directacting antivirals can trigger lymphoma responses as viremia clears implicatingthevirusindiseasemaintenance

Theoretical note. These links illustrate antigen-driven clonal selection and virus-mediated oncogenesis as credible causal pathwaysinhematologicmalignancy.

5) Therapy-related disease: when cures create risk

Prior chemotherapy/radiotherapy can initiate therapy-related myeloid neoplasms (t-MNs)—now formalized in contemporary classifications (WHO-HAEM5 and ICC) as myeloid neoplasms post-cytotoxic therapy (MN-pCT). Typical mutational patterns reflect alkylating agents, topoisomerase II inhibitors, and prior radiation.

Understanding the Causes of Blood Cancer

6) Bone-marrow “soil”: the niche and microenvironment

Beyond the “seed” (mutant HSC), the bone-marrow microenvironment (BMME) can foster or restrain malignancy. PubMed Haematologica talks about elegant mouse genetics showed that disrupting osteolineage progenitors (e.g., Dicer1 deletion) can initiate MDS-like disease and secondary AML, even though leukemic cells retain wild-type Dicer1 evidence that a defective niche can be causal.

Human and preclinical studies also show that the BMME supports leukemic cell survival, immune evasion, and therapy resistance, making it an emerging therapeutic target.

7)

Inherited predisposition: when risk runs in families

Puttingittogether:aunifyingcausalpicture

A minority of blood cancers arise on a germline foundation. Recognized syndromes include RUNX1, GATA2, ETV6, ANKRD26 and, notably in adults, DDX41 germline variants (often with a second somatic DDX41 hit), typically presenting as MDS/AML in mid-to-late adulthood. Identification affects surveillance, donor selection, and counseling.

Most blood cancers reflect stepwise somatic evolution in long-lived HSCs. Aging seeds the field with CH clones; exposures (radiation/chemicals), chronic immune stimuli or infections, therapy, andnichedysfunctionapplyselectivepressures;germlinevariantssetbaselinerisk.Thedisease youseeintheclinicistheend-productofthisyears-long“competition”

Understanding the Causes of Blood Cancer

What remains uncertain?

Why do some CH clones progress while most remain indolent? (Work continues on clone size, specific genes, and inflammatory stressors.)

Which microenvironmental alterations are initiating versus permissive in humans and how best to therapeutically re-educate the niche.

Practical takeaways

Most blood cancers arise from acquired mutations interacting with ageing biology; a minority are rooted in germline predisposition.

Established external causes include ionizing radiation, benzene, cigarette smoking, and specific infections (e.g., H. pylori, EBV, HCV) in defined entities.

The bone-marrow niche and inflammation are active players—not just bystanders offering new preventive and therapeutic targets.

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Emerging Treatments for Blood Cancers in Canada (2025) by Dr. Kemi Okwuegbuna and

Cheryl Petruk, MBA

Blood-cancer care is moving fast. In just the last few years, Canada has seen a surge of advanced immunotherapies—especially CAR T-cell therapies and bispecific antibodies—alongside nextgeneration targeted medicines that home in on the mutations or pathways that drive disease. Some of these options are already approved and funded; others are newly authorized and working their way through pan-Canadian review and provincial adoption. For patients and families, the result is more choice, more personalization, and (in some settings) treatment earlier in the journey, not just after many lines of therapy. Health Canada and national health technology bodies have also sharpened their focus on safety and real-world value, which directly shapes what’s offered where you live.

The big immunotherapy shift

CAR T-cell therapy extends to earlier

lines in myeloma

A headline development for Canadians living with multiple myeloma is ciltacabtagene autoleucel (CARVYKTI) being authorized by Health Canada for use as early as second line—for adults who’ve had 1–3 prior therapies and are lenalidomide-refractory. That means some patients can access a one-time, highly active cellular therapy sooner than before, potentially changing the trajectory of their disease and time on continuous therapy. Funding decisions remain provincial, but several jurisdictions have begun working through implementation.

CAR-T safety: honest talk about rare risks

At the same time, Canadian regulators have been transparent about a rare risk of secondary Tcell malignancies after CAR-T. Health Canada completed a safety review and is aligning product information across all CAR-T products; clinicians are advised to monitor long-term. The U.S. FDA added a class-wide boxed warning in 2024, and Canada’s InfoWatch outlined label updates in December 2024. The message isn’t “don’t use CAR-T”—it’s “use CAR-T wisely, counsel clearly, and monitor for years” Benefits still outweigh risks in approved uses

Made-in-Canada manufacturing and access research

Canada continues to build domestic CAR-T capacity and clinical networks (for example, Canadian-led initiatives to manufacture CAR-T in Canada and study access/feasibility). Local capacity can shorten vein-to-vein times, reduce logistical risk, and potentially expand access outside major hubs over time

Emerging Treatments for Blood Cancers in Canada (2025)

Bispecific antibodies: “off-the-shelf” T-cell redirection

While CAR-T re-engineers your T cells, bispecific antibodies act like pre-built matchmakers that bring your T cells to the cancer Canada has seen progress in lymphomas and myeloma with CD20×CD3 and BCMA×CD3 agents (availability and funding vary by province and indication, and some products are still in regulatory or HTA pipelines). These off-the-shelf options can be critical when speed matters or CAR-T isn’t feasible due to comorbidities, geography, or manufacturing slots. As with CAR-T, teams watch closely for cytokine release syndrome (CRS) and neurologic events—but centers have become skilled at prevention and management. (Note: availability and lines of therapy differ by agent and province; ask your team which bispecifics are active where you receive care.)

Disease-by-disease highlights (Canada)

Multiple Myeloma

Earlier-line CAR-T (CARVYKTI): As noted, Health Canada authorized cilta-cel in the second-line setting for lenalidomide-refractory patients with 1–3 prior lines This can offer deep, durable responses after only one infusion. Provincial funding pathways are catching up, so verify local coverage and referral logistics. Innovative Medicine Globalcda-amc.ca

Where bispecifics fit: BCMA- or GPRC5D-directed bispecifics offer “off-the-shelf” immunotherapy. Their place relative to earlier-line CAR-T is an active discussion in Canadian myeloma tumour boards; expect evolving provincial criteria and sequencing guidance What to ask your team

1) Am I potentially eligible for CAR-T earlier than I thought?

2) If CAR-T isn’t right now, is a bispecific appropriate—and how does monitoring work?

Emerging Treatments for Blood Cancers in Canada (2025)

Lymphomas(DLBCLandHodgkinlymphoma)

Second-line CAR-T in DLBCL: Lisocabtagene maraleucel (BREYANZI) has been moving earlier in the DLBCL journey. In January 2025, BMS announced Canadian approval for second-line R/R DLBCL, an “earlier access” milestone that may reduce reliance on high-dose chemo plus transplant in some patients. Provincial listings follow CADTH recommendations and pCPA negotiations askabouttiminginyourprovince.

First-line Hodgkin lymphoma pivot (nivolumab + AVD): A pivotal trial (SWOG S1826) published in NEJM (Oct 2024) showed nivolumab + AVD outperformed BV-AVD with fewer adverse effects, prompting programs to consider N-AVD a new standard in advanced cHL. Ontario Health has alreadycreatedaregimenmonographreferencingtheNEJMdata;broaderCanadianadoptionis inmotionasfundingandguidelinesalign.

Whattoaskyourteam

If I relapse early after R-CHOP, is second-line CAR-T an option instead of transplant? 2) For advancedHodgkinlymphoma,isnivolumab+AVDavailableatmycenter?

AcuteleukemiasandMDS

Menininhibitiononthehorizon:IntheU.S.,revumenib(first-in-classmenininhibitor)gainedFDA approval in Nov 2024 for R/R acute leukemia with KMT2A translocation including pediatric patients. Canadian clinicians are watching closely as Health Canada’s SUR lists evolve; some patients may access menin inhibitors via trials. If your disease harbours a KMT2A translocation (orNPM1mutation),askaboutlocaltrialsorspecialaccessmechanisms.

New therapy for lower-risk MDS (US first): The FDA approved imetelstat (RYTELO) in June 2024 for transfusion-dependent anemia in adults with low- to intermediate-1 risk MDS In Canada, imetelstat remains under evaluation; monitor CADTH and provincial updates If you’re ESArefractoryandtransfusion-dependent,askwhetheratrialorcompassionateaccessispossible

Whattoaskyourteam

1)Hasmyleukemia/MDSbeenprofiledforKMT2AorNPM1?

2)ArethereCanadiantrialsformenininhibitorsortelomeraseinhibition?

Emerging Treatments for Blood Cancers in Canada (2025)

Myeloproliferative neoplasms (MPNs)

Momelotinib for myelofibrosis with anemia: OJJAARA (momelotinib) received a Health Canada Notice of Compliance in November 2024, and in February 2025, Canada’s Drug Agency recommended reimbursement with conditions for MF patients with moderate-to-severe anemia This matters because momelotinib can improve symptoms and anemia, addressing a stubborn need in MF. Ask your hematologist about provincial funding status and referral pathways.

Ropeginterferon (PV): Canada permitted exceptional importation of U.S.-authorized ropeginterferon alfa-2b (BESREMi) in March 2025 to mitigate a peginterferon shortage; that step supports continuity of care while full Canadian regulatory pathways proceed If you’re on interferon or considering it, ask whether ropeginterferon is an option under exceptional importation in your province.

What to ask your team

1) For MF with anemia, am I a candidate for momelotinib now that a Canadian NOC exists?

2) For PV, would interferon (including ropeg under exceptional importation) fit my goals?

How Canadian access decisions get made (and what you can do)

After Health Canada authorizes a therapy, CADTH/pCODR assesses clinical value and costeffectiveness; the pan-Canadian Pharmaceutical Alliance (pCPA) then negotiates with manufacturers; finally, each province decides how and when to fund. This multi-step model explains why a drug can be “approved” nationally but available on different timelines across provinces Patients and clinicians can track CADTH recommendations and ask cancer agencies for program timelines, bridging options, or clinical trials nearby.

Emerging

Treatments for Blood Cancers in Canada (2025)

Safety,survivorship,andlivingwell

Modernimmunotherapiesarepowerful.Theyalsobringspecificmonitoring:

CAR-T: CRS/ICANS in the short term; very rare secondary T-cell malignancies long term so lifetimefollow-upiskey.

Bispecifics:CRSisgenerallylower-gradeandoftenmanageableasteamshavebecomeexpertat premedication,earlydetection,andsupportivecare.

Targeted therapy: Side-effects vary by pathway (e.g., cytopenias, infections, or cardiac signals). Canadiancentreshavestandardizedpathwaystoprevent,detect,andtreatthese.

Fivesmartquestionstobringtoyournextvisit

Eligibility: Given my exact diagnosis, genetics, and prior treatments, which emerging options applytomenowvs.later?

Timing: If a therapy is authorized but not yet funded, are there trials, compassionate access, or out-of-provincereferraloptions?

Safety: How will you monitor and manage CRS, neurologic effects, and rare long-term risks (e.g., secondaryT-cellmalignanciesafterCAR-T)?

Logistics: For CAR-T, what’s the realistic vein-to-vein time and where will I stay during monitoring?Forbispecifics,what’stheinfusioncadence?

Life goals: How will this plan affect work, caregiving, travel, fertility, or finances? Is there a navigatororsocialworkerwhocanhelp?

Bottom line for Canadians

Canada is entering a period where earlier-line cellular therapy, rapid-acting bispecifics, and mutation-targeted medicines are not future ideas—they’re here, expanding, and being integrated into provincial programs. The exact timing and sequencing will differ by province, but the direction is clear: more personalized treatment choices, more emphasis on outcomes that matter to patients, and transparent discussion of safety and long-term monitoring. Bring questions. Your care team is ready to tailor these new tools to your situation.

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Types of Blood Cancers

These article is for education through My Blood My Health and is not a substitute for medical advice. If you have concerns or symptoms, please consult a qualified healthcare professional.

Leukiemia

What is Leukemia?

Leukemia is a blood cancer that begins in the bone marrow—the factory for new blood cells and leads to the production of abnormal blood cells that crowd out healthy ones. The word itself comes from the Greek for “white blood,” reflecting how many leukemias start in white blood cells Clinicians classify leukemia by (1) the kind of cell that becomes cancerous—myeloid or lymphoid—and (2) how quickly it develops acute (fast-growing) or chronic (slower-growing). Those two axes create the four major types you’ll hear about: AML (acute myeloid leukemia), ALL (acute lymphoblastic leukemia), CLL (chronic lymphocytic leukemia), and CML (chronic myeloid leukemia).

How leukemia happens (in brief)

Healthy marrow makes red cells (carry oxygen), platelets (clotting), and white cells (immune defence). In leukemia, acquired genetic changes (mutations or chromosomal alterations) push a developing blood cell to multiply abnormally and evade normal maturation and death signals. One well-known example is the Philadelphia chromosome, a swap of DNA between chromosomes 9 and 22 that fuses the BCR and ABL1 genes This driver change produces an overactive enzyme (a tyrosine kinase) and is the hallmark of CML (and present in a subset of ALL).

