Practical advice, personal experiences and pertinent news for people touched by ALPHA-1
Alpha-1 Foundation National Conference Page 4
Building Friends for a Cure (BFC) Summer 2024 Highlights Page 22
As summer comes to an end, we are excited to kick-off a busy autumn. It was wonderful to see so many Alphas at the Alpha-1 Foundation National Conference in Miami and online. The theme “The Power is in You to Make a Difference” acknowledged the spirit of the Alpha-1 community and the power we all have to make a difference. (Page 4) The force driving all our success is you, the Alpha-1 community. Your passion fuels our shared mission, leaving no doubt that together we will achieve our goals.
The power is in you to participate in research and join the Alpha-1 Research Registry to be part of the cure. At the A1F National Conference we heard from industry partners with potential paradigm shifting emerging therapies as we continue to emphasize the importance of participating in clinical trials. We look forward to hearing from our past grant awardees this October at the Investigator’s Meeting as they share their findings and collaborate with Alpha-1 experts from around the world. (Page 6)
Thank you to everyone who participated in the Alpha-1 community-wide market survey. The survey comes at a very exciting and hopeful time, helping us develop a strategic plan that will have a meaningful impact on patients, families and the multitude of stakeholders we serve. Your input is invaluable as it will help us shape our collective future to achieve our mission, a cure for Alpha-1.
We extend our heartfelt gratitude to the members of the Board of Directors (BOD) that have completed their terms. Thank you to Dr. Noel “Gerry” McElvaney for his countless years to enhance our global efforts, research, detection and the lives of many Alphas. A special thank you to Faron Schonfeld for his participation on the board and leadership on the development, finance and investment committees. Excitingly, A1F welcomes two new members of the BOD, Erin Carr and Alice Turner, MBChB, PhD. Both members assume their position on the board with a strong passion and commitment to the Alpha-1 community.
We hope to see all of you this fall at events to help raise awareness and funds toward our mission. You challenged yourself this September by joining Alpha-1 Riding for a Reason and joined us in person in Cape Cod, MA for the annual Escape to the Cape Bike Trek celebrating its 40th anniversary. (Scan to register on page 29). November is Alpha-1 Awareness Month and this year’s theme “The Power to Connect” highlights the importance of connecting with your fellow Alphas through Alpha-1 support groups, Alpha-1 specialists in Clinical Resource Centers, legislators on Capitol Hill and with your local community to raise #Alpha1Awareness. (Page 16) All A1F resources are available at your fingertips on alpha1.org. Visit frequently to register for events and access educational materials and recordings to help you manage your journey.
In 2025, A1F will proudly commemorate its 30th anniversary. This milestone celebrates and honors the legacy of all who impacted the Alpha-1 community, especially the patients and families A1F serves. Over the course of three decades, and with the insight of the Alpha-1 community, A1F has designed and launched innovative programs and services to help patients along their journey; this includes a solid infrastructure to promote research and pioneer science, and the establishment of collaborative partnerships to advance the development of novel therapies.
The essence of A1F’s work has resulted in improving the quality of life for those diagnosed with Alpha-1. Please join A1F in recognizing the last 30 years of insights, innovation, investment and impact as the journey continues toward a cure for Alpha-1. We have a lot in store for next year’s celebration and can’t wait to join together as a community to commemorate this special year.
Sincerely,
Scott Santarella
President & CEO Alpha-1 Foundation
Practical advice, personal experiences and pertinent news for people touched by Alpha-1, their families and friends
VOL. 22, NO. 2 (Fall 2024)
Published by the Alpha-1 Foundation 3300 Ponce de Leon Blvd., Coral Gables, FL 33134 (877) 2 CURE A1 (228-7321)
alpha1.org
Alpha-1 Foundation Board of Directors
Executive Committee
Jon Hagstrom*, Chair
Fred Walsh*, Vice Chair
Peg Iverson*, Secretary
Kenneth A. Irvine +, Treasurer
Virginia Clark, MD, Physician-Director
Members
Mark L. Brantly, MD
Erin Carr
Jennifer Jopp*
Ann Knebel, PhD, RN
Darrell N. Kotton, MD
Alice Turner, MBChB, PhD
Catherine Vernon*
Martin R. Zamora, MD, Director Emeritus
Alpha-1 Foundation Executive Staff
Scott Santarella, President & Chief Executive Officer
Mark B. Delvaux, Chief Financial Officer
Andrew A. Wilson, MD, Scientific Director
Robert A. Sandhaus, MD, PhD, FCCP, Clinical Director
Jeanine D’Armiento, MD, PhD, Medical Liaison
Alpha-1-To-One Editorial Board
Gordon Cadwgan, PhD*;Erin Carr+; Catriona Garry*; Jon Hagstrom*; Andrew A. Wilson, MD; William J. Martin II, MD: Robert A. Sandhaus, MD, PhD, FCCP; Patricia Tew*; Bruce C. Trapnell, MD
Managing Editor
Jeanne Kushner
Contributing Editors
Gennesis Corado; Cathey Henderson; Richard Lovich; Angela McBride; Shakira Molet; Miriam O’Day; Alexis Artiles Ojeda; Randel Plant; Linda Rodriguez
ALPHA-1-TO-ONE is published by the Alpha-1 Foundation with support from its advertisers. No part may be reproduced in any form by any means without prior written permission of the Alpha-1 Foundation. The contents are not intended to provide medical advice, which should be obtained directly from a physician. The Foundation is not responsible for the accuracy of information expressed in advertisements in this publication.
Advertising: For advertising inquiries, please contact Angela McBride, Senior Director of Corporate Relations at (877) 228-7321 ext. 233, or amcbride@alpha1.org
Letters to the Editor: Please send letters to the editor to Jeanne Kushner, Senior Director of Communications and Policy, at jkushner@alpha1.org. Letters may be edited for clarity and length.
The Alpha-1 Foundation is committed to finding a cure for Alpha-1 Antitrypsin Deficiency (Alpha-1) and to improving the lives of people affected by Alpha-1 worldwide.
*Diagnosed with Alpha-1 Antitrypsin Deficiency + Diagnosed Family Member
SPOTLIGHT
Alpha-1 Foundation National Conference in Miami, Florida ........................................................................4
FRONTIERS
Alpha-1 Foundation
26th Gordon L. Snider Critical Issues
2024
ASK THE ALPHA DOC
Your Virtual Alpha-1 Questions Answered by Dr. Robert A. Sandhaus ......................................................................18
ALPHA-1 COMMUNITY
“You’re Not Alone in the Fight” Douglas Hipperling’s Alpha-1 Story............................................................................21
The 2024 Alpha-1 Foundation National Conference
The Alpha-1 Foundation (A1F) recently hosted the 2024 A1F National Conference with the theme “The Power is in You to Make a Difference,” from June 7th to 9th in Miami, FL. This year’s conference was a mix of in person and virtual experiences, drawing over 800 participants in person and virtually. Over three conference days, those in attendance learned from top Alpha-1 experts, connected with fellow Alphas, and discovered the many resources and programs A1F offers.