Over the past few years, expert groups have refined how these diseases are categorized using genetics and molecular features, because biology guides both prognosis and treatment. The World Health Organization’s 5th edition classification (2022–2024) and companion updates in Leukemia reflect this shift to a genetics-first framework.

How common is it?

Leukemia accounts for a meaningful share of blood cancers in both adults and children. In Canada, the Canadian Cancer Society provides up-to-date statistics and trends (incidence and mortality) for leukemia overall and by subtype, which helps patients and policymakers understand the burden across provinces and over time. In the United States, Surveillance, Epidemiology, and End Results (SEER) data show an incidence of roughly 14 4 per 100,000 and a death rate of 5 8 per 100,000 (age-adjusted; most recent periods). Survival varies widely by subtype, age, and biology.

Signsandsymptoms

Becauseleukemiadisruptsnormalbloodcellproduction,manysymptomsaretiedtolowcounts: Anemia(lowredcells):fatigue,shortnessofbreath,paleness

Neutropenia(lowinfection-fightingwhitecells):recurrentorsevereinfections,fevers Thrombocytopenia(lowplatelets):easybruising,nosebleeds,bleedinggums

Some people notice night sweats, weight loss, swollen lymph nodes, a feeling of fullness (enlargedspleen),orbone/jointpain.Symptomscanbenon-specificandmayresemblecommon infections,sopersistentorunusualpatternswarrantmedicalattention.

Howleukemiaisdiagnosed

There’snopopulationscreeningtestforleukemia.Manydiagnosesbeginwithacompleteblood count(CBC)andbloodsmearwhensymptomsorroutinetestsshowsomethingoff.Ifleukemiais suspected, clinicians confirm and classify it using a bone marrow aspiration and biopsy plus specializedtests:

Flowcytometry/immunophenotyping(identifiescellsurfacemarkers)

Cytogenetics(looksforchromosomalchangeslikethePhiladelphiachromosome)

Moleculartesting(detectsmutationsthatinformriskandtreatment)

These studies together determine the exact subtype and risk group, which is essential for treatmentplanning.

Riskfactors:whatweknow

Most leukemias are not inherited. Documented risk factors include ionizing radiation, certain chemicals(notablybenzene),previouschemotherapyorradiationforanothercancer,somerare inherited syndromes (e.g., Down syndrome), and specific prior marrow disorders. Having a risk factordoesnotmeanleukemiaisinevitable;itmeansriskishigherthanaverage.

The major types at a glance

Acute lymphoblastic leukemia (ALL): Rapid-onset lymphoid leukemia seen in children and in older adults. Treatments often combine multi-agent chemotherapy with targeted drugs; CAR Tcell therapy is an option for some relapsed/refractory B-ALL (children/young adults with tisagenlecleucel; adults with brexucabtagene autoleucel).

Acute myeloid leukemia (AML): An aggressive myeloid leukemia of older adults, though it can occur at any age. Care is guided by genetics (e g., FLT3, IDH1/2, NPM1), overall fitness, and goals Diagnostic workup and treatment algorithms are detailed in NCCN patient guidelines and NCI PDQ resources.

Chronic lymphocytic leukemia (CLL): Often found incidentally on routine bloodwork and may be monitored (“watch and wait”) until treatment is needed. Modern targeted therapies (e.g., BTK inhibitors or BCL-2 inhibitor–based combinations) have transformed outcomes; staging uses Rai/Binet systems

Chronic myeloid leukemia (CML): Driven by BCR-ABL1. Daily tyrosine kinase inhibitors (TKIs) like imatinib and newer agents can induce deep remissions; some patients achieve treatment-free remission under strict monitoring.

How leukemia is treated

Treatment plans reflect the leukemia type, genetics, patient age/fitness, and preferences. Broad tools include:

Chemotherapy and combinations (especially for acute leukemias)

Targeted therapy (e g., TKIs for CML; targeted agents in AML and CLL)

Immunotherapies (monoclonal antibodies; CAR T-cell therapy for certain B-ALL)

Stem cell transplantation (allogeneic transplant) for selected higher-risk or relapsed cases

Shared decision-making is key—balancing the chance of long-term disease control with sideeffects and quality of life. National resources like NCI’s PDQ and NCCN’s patient guidelines explain options, potential benefits, and risks in plain language

Living well with leukemia

Beyond disease control, supportive care matters: preventing and treating infections, managing anemia or bleeding, addressing fatigue and mood, and staying current with vaccinations (as advised by your care team). Many adults live for years with chronic forms like CLL or CML, and even in acute leukemias, outcomes continue to improve as precision-guided therapies and cellular therapies evolve. For practical information and peer support, organizations such as the Leukemia & Lymphoma Society offer education and patient services.

When to seek medical advice

See a clinician if you have persistent fatigue, frequent infections, unexplained bruising/bleeding, night sweats, or enlarged lymph nodes or spleen—especially if symptoms last more than a couple of weeks. Prompt evaluation allows earlier diagnosis and, when needed, faster treatment. Remember, these symptoms are not specific to leukemia; many benign conditions can look similar, which is why proper testing is important.

These article is for education through My Blood My Health and is not a substitute for medical advice If you have concerns or symptoms, please consult a qualified healthcare professional.

Myeloma

What Is Myeloma—and Is It a Blood Cancer?

Multiple myeloma (MM)—often simply referred to as myeloma—is indeed a type of blood cancer, one that specifically affects plasma cells, a mature form of white blood cell responsible for producing antibodies. In myeloma, genetically abnormal plasma cells multiply uncontrollably within the bone marrow, producing large amounts of a single type of antibody (a “monoclonal protein”), crowding out healthy blood cells, and causing damage to bones, kidneys, and other organs

It is classified as a hematological lymphoid malignancy of tumour plasma cells. It is unquestionably within the spectrum of blood cancers, along with leukemia and lymphoma.

How Does It Arise? Biology and Classification

Multiple myeloma develops through a multistep biological process driven by genetic changes and interactions with the bone marrow microenvironment. It's genetically heterogeneous and remains incurable mainly due to relapse or treatment resistance.

Diagnosis uses the International Myeloma Working Group (IMWG) criteria, which require:

≥10% of plasma cells in bone marrow being clonal.

At least one myeloma-defining event, which may include end-organ damage captured in the CRAB acronym: Calcium elevation, Renal dysfunction, Anemia, or Bone lesions, or biomarkers indicating high likelihood of progression.

Myeloma is the second most common blood cancer after lymphoma globally, accounting for approximately 10% of hematologic malignancie.

These article is for education through My Blood My Health and is not a substitute for medical advice If you have concerns or symptoms, please consult a qualified healthcare professional.

Myeloma

Epidemiology&CanadianPerspective

Canada:Roughly4,100Canadiansareforecasttobediagnosedwithmultiplemyelomaannually, andaround1,750diefromit.

Prevalencedatashowthatabout1in2,505Canadiansarelivingwithmyelomaasof2022.The annual incidence equates to about 5.2 new cases per 100,000 people, representing approximately1.3%ofallcancersand10%ofbloodcancersinCanada.

AlargepopulationstudyfoundanincidenceinCanadaaveraging54.3casespermillionpeople peryear(≈5.43per100,000),withasteadyannualincreaseofnearly0.96casespermillionper year.Themeanageatdiagnosiswas70years,withabout54%ofcasesinmen.

Worldwide, in 2020, nearly 175,000 people were diagnosed, and 117,000 died from multiple myeloma IntheUS,projectionsestimatearound35,000newcasesand12,000deathsin2023

Approximately 170,405 Americans were living with the disease in 2020 Survival has improved: about60%surviveatleast5years,andaround34%live10yearsormoreafterdiagnosis

Thediseasetypicallyaffectsindividualsaroundage60or69,ismorecommoninmen,andit's rareunderage40

Signs&Symptoms

Myeloma’ssymptomscommonlyreflectbonemarrowinfiltrationandend-organeffects:

Bone pain, particularly in the spine and ribs; osteolytic lesions lead to fractures and are part of theCRABcriteria

Anemia,causingfatigueandpallor

Hypercalcemia(elevatedcalcium)canleadtoconfusion,constipation,anddehydration

Renalimpairmentfromlightchainproteinscloggingthekidneys

Recurrentinfectionsduetoineffectiveantibodyproduction.

Importantly, the disease often starts subtly and without major symptoms, indicated by monoclonal gammopathy of undetermined significance (MGUS) or smouldering myeloma, precursorstatesthatmayprogressovertime.

Diagnosis&Staging

Diagnosis combines blood work (monoclonal proteins), imaging (X-ray, MRI, PET), and bone marrowbiopsy(clonalplasmacells,≥10%).TheIMWGcriteriarefinethresholdstodetectdisease earlierandimproveoutcomes

Staging uses the International Staging System (ISS), later revised (R ISS) to include cytogenetic markersandLDHlevels,offeringrobustprognosticstratification

These article is for education through My Blood My Health and is not a substitute for medical advice If you have concerns or symptoms, please consult a qualified healthcare professional.

Myeloma

Treatment Landscape

Multiple myeloma is considered treatable but not curable in most cases, though remission is often achievable. Therapeutic approaches include: Chemotherapy and steroids.

Targeted therapies: proteasome inhibitors (e.g., bortezomib), immunomodulatory drugs (lenalidomide, thalidomide), and monoclonal antibodies (e.g., elotuzumab)

Stem cell transplantation, especially in eligible younger/fit patients.

CAR T-cell therapies targeting BCMA, such as idecabtagene vicleucel (Abecma), a breakthrough gene therapy now FDA-approved for relapsed/refractory myeloma

Supportive treatment with bisphosphonates to prevent bone fractures and pain.

Palliative care early in advanced stages improves quality of life through symptom management

Ongoing research includes AI-based monitoring of minimal residual disease (MRD) for prognostication and treatment decisions.

Has a significant presence in Canada, with thousands diagnosed annually and a continual rise in prevalence.

Benefitsfromstrongadvocacyeffortsandongoingclinicalresearch helpingpatientslivelonger, healthierlives

Chronic Lymphocyte Luekemia

WhatItIsandHowIt’sTreated

Chronic lymphocytic leukemia (CLL) is a slow-growing cancer of B lymphocytes (a type of white blood cell). In CLL, abnormal B cells build up in the blood, bone marrow, lymph nodes, and spleen. Many people are diagnosed after a routine blood test shows a high lymphocyte count; others notice swollen lymph nodes, fatigue, night sweats, unintentional weight loss, or frequent infections.CLLusuallyprogressesgradually,andtodaytherearemanyeffectivetreatmentsthat cancontrolthediseaseforyears.

HowCLLisDiagnosedandRisk-Stratified

Diagnosistypicallybeginswithacompletebloodcountshowinglymphocytosisandconfirmation by flow cytometry that the cells are clonal B lymphocytes (often CD5+, CD19+, CD23+) Additional tests help guide therapy, including genetic studies of the leukemia cells Two of the most important markers are TP53 disruption (17p deletion and/or TP53 mutation) and IGHV mutation status: TP53 abnormalities signal resistance to chemotherapy, while mutated vs. unmutated IGHV helps predict disease behaviour and response to certain treatments. These factors along with clinical stage and other labs are combined in prognostic tools like the CLLIPItoestimateriskandtailorcare.

“WatchandWait”(ActiveSurveillance)

Becauseearlytreatmenthasnotbeenshowntoimproveoverallsurvivalinasymptomatic,earlystage CLL, the standard approach for many newly diagnosed people is active surveillance (“watch and wait”). This includes regular checkups and labs to monitor for progression. Treatmentgenerallystartsonlywhenspecificcriteriaaremet:significantsymptoms(e.g.,fevers, night sweats, weight loss), bulky or progressive lymph nodes/spleen, rapidly rising lymphocyte counts, autoimmune complications not controlled by steroids, or declining red blood cells/plateletsfrommarrowfailure.

Today’sMainTreatmentOptions

ModernCLL carehasshifteddecisively from chemotherapy totargetedtherapiesandantibodybasedcombinations Themostwidelyusedcategoriesare:

Chronic Lymphocyte Luekemia

BTKinhibitors(BTKi)

BTK inhibitors block Bruton’s tyrosine kinase, a key signal that CLL cells use to survive. They’re pillstakendailyandcanbeusedupfrontorafterrelapse.

Acalabrutinib and zanubrutinib are next-generation (covalent) BTK inhibitors often preferred over ibrutinib due to similar efficacy with fewer certain side effects in many patients. They are commonlyusedaloneorwithananti-CD20antibody.

Pirtobrutinib is a non-covalent BTK inhibitor designed to work even when the cancer has developed resistance to covalent BTKis. In the U.S., it received accelerated approval (Dec 2023) for adults with CLL/SLL previously treated with both a BTK inhibitor and a BCL-2 inhibitor (see below),andongoingstudiescontinuetodefineitsroleearlierintherapy.