The event kicked off with a warm welcome from A1F President and CEO Scott Santarella, who set an inspiring tone for the weekend alongside Jon Hagstrom, Chair of the A1F Board of Directors. Scott’s opening words
“This year’s theme acknowledges the spirit of the Alpha-1 community and the power we all have to make a difference. Your passion fuels our mission and there is no doubt that together we will achieve our goals,” shared Santarella.
A favorite session from the 2024 conference, the Alpha-1 Empowerment Hour featured four panelists: Erin Carr, Education & Support, Dan Grimm, Awareness & Advocacy, Jessica Brown, Fundraising & Getting Involved, and Melissa Hale, Participate in Research. They each shared their personal Alpha-1 journeys as patients, parents, and advocates, vividly demonstrating the theme, “The Power is in You to Make a Difference.”
Their stories were touching and profoundly powerful, underscoring the incredible impact everyone can have by educating themselves and others about Alpha-1, raising awareness of Alpha-1 in their communities, advocating for Alpha-1, supporting others affected by the condition, and participating in research to find a cure. This session truly highlighted how every member of the Alpha-1
A1F Staff
community has the power to make a difference.
The Alpha Empowerment Hour was followed by the Alpha-1 Research Registry Presentation, where Dr. Jeanine D’Armiento, Alison Keaveny, and Nadine Nuchovich discussed the impact of the Alpha-1 Research Registry, with patient Douglas Hipperling highlighting his experiences with Alpha-1 research. (Read more on page 21)
Afternoon sessions included the newly diagnosed and first-time attendee track with Alpha-1 101 Lung and Liver, and an Alpha-1 physician panel. A “Miami” Dinner Reception concluded day one of the conference with a lively reception featuring Miami-inspired cuisine and entertainment, fostering community and celebration.
Day two of the A1F National Conference was all about Alpha-1 research and the hope for the future. The day kicked off with the Alpha Angels Memorial Service; a heartfelt tribute to those we have lost in the Alpha-1 community, led by Fred Walsh and Dan Coffin.
A1F’s Scientific Director Andrew Wilson, MD shared the A1F Research Agenda, followed by the Alpha-1 Research Panel where esteemed researchers, moderated by Dr. Monica Goldklang, presented the latest advancements in Alpha-1 therapies.
The day two general session wrapped up with the favorite session of the weekend: Emerging Therapies in Alpha-1. Special thank you to the Emerging Therapies Sponsors: Beam Therapeutics, BioMarin Pharmaceutical, Inhibrx, Korro Bio, Takeda, and Wave Life Sciences, who showcased their innovative approaches to treating Alpha-1 and participated in a panel Q&A with the audience.
Three afternoon breakout tracks covered diverse topics, allowing attendees to choose what was most pertinent to them. Presentations ranged from patient advocacy to everyday living with Alpha-1 and provided valuable insights and practical advice for Alphas and their families. The day concluded with the GLOW PARTY Dinner Reception, a vibrant glow-themed party, celebrating the community’s spirit and resilience with a Miami flair.
Day Three of the conference featured the Bring You Back 2025 Beach Bash Brunch: A farewell brunch with fun beach themed giveaways that excited attendees for next year’s conference in 2025, celebrating A1F’s 30th Anniversary.
Throughout the event, A1F ensured that both in-person and virtual participants had access to all presentations, which were live-streamed and later available on-demand. This hybrid approach exemplified A1F’s commitment to inclusivity and accessibility, ensuring everyone could benefit from the knowledge shared during the conference.
Special thanks to all our 2024 sponsors for giving their valuable time for this conference and throughout the
entire year to make our educational programming topnotch. Thank you to our sponsors for their commitment and dedication to the Alpha-1 Community. Platinum Sponsors: AlphaNet, CSL Behring, Grifols, Takeda and Vertex, Gold Sponsor: Accredo by Evernorth, Emerging Therapies Sponsors: Beam Therapeutics, BioMarin Pharmaceuticals, Inhibrx, Korro Bio, Takeda and Wave Life Sciences.
The 2024 A1F National Conference was a testament to the strength and unity of the Alpha-1 community, reinforcing that the power to make a difference truly lies within everyone.
Recordings from the conference can be found at alpha1.org in the video library. 1 ALPHA-1 FOUNDATION
Alpha-1 Foundation recognizes 2024 Grant Awards Recipients at ATS
Reception
On Monday, May 20th over 160 attendees joined together in San Diego, California during the annual American Thoracic Society (ATS) International Conference to recognize the 2024 Alpha-1 Grant Awards recipients and celebrate the research that has recently been funded by the Alpha-1 Foundation (A1F).
A1F continues to invest boldly in those working in the Alpha-1 research field, funding the most promising science to accelerate treatments for the underlying cause of Alpha-1 and to hopefully find a cure for every person diagnosed with the condition.
“Tonight, we celebrate an incredible milestone. Since its inception, the Alpha-1 Foundation’s investment in research around the globe has reached the $100 million mark. As the largest funding source of Alpha-1 research, we are proud to support 130 institutions around the world over three decades and we are not done yet. Thanks to the brilliance of many of you in this room tonight, the Foundation will continue moving the needle towards a cure,” announced Scott Santarella, President, and CEO of the A1F.
Members of the Grants Advisory Committee (GAC) were recognized for their outstanding commitment and dedication to A1F, and their scientific expertise
and guidance. Special recognition was given to two members who cycled off the committee, Dr. Craig Hersh and Dr. Christine H. Wendt. Dr. Hersh, from Brigham & Women’s Hospital, served on GAC for 12 years, and Dr. Wendt, from the University of Minnesota, served for 16 years. We are grateful for their expertise and the gift of their time.
“We have an incredible community and seeing all the talented and dedicated people gathered in one place reminds me of that and reinforces my optimism that we can accomplish great things for the Alpha-1 community together. A second reason, I am feeling optimistic tonight is because for the first time since this disease was discovered there are now numerous potential therapies in the pipeline,” stated Dr. Andrew Wilson, A1F Scientific Director.
Dr. Wilson announced the 2024 Grant Awards Recipients and recognized those in attendance. This year’s awards consisted of eleven research grants, four pilot and feasibility grants, and four postdoctoral research fellowship grants. A1F was challenged to double the number of grant recipients this cycle from last year, which we achieved with a total of 19 grant recipients receiving over $3 million for their meritorious projects.
In-Cycle Research Grant Recipients: (Awarded in-cycle for basic and translational research and provide funds to encourage the development of new information that contributes to the understanding of Alpha-1.)
Promoting Progenitor-Driven Liver Regeneration as an AATDAssociated Liver Disease Therapy
Diversity of SERPINA1 Mutations as Reflected in an Indian Population
Multi-omic Sex Differences in Alpha-1 Antitrypsin Deficiency Associated COPD
A Novel Strategy to Deliver Intrapulmonary AAT & Limit Lung Destruction in Models of AATD
Production and Characterization of a Biobetter Recombinant AAT-Fc Therapeutic Protein
Nicotine as a Cause of Airway Inflammation and Lung Emphysema in a Novel AATD Mouse Model
B Cell Adaptive Immune Profile in A1AT Deficiency-Associated
Valerie Gouon-Evans, PhD, Boston Medical Center
Devipriya Harinath, PhD, Columbia University
Kristen Hudock, PhD, University of Cincinnati
Jorge Lascano, MD, University of Florida
Camila Lopes-Ramos, PhD, Brigham and Women’s Hospital
Karen McDonald, PhD, University of California, Davis
Francesca Polverino, MD, PhD, Baylor College of Medicine
Emphysema
Sheikh Tamir Rashid, MD, PhD, Imperial College of Science, Technology and Medicine
Deciphering the Molecular Landscape in Alpha-1 Antitrypsin Deficiency Liver Disease
Leonard Riley, MD, University of Kansas Medical Center, Research Institute
Impact of a Multicenter Best Practice Alert to Improve AATD Testing and Detection
Florian Rosenberger, PhD, Max Planck Institute of Biochemistry
Alpha-1 Hepatocyte Dynamics: A Single-Cell and Spatial Proteomics Study
Konstantinos
University College London
Cellular Effects of A1AT Aggregation with Single-Cell and Crosslinking Proteomics
Pilot and Feasibility Grant Recipients: (The objective of the Pilot and Feasibility grants are to provide funds to encourage the development and testing of new hypothesis and/or new methods in research areas relevant to Alpha-1.)
Suzanne Roche, MB, BCh, BAO, Royal College of Surgeons in Ireland
Genetic Discrimination – Tackling a Growing Issue in Alpha-1 Antitrypsin Deficiency
Postdoctoral Research Fellowship Grants:
Vera Khodzhaeva, PhD, Cambridge Institute for Medical Research- University of Cambridge
FGF21 and Cellular Homeostasis in Alpha-1 Antitrypsin Deficiency
MD, PhD, Boston University
Regeneration of Lung Epithelial Stem Cell Compartments in a Novel Z-AAT Mouse Model
Huiliang Wang, PhD, University of Texas at Austin
Genetic Engineered Exosomes for Efficient DNA Delivery for Alpha-1 Antitrypsin Deficiency
Rhiannon Werder, PhD, Murdoch Children’s Research Institute
Investigating the Cellular and Molecular Mechanisms of Respiratory Infections in AATD
Mark Murphy, PhD, Royal College of Surgeons in Ireland
A Detailed Study of Lung Immune Cell Outcomes in AATD
Valentina Schiano, PhD, Fondazione Telethon ETS
Targeting Inflammation as Therapeutic Approach for AATD
A1F congratulates the Grant Awards recipients. The mission cannot be achieved without the dedication of the scientific community, and we are so grateful for the leadership of Dr. Wilson and his commitment to engage new, young investigators in the Alpha-1 research community to ensure that we continue to benefit from new ideas and high-quality research for years to come.
Thank you to the event sponsors: AlphaNet, CSL Behring, Grifols and Takeda 1 ALPHA-1 FOUNDATION
Hirofumi Kiyokawa,
Thalassinos, PhD,
ARE YOU ON A JOURNEY WITH ALPHA-1 LIVER DISEASE?
The Redwood Study is looking for adults 18 to 75 years of age who have a confirmed or suspected diagnosis of Alpha-1 Liver Disease with PiZZ genotype. There is currently no approved treatment available for Alpha-1 Liver Disease. The investigational study drug aims to reduce the production of the abnormal Z-AAT protein and its buildup in the liver. To learn more and see if you may qualify, talk to your doctor, visit a1f.org/redwood-study-prescreening, or scan the QR code today.
26th Gordon L. Snider Critical Issues Workshop: Pragmatic Solutions to Clinical Trial Endpoints for Lung Disease in AATD
and AAT Variant
Nomenclature
Two topics important to Alpha-1 Antitrypsin Deficiency (Alpha-1) research were the focus of the 26th Gordon L. Snider (GLS) Critical Issues Workshop convened in Miami, Florida in March: (1) how best to name the many variants of the gene that causes the condition, and (2) refining better strategies for optimizing clinical trials of new treatments. Speakers and attendees from the research, pharmaceutical, biotech, regulatory, and patient communities engaged in the 2-day conference.
What’s in a Name?
In Alpha-1, individuals with two copies of the most common mutation associated with the condition, designated as the Z allele, have a low level of the alpha-1 antitrypsin (AAT) in the blood because the misfolded Z version of AAT cannot be secreted from the liver in normal amounts, causing lung and/or liver disease. In clinical practice, 96 percent of individuals with diseases associated with Alpha-1 have the ZZ genotype, and 4 percent have combinations of the “rare” or “null” Z or S mutations (also called alleles or variants).
More than 500 variants in the AAT gene (called SERPINA1) have been described in the literature. Outside of the few common and well-known variants, the names assigned the less common ones are unconventional and variable. This unsystematic and nonstandard naming system (nomenclature) has resulted in miscommunication, spurred duplication of efforts, and stymied therapeutic discovery for this rare disorder. Some identical variants even have multiple names depending on where they were found.
Importantly, these variants differ in their effects, and because of the diversity and novelty of especially rare variants, the clinical significance of many is largely unknown. Participants discussed lessons learned from the naming and classification of the gene that causes cystic fibrosis, assessed the current landscape for SERPINA1 nomenclature, and considered options for a simplified, rational approach to classifying and naming variants in the AAT gene so that research can be streamlined and advanced.
Faster, Better Clinical Trials
A second workshop topic focused on how the scientific community might optimize outcomes in clinical trials of Alpha-1 therapeutics in ways that can meet the regulatory requirements for efficacy and safety. Alpha-1 lung disease is a rare condition, affecting roughly 10,000 to 15,000 individuals in the United States, which makes
it more difficult to identify patients, recruit them to clinical trials, develop reliable outcomes measures for trials, and reach sufficient statistical power to assess dosing, safety, and efficacy of new drugs.
The clinical trials challenges in Alpha-1 are well documented. Large databases of cases suggest that more than half of Alphas have severe or very severe lung disease and are therefore not currently eligible for most clinical trials. The small number of remaining eligible participants is not sufficient to enroll planned trials in the pipeline, particularly if studies require 2 to 3 years of complex data to demonstrate efficacy. As an alternative, pragmatic trial design would allow the best biomarker of Alpha-1, AAT level, to serve as an outcome measure.
Because augmentation with AAT is the only available therapy for lung disease and has been shown to be effective, more clinical studies are needed not only to improve the dosing, timing, and use of augmentation therapy, but also to study what biomarkers other than serum AAT level will inform treatment. Other markers, such as patient-reported outcomes, exacerbations, proinflammatory molecules, lung density, and survival have been the focus of several studies over many decades and were discussed as alternative or companion markers of Alpha-1 that could be tracked in clinical trials. Participants also said that inclusion and exclusion criteria for trials should be revisited to accelerate the generation of data to improve patient care with the lowest possible burden on patients. The workshop left participants with much to consider for developing trials that meet regulatory requirements and advance research in Alpha-1 lung disease.
Discover the ZEMAIRA difference...
...available in 1-, 4-, and 5-gram vials
The first and only Alpha-1 therapy to offer 1-, 4-, and 5-gram vials to help streamline preparation and offer greater simplicity for people with Alpha1 antitrypsin deficiency (AATD).