Common side effects of BTK inhibitors include bruising/bleeding, high blood pressure (notably with ibrutinib), heart rhythm issues (atrial fibrillation), headaches (more with acalabrutinib), and infections. Your care team will assess cardiovascular risk and drug interactions when choosing amongBTKis

BCL-2inhibitor–based,time-limitedtherapy

Venetoclax inhibits BCL-2, a protein that helps CLL cells avoid cell death. It can be paired with obinutuzumab (frontline) or rituximab (relapsed). A key benefit is fixed-duration treatment often 12 months in the frontline venetoclax-obinutuzumab regimen allowing a defined course with deep responses, including undetectable measurable residual disease (uMRD) for many patients.Carefuldoseramp-upmitigatestumorlysissyndromerisk.

Researchers are actively studying MRD-guided treatment lengths stopping earlier when uMRD isachieved butoutsidetrialsmostguidelinesstillrecommendfollowingthestandard,validated durations.

Anti-CD20monoclonalantibodies

Obinutuzumab and rituximab target CD20 on B cells, aiding immune clearance of CLL cells Today, they are typically used in combination with BTK inhibitors or venetoclax to deepen responses rather than as stand-alone therapies Infusion reactions and infections are the main considerations

Chronic Lymphocyte Luekemia

Chemotherapy (chemoimmunotherapy)

Regimens like FCR (fludarabine, cyclophosphamide, rituximab) or BR (bendamustine-rituximab) used to be standard but are now less common, especially if TP53 is disrupted (where chemo performs poorly). In highly selected younger patients with mutated IGHV, FCR can still induce very long remissions but comes with higher short- and long-term toxicity. Most patients today start with targeted therapies

Cellulartherapyandtransplant(selectsituations)

Allogeneicstemcelltransplantationcanofferlong-termdisease control for a small subset of high-risk, multiply relapsed patients who respond to therapy and are transplant-eligible, balancing potential benefits with significant risks. CAR T-cell therapy has transformed other blood cancers and is under study in CLL; it is available in trials and select settings with encouragingresults,butitisnotyetstandardfrontlinecare.

ChoosingaFirstTreatment

There isn’t a single “best” regimen for everyone Decisions consider:

Genetics: TP53/17p deletion → favor BTKi or venetoclax-based regimensoverchemo

Preference for a daily pill (often indefinite) vs finite therapy: BTKi is typically continuous; venetoclax-obinutuzumab is timelimited.

Comorbidities/risks: Cardiovascular history (BTKi considerations), kidney function (TLS risk with venetoclax), drug interactions,andinfectionrisk.

Age/fitness and logistics: Clinic visits for infusions, monitoring needs,andpersonalpriorities.

Major guidelines (e.g., ESMO) and national resources (e.g., NCI PDQ) list BTKi monotherapy (acalabrutinib or zanubrutinib) and venetoclax + obinutuzumab (fixed duration) among the leading frontline choices for many patients without TP53 disruption, withadjustmentsinspecialpopulations.

Chronic Lymphocyte Luekemia

TreatmentatRelapse

If CLL returns, doctors often switch classes: someone who had venetoclax first might receive a BTKi next, and vice versa After exposure to both classes, pirtobrutinib is an option in the US under accelerated approval, and clinical trials exploring combinations and sequencing are rapidly evolving Supportive care (vaccinations, infection prevention, management of autoimmunecomplications)remainsessentialateverystage

MeasuringSuccessandLong-TermOutlook

Response is assessed by symptoms, blood counts, lymph node size (exam or imaging), and sometimes measurable residual disease (MRD) testing in blood or marrow. Achieving uMRD is associated with longer remissions, especially after fixed-duration therapies, but routine MRDdrivendecisionsoutsidetrialsarestillbeingdefined.Overall,thankstotargetedtherapies,many people with CLL now experience years to decades of good quality of life with sequential treatmentsasneeded.

LivingWellWithCLL

Regularvaccinations(followingyouroncologyteam’sguidance),promptevaluationofinfections, skin cancer screening, and heart-healthy habits are practical steps that complement treatment. Emotional health matters too “watch and wait” can be stressful, and connecting with patient organizations,supportgroups,orcounsellingcanhelp.

KeyTakeaways

CLL is a slow-growing B-cell leukemia; many patients start with active surveillance rather than immediatetreatment.Cancer.gov

When treatment is needed, BTK inhibitors (acalabrutinib, zanubrutinib, and others) and venetoclax-basedcombinationsarethemainstays,oftenpreferredoverchemotherapy. Genetics (especially TP53 status) and IGHV mutation guide choices; TP53-aberrant CLL should avoidchemotherapy.

After both BTKi and BCL-2 therapy, pirtobrutinib is an FDA-approved option in the US under acceleratedapproval(Dec2023)

MRD testing is promising, especially with time-limited venetoclax regimens, but routine MRDguidedstoppingoutsidetrialsisstillevolving.

Chronic Myleoid Luekemia

Chronic Myeloid Leukemia (CML) is a type of cancer that originates in the bone marrow, affecting the blood-forming cells responsible for producing white blood cells. Unlike acute leukemias, which progress rapidly, CML develops more slowly and is often detected incidentally through routine blood work Over the past two decades, advances in targeted therapies have transformed CML from a once life-threatening disease into a largely manageable chronic condition for many patients. This article explores the biology of CML, its stages, diagnosis, treatment strategies, and future outlook.

What is Chronic Myeloid Leukemia?

CML is a myeloproliferative neoplasm (MPN), a category of blood cancers characterized by abnormal growth of bone marrow stem cells. The hallmark of CML is the presence of the Philadelphia chromosome, a genetic abnormality caused by a translocation between chromosomes 9 and 22. This translocation creates a BCR-ABL1 fusion gene, which encodes an abnormal tyrosine kinase enzyme. The enzyme drives uncontrolled cell division and survival of myeloid cells, leading to elevated white blood cell counts in the blood and bone marrow CML accounts for about 15% of adult leukemias and has an annual incidence rate of 1–2 cases per 100,000 people worldwide (Hochhaus et al., 2020). It is more common in adults, with a median age of diagnosis around 60 years, though it can occur at any age.

Phases of CML

CML typically progresses through three distinct clinical phases: Chronic Phase: The most common stage at diagnosis (85–90% of patients).

Patients may be asymptomatic or present with fatigue, night sweats, weight loss, or splenomegaly (enlarged spleen). Blood counts reveal elevated white blood cells and platelets. Accelerated Phase: The disease becomes less stable, with rising blast counts (immature white blood cells) and worsening symptoms. Resistance to therapy may begin to emerge. Prognosis worsens without effective treatment.

Blast Phase (Blast Crisis): CML transforms into an acute leukemia, resembling acute myeloid leukemia (AML) or acute lymphoblastic leukemia (ALL). Blast cells exceed 20% of blood or marrow cells. This phase is life-threatening and associated with poor outcomes. These article is for education through My Blood My Health and is not a substitute for medical advice If you have concerns or symptoms, please consult a qualified healthcare professional.

Chronic Myleoid Luekemia

Symptoms and Diagnosis

Many patients with CML are diagnosed incidentally through routine blood tests showing high white blood cell counts. When symptoms do occur, they may include:

Fatigue

Abdominal fullness or discomfort (due to splenomegaly)

Night sweats and fevers

Unintentional weight loss

Bone or joint pain

Diagnosis is confirmed through:

Complete blood count (CBC): Elevated white blood cells with a left shift (immature cells).

Bone marrow biopsy: Increased myeloid cells and presence of Philadelphia chromosome

Cytogenetic and molecular testing: Detection of the BCR-ABL1 fusion gene via fluorescence in situ hybridization (FISH), polymerase chain reaction (PCR), or karyotyping.

Molecular monitoring of BCR-ABL1 levels by PCR is critical throughout treatment to assess response and guide therapy.

Treatment of CML

Tyrosine Kinase Inhibitors (TKIs)

The discovery of imatinib (Gleevec) in 2001 revolutionized CML treatment. TKIs specifically inhibit the BCR-ABL1 protein, halting the uncontrolled proliferation of leukemic cells. Today, several TKIs are available:

Imatinib (first-generation)

Dasatinib, nilotinib, bosutinib (second-generation)

Ponatinib (third-generation, effective against resistant mutations such as T315I)

These oral drugs have transformed CML into a chronic disease with near-normal life expectancy for many patients. Therapy response is measured through hematologic, cytogenetic, and molecular milestones, such as achieving a major molecular response (MMR) or deep molecular response (DMR).

Allogeneic Stem Cell Transplantation

Before TKIs, bone marrow or stem cell transplantation was the only curative option. Today, transplantation is reserved for patients with TKI resistance, intolerance, or advanced-phase disease.

Interferon-alpha and Chemotherapy

Historically used before TKIs, interferon-alpha can still play a role in certain cases, such as pregnancy, where TKIs are contraindicated. Hydroxyurea may be used to control high white blood cell counts temporarily.

Chronic Myleoid Luekemia

MonitoringandTreatment-FreeRemission

An important development in CML management is the concept of treatment-free remission (TFR). Patients who achieve sustained deep molecular responses on TKI therapy may discontinue treatment under close monitoring, with about 40–60% maintaining remission (Mahon et al., 2018). This strategy offers hope for reducing long-term drug toxicity, financial burden, and quality-of-lifechallenges.

Prognosis

The prognosis of CML has improved dramatically. With effective TKI therapy, 5-year survival rates exceed 90% for patients diagnosed in the chronic phase (Hochhaus et al., 2020). Prognostic scoring systems such as the Sokal, Hasford, and EUTOS scores help predict treatment outcomes, althoughmolecularmonitoringremainsthemostreliablemeasureoflong-termdiseasecontrol.

FutureDirections

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BCR-ABL1

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Long-term metabolic,andg

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Cure stra vaccines, minimalresidualdiseaseandpr

Conclusion

Chronic My greatest success limited trea chronic disease

Continued and immunother outcomes molecular essentialpillarsinmanagingthisdisease

Myleoproliferative Neoplasms

Myeloproliferative neoplasms (MPNs) are a group of rare blood cancers that arise from abnormal growth of stem cells in the bone marrow, the body’s blood-forming factory They are characterized by the overproduction of one or more types of blood cells—red blood cells, white blood cells, or platelets. While some MPNs may remain stable for years, others can progress to more aggressive forms of blood cancer, including acute myeloid leukemia (AML). Advances in research have greatly improved our understanding of these diseases, leading to new treatment strategies and better outcomes for many patients

What Are Myeloproliferative Neoplasms?

The term myeloproliferative neoplasm describes a group of clonal hematologic cancers in which a genetic mutation causes uncontrolled proliferation of blood cell precursors in the bone marrow. These cells mature but accumulate in excessive numbers, leading to abnormal blood counts and complications

The hallmark feature of most MPNs is the presence of specific driver mutations in genes that regulate blood cell growth and signaling pathways, most notably JAK2, CALR, and MPL. These mutations activate the JAK-STAT signaling pathway, driving constant signals for cell division even in the absence of normal growth factors.

MPNs are classified as Philadelphia chromosome–negative (classic MPNs such as polycythemia vera, essential thrombocythemia, and primary myelofibrosis) or Philadelphia chromosome–positive, represented by chronic myeloid leukemia (CML).

Myleoproliferative Neoplasms

Types of Myeloproliferative Neoplasms

Polycythemia Vera (PV)

PV is characterized by the overproduction of red blood cells, often accompanied by elevated white blood cells and platelets. Symptoms may include:

Headaches, dizziness, and blurred vision

Itching (pruritus), especially after warm showers

Blood clotting events (deep vein thrombosis, stroke, or heart attack)

Enlarged spleen (splenomegaly)

Most patients with PV harbor the JAK2 V617F mutation. Left untreated, PV can increase the risk of life-threatening clots or progress to myelofibrosis or AML.

Essential Thrombocythemia (ET)

ET involves the overproduction of platelets, the blood cells responsible for clotting. Patients are at risk for both blood clots and bleeding complications due to dysfunctional platelets. Common symptoms include headaches, visual disturbances, and tingling in the hands or feet. Mutations in JAK2, CALR, or MPL are most commonly found.

Primary Myelofibrosis (PMF)

PMF is a more aggressive MPN characterized by scarring (fibrosis) of the bone marrow, which disrupts normal blood cell production. As a result, blood formation may shift to organs like the spleen and liver, causing enlargement and related symptoms. Hallmarks include:

Severe fatigue and weight loss

Night sweats and fevers

Anemia and low platelet counts in advanced stages

PMF can progress to acute leukemia and is associated with significant morbidity and mortality

Other Rare MPNs

There are less common subtypes such as:

Chronic neutrophilic leukemia (CNL)

Chronic eosinophilic leukemia (CEL)

MPN-unclassifiable (MPN-U), where features overlap but don’t fit neatly into one category

Myleoproliferative Neoplasms

Symptoms of MPNs

Symptoms vary by subtype but can include:

Fatigue and weakness

Unexplained weight loss

Night sweats

Enlarged spleen causing abdominal fullness or pain

Headaches, dizziness, or visual disturbances

Increased risk of thrombosis or bleeding

Importantly, some patients remain asymptomatic for years and are diagnosed through routine blood tests.

Diagnosis

Diagnosis begins with a complete blood count (CBC) showing abnormal red cells, white cells, or platelets.Furthertestingincludes:

Bonemarrowbiopsy:toevaluatemarrowcellgrowthandfibrosis.