IMPORTANT SAFETY INFORMATION
ZEMAIRA®, Alpha1-Proteinase Inhibitor (Human), is indicated to raise the plasma level of alpha1-proteinase inhibitor (A1-PI) in patients with A1-PI deficiency and related emphysema. The effect of this raised level on the frequency of pulmonary exacerbations and the progression of emphysema have not been established in clinical trials.
ZEMAIRA may not be suitable for everyone; for example, people with known hypersensitivity to components used to make ZEMAIRA, those with a history of anaphylaxis or severe systemic response to A1-PI products, and those with certain IgA deficiencies. If you think any of these may apply to you, ask your doctor.
Early signs of hypersensitivity reactions to ZEMAIRA include hives, rash, tightness of the chest, unusual breathing difficulty, wheezing, and feeling faint. Immediately discontinue use and consult with physician if such symptoms occur.
In clinical studies, the following adverse reactions were reported in at least 5% of subjects receiving ZEMAIRA: headache, sinusitis, upper respiratory infection, bronchitis, fatigue, increased cough, fever, injection-site bleeding, nasal symptoms, sore throat, and swelled blood vessels.
Because ZEMAIRA is made from human blood, the risk of transmitting infectious agents, including viruses and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent and its variant (vCJD), cannot be completely eliminated.
Please see brief summary of prescribing information on following page and full prescribing information for ZEMAIRA available at ZEMAIRA.com.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov.medwatch, or call 1-800-FDA-1088.
You can also report side effects to CSL Behring’s Pharmacovigilance Department at 1-866-915-6958.
ZEMAIRA®, Alpha1-Proteinase Inhibitor (Human) lyophilized powder for reconstitution for intravenous use
Initial U.S. Approval: 2003
BRIEF SUMMARY OF PRESCRIBING INFORMATION
These highlights do not include all the information needed to use ZEMAIRA safely and effectively. See full prescribing information for ZEMAIRA.
-----------------------------------INDICATIONS AND USAGE----------------------------------
• ZEMAIRA is an alpha1-proteinase inhibitor (A1-PI) indicated for chronic augmentation and maintenance therapy in adults with A1-PI deficiency and clinical evidence of emphysema (1).
• The effect of augmentation therapy with ZEMAIRA or any A1-PI product on pulmonary exacerbations and on the progression of emphysema in A1-PI deficiency has not been demonstrated in randomized, controlled clinical studies (1).
• ZEMAIRA is not indicated as therapy for lung disease patients in whom severe A1-PI deficiency has not been established (1).
-------------------------------DOSAGE AND ADMINISTRATION------------------------------
For intravenous use after reconstitution only (2).
• The recommended weekly dose of ZEMAIRA is 60 mg/kg body weight. Dose ranging studies using efficacy endpoints have not been performed with ZEMAIRA or any A1-PI product (2).
• Administer through a suitable 5 micron infusion filter (not supplied) at room temperature within 3 hours after reconstitution (2.2).
• Do not mix with other medicinal products. Administer through a separate dedicated infusion line (2.2).
• Administer at a rate of approximately 0.08 mL/kg/min as determined by the response and comfort of the patient (2.2).
• Monitor closely the infusion rate and the patient’s clinical state, including vital signs, throughout the infusion. Slow or stop the infusion if adverse reactions occur. If symptoms subside promptly, the infusion may be resumed at a lower rate that is comfortable for the patient (2.2).
--------------------------------DOSAGE FORMS AND STRENGTHS---------------------------
• History of anaphylaxis or severe systemic reactions to ZEMAIRA or A1-PI protein (4).
• Immunoglobulin A (IgA)-deficient patients with antibodies against IgA, due to the risk of severe hypersensitivity (4). -------------------------------WARNINGS
AND PRECAUTIONS-------------------------------
• Observe any signs of hypersensitivity such as tachycardia, hypotension, confusion, syncope, oxygen consumption decrease, and pharyngeal edema when administering ZEMAIRA to patients with known hypersensitivity to an A1-PI product (5.1).
• Patients with selective or severe IgA deficiency can develop antibodies to IgA and, therefore, have a greater risk of developing potentially severe hypersensitivity and anaphylactic reactions. If anaphylactic or severe anaphylactoid reactions occur, discontinue the infusion immediately (5.2).
• Because ZEMAIRA is made from human blood, it may carry a risk of transmitting infectious agents, e.g., viruses, the variant Creutzfeldt-Jakob disease (vCJD) agent and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent (5.3). -------------------------------------ADVERSE REACTIONS--------------------------------------
• Serious adverse reactions reported following administration of ZEMAIRA in prelicensure clinical trials included one event each in separate subjects of bronchitis and dyspnea, and one event each in a single subject of chest pain, cerebral ischemia and convulsion.
• The most common adverse reactions occurring in at least 5% of subjects receiving ZEMAIRA in all pre-licensure clinical trials were headache, sinusitis, upper respiratory infection, bronchitis, asthenia, cough increased, fever, injection site hemorrhage, rhinitis, sore throat, and vasodilation (6).
To report SUSPECTED ADVERSE REACTIONS, contact CSL Behring Pharmacovigilance at 1-866-915-6958 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Based on September 2022 revision
ZEMAIRA is supplied in a single-dose vial containing approximately 1000 mg, 4000 mg, or 5000 mg of functionally active A1-PI as a white to off-white lyophilized powder for reconstitution with 20 mL, 76 mL, or 95 mL of Sterile Water for Injection, USP. The amount of functional A1-PI is printed on the vial label and carton (3). ---------------------------------------CONTRAINDICATIONS -----------------------------------
2024 Alpha-1 Educational Scholarships
Each year the Alpha-1 Foundation (A1F) awards Alpha-1 Educational Scholarships to Alphas and family members seeking to further their higher education. This year, ten scholarships were awarded to deserving individuals from across the country. Congratulations to these outstanding students.
Elizabeth Hipperling and Grace Johnson were awarded the James (Jim) Quill Memorial Scholarships. These scholarships honor the memory of Jim Quill, who served as an AlphaNet Coordinator, and then Manager of AlphaNet. Jim also served on the A1F Board of Directors. He consistently solved problems for the entire Alpha-1 community. Not only was Jim the consummate mentor to all AlphaNet Coordinators and staff, but he was always available to listen and offer counsel to any Alpha.
Elizabeth is a sophomore at the University of New Haven majoring in Forensic Science and is from Sound Beach, New York.
“Although Alpha-1 is a terrible and scary disorder, it made me who I am today. I believe that I would not be on the path I am today without my father’s diagnosis. Alpha-1 made me fall in love with science and what I want my life to be. It also made me appreciate life, teaching me the importance of being grateful for what I have.”
Grace is a freshman at Florida State University majoring in Marketing and is from St. Johns, Florida.
“Through conversations with my parents, I now realize Alpha-1 is passed on through families. In my family alone, we have tested 26 and discovered 19 of the 26 are carriers. My brother and I are both ZZ Alphas. Genetic testing has advanced so much that 23andMe now tests for Alpha-1. Because of this, I have found out 6 friends are carriers. I will continue to advocate for the awareness of Alpha-1 and contribute to the Alpha-1 Foundation’s mission to find a cure and improve the lives of those affected by this genetic condition.”
Stella Guthmann was awarded the Robert (Bob) J. Haggerty Memorial Scholarship. This scholarship honors the memory of Bob Haggerty, who was the face and voice of the Alpha-1 community for a decade. He served as a leader for many years, as well as Master of Ceremonies at many events.
Stella is a junior at the University of Nebraska majoring in Graphic Design and is from Omaha, Nebraska.
“I don’t see my diseases as barriers, but as an opportunity for growth and development. I have learned so much from each condition, especially Alpha-1 and Cystic Fibrosis. Both have gone hand in hand with everything in my life. Instead of letting them control me, I use them to see the world a little differently and to strengthen myself rather than let them tear me down.”
2024 Alpha-1 Educational Scholarships
Kelly Grant was awarded the John W. Walsh III (Jack) Memorial Scholarship. This scholarship honors the memory of Jack Walsh, affectionately known as “Coach.” Having celebrated 94 birthdays, Coach was an icon in the Alpha-1 community. Father to John, Fred, Judy, and Sue, he perpetuated the importance of education and family within the community.
Kelly is a sophomore at Portland Community College majoring in Physics and is from Klamath Falls, Oregon.
“I have learned that it is essential to educate the public and the medical community to understand Alpha-1 as a starting point, but just as importantly that those affected by it need creative and extensive support to lead their best possible lives. The more that I learn about Alpha-1, the more excited I am for the many possibilities ahead in improved treatment, understanding, caring, and outcomes for Alphas such as my wife.”
Eliana Ainsa was awarded the E. Lou Glenn Memorial Scholarship. This scholarship honors the memory of Lou Glann, a dedicated caregiver of her daughter who was diagnosed with Alpha-1. A force in the Alpha-1 community, she served on various committees, always supported programs for Alphas, and represented the voice of caregivers.
Eliana is a freshman at Brigham Young University-Idaho majoring in Exercise Kinesiology and is from Bluffdale, Utah.
“Alpha-1 has always been a part of my life. I was humbled by how incredibly hard my parents worked to afford my dad’s medication for his Alpha-1, never once abandoning me and always making time for me.”
Tara Mulholland was awarded the Terry L. Young Memorial Scholarship. This scholarship honors Terry Young, co-founder of AlphaNet and the first AlphaNet Coordinator. A pillar in the Alpha-1 community, he perpetuated the AlphaNet slogan, “Alphas serving Alphas.”
Tara is a sophomore at Pennsylvania State University, University Park majoring in Supply Chain Management and is from Gaithersburg, Maryland.
“During my second week of college, I lost the person who I loved the most in this entire universe, my dad. Even in the midst of my grief, I find comfort in the words of my father’s childhood friend ‘the last time I was talking to your dad, he said he was grateful for all the fun he had in his life and that it is now your turn to do the same.”
The 2025 Alpha-1 Educational Scholarships applications will open in January. Scholarships are available to Alphas and their immediate family members and can be used for study at an approved institution for post-high school education and career change/returning adult students. The deadline to submit a completed application with all attachments is April 1, 2025. To learn more, visit alpha1.org/alpha-1-educational-scholarships
This year’s Peter Smith Scholarships have been awarded to four deserving awardees, Hannah Cathrae, Tiffany Nutter, Joseph Reidy, and Gabrielle Salzman. Peter Smith wrote, edited, and published Alpha-1 News from 1989 to 1992. Before his early death due to Alpha-1, he was spreading the word to over 1,200 households. His efforts encouraged, enlightened, and inspired those with Alpha-1 to reach out to one another and to learn. The intent of the Peter Smith Scholarship is to continue this tradition by helping those with Alpha-1 and their families to learn and achieve.
Hannah is a sophomore at Idaho State University majoring in Nursing and is from Boise, Idaho.
“Alpha-1 has been much like a teacher in my life, shaping my journey in the medical field and creating my dedication to helping others. I know the fight for Alpha-1 isn’t over, but I hope to bring awareness to this condition to help bring us closer to a cure.”
Tiffany is a junior at Georgia Southern University majoring in Psychiatric Mental Health Nurse Practitioner and is from Richmond Hill, Georgia.
“The Alpha journey has not only shaped our family’s narrative but has also ignited a passion within me to contribute meaningfully to the mental health landscape. My goal as a psychiatric mental health nurse practitioner is to extend a compassionate hand to individuals facing similar battles, fostering a community where individuals can find solace and support.”
Joseph is a junior at Rochester Institute of Technology majoring in Cybersecurity and is from New Jersey, Waldwick.
“Alpha-1 has made me a stronger person and has helped me face any challenge that I come across head on. From staying resilient in negative situations to being able to work on a team, I’ve learned that people can do anything, as long as they do it together.”
Gabrielle is a freshman at the University of Alabama majoring in Forensic Science and Laboratory Sciences and is from Vance, Alabama.
“Alpha-1 not only has affected my life, but it is my life. Growing up attending Alpha-1 conferences, I discovered that I could change the outcome of this condition, which ignited my passion for S.T.E.M. research.”
THE P
C NNECT
2024 ALPHA-1 AWARENESS MONTH
November is Alpha-1 Awareness Month, a 30-day initiative that encourages the Alpha-1 community to share stories and resources and raise funds to support the search for a cure.
This year’s theme is “The Power to Connect,” highlighting the importance of connecting with your fellow Alphas through Alpha-1 Support Groups, Alpha-1 specialists in Clinical Resource Centers, legislators on Capitol Hill, and your local community to raise #Alpha1Awareness.
Whether you post on social media, fundraise for the 2024 Alpha-1 Foundation Virtual Walk, or sign up for the Alpha-1 Research Registry, you have the power to connect.
To learn more about events happening during Alpha-1 Awareness Month, visit: alpha1.org/alpha-1-awareness-month
WER TO NNECT
2024 ALPHA-1 FOUNDATION VIRTUAL WALK
Help us raise funds and awareness for Alpha-1!
You have “The Power to Connect” to help find a cure.
Connect with friends and family to boost your fundraising, whether at home, in your neighborhood, or at your local park. Use the “Good Move” app to make it easy and fun!
Start a team or join a team in your area. You have the power to connect to the Alpha-1 community!
Registration is $25 and includes the official 2024 A1F Virtual Walk T-shirt. For more information, contact Irene Calderon at icalderon@alpha1.org.
Ask the Alpha Doc: Your Alpha-1 Questions Answered by Dr.
Robert Sandhaus
During the Alpha-1 Foundation (A1F) National Conference in Miami, Florida online participants submitted questions on the virtual environment (VE). While conference speakers answered many of the questions during the event, below are frequently asked questions that were received.
Q: What vaccines do you recommend an Alpha get?
A: Vaccines are preventative measures to boost your immunity against certain infections, including both viruses and bacteria. For Alphas, vaccines can be helpful at preventing infections that can worsen your lung and liver disease. These include vaccines against types of viral hepatitis (Hepatitis A and Hepatitis B), vaccines that prevent types of bacterial pneumonias (pneumococcal vaccines), and vaccines against respiratory viruses (influenza, RSV, and COVID vaccines). There are vaccines that everyone should receive regardless of their Alpha-1 status including polio, measles, mumps, rubella, tetanus, pertussis, diphtheria, etc.