Cytogeneticandmoleculartesting:toidentifykeymutations(JAK2,CALR,MPL,BCR-ABL1). Imaging:suchasultrasoundorMRI,todetectspleenorliverenlargement.

Accuratemoleculardiagnosisisessentialnotonlyforconfirmingthediseasebutalsoforguiding therapy.

TreatmentApproaches

PolycythemiaVera

Phlebotomy:periodicremovalofbloodtoreduceredcellmass.

Low-doseaspirin:tolowerclotrisk.

Cytoreductivetherapy:hydroxyureaorinterferon-alphainhigh-riskpatients.

Targetedtherapy:JAKinhibitorssuchasruxolitinibforresistantorintolerantPV.

EssentialThrombocythemia

Low-doseaspirin:formostpatients.

Cytoreduction:hydroxyurea,interferon-alpha,oranagrelideforhigh-riskpatients.

PrimaryMyelofibrosis

JAKinhibitors:ruxolitinib,fedratinib,andpacritinibhelpreducesymptomsandspleensize. Stem cell transplantation: the only potentially curative therapy, though suitable only for select patientsduetorisks.

Supportivecare:transfusionsandsymptommanagement.

Myleoproliferative Neoplasms

ComplicationsandRisks

UntreatedoradvancedMPNscanleadtoseriouscomplications: Thrombosisandbleeding:commoninPVandET.

Progressiontomyelofibrosis:PVandETcanevolveintosecondarymyelofibrosis. Transformationtoacuteleukemia(blastcrisis):occursinupto20%ofpatientsovertime.

Burden of symptoms: chronic fatigue, pruritus, and psychological distress significantly impact qualityoflife.

LivingwithanMPN

With regular monitoring and appropriate therapy, many patients live for years with controlled disease. Quality-of-life challenges, such as fatigue, emotional stress, and treatment side effects, remain central to patient care Support groups, advocacy organizations, and patient education playanimportantroleinhelpingpatientsandcaregiversnavigatethesechronicconditions

ResearchandFutureDirections

ThefutureofMPNtreatmentisrapidlyevolving:

Next-generationJAKinhibitorsaimtoimproveefficacyandreducetoxicity.

Combinationtherapiesarebeingtestedtoovercomeresistance.

Novel targets beyond JAK-STAT, such as epigenetic regulators and telomerase inhibitors, are underinvestigation.Immunotherapyapproachesmayonedayhelperadicatemalignantclones. Effortstoachievetreatment-freeremissioninCMLandpotentiallyinotherMPNsareongoing.

Myeloproliferative neoplasms represent a complex but increasingly manageable group of blood cancers. From PV and ET, which often remain indolent for years, to MF and CML, which can be life-threatening without treatment, the spectrum of MPNs reflects the importance of early diagnosis, molecular testing, and targeted therapy Advances in understanding the genetic and molecular basis of these diseases have transformed patient care and opened doors to personalized medicine With continued research, the outlook for patients with MPNs continues toimprove,offeringhopeforlonger,healthierlivesandevenpotentialcures

Myleodisplatic Syndrome

Myelodysplastic syndromes (MDS) are a group of rare blood cancers characterized by abnormal development of blood-forming cells in the bone marrow. Once known as “pre-leukemia,” MDS occurs when immature blood cells (called blasts) do not mature properly and fail to function normally. This leads to low counts of red blood cells, white blood cells, and/or platelets, a condition known as cytopenia Although some people with MDS may live with stable disease for years, others may progress to acute myeloid leukemia (AML), a more aggressive form of blood cancer.

MDS is classified as a clonal hematopoietic disorder, meaning it begins with a genetic change in a single bone marrow stem cell that gives rise to defective blood cells. These abnormal cells either die prematurely in the marrow or enter circulation without functioning properly.

As a result, patients with MDS often experience: Anemia (low red blood cells) leading to fatigue, weakness, and shortness of breath. Neutropenia (low white blood cells), which increases susceptibility to infections. Thrombocytopenia (low platelets), causing easy bruising, bleeding, and petechiae. The disease is heterogeneous, ranging from mild forms requiring little intervention to aggressive types that rapidly evolve into AML.

Causes and Risk Factors

The exact cause of MDS is often unknown, but several risk factors are recognized: Age: MDS is most common in individuals over 60 years old.

Previous chemotherapy or radiation: Known as therapy-related MDS, this subtype arises after cancer treatment.

Genetic predisposition: Rare inherited syndromes (e g., Fanconi anemia, Shwachman-Diamond syndrome) can predispose individuals.

Environmental exposures: Long-term contact with benzene, tobacco smoke, or heavy metals may increase risk.

Most cases of MDS, however, occur sporadically without a clear cause.

Myleodisplatic Syndrome

Classification of MDS

MDS is classified by the World Health Organization (WHO 2022) into subtypes based on blood counts, blast percentage, and genetic abnormalities. The main categories include:

MDS with low blasts (MDS-LB) – mild cytopenias, fewer immature cells

MDS with SF3B1 mutation and ring sideroblasts (MDS-SF3B1) – characterized by defective red cell maturation.

MDS with biallelic TP53 inactivation – associated with poor prognosis.

MDS with excess blasts (MDS-EB) – higher risk of progression to AML.

Therapy-related MDS – occurs after exposure to chemotherapy or radiation. This classification guides both prognosis and treatment

Symptoms

Symptomsvarybutofteninclude:

Persistentfatigueandweakness

Paleskin(fromanemia)

Frequentinfections(fromneutropenia)

Easybruisingorbleeding(fromthrombocytopenia)

Enlargedspleenorliver(insomecases)

Because these symptoms overlap with many other conditions, MDS is often diagnosed through detailedbloodandbonemarrowtesting.

Diagnosis

DiagnosisofMDSrequiresacombinationoftests:

CompleteBloodCount(CBC):Showscytopenias.

PeripheralBloodSmear:Revealsabnormalcellmorphology.

BoneMarrowBiopsy:Confirmsdysplasia,blastcount,andcellularity

Cytogenetic and molecular testing: Detects chromosomal abnormalities (e g., deletion 5q, monosomy7,complexkaryotype)andgenemutations(e.g.,TP53,TET2,ASXL1).

PrognosisandRiskStratification

Prognosis varies widely. Some patients live for a decade with low-risk disease, while others progress quickly to AML. Doctors use scoring systems like the Revised International Prognostic ScoringSystem(IPSS-R),whichconsiders:

Cytogeneticabnormalities

Numberandseverityofcytopenias

Blastpercentageinbonemarrow

Thisriskstratificationguidestreatmentdecisions.

This article is informational and not a substitute for medical advice Individual situations vary please discuss your results and options with your hematologist/oncologist

Myleodisplatic Syndrome

PrognosisandRiskStratification

Prognosis varies widely. Some patients live for a decade with low-risk disease, while others progress quickly to AML. Doctors use scoring systems like the Revised International Prognostic ScoringSystem(IPSS-R),whichconsiders:

Cytogeneticabnormalities

Numberandseverityofcytopenias

Blastpercentageinbonemarrow

Thisriskstratificationguidestreatmentdecisions.

TreatmentApproaches

SupportiveCare

Bloodtransfusionsforanemia

Erythropoiesis-stimulatingagents(ESAs)toboostredcellproduction

Antibioticsforinfections

Platelettransfusionsforbleeding

Disease-ModifyingTherapies

Hypomethylating agents (HMAs): Azacitidine and decitabine can improve survival and delay AMLprogression.

Lenalidomide: Effective for patients with an isolated deletion 5q (del(5q)) chromosomal abnormality

Immunosuppressive therapy: Antithymocyte globulin (ATG) and cyclosporine for select patients

CurativeTherapy

Allogeneic stem cell transplantation remains the only potentially curative treatment, but it is oftenlimitedtoyoungerorfitpatientsduetorisks.

LivingwithMDS

MDS can be a chronic condition requiring ongoing monitoring and management Patients benefit from a multidisciplinary approach, including hematologists, transplant specialists, and supportive care teams. Fatigue, emotional stress, and treatment side effects can significantly affectqualityoflife,makingpsychologicalsupportandpatientadvocacycrucial.

Myleodisplatic Syndrome

FutureDirections

ResearchinMDSisadvancingtoward:

Targetedtherapies:Drugsdesignedtoinhibitspecificmutations(e.g.,IDH1/2inhibitors). Combinationtherapies:HMAsplusvenetoclaxornovelimmunecheckpointinhibitors. Genomicprofiling:Personalizingtreatmentbasedonindividualmutationalprofiles. Improvedtransplantapproaches:Reducingcomplicationsandexpandingeligibility.

Myelodysplastic syndrome is a complex group of bone marrow cancers that disrupt normal blood cell development and function With its variable course, ranging from mild to aggressive, MDS presents both clinical challenges and opportunities for innovation in treatment Advances in molecular genetics and targeted therapy are offering new hope, and ongoing research continuestotransformtheoutlookforpatientslivingwiththisdisease.

Iron Deficiency Anemia

Iron Deficiency Anemia: Understanding the Most Common Blood Disorder

Iron Deficiency Anemia (IDA) is the most common type of anemia worldwide, affecting billions of people regardless of age, gender, or geography. It occurs when the body doesn’t have enough iron to produce hemoglobin—a protein in red blood cells that carries oxygen throughout the body. Without adequate hemoglobin, the body’s tissues and organs don’t get the oxygen they need to function optimally, leading to fatigue, weakness, and a range of other symptoms

This article explores what IDA is, who it affects, the symptoms to watch for, treatment options, and what the long-term outlook looks like for patients living with this condition.

What Is Iron Deficiency Anemia?

Iron is an essential mineral that plays a critical role in producing hemoglobin. When iron stores are depleted or when the body’s demand for iron exceeds supply, red blood cell production drops This leads to fewer red blood cells, smaller cell size, and reduced oxygen-carrying capacity.

Causes of IDA

Iron deficiency anemia can result from one or a combination of these factors:

Inadequate dietary intake

Diets low in iron-rich foods such as red meat, leafy greens, or fortified grains.

Blood loss

Heavy menstrual periods, gastrointestinal bleeding (from ulcers, hemorrhoids, or cancers), or frequent blood donation.

Increased demand

Pregnancy, breastfeeding, rapid growth during childhood or adolescence, or endurance training. Poor absorption

Conditions like celiac disease, inflammatory bowel disease, or bariatric surgery can limit iron absorption.

Who Does IDA Affect?

IDA is widespread, but certain populations are more at risk:

Women of Childbearing Age: Due to menstrual blood loss, women between 15 and 50 are at high risk. Up to 20% of women in North America may have low iron levels, according to the Centers for Disease Control and Prevention (CDC, 2023).

Pregnant Women: Iron requirements double during pregnancy to support fetal growth and maternal blood volume expansion.

Children and Adolescents: Rapid growth increases iron demand.

Dietary habits (picky eating or lack of iron-rich foods) often compound the risk.

Older Adults: Chronic conditions, medications, and age-related changes in absorption increase the risk.

Patients with Chronic Diseases: Conditions like kidney disease, cancer, inflammatory bowel disease, or chronic bleeding disorders frequently lead to secondary IDA

Athletes: Endurance sports can increase iron losses through sweat, urine, or gastrointestinal microbleeding.

Signs and Symptoms of IDA

Iron deficiency anemia often develops slowly, and symptoms may be mild or overlooked until anemia becomes severe.

Common Symptoms

Persistent fatigue and weakness

Pale skin, especially noticeable in the face or inner eyelids

Shortness of breath during routine activities

Rapid heartbeat or palpitations

Cold hands and feet

Other Indicators

Headaches, dizziness, or lightheadedness

Brittle nails or hair loss

Pica (craving non-food items like ice, dirt, or starch)

Restless legs syndrome (RLS)

If you suspect iron deficiency, it’s critical to see a healthcare provider for a blood test. Diagnosis typically involves:

Complete Blood Count (CBC)

Serum ferritin and iron studies

Additional testing to find the cause of iron loss if needed

DiagnosisofIDA

Accurate diagnosis is essential to guide treatment and rule out underlying causes, especially gastrointestinalbleedingorchronicdisease.

Keytestsinclude:

HemoglobinandHematocritlevels–toassessthedegreeofanemia. Ferritin–ameasureofstorediron(lowlevelsindicatedeficiency).

SerumironandTotalIronBindingCapacity(TIBC)–toevaluateavailableiron. Insomecases,additionaltesting,suchasacolonoscopyorupperendoscopy,maybeorderedto investigategastrointestinalsourcesofbloodloss.

TreatmentOptionsforIDA

Effectivetreatmentaddressesbothirondeficiencyanditsrootcause. DietaryChanges

Inmildcases,dietalonemayhelprestoreironlevels.

Iron-richfoods:

Redmeat,poultry,fish

Lentils,beans,tofu

Darkleafygreens(spinach,kale)

Fortifiedcerealsandgrains

Pai

Avo

OralIronSupplements

First-linetherapyformostpatients.

Commonforms:ferroussulfate,ferrousgluconate,orferrousfumarate. Usuallytakenfor3–6monthstorebuildstores.

Side effects: gastrointestinal upset, constipation, or dark stools. Slow-release formulations or alternate-daydosingcanhelp.

Intravenous(IV)IronTherapy

Forpatientswho:

Cannottolerateoraliron

Havemalabsorptionsyndromes

Needrapidreplenishment(e.g.,severeanemia,latepregnancy,chronicdisease)

IVformulations(likeferriccarboxymaltoseorironsucrose)quicklyrestorelevels,ofteninone ortwoinfusions.

AddressingtheRootCause

Treatgastrointestinalbleedingorulcers.

Manageheavymenstrualbleedingwithhormonaltherapies.

Addressunderlyingchronicdiseases.

5.Erythropoiesis-StimulatingAgents(ESAs)

Inpatientswithchronickidneydiseaseorcancer,ESAscombinedwithirontherapymaybeused tostimulateredbloodcellproduction.

Long-Term Outlook

Iron deficiency anemia is highly treatable, and the prognosis is generally excellent once the underlying cause is addressed.

Recovery Timeline

Symptom improvement – within a few weeks of starting therapy.

Normal hemoglobin – typically within 2–3 months

Iron stores – may take 6 months or longer to replenish fully Challenges

Non-adherence due to side effects from oral supplements.

Unidentified or ongoing blood loss leading to recurrent anemia.

Complications of Untreated IDA

Severe fatigue is impacting daily function.

Increased risk of infections

Heart complications, such as a rapid heartbeat or heart failure, can occur in severe cases. In pregnancy, risks include low birth weight or premature birth.

LivingWellwithIDA

Withpropermanagement,mostpeoplewithIDAcanleadfull,healthylives.

Self-ManagementTips:

Takesupplementsasprescribedandfollowupwithyourhealthcareprovider

Eatabalanceddietwithiron-richfoods

Reportanyrecurringsymptomspromptly

Scheduleregularbloodteststomonitorprogress

IDAandSpecialPopulations

Children: Early detection is critical as iron deficiency can affect cognitive development and behaviour.Pediatriciansoftenrecommendironsupplementationinat-riskchildren.

PregnantWomen:Screeningforanemiaisroutine.Supplementationimprovesoutcomesforboth motherandbaby.

Older Adults: Regular screening and nutritional assessments help prevent complications related tocognitivefunctionandcardiovascularhealth.

Research and Emerging Therapies

Recent advancements focus on improving the safety, efficacy, and convenience of iron therapies:

New IV iron formulations with fewer side effects

Alternate-day dosing regimens have been shown to improve absorption and reduce side effects of oral iron.

Digital tools and apps for monitoring symptoms and treatment adherence.

Key Statistics

Approximately 1.2 billion people worldwide are affected by IDA (World Health Organization, 2023).

Up to 50% of anemia cases globally are due to iron deficiency

In Canada, about 4–6% of men and 12–15% of women have low iron levels (Statistics Canada, 2023).

Iron Deficiency Anemia may be common, but it is far from trivial Recognizing symptoms early, understanding risk factors, and seeking timely medical care are essential steps toward recovery With proper treatment and lifestyle adjustments, patients can restore their iron levels, reclaim their energy, and prevent long-term complications.

Quality of Life Survey for IDA

If you have Iron Deficiency Anemia, please take the survey https://www.surveymonkey.com/r/MRQ3VXL

Podcast - for IDA

https://www.healcanada.org/podcasts

Patient Advocacy Support Group IDA

If you would like to join an IDA Support Group, please email us at admin@healcanada.org

Overview of Iron Deficiency Anemia Treatment in Canada

First-line Therapy: Oral Iron Supplementation

Standard of Care: Oral iron salts—like ferrous sulphate, fumarate, or gluconate—remain the primary treatment for IDA due to their affordability, effectiveness, and widespread availability

Dosing Considerations: Traditional dosing (e.g., ferrous sulphate 325 mg three times daily) is still used, though emerging evidence supports lower or alternate-day dosing to reduce gastrointestinal side effects while maintaining efficacy.

Challenges: Around 30–70 % of patients report side effects such as constipation, nausea, abdominal pain, or diarrhea, limiting adherence KHSC Kingston Health Sciences.

Monitoring: Hemoglobin and ferritin levels are typically reassessed in 2–4 weeks for response, and treatment continues for 4–6 months after anemia correction to replenish iron stores—target ferritin levels are often set above 100 µg/L in Canadian guidelines.

When Oral Iron Isn't Enough: Intravenous Therapy

New Approval—Ferinject® (Ferric Carboxymaltose): As of March 2024, Health Canada approved Ferinject for use in adults and children aged one year and older in cases where oral iron is intolerable or ineffective. It's also indicated for adults with iron deficiency associated with heart failure (NYHA class II/III) to improve exercise capacity.

Other IV Options in Canada: Health Canada had previously approved ferumoxytol (marketed as Feraheme) for IDA treatment in patients with chronic kidney disease.

Clinical Use of IV Iron: IV infusions provide fast, effective iron replenishment and are particularly helpful when oral therapy fails or when quick correction is necessary, such as in pregnancy or severe anemia. Common IV formulations in clinical use include iron sucrose (Venofer®), ferric carboxymaltose (Ferinject), ferumoxytol (Feraheme), and iron isomaltoside (Monofer). Each has different dosing protocols and safety profiles.

Safety Considerations: Serious allergic reactions are rare, especially with newer IV preparations, but mild reactions like dizziness or metallic taste may occur Most institutions monitor patients during infusion to ensure safety.

Dietary and Public Health Measures

Food Fortification & Dietary Counseling: Promoting iron-rich foods (e g., red meats, legumes, leafy greens) and dietary counseling is a complementary strategy. Fortified foods—like ironfortified flour or cereals are widely utilized public health approaches to help prevent IDA. Supplements for At-Risk Groups: Preventive supplementation is sometimes recommended for those with increased iron needs (e.g., pregnant individuals, those with restrictive diets, or malabsorption conditions).

Diagnostics and Broader Clinical Pathway

Identifying Underlying Causes: Diagnosis involves CBC, ferritin testing, and investigation of potential etiologies especially with microcytic anemia presentations. If ferritin is normal but anemia persists, hemoglobinopathies like thalassemia may be considered.

Further Investigations: If there’s an inadequate response to iron treatment, guidelines recommend GI evaluations (upper/ endoscopy, colonoscopy) and, if these are negative, exploring small bowel causes using capsule endoscopy or CT/MR enterography Medsc

Summary Table: Treatment Landscape in Canada

Treatment Modality Use Case / Benefits Limitations / Monitoring Requirements

Oral Iron Supplements First-line, cost-effective, convenient GI side effects; adherence challenges; ongoing monitoring

IV Iron (Ferinject®, Feraheme, etc.) Effective when oral therapy fails or is urgent; newly approved options are expanding treatment access. Requires a clinical setting; potential infusion reactions

Dietary & Fortification Strategies Population-level prevention; supports therapy. May not address existing anemia; adherence relies on education

Diagnostics / Investigations Essential for identifying causes and guiding therapy

Resource-dependent; may delay treatment initiation temporarily

FinalThoughts

In Canada, the primary approach remains oral iron supplementation, supported by evidencebased guidelines. However, treatment is becoming more flexible and effective with the introduction of new IV iron options like Ferinject, expanding the capacity to manage patients whocannottolerateorrespondtooraliron.

Public health measures—such as improved dietary intake and food fortification—continue to play a key role in prevention, while robust diagnostic pathways help ensure that underlying conditionsaren’tmissed

Shouldyoulike,Icandivedeeperintodosingprotocols,compareoutcomesbetweenIVagents, orexploreregionalaccessandreimbursementissuesacrossCanadianprovinces.

Spotlight on Innovation: Advances in Blood Cancer Treatment (2025)

Blood cancer care is changing fast and for the better. In just the last couple of years, new immune therapies, smarter targeted drugs, and practical digital tools have opened real options for people living with leukemia, lymphoma, myeloma, myeloproliferative neoplasms (MPNs), and related conditions. This spotlight walks you through the biggest developments—what they are, why they matter, and how to talk about them with your care team—using plain language and a patient-first lens.

CAR T-cell therapy: Powerful, expanding, and easier to access

What it is (in a sentence): Doctors collect some of your T-cells, engineer them to recognize a marker on your cancer cells, multiply them, and infuse them back to hunt the cancer.

What’s new

Earlier use in myeloma. In 2024, the FDA moved BCMAdirected CAR T earlier in treatment: ciltacabtagene autoleucel (Carvykti) became available in the second line, not just after many prior therapies, and idecabtagene vicleucel (Abecma) was cleared for earlier use in relapsed/refractory myeloma. That’s a big shift patients may no longer have to wait through multiple regimens to access a one-and-done cell therapy. More lymphomas and now CLL/SLL In 2024, the CD19 CAR T lisocabtagene maraleucel (Breyanzi) gained new indications: for follicular lymphoma and mantle cell lymphoma, and crucially for CLL/SLL after both a BTK inhibitor and venetoclax offering a cell therapy option where choices have been limited.

Access improvements In June 2025, the FDA eliminated the special REMS programs that used to be required for all CAR Ts (the boxed warnings for CRS/ICANS remain). This reduces administrative friction for hospitals and may help expand the number of centers offering these treatments.

Safety clarity In April 2024, the FDA added a class boxed warning about rare cases of T-cell malignancies (new Tcell cancers) after CAR T, across both CD19- and BCMAdirected products. The overall risk appears low, but it’s important to discuss it alongside the well-known risks of cytokine release syndrome (CRS) and neurotoxicity.

Whatthismeansforyou

If you or your loved one has myeloma and has relapsed after initial therapy, you may be eligible for CAR T soonerthanbefore.

If you have DLBCL, FL, MCL, or CLL/SLL, ask whether CARTisafitnoworasaplannedoptionlater.

Because manufacturing takes weeks, teams often use “bridging” therapy to control disease while your cells are prepared Knowing timelines helps you plan supports around work, caregiving, travel, and infection prevention.

Bispecificantibodies:“Off-the-shelf”immunetherapies What they are: Antibodies that latch onto a T-cell with one“hand”andyourcancercellwiththeother,bringing themtogethertokillthecancer withoutcollectingand engineeringyourowncells.

Highlights

Epcoritamab (Epkinly), a CD20×CD3 bispecific, added an FDA indication in follicular lymphoma in June 2024 (building on its DLBCL indication) It’s given subcutaneously with step-up dosing and careful monitoringearlyon

Teclistamab (Tecvayli), a BCMA bispecific for myeloma, earned a reduced dosing frequency option (every two weeks) for patients in deep remission; that can ease clinicburdenonceyou’restable.JNJ.

Talquetamab (Talvey), first-in-class GPRC5D×CD3 for myeloma, received accelerated approval in 2023 and remains a key option, especially for patients previously exposedtoBCMA-directedtherapies.

Safetynotes

Bispecifics can cause CRS and neurologic effects, especially in the first cycles, plus infection risk (including opportunistic viruses) Your team will do step-up dosing, premedication, and infection prevention; calling early about fevers or confusion is crucial.

Why patients like them

They’re available immediately (no manufacturing wait), and many are subcutaneous or IV on a clinic schedule that may become less frequent over time—useful if travel to a CAR T center is hard.

Precision & targeted therapies: Sharper tools for specific mutations or pathways

Blood cancers are driven by different genetic “wiring.” Matching drugs to those drivers can deepen responses and reduce broad toxicity

Breakthroughs and updates

Menin inhibition for acute leukemia. In November 2024, the FDA approved revumenib (Revuforj) for KMT2A-rearranged relapsed/refractory acute leukemia in adults and children ≥1 year—the first approval in this class. Another menin inhibitor, ziftomenib, is under FDA priority review in 2025 for NPM1-mutant AML. Ask about mutational testing if you or a loved one has AML

Next-gen BTK inhibition in CLL/SLL. Pirtobrutinib (Jaypirca), a non-covalent BTK inhibitor, gained an FDA indication in December 2023 for adults who’ve already had both a BTK inhibitor and a BCL-2 inhibitor an important option when resistance emerges. Zanubrutinib continues to mature with additional data and new combinations (including an FL indication with obinutuzumab in 2024).

Anemia-focused advances in MDS and MPNs

Imetelstat (Rytelo), the first-in-class telomerase inhibitor, was approved in June 2024 for lowerrisk MDS with transfusion-dependent anemia after ESAs, offering meaningful transfusion independence for some patients.

Momelotinib (Ojjaara) became the first myelofibrosis therapy specifically indicated for patients with anemia (approved September 2023). Managing anemia and spleen/symptoms together can improve everyday functioning. Pacritinib (Vonjo) emains option thrombocytopenia

CAR-T beyond DLBCL: more doors opening. Beyond the myeloma expansions above, Breyanzi also added follicular lymphoma and mantle cell lymphoma indications in 2024 evidence that celltherapyisbecomingaplatformacrossB-cellcancers.

Bottom line: Ask your clinician whether your cancer has a target (e.g., KMT2A rearrangement, NPM1 mutation in AML; BTK resistance in CLL; cytopenias in MF) that matches one of these neweroptions.