The timing and dosing of vaccines can be complex and is based on age, risk, and previous vaccinations. The best resource to learn about vaccines is the CDC: https://www.cdc.gov/ vaccines/imz-schedules/index.html
Q: How does a CPAP machine help an Alpha and when should you consider a BiPAP?
A: CPAP (Continuous Positive Airway Pressure) machines are used to treat obstructive sleep apnea (breathing that intermittently stops during sleep due to the tongue and throat relaxing to the point that air can’t move in and out of the lungs while steeping). CPAP applies an adjustable amount of positive pressure to the air entering your mouth and/or nose (depending on the type of mask you are given) that keeps the upper airways open during sleep. BiPAP (Bilevel Positive Airway Pressure) provides different pressure during inspiration and expiration to improve comfort compared to CPAP and some units can even help breath for you if you’ve stopped breathing. Many modern CPAP machines can be adjusted to act as BiPAP if needed.
Q: What level is considered “severe” Alpha-1?
A: Measurement of alpha-1 antitrypsin (AAT) blood levels is a simple and inexpensive lab test. However, the results can be confusing. This is primarily because different labs and different
countries use different units to report their results. In the U.S. most labs report AAT levels in units of either mg/dL (milligrams per deciliter) or mmoles (micro-moles per liter). These two units can be converted between one another by either dividing or multiplying by 5.2. Generally, any level below 57 mg/dL or 11 mmol is considered severely deficient.
Q: How does vaping affect the lungs of an Alpha?
A: The risks of vaping have been under intense investigation. The results this far have been affected by the fact that there is no standardization or regulation of the contents of the vaping cartridges. When analyzed, many contained dangerous ingredients such as formaldehyde which has been shown to be a cancer-causing chemical. Even for an individual product, the formulations can change from batch to batch. Therefore, there is no easy way to assess the actual risks for vaping in the general population or in Alphas. Many feel vaping can be safer than smoking. Unfortunately, many individuals who use vaping as a smoking cessation aid have become more addicted to nicotine and even add cigarette smoking to their vaping.
Q: As a ZZ lung affected Alpha, should I have my liver checked? What specific liver tests should I request from my physician?
A: Anyone with Alpha-1 who develops symptoms related to liver disease should be evaluated with measurement of liver enzymes in blood (AST, ALT, GGT, alkaline phosphatase, bilirubin) and imaging studies (liver ultrasound, Fibroscan, MRI). Guidelines recommend that all Alphas over 50 should have these tests even if they have no symptoms. Most healthcare providers who see Alpha-1 patients generally do these tests on an annual basis.
At each Alpha-1 Foundation Education Day, you have the opportunity to attend virtually and have your Alpha-1 related questions answered in realtime by Alpha-1 experts. To learn more about upcoming events, please see page 31.
PROLASTIN®-C LIQUID
Alpha1-Proteinase Inhibitor (Human)
HIGHLIGHTS OF PRESCRIBING INFORMATION
These highlights do not include all the information needed to use PROLASTIN-C LIQUID safely and effectively. See full prescribing information for PROLASTIN-C LIQUID.
PROLASTIN-C LIQUID
(Alpha1-Proteinase Inhibitor [Human])
Solution for Intravenous Injection
Initial U.S. Approval: 1987
---------------------INDICATIONS AND USAGE --------------------
PROLASTIN®-C LIQUID is an Alpha1-Proteinase Inhibitor (Human) (Alpha1-PI) indicated for chronic augmentation and maintenance therapy in adults with clinical evidence of emphysema due to severe hereditary deficiency of Alpha1-PI (alpha1-antitrypsin deficiency).
Limitations of Use:
• The effect of augmentation therapy with any Alpha1-PI, including PROLASTIN-C LIQUID, on pulmonary exacerbations and on the progression of emphysema in Alpha1-PI deficiency has not been conclusively demonstrated in randomized, controlled clinical trials.
• Clinical data demonstrating the long-term effects of chronic augmentation or maintenance therapy with PROLASTIN-C LIQUID are not available.
• PROLASTIN-C LIQUID is not indicated as therapy for lung disease in patients in whom severe Alpha1-PI deficiency has not been established.
-----------------DOSAGE AND ADMINISTRATION----------------
For intravenous use only.
• Dose: 60 mg/kg body weight intravenously once per week.
• Dose ranging studies using efficacy endpoints have not been performed with any Alpha1-PI product, including PROLASTIN-C LIQUID.
• Administration: 0.08 mL/kg/min as determined by patient response and comfort.
---------------DOSAGE FORMS AND STRENGTHS --------------
For injection: approximately 500 mg (10 mL), 1,000 mg (20 mL) and 4,000 mg (80 mL) of a solution for injection in single-dose vials.
• Immunoglobulin A (IgA) deficient patients with antibodies against IgA.
• History of anaphylaxis or other severe systemic reaction to Alpha1-PI.
-----------------WARNINGS AND PRECAUTIONS ----------------
• Severe hypersensitivity and anaphylactic reactions may occur in IgA deficient patients with antibodies against IgA. Discontinue administration of the product and initiate appropriate emergency treatment if hypersensitivity reactions occur.
• Because PROLASTIN-C LIQUID is made from human plasma, it may carry a risk of transmitting infectious agents, e.g., viruses, the variant Creutzfeldt-Jakob disease (vCJD) agent, and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent.
The most common adverse reactions during PROLASTIN-C LIQUID clinical trials in > 5% of subjects were diarrhea and fatigue, each of which occurred in 2 subjects (6%).
To report SUSPECTED ADVERSE REACTIONS, contact Grifols Therapeutics LLC at 1-800-520-2807 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Grifols Therapeutics LLC
Research Triangle Park, NC 27709 USA
U.S. License No. 1871 Revised: 5/2020 3062338/3062339
“You’re Not Alone in the Fight”: Douglas
Hipperling’s Alpha-1 Story
Douglas Hipperling, a ZZ Alpha, was diagnosed with Alpha-1 Antitrypsin Deficiency (Alpha-1) at the age of twenty-five. A lifelong New York State resident, Douglas is a Civilian Technician for the Suffolk County Police Department known for his automotive and marine repair expertise. He is a proud father of two adult daughters and has one grandchild. Douglas’s interests include all things mechanical, especially antique items and vintage Lionel trains. His story is of strength, resilience, and an unwavering commitment to living life to its fullest.
Douglas’s journey with Alpha-1 began with a painful loss: his maternal grandfather passed away at the young age of 41 due to complications from the condition. Aware of his genetic risk, Douglas got tested, only to face the grim reality of his genetic diagnosis. Lacking information and support, he feared the worst and resolved to make the most of his life until he reached 41, considering every day beyond that a gift.
Ironically, at 41, Douglas faced the terrifying reality of his underlying condition. After managing to avoid catching the highly contagious virus for a year, he was hospitalized with severe back pain and tested positive for COVID-19. The hospital’s lack of treatment options for Alpha-1 patients left him feeling hopeless. Desperate for answers, Douglas turned to the internet, where he discovered the Alpha-1 Foundation (A1F).