Digitalhealth&symptomtracking:Smallhabits,bigimpact

Whytracksymptomsdigitally?Becausewhatyounoticeday-to-day fatigue,fevers,neuropathy, mood, sleep, pain often predicts complications before labs or scans do When clinics invite patientstoreportsymptomsregularlythroughanapporwebportal(ePROs),outcomesimprove: Randomizedtrialsshowbetterqualityoflife,fewerERvisits/hospitalizations,andevenimproved survival when patients use electronic symptom reporting with rapid team feedback, compared withusualcare.

Practicaltoolsandframeworks

Validated instruments matter. In MPNs, the MPN-10 / MPN-SAF Total Symptom Score is a short, validated questionnaire used in clinics and recommended in guidelines; digital versions are increasinglyusedforroutinetracking.

Clinic portals and stand-alone apps. Many cancer centers activate ePRO modules inside the patient portal (e.g., weekly check-ins that page your nurse if a score spikes). Disease-specific platforms such as HealthTree Cure Hub help myeloma and other blood-cancer patients track labs, symptoms, and treatments in one place and contribute anonymized real-world data to research.

Plain-language care pathways. The NCCN launched a refreshed Guidelines Navigator to make high-quality, plain-language guidance easier to use on mobile handy to prepare for appointmentsandconfirmwhatyou’veheard.

Howtostart(amini-playbook)

1.AskyourclinicwhethertheyofferePROsymptommonitoringandhowalertsarehandledafter hours.(Whocallsyouback?Howfast?)

2.Pickonetrackeranduseitconsistently ideally,theoneyourcareteamcansee.Ifyouusean outsideapp,bringtheprintoutorscreenshottovisits.

3. Track what matters: fatigue/energy, pain, fevers, weight change, mood/sleep, bowel patterns, neuropathy,bleeding/bruising,newlumpsorswelling,infections,medicationsideeffects.

4. Set a weekly check-in. Five minutes on Sunday night can catch trends you might otherwise miss.

5)Puttingittogether:Choosingamongoptions

There’s no single “best” therapy the right next step depends on your disease type, prior treatments, genetics, age, other health issues, and your priorities (e.g., fewer clinic days vs. deepestresponse).Here’saquickcomparisontosparkproductiveconversationswithyourteam: Option;Howitworks;Whoithelpsmosttoday;Pros;Watch-outs

CAR T-cell therapy; Retrain your own T-cells to target CD19 or BCMA Many B-cell lymphomas; myeloma (now earlier); CLL/SLL after BTK + BCL-2; One-time infusion, high response rates, durable remissions CRS/ICANS risk; manufacturing wait; rare T-cell malignancy warning; caregiver/timecommitments.

Bispecificantibodies“Off-the-shelf”T-cellengagers(eg,CD20×CD3,BCMA×CD3,GPRC5D×CD3) DLBCL/FL (epcoritamab), myeloma (teclistamab/talquetamab) Available immediately; can transition to less-frequent dosing Early-cycle CRS/neurologic toxicity; infection risk; regular clinictime

Targeted therapies: Aim at specific mutations/pathways (menin, BTK, JAK/ACVR1) AML with KMT2A rearrangement (revumenib); CLL with BTK resistance (pirtobrutinib); MF with anemia (momelotinib); MDS anemia (imetelstat). Oral options in some cases; precision matching; can be combined.Needmutationtesting;somearenewwithevolvinglong-termdata

Questionstobringtoyournextappointment

“Where am I on my treatment journey?” (First line? Secondline?Later?)

“Do my labs or genetics open doors to targeted options?” (e.g., KMT2A/NPM1 in AML; BTK resistanceinCLL;cytopeniaprofileinMF.)

“Could CAR T or a bispecific be right for me now orasaplannednextstep?”

“How do we monitor and prevent infections?” (Vaccines,antivirals,IVIG,growthfactors.)

“Can we use ePRO symptom tracking?” (What app/portal?Whowatchesmyalerts?)

“What’s the clinic time commitment and caregiver supportneeded?”

“Aretheretrialsnearbythatfitmysituation?”

g well through treatment: small choices, big ends

ever therapy you and your team choose, patient-testedhabitsmakeadifference: symptomsweeklyandcallearlyaboutfevers, ness of breath, confusion, chest pain, ing, or new neurologic symptoms. (ePRO amsexistbecauseearlyactionworks.)

nt infections: hand hygiene, masks in ded indoor spaces, and keep vaccines up-toperyourclinician.

Protect energy: fatigue is common; plan “big energy” tasks for better days, take short walks to fight deconditioning, and ask about sleep or mood supports

Bring a buddy (in person or by speakerphone) to important visits to help with notes and shared decisions.

Ask about financial and logistical help: social work, travel programs, and assistance foundations can reducetheburdenofhigh-touchtherapies.

Glossary (quick & friendly)

CAR T-cell therapy: Your own T-cells are engineered to recognize a cancer marker (like CD19 in many lymphomas or BCMA in myeloma) and infused back.

Bispecific antibody: An antibody that binds to a T-cell and a cancer cell simultaneously to bring them together.

CRS/ICANS: Common early CAR T/bispecific side effects—fevers, low blood pressure (CRS), and temporary brain-related symptoms (ICANS).

Menin inhibitor: A drug class (e g., revumenib) that disables a protein complex driving certain leukemias (KMT2A-rearranged, NPM1-mutant).

ePRO: Electronic patient-reported outcomes your symptoms, tracked regularly via a portal or app. These programs improve outcomes when teams respond to alerts.

The horizon: what we’re watching

Earlier-line use of CAR T and bispecifics across more blood cancers, including combinations (e g., dual bispecifics in myeloma).

More precision drugs (e.g., additional menin inhibitors for AML under review in 2025).

Smarter digital tools that integrate symptom alerts, lab trends, and home vitals in one patientfriendly dashboard—now appearing in major guideline apps and portals.

New Treatements for Polycthemica Vera

Author: Lindsay Kaun, PharmD, BCPS

Current treatment strategies are aimed at reducing hematocrit and symptom burden through phlebotomy,aspirin,andcytoreductiveagents,whileemergingtherapiesofferpromiseforbetter diseasecontrolandqualityoflife.

Polycythemia vera (PV) is a rare and chronic blood cancer that is classified as a myeloproliferative neoplasm (MPN) The 3 classical Philadelphia chromosome-negative MPNs, consisting of PV, essential thrombocythemia (ET) and myelofibrosis (MF), originate in the hematopoieticstemcellsofthebonemarrowandarecharacterizedbyincreasedproliferationof themyeloidlineagesowingtoanoveractiveJAK-STATpathway

Bonemarrowstudyofamyeloproliferativedisorder

MPNs were once considered disorders rather than cancers and were largely ignored9 This identification changed in 2008 once it was understood that these diseases involve all types of myeloidcellsandareclonallydrivenbyspecificdrivermutations

TherecognitionofMPNsasbloodcancersin2008broughtlong-overdueattentionandresearch investment.2PVisthemostcommonMPNandtheonlyMPNinwhicherythrocytosisoccurs.

Acquired somatic gain-of-function mutations of JAK2, primarily JAK2V617F and less commonly exon12,arepresentinvirtuallyallpatientswith ThesemutationsleadtoanoveractiveJAK-STAT signalling pathway, which is responsible for transmitting signals from growth factors, cytokines andotherligandsoutsideofastemcelltoitsnucleus.Thisleadstoexcessiveerythropoiesisand a subsequent increase in red blood cell (RBC) mass, which may lead to clinically significant thrombotic and hemorrhagic complications. Because pluripotent stem cells are involved, PV is considered to be a tri-lineage disease and can overproduce white blood cells and platelets as well.

PV is estimated to affect 44 to 57 per 100,000 people, equating to an estimated 150,000 to 190,000patientsintheUS.

PV is usually diagnosed between 50 and 70 years of age and is more common in men than women.ThemediansurvivalforpatientswithPVisapproximately14years,withthosediagnosed atanearlieragemorelikelytosurvivelonger.

These patients have a 1.6-fold higher mortality. Despite its later diagnosis and indolent course, PVisstillexpectedtoshortensurvivalbyapproximately5to10years

PVsymptomsareoftennon-specificbutcangreatlyimpactqualityoflife(QOL).Fatigueisoften reportedbypatientsasthemostprevalentandseveresymptom,with84%ofpatientsreporting fatigueintheinternationalMPNLandmarksurvey.

Inaddition,ofpatientswithPVwithhighsymptomburden,94%hadreducedQOLand483%had decreasedworkproductivity Othersymptomsincludedrenchingsweats,weightloss,abdominal discomfort, bone pain, sexual dysfunction, headache, shortness of breath, blurry/double vision, eye pain, dizziness, brain fog, lack of concentration, and aquagenic pruritus Many of these symptoms (ie, fatigue and pruritus) are thought to be caused by pro-inflammatory cytokines fromtheJAK-STATpathway

Clinical signs may include hypertension and splenomegaly

It is important to note that patients with PV may not always exhibit obvious signs of illness but often feel “sick” as these non-specific symptoms compound and impact QOL This may make their experience difficult to understand for others.

The complications of PV can be serious and lead to significant morbidity and mortality The increased blood viscosity caused by erythrocytosis, qualitative abnormalities of RBCs, and leukocytosis has been associated with an increased risk of thromboses and other microcirculatoryabnormalities

Thromboses can be either venous or arterial and may occur in any area of the body. The incidence range of arterial and venous thrombosis is 12% to 39% in PV. Arterial thrombosis, includingacutemyocardialinfarction,cerebrovascularischemicepisodes,andperipheralarterial occlusion,represent60%to70%ofallcardiovasculareventsinPV.

Deep vein thrombosis and pulmonary embolism may also occur in these patients. Additionally, patients with PV show a high prevalence of rare forms of thrombosis, such as abdominal vein thrombosis,includingextrahepaticportalveinocclusion,Budd-Chiarisyndrome,andmesenteric veinthrombosis

These events tend to occur most frequently shortly before or after diagnosis and decrease over time, likely due to PV treatment. Cardiovascular mortality accounts for 41% of deaths (1.5 death events per 100 persons per year), with an incidence of non-fatal thrombosis of 3.8 events per 100patient-years.

Another serious complication of PV is the risk of progression to MF or transformation to acute myeloidleukemia(AML).Approximately20%ofpatientswithPVprogresstoMForanaggressive formofAMLknownasMPNblastphase(MPN-BP).

Elevatedhematocrit(HCT)isahallmarkofPV,indicatingoverproductionofRBCsandhighblood viscosity. Other lab abnormalities include increased hemoglobin (HGB), abnormal red cell morphology (mean corpuscular volume, mean corpuscular hemoglobin), increased lactate dehydrogenase,decreasederythropoietin,andirondeficiency.

The 2016 World Health Organization diagnostic criteria for PV require the presence of either all threemajorcriteriaorthefirsttwomajorcriteriaandtheminorcriterion

ThemajorWHOcriteriaare:

HGB >165 g/dL in men and >16 g/dL in women, or HCT >49% in men and >48% in women, or red cellmass>25%abovemeannormalpredictedvalue

Bonemarrowbiopsyshowinghypercellularityforagewithtrilineagegrowth,and PresenceofaJAK2V617Forexon12mutation.

Theminorcriterionisasubnormalerythropoietinlevel WorkupforPVtypicallyincludesabone marrow biopsy to detect the JAK2 mutation and look for the presence of fibrosis, which may indicateprogressiontoMF.

Risk stratification per the National Comprehensive Cancer Network (NCCN) classifies patients with PV under 60 years of age and no history of thrombosis as low risk and those over 60 years ofageand/orwithapriorhistoryofthrombosisashighrisk.

The main goals of treatment are to reduce the risk of thrombosis, which is the most common causeofmortality,preventtransformationtoMForMPN-BP,andtotreatsymptoms. Atthistime,nodrugtherapyhasbeenshowntoprolonglifeorpreventtransformationtoMFor AML. The mainstays of PV treatment are low-dose aspirin and therapeutic phlebotomy (bloodletting) to mitigate thrombotic risk. The purpose of phlebotomy is to reduce the red cell massandsubsequenthyperviscosity,whichcandirectlyleadtothrombosis.

Current guidelines recommend that patients with PV maintain a target HCT under 45%.16 This cutoffisbasedonthe2013CYTO-PVstudy,whichrandomized365patientswithPVtreatedwith phlebotomyand/orhydroxyureatoeitheraHCTtargetoflessthan45%or45%to50%18Aftera median follow-up of 31 months, death from cardiovascular causes or major thrombotic events wasrecordedin3%ofpatients(5of182patients)withaHCTleveloflessthan45%comparedto 10%(18of183patients)ofpatientswithaHCTlevelof45%to50%(P= 007)18Somephysicians useaHCTtargetof42%inwomenorinthosewithsymptoms

An argument can be made that because men have higher testosterone levels, which boost RBC production, and a higher normal red cell mass than women, women should have lower HCT targets. While there is no data to support this practice, HCT targets can be individualized for those who may feel better at lowerHCTs.

Phlebotomy is not a benign intervention as it can exacerbate disease-related iron-deficiency Recurrent phlebotomy may lead to or worsened iron-associated symptoms, such as fatigue, lack of concentration, shortness of breath, depression, paresthesia, and restlesslegssyndrome.

It also results in volume shifts which can lead t hypotension, lightheadedness and dizzines

Phlebotomies are also time-consuming and resource intensive,andaccessmaybedifficultforpatientslivin inmoreruralareas ThetreatmentofPVisdrivenbyth patient’s risk stratification Low-risk patients ar managed with phlebotomy and low-dose aspiri

Cytoreductive therapy may be added in the case o frequent phlebotomies, phlebotomy intoleranc disease-related symptoms, progressive thrombocytos and/orleukocytosis,orsplenomegaly.

Cytoreductive agents used in PV include hydroxyurea (HU), pegylated interferon-alpha (INF-α) and ruxolitinib.HU was first used as an antineoplastic in the 1960s and, despite not being FDAapproved for PV, it is the most commonly used cytoreductive agent It has the dual advantages of being oral and inexpensive. Important adverse effects (AEs) that are often dose-related and may lead to discontinuation include leg ulcers and other mucocutaneous manifestations such as mouth sores, gastrointestinal symptoms, pneumonitis, and fever.

There is a risk of HU-induced secondary malignancies, particularly non-melanoma skin cancers, and approximately 25% of patients with PV become intolerant or resistant to HU.

Two INF-α products are used to treat PV: peginterferon alfa-2a (Pegasys; pharma& GmbH) and ropeginterferon alfa-2b (Besremi; PharmaEssentia Corporation). Peginterferon alfa-2a is not FDAapproved for PV but has been used off-label for many years. Ropeginterferon alfa-2b is a longeracting formulation that requires less frequent dosing than peginterferon alfa-2a. Both are administered as subcutaneous injections and typically take several months to reach full therapeutic effect.

A recent update to the NCCN Guidelines for PV recommends ropeginterferon alfa-2b as a preferred cytoreductive therapy, while HU and peginterferon alfa-2a are recommended as alternatives. The AE profile of INF-α is extensive and includes a black box warning for neuropsychiatric,autoimmune,ischemicandinfectiousdisorders.

Other AEs include injection site reactions, flu-like symptoms, headache, fatigue, nausea, diarrhea,musculoskeletalpain,dizziness,arthralgias,andcytopenias

Ruxolitinib (Jakafi; Incyte Corporation) is a JAK1/2 inhibitor that is approved for use in patients with PV who are intolerant to or refractory to HU. This drug may be especially useful in those withsymptomsofpruritusandsplenomegaly.

The phase 2 MAJIC-PV (ISRCTN61925716) study demonstrated that ruxolitinib had a superior complete response within 1 year compared to best available treatment (BAT) in patients who were resistant to or intolerant of HU: 40 (43%) patients on ruxolitinib vs 23 (26%) on BAT (OR 2.12; 90% CI 1.25-3.60, P=0.02).24 Ruxolitinib is associated with cytopenias, particularly anemia and thrombocytopenia. Its immunosuppressive effects and propensity for weight gain can make itdifficulttotolerateformanypatients.

There are new developments on the horizon for PV that have the potential to alleviate phlebotomy burden, consistently maintain HCT under 45% and improve patient QOL. Rusfertide, a hepcidin mimetic, is the furthest along in development. Rusfertide binds to an iron exporter on key cells involved in iron metabolism called ferroportin, reducing iron availability in the bone marrow that is needed for erythropoiesis.

The ongoing phase 3 VERIFY study achieved its primary endpoint, showing a significantly higher proportion of clinical responders defined as patients no longer meeting phlebotomy eligibility among those treated with rusfertide for PV (77%) compared to those who receivedplacebo(33%)duringweeks20to32(P<00001)

Givinostat (Duvyzat; Italfarmaco SpA) is a histone deacetylase inhibitor specific for JAK2 V617Fmutatedcells.Thephase3GIV-INPVtrialinvestigatinggivinostatiscurrentlyinprogress.

There are several other molecules currently in phase 2 development. Sapablursen (ISIS 702843; Ono Pharmaceutical) is a ligand-conjugated antisense molecule that targets the TMPRSS6 gene to modulate hepcidin production and subsequent regulation of iron metabolism20 Bomedemstat inhibits LDS1, an enzyme involved in increasing cell proliferation and hematopoieticdifferentiation

Divesiran is a double-stranded small interfering RNA targeting TMPRSS6 mRNA.20 Lastly, the combination of 2 frequently used cytoreductive therapies, ruxolitinib and ropeginterferon alfa2b, are being studied in a phase 2 trial in HU-resistant patients with PV; results are expected in 2028.

Pharmacistsareinauniquepositiontoimprove the care of patients with PV. They can ensure that patients are treated to guideline recommendations, ie, that all patients with PV are on low-dose aspirin and high-risk patients additionally receive cytoreductive therapy, if notcontraindicated.

Pharmacists can intervene in cases where HCT is not maintained at less than 45% and recommend the addition of therapy or dose adjustments as needed to achieve this goal. As many new PV diagnoses are missed, pharmacists can also help identify cases of untreatederythrocytosisforfurtherworkup.

Lastly, pharmacists may serve a role in educating patients and providers on the importance of recognizing and managing PV symptoms, as well as managing HCT levels to mitigatethromboticrisk.

Interferon Therapy for MPNs in Canada in 2025: Shortages,

Stopgaps, and Why BESREMi Matters for PV

Interferons have long been part of the therapeutic backbone for Philadelphia–chromosome–negative myeloproliferative neoplasms (MPNs) especially polycythemia vera (PV) because they control blood counts and, uniquely, can drive down the JAK2 V617F mutation burden over time(apotentialmarkerofdiseasemodification).Contemporaryreviewsandguidelinescontinue to position pegylated interferon-α alongside hydroxyurea as a first-line cytoreductive option in PV,particularlyforyoungerpatientsandthoseaimingtoavoidorcomeoffhydroxyurea.

TheCanadiancontext:what’sbeenused andwhat’smissing

Historically in Canada, peginterferon alfa-2a (PEGASYS) authorized for hepatitis B/C has been prescribed off-label for PV and related MPNs. Other interferon products have dwindled: interferonalfa-2b(IntronA)hasbeendiscontinuedinCanada(andabroad),leavingpeginterferon alfa-2aasthepracticalinterferonoptionformanyhematologists.

Theshortage:aTier3supplycrunch

Canada entered a Tier 3 (most serious) shortage of PEGASYS beginning October 29, 2024, with theshortagelistedasongoingthroughDecember31,2025 TheDrugShortagesCanadadatabase and Health Canada’s InfoWatch both flagged this as critical, noting real risks for MPN patients whodependoninterferontherapy eventhoughPEGASYSisnotformallyauthorizedforMPNs.

To mitigate the impact, Health Canada (March 17, 2025) permitted the exceptional, temporary importation of US-authorized BESREMi (ropeginterferon alfa-2b-njft). Crucially, Health Canada emphasizes that BESREMi is “similar but not identical” to PEGASYS; it carries different dosing, indication,andmonitoringrequirementsandshouldnotbeconsideredadirectsubstitute.Italso lacksaCanadianDINandmaynotappearinstandardpharmacysystems,requiringspecialbilling processes.

RecallsandSafetyAlerts

Professional bodies and pharmacy groups circulated practical guidance on converting appropriate patients, while also clarifying important exceptions (eg, pregnant patients and thosewithCTCLshouldgenerallyremainonPEGASYSwhenpossible)

Following Health Canada’s temporary pathway, distributor FORUS Therapeutics announced USauthorized BESREMi is available in Canada via Exceptional Importation, with technical details (e.g.,OntarioPIN09858357andQuebecRAMQcode99114432)toenablebilling/logistics.

BESREMiremainsnotyetreviewedunderHealthCanada’sstandarddrugreviewprocessin2025, butalicensing/registrationtrackwiththeoriginatorcompany(PharmaEssentia)isunderway

Whatmakesinterferon andBESREMi importantforPV?

Diseasecontrolthatcanbe“disease-modifying.”

Randomized and long-term extension studies demonstrate that ropeginterferon alfa-2b (BESREMi) achieves durable complete hematologic responses and deep molecular responses (reductionsinJAK2V617F alleleburden),withoutcomesthatstrengthenovertime.ThePROUDPV trial and CONTINUATION-PV extension (to 5–6 years) showed sustained efficacy and, in later follow-up,signalssuchasimprovedevent-freesurvivalcomparedwithcontrolarms.PMCNature Emerging analyses (and real-world/clinical experience reports) continue to link deeper JAK2 burden reductions with favourable clinical trajectories, underscoring why many clinicians view interferonastheonlycytoreductiveoptionwithrobust,prospectivemolecular-responsedatain PV.

Tolerabilityandcadence.

Ropeginterferon’s long half-life enables dosing every 2–4 weeks (after titration), which many patients find easier than weekly regimens associated with older interferons Several reviews highlight comparatively lower long-term discontinuation rates with ropeginterferon versus historicalinterferonformulationsinPVcohorts

Aviablefirst-lineoption especiallyforyoungerPVpatients

Reviews and expert algorithms place pegylated interferon-α alongside hydroxyurea as first-line cytoreduction in PV. For younger patients who may be on therapy for decades, the potential for sustainedhematologiccontrolandmolecularremissionsmakesinterferonparticularlyattractive.

PracticalimplicationsofthePEGASYSshortage Whoshouldswitch?

Canadian hematology groups and pharmacy bodies recommend considering BESREMi for appropriate patients affected by the shortage, while noting specific groups should not be switched(e.g.,pregnancyandCTCL,wherePEGASYSremainspreferred).Thedecisionbelongsto the treating hematologist, who will individualize based on prior response, comorbidities, and goals(e.g.,fertility,molecularresponse).

Dosingandmonitoringarenotinterchangeable.

Health Canada’s risk communication stresses that BESREMi has different dosing and monitoring thanPEGASYS;cliniciansaredirectedtotheUSPrescribingInformationandrelevantguidelines, andpatientsrequirecounselingonsyringeformatandhandling(BESREMiarrivesasasingle-use prefilledsyringewithEnglish-onlylabels).

Coverageandlogistics

Because BESREMi’s importation is exceptional (without a DIN), ordering and billing require special processes and may vary by province/plan. FORUS has published provincial billing identifierstofacilitatedispensing;patientsandprovidersshouldworkwithspecialtypharmacies andinsurersoncase-by-casearrangements.

HowBESREMichangesthenear-termlandscapeforCanadianPVpatients

WhileCanadaaddressesthePEGASYSshortagethroughatleastDecember2025,theavailability of BESREMi offers a credible, evidence-based interferon path for many PV patients who otherwise faced therapy disruption or suboptimal alternatives It is particularly impactful for patients aiming for long-term disease control with potential molecular remissions, for those intolerant or resistant to hydroxyurea, and for clinicians striving to reduce phlebotomy burden whileadheringtohematocritandthrombotic-risktargets.

That said, not every patient should be switched, and interferon choice in pregnancy remains a special case (PEGASYS preferred when feasible). The message from Canadian advisories is consistent: coordinate closely with your hematology team to determine suitability, dosing, and monitoring andtonavigatethetemporarylogisticsofanimportedproduct.

Bottomline

Interferon remains a cornerstone for PV in Canada, valued for durable hematologic control and theuniquepotentialtoreduceJAK2V617Falleleburdenovertime.WileyOnlineLibrary CanadaisexperiencingaTier3PEGASYSshortage(Oct2024→Dec2025),officiallyrecognizedby HealthCanada.

In response, US-authorized BESREMi is temporarily available through exceptional importation, withdistinctdosing/monitoringandspecialhandling(noDIN)

Why it matters for PV: BESREMi brings long-term, disease-modifying potential with convenient dosing and robust trial data helping preserve interferon access during the shortage and potentiallyimprovinglong-runoutcomesformanyCanadianPVpatients.

Switching from PEGASYS® to BESREMI®: What Patients Should Ask

Ask About Your Suitability

Am I a good candidate for BESREMi®?

• Are there reasons I should stay on PEGASYS®, such as pregnancy or other conditions?

• How will switching impact my treatment goals, such as controlling hematocrit or reducing my JAK2 mutation burden?

Know the Side Effects

What side effects should I expect with BESREMi®?

• How do I report side effects or concerns early?

• What should I do if I miss a dose?

Understand the Differences

How is the dose and schedule different from PEGASYS®?

• What new monitoring (labs, visits) will I need?

• Will I be able to self-inject, and how often

Learn About Logistics

Which pharmacy will dispense BESREMi®?

• How is the medication stored and handled?

• How is insurance coverage or reimbursement arranged, and who can help with paperwork?

Plan for Follow-Up

How often will we check my blood counts and liver function after the switch?

• How will we measure whether the medication is working for me?

• What are the steps if BESREMi® isn’t effective or tolerated?