A life-changing phone call to the Patient Information Line with Cathey Henderson, Director of Patient Services at A1F marked a turning point. Cathey connected Douglas with Dr. Monica Goldklang at Columbia University Irving Medical Center in New York City. Together with the expertise of Dr. Jeanine D’Armiento, Dr. Goldklang provided him with the personalized care he desperately needed. Their dedication and compassion renewed his hope.
Through A1F, Douglas found medical support and a community. He has participated in clinical trials through the A1F Research Registry, which makes him feel part of something bigger than himself. These experiences highlight the importance of research and the collective effort to combat Alpha-1.
Douglas’s journey is a testament to the power of community and the impact of dedicated medical professionals. A1F’s network of support and resources for patients has transformed his outlook on life. Douglas now knows that Alpha-1 is not a death sentence but a manageable condition with the right care and support.
Douglas encourages others to get involved, raise awareness, and support the Alpha-1 community. “When we stand as one, we will dominate,” he says. His heartfelt gratitude goes to the Alpha-1 Foundation, Dr. Goldklang, Dr. D’Armiento, and everyone who has supported him in his journey. 1 ALPHA-1 FOUNDATION
Building Friends for a Cure (BFC)
Summer 2024 Highlights
The first annual Cones for a Cure was held on Saturday, June 29th at the Chalco Hills Recreation Area in Omaha, Nebraska. Hosted by Alpha moms Nora Kemmerer and Chelsea Boring, the dynamic duo brought together over 100 participants including members of the Big Red Alphas Support Group and the local Alpha-1 community. The warm summer day included face painting, raffles, and of course, ice cream. Together with the community, they raised more than $2,500 for Alpha-1 research and related programs.
“It was wonderful to see Cones for a Cure come to life this weekend. Alphas, along with family and friends, came together to enjoy delicious ice cream, great fellowship, and, most importantly, to support a cause close to all our hearts. The cure is so near, and I’m so grateful to have had the opportunity to do whatever I can to bring people together to support that mission,” exclaimed Nora Kemmerer.
“The Big Red Alphas Support Group first ever Cones for a Cure was a great event allowing Alphas to share their stories with community members, raise funds for research, and increase awareness. Thank you to the Alpha-1 Foundation, community sponsors and volunteers for their help and support to pull off this event. It was a huge success and we had so much fun,” stated Chelsea Boring.
A special thank you to event sponsors and supporters: Turnkey Solutions for underwriting directional signage, Heartland Hearing Center for the raffle basket and donated gift cards, Ima the Clown for donating face painting services, Hyvee for gift cards and all the generous donors, volunteers, and sponsors. Thank you to Peg Iverson, Alpha-1 Foundation (A1F) Board of Director, for joining the BFC and supporting the event.
Thank you to Nora, Chelsea, and their families for supporting the BFC Program. The BFC Program is centered on facilitating and empowering the Alpha-1 community to increase awareness and raise funds to support A1F’s mission by providing the tools for fundraising success all while building and maintaining relationships in the community. Nora and Chelsea attended a BFC training earlier this year in Boston, Massachusetts. Participants of the training joined the Celtic Connection event committee to prepare for the largest annual BFC event in the country. It was a perfect way to get hands-on training leading into the workshop, including silent auction set-up and closeout, décor set-up, and registration.
A1F is here to help! We have online platforms to make peer-to-peer fundraising easy. Donation pages are easy to build and personalize for your event and share with family and friends. Your success is our success!
If you are interested in creating an ice cream social fundraiser or learning more about the BFC program, please contact Irene Calderon at icalderon@alpha1.org.
Batter Up… It’s Time to Strike Out Alpha-1!
Blake Aaronson is a 12-year-old Alpha and baseball star that helped Strike Out Alpha-1 this summer. Together with his team, The Sacred Heart, he prepared for their big tournament this July in America’s hometown of baseball, Cooperstown, New York. Blake and his teammates wore jerseys with the purple A1F logo prominently displayed, raising awareness on the field with each homerun. In their last year of little league, this was great way to end the season together.
Parents Kelly and Kyle Aaronson are so excited for Blake and his team and so proud that he is helping to raise awareness and funds for Alpha-1. They are no strangers to fundraising, having hosted four successful golf tournaments raising over $60,000 for Alpha-1 over the years. Blake was seen on the A1F social media outlets sharing his videos as he got ready for the tournament. He started promoting his campaign on Memorial Day weekend and continued throughout July to the National Championship.
The Alpha-1 Foundation thanks the Aaronson Family for their continued dedication and commitment to raising awareness for Alpha-1 during each inning, hit and home run. Congratulations to the Sacred Heart Baseball team for making it to the playoffs. They finished number 24 of 100 teams - A feat never accomplished in the team’s history.
Thank you to the continued support of our Building Friends for a Cure (BFC) Sponsors: AlphaNet, CSL Behring, Grifols, and Takeda. 1 ALPHA-1 FOUNDATION
Natasha Durant, Alpha Mom & Support Group Leader Raising Awareness
Natasha Durant, Alpha Mom and Oregon Support Group Co-Leader began planning for her local 4th of July parade early this year!
In February, she reached out to the Alpha-1 Foundation (A1F) excited to participate in the annual parade with an Alpha-1 Antitrypsin Deficiency (Alpha-1) themed float. Friends and family joined together in their purple A1F Virtual Walk t-shirts to raise awareness in Oregon, passing out Alpha-1 Fast Facts flyers on the parade route.
In her own words, “I know being involved with the Alpha-1 Foundation is the best thing I can do for my children’s futures, and for the Alpha-1 community. I came up with a great idea! What better way to raise awareness for Alpha-1 than by broadcasting it to a captive audience? We asked our local community for donations of lumber, decorations, candy and entry fees to help build this Alpha-1 awareness 4th of July float and they delivered graciously.
We were excited to ride along in the parade on our purple Alpha-1 themed float, throwing candy, waving, and handing out Alpha-1 Fast Facts Flyers with the hope of reaching new patients, family members, doctors, and/or medical staff. Maybe we could educate someone on Alpha-1 and get someone detected and diagnosed early. Several people who did know about Alpha-1 thanked us for being there and sharing our cause.
Awareness elevates the desire to find a cure. You never know who might be inspired if you don’t share the information. By gaining awareness, we hope to gain supporters and donors who can help us in the search for a cure.
The Alpha-1 community is worth the fight. My children are worth the fight. I implore each of you to advocate for your fellow Alphas. Raise awareness, participate in research, and fundraise with Building Friends for a Cure (BFC) to help move the mission forward.
The power is in you to make a difference! Together, we can be even stronger.” 1 ALPHA-1 FOUNDATION
For patients with emphysema caused by severe Alpha-1 antitrypsin deficiency. Neither are their needs.
GLASSIA® offers the most infusion setting options; with your doctor, you can decide which works best for you.
*If self-infusion is deemed appropriate, ensure that you receive detailed instructions and adequate training on how to infuse at home or other appropriate setting and have demonstrated the ability to independently administer GLASSIA.
Scan the code or visit glassialiquid.com to learn about which infusion setting option may be right for you.