Clinical Trials and Surveys

Surveys

AML - Acute Myleod Leukemia

Patients Quality of Life

PV - Polycythemia vera

Patients Quality of Life

Quality of life - Blood

Cancer Patients

Iron Deficiency Anemia

Quality of Life

If you are living with a hematological condition or care for someone who is, consider exploring clinical trials. Talk to your healthcare team, reach out to advocacy organizations and use platforms such as ClinicalTrials.gov or Canadian Cancer Trials to learn more. Your voice and experience matter—more than you know.

Clinical Trial Search - Canada

HealthCanada'sClinicalTrialsDatabase-Website

Health Canada, through its Clinical Trials Database, is providing the public with a listing of specific information relating to phase I, II, and III clinical trials in patients. The database is managed by Health Canada and provides a source of information about Canadian clinical trials involving humanpharmaceutical andbiological drugs

AccesstheClinicalTrialsDatabase

Patients can access the database to determine if a clinical trial has met the regulatory requirements. The database may also assist Canadians in finding clinical trials that might be relevanttotheirmedical condition.

The Clinical Trials Database is not a registry, and therefore, it does not contain comprehensive information about each clinical trial. To maximize the use of the database and available information, users are advised to link to external resources, including publicly available registries,toobtainfurtherinformationsuchastrial objectivesandpatienteligibility.

Note, however, that not all clinical trials are necessarily registered and thus found in these registries.

Health Canada continues to encourage sponsors to register their clinical trials in publicly accessibleregistriessuchasClinicalTrials.gov andISRCTN.

A Canadian-based registry for cancer trials is also available at Canadian Cancer Trials Additionally, the search portal provided by the World Health Organization (WHO) can be used to access a central database that contains information about trials registered in several international registries.

Health Canada is the federal regulator responsible for authorizing the importation and sale of drugs for the purpose of clinical trials This responsibility is fulfilled through the review of clinical trial applications (CTAs) for phase I, II, and III clinical trials, filed by clinical trial sponsors One of the objectives of Health Canada's review is to ascertain that subjects participating in the trial will notbeexposedtounduerisks.

Clinical Trial Search - Canada

Every year, Health Canada authorizes approximately 900 clinical trials in patients. The database lists trials that were authorized by Health Canada starting April 1, 2013. The database will be populated with information about each clinical trial after Health Canada issues its No-Objection Letter (see terminology section for a definition). Thus, following the launch, the number of clinicaltrialsavailableinthedatabaseisexpectedtobesmall,butthenumberwillincreasewith timeasthedatabaseispopulated.

InformationListedintheDatabase

The database will provide the following information on clinical trials for which a CTA has been authorized:

Protocol Number;

Protocol Title;

Drug Name;

Medical Condition; Study population; Date of No Objection Letter;

Sponsor Name; Control Number; Study Start Date; Study End Date; Trial Status

Health Canada's Clinical Trials Database serves as an essential resource for patients, healthcare providers, and researchers by offering regulatory insights into authorized clinical trials in Canada. While not a comprehensive registry, the database helps users identify trials that have met Health Canada’s safety and regulatory standards, offering a level of trust and transparency forCanadiansexploringparticipationinclinicalresearch.

By supplementing searches with additional global registries such as ClinicalTrialsgov, ISRCTN, and the Canadian Cancer Trials portal, individuals can gain a more complete understanding of trial options and eligibility As the database continues to expand over time, it reinforces Health Canada's commitment to safeguarding patient well-being while promoting access to innovation andinformeddecision-makinginclinicaltrialparticipation

Resources

Hematolgy Advocacy Groups

List of Hematology Advocacy Groups - Canada

National & Regional Hematology Advocacy Organizations

Network of Rare Blood Disorder Organizations (NRBDO)

A pan-Canadian coalition representing patients with rare blood disorders such as hemophilia, thalassemia, aplastic anemia, and von Willebrand disease. NRBDO focuses on advocacy, healthcare access, and best practices in care delivery

Canadian Hemophilia Society (CHS)

Founded in 1953, CHS supports individuals with inherited bleeding disorders through education, research, and advocacy. It operates chapters in every province

Aplastic

Anemia & Myelodysplasia Association of Canada (AAMAC)

Provides education, peer support, and research funding for Canadians living with aplastic anemia, myelodysplastic syndromes (MDS), and paroxysmal nocturnal hemoglobinuria (PNH). Offers local support meetings and webinars.

Leukemia

& Lymphoma Society of Canada (LLSC)

A national charity dedicated to curing leukemia, lymphoma, Hodgkin's disease, and myeloma. LLSC provides patient support services, education, and funds research across Canada.

Lymphoma Canada

Focuses on education, support, and advocacy for lymphoma patients. Offers virtual and in-person support groups across provinces, including Ontario.

List of Hematology Advocacy Groups - Canada

Myeloma Canada

The only national organization exclusively focused on multiple myeloma. Provides educational resources, promotes clinical research, and advocates for patient access to new therapies.

CLL Canada

A national advocacy group supporting Canadians affected by Chronic Lymphocytic Leukemia (CLL) and Small Lymphocytic Lymphoma (SLL). Offers education and works to improve treatment access

Alpha-1 Canada

Provides peer-to-peer support and disease management services for Canadians diagnosed with Alpha-1 Antitrypsin Deficiency. Engages in national and international advocacy efforts.

aHUS Canada

Advocates for individuals with atypical Hemolytic Uremic Syndrome (aHUS), focusing on education, support, and access to treatment.

Canadian Hemochromatosis Society

Founded by Marie Warder, this organization raises awareness and provides support for those affected by hereditary hemochromatosis. Offers educational materials and advocacy.

The Canadian CML Network

is a community of people living with Chronic Myeloid Leukemia. We are dedicated to providing emotional, social and educational support to people living with CML and their families.The Canadian CML Network works with patients and healthcare providers from all across Canada, and is focused on building CML patient groups nationally

Canadian MPN Network Patient Advocacy Group

A Formal Canadian network of patients and caregivers Provides a platform for sharing best practices and connecting with local support groups.

Know your Blood

A Formal Canadian organization dedicated to the Black/Caribbean community. Provides a platform for sharing and connecting for specific diseases to this community

Informal Groups (Facebook, other social media)

CLL/SLL Information & Support (Canada)

A Canadian community for individuals living with Chronic Lymphocytic Leukemia (CLL) and Small Lymphocytic Lymphoma (SLL), their families, and professionals. Members share experiences, ask questions, and offer mutual support. Facebook CML Friends of Canada

A group for Canadians living with or supporting someone with Chronic Myeloid Leukemia (CML). It serves as a space for sharing experiences, information, and support among members. Lymphoma Canada – Facebook Page

While not a group, Lymphoma Canada's Facebook page offers resources, updates, and information on virtual support groups, including monthly sessions co-hosted with Wellspring for lymphoma and CLL patients. Lymphoma Canada

List of Hematology Advocacy Groups - USA

Leukemia & Lymphoma Society (LLS)

LLS is the largest nonprofit dedicated to curing leukemia, lymphoma, Hodgkin's disease, and myeloma. It provides patient support, funds research, and advocates for policies to ensure access to quality, affordable care

National Bleeding Disorders Foundation (NBDF)

Formerly the National Hemophilia Foundation, NBDF focuses on awareness, care, and treatment of inheritable blood and bleeding disorders like hemophilia and von Willebrand disease

International Myeloma Foundation (IMF)

IMF is dedicated to improving the quality of life for myeloma patients through research, education, support, and advocacy. It offers resources for patients, caregivers, and healthcare professionals

Leukemia Research Foundation

This foundation's mission is to cure leukemia by funding research and supporting patients and families. It provides educational resources and financial assistance.

Lymphoma Research Foundation

As the nation's largest lymphoma-focused health organization, it is devoted to funding lymphoma research and providing patients with education and support services

CancerCare

CancerCare offers free, professional support services for people affected by cancer, including counselling, support groups, educational workshops, publications, and financial assistance

List of Hematology Advocacy Groups - USA

National Marrow Donor Program (NMDP)

NMDP manages the Be The Match Registry, facilitating bone marrow and umbilical cord blood transplants for patients with life-threatening blood cancers

Aplastic Anemia and MDS International Foundation (AAMDSIF)

AAMDSIF provides support and education for patients with aplastic anemia, myelodysplastic syndromes (MDS), and related bone marrow failure diseases.

MDS Foundation

The MDS Foundation offers support and resources for patients with myelodysplastic syndromes, including information on support groups and treatment options.

International Waldenstrom's Macroglobulinemia Foundation

This foundation provides support and education for patients with Waldenström macroglobulinemia, a rare type of non-Hodgkin lymphoma, and funds research for improved treatments.

Children's Cancer and Blood Foundation (CCBF)

CCBF is the first and largest charitable organization in the U.S. dedicated to supporting the care of children with cancer and blood diseases

MPN Advocacy & Education International

This organization offers educational programs, patient and caregiver conferences, and resources tailored to MPN patients. They aim to empower patients through knowledge and support.

MPN Education Foundation

The foundation provides information and support for MPN patients, including the MPN-NET online support group, which facilitates discussions among patients, caregivers, and healthcare professionals.

List of Hematology Advocacy Groups - Global

The Acute Leukemia Advocates Network (ALAN)

A global patient advocacy organization dedicated to improving the lives of people affected by acute leukemias. By connecting patients, caregivers, and advocates, ALAN promotes awareness, facilitates access to information, and supports research to ensure patient voices shape leukemia care and policy worldwide.

CML Advocates Network

An informal global network moderated by Chronic Myeloid Leukemia (CML) patients and caregivers. Provides a platform for sharing best practices and connecting with national support groups

The Global MPN Scientific Foundation (Global MPNSF)

A non-profit organization committed to improving the lives of individuals affected by myeloproliferative neoplasms (MPNs), a group of rare blood cancers. Through education, advocacy, and research support, Global MPNSF raises awareness, connects patients with specialists, and promotes advancements in treatment and care.

List of Hematology Advocacy Groups - Online

MPN-NET Online Support Group

Hosted by the MPN Education Foundation, MPN-NET is an email-based discussion group where patients and caregivers share experiences, information, and support related to MPNs.

Facebook Groups

Several Facebook groups offer informal support for MPN patients: Myeloproliferative Neoplasms (MPN) Support Group: A global community where members discuss various MPN-related topics.

Polycythemia Vera (PV) Support Group: Focused on individuals diagnosed with PV, sharing experiences and coping strategies.

Essential Thrombocythemia (ET) Support Group: A space for those with ET to connect and support each other.

Myelofibrosis (MF) Support Group: Dedicated to discussions around living with MF, treatments, and research updates.

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NCI PDQ®. Adult AML—Patient/Health Professional Versions. Updated 2024–2025. Cancer.gov+1

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NCI PDQ®. CLL—Patient Version. Updated 2024. Cancer.gov

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Goldstein BD. Benzene and hematologic cancers (IARC/NCBI Bookshelf). 2019. NCBI FDA & NCI Cancer Currents. CAR T approvals in B-ALL (tisagenlecleucel; brexucabtagene autoleucel). U.S. Food and Drug AdministrationCancer.gov

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Kiladjian JJ, Winton EF, Talpaz M, Verstovsek S Ruxolitinib for the treatment of patients with polycythemia vera. Expert Rev Hematol. 2015;8(4):391-401. doi:10.1586/17474086.2015.1045869

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Tremblay D, Yacoub A, Hoffman R. Overview of Myeloproliferative Neoplasms: History, Pathogenesis, Diagnostic Criteria, and Complications. Hematol Oncol Clin North Am. 2021;35(2):159-176. doi:10.1016/j.hoc.2020.12.001.

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Cheryl Petruk, MBA B.Mgt.

Cheryl A. Petruk is a multifaceted professional whose career spans across patient advocacy,business,andpost-secondaryeducation,showcasingherdedicationtomakinga significantimpactineachoftheseareas.

Cheryl's transition into patient advocacy was driven by a passion from her family circumstances and a deep commitment to ensuring patients' rights and access to care Cheryl has worked tirelessly to bridge the gap between the healthcare system, patients, andpharmastakeholders,ensuringthatpatients'voicesareheardandtheirneedsaremet Her work involves collaborating with Stakeholders and Patient Advocacy Organizations, lobbying for patient centricity, and providing patient support and guidance Cheryl's empatheticapproachanddedicationtoadvocacyhavemadeherarespectedfigureinthis field,admiredbypatients,healthcareprofessionals,andfellowadvocates

Cheryl also leads and is the lead faculty member at CACHEducation, Patient Advocacy Training CherylisacertifiedgreenbeltinVBHC,acertifiedTrainerinVBHC,andisaDBA Student My Blood My Health Digital Magazine –

Heal Canada and its signature initiative, My Blood My Health, collaborate with a diverse array of consultants who bring specialized expertise to support our mission of advancing patient advocacy, education, and engagement in hematological health. These consultants include healthcare professionals, patient engagement strategists, medical writers, clinical research advisors, digital health experts, legal and regulatory specialists, and communications and media consultants. Each contributes uniquely—whether it's crafting patient-centered educational content, guiding ethical and regulatory compliance for clinical outreach, developing digital tools for patient engagement, or ensuring that advocacy messaging resonates across platforms. This multidisciplinary approach ensures our programs are informed, inclusive, and impactful, amplifying the patient voice while driving meaningful change in healthcare.

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