When you’re prescribed GLASSIA, Takeda Patient Support is here for you.
What is GLASSIA?
GLASSIA is a medicine containing human Alpha₁-Proteinase
Inhibitor (Alpha₁-PI) that is used to treat adults with lung disease (emphysema) because of severe Alpha₁-antitrypsin (Alpha₁) deficiency. GLASSIA is not meant to be used as a therapy for lung disease other than severe Alpha₁ deficiency. Effects of GLASSIA on worsening lung function and emphysema progression have not been proven in clinical trials. Long-term effects of Alpha₁ replacement and maintenance therapy have not been studied.
IMPORTANT SAFETY INFORMATION
What is the most important information I need to know about GLASSIA?
• GLASSIA can cause severe allergic reactions including hives, swelling in the mouth or throat, itching, tightness in the chest, trouble breathing, wheezing, faintness or low blood pressure
• If you will be taking GLASSIA outside a healthcare setting, ask your healthcare provider (HCP) about an epinephrine pen and/or other supportive care for certain severe allergic reactions.
Who should not use GLASSIA?
Do not use GLASSIA if you:
• Have immunoglobulin A (IgA) deficiency with antibodies to IgA
• Have a severe allergic reaction to human Alpha₁-PI products.
IMPORTANT SAFETY INFORMATION (continued)
What are the possible or reasonably likely side effects of GLASSIA? If any of the following problems occur contact your healthcare provider (HCP) or call emergency services right away:
• Worsening or flare-up of your chronic obstructive pulmonary disease (COPD)
• Hives, swelling in the mouth or throat, itching, chest tightness, trouble breathing, wheezing, fainting or dizziness. These could be signs of a serious allergic reaction.
The most common side effects that may occur are headache and upper respiratory tract infections.
Other possible side effects of GLASSIA include:
• Cough
• Sinus infection
• Chest discomfort
• Dizziness
• Increased liver enzymes
• Shortness of breath
• Nausea
• Fatigue
These are not all the possible side effects. Tell your HCP about any side effect that bothers you or that does not go away. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
Please see the Important Facts About GLASSIA on the next page.
All rights reserved. TAKEDA®, the TAKEDA Logo®, and the TAKEDA Patient Support LogoTM are trademarks or registered trademarks of Takeda Pharmaceutical Company Limited. GLASSIA® is a registered trademark of Kamada Ltd., and used under license. US-GLA-0456v1.0 04/24
Glassia
[Alpha,-Proteinase Inhibitor (Human)]
Patient Information
GLASSIA
(Alpha1-Proteinase Inhibitor (Human)) Injection, For Intravenous Use
The following summarizes important information about GLASSIA (pronounced glass-see-ă). Please read it carefully before using this medicine. This information does not take the place of talking with your healthcare professional, and it does not include all of the important information about GLASSIA. If you have any questions after reading this, ask your healthcare professional.
What is the most important information that I should know about GLASSIA?
Severe allergic reactions can occur with GLASSIA. Your doctor will inform you about signs of allergic reactions which include hives, swelling in the mouth or throat, itching, tightness in the chest, trouble breathing, wheezing, faintness, low blood pressure, or serious allergic reaction. If you have any of these reactions, discontinue use of the product and contact your physician and/or seek immediate emergency care, depending on the severity of the reaction. If you or your caregiver will be administering GLASSIA outside a healthcare setting, ask your doctor about an epinephrine pen and/or other supportive care for certain severe allergic reactions.
Ask your doctor to make sure you receive training on how and when to use any prescribed supportive care medicine and keep it close at hand when administering GLASSIA.
What is GLASSIA?
GLASSIA is a liquid medicine containing human Alpha1Proteinase Inhibitor (Alpha1-PI) also known as alpha1-
main purpose of infusing GLASSIA is to increase the levels of the AAT protein in your blood and lungs. AAT protein protects the lung tissue by blocking certain enzymecaused damage. Such damage can lead to severe lung disease, such as emphysema.
Limitations of Use:
• GLASSIA or any other Alpha1-PI product on worsening pulmonary function and progression of emphysema have not been proven in clinical trials.
• maintenance therapy with GLASSIA have not been studied.
• GLASSIA is not intended as a therapy in individuals with lung disease other than severe Alpha1
Who should not take GLASSIA?
You should not use GLASSIA if you:
• to IgA
• Have had a severe allergic reaction to human Alpha1-PI products
How should I take GLASSIA?
• GLASSIA is given directly into the bloodstream.
• You can get GLASSIA at your healthcare professional’s by a healthcare professional from a limited network of specialty pharmacy providers.
• Your healthcare professional will decide if self-infusion in your home is right for you. You should be trained on how to do infusions by your healthcare professional.
What should I tell my healthcare professional before I start using GLASSIA?
Before starting GLASSIA, tell your healthcare professional if you:
•
• Have a history of severe allergic reactions to Alpha1-PI products.
of GLASSIA?
• (COPD) in which your breathing gets worse than usual.
• Call your healthcare professional or go to your emergency department right away if you get: Hives, swelling in the mouth or throat, itching, chest tightness, trouble breathing, wheezing, fainting or dizziness. These could be signs of a serious allergic reaction.
• upper respiratory tract infections. Other possible side chest discomfort, dizziness, increased liver enzymes, shortness of breath, nausea, and fatigue.
You can ask your healthcare professional for information that is provided to healthcare professionals. Talk to your
you or that don’t go away.
How do I store GLASSIA?
Store GLASSIA refrigerated or at room temperature.
• You can store GLASSIA in the refrigerator (36°F to 46°F [2°C to 8°C]). Do not freeze.
• You can store GLASSIA at room temperature (up to 77°F [25°C]) for up to one month. You must use GLASSIA within one month once you remove it from the refrigerator Do not re-refrigerate GLASSIA once the product has been stored at room temperature.
• Keep the GLASSIA vial in the box until you are ready to administer the product.
Check the expiration date on the carton and vial label. Do not use GLASSIA after the expiration date.
Manufactured by: Takeda Pharmaceuticals U.S.A., Inc. Lexington, MA 02421 USA
U.S. License No. 1898
Revised: September 2023
GLA378 US-GLA-0459v1.0 03/24
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SUPPORT GROUP MEETINGS
September 14, 2024Big Red Alphas
September 17, 2024Arizona and New Mexico
September 21, 2024
September 28, 2024
October 5, 2024
October 6, 2024
October 10, 2024
October 12, 2024
Hampton Roads VA/Alpha-1 Connections
Iowa Alpha-1 SG
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Omaha, NE
VIRTUAL
Chesapeake, VA
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Lewiston, ME
Oceanside, CA Point Lookout, MO Attleboro, MA VIRTUAL
Middleton, ID
Reno-Tahoe/Sacramento Alphas/San Francisco Bay Area Alphas VIRTUAL, NV
Alpha-1 Tampa Illiana Alphas
Clearwater, FL Terre Haute, IN
October 16, 2024 Hoosier Alphas Indianapolis, IN
October 26, 2024 Alpha Opportunities Phoenixville, PA
November 9, 2024Oregon Alphas Happy Valley, OR
ALPHA-1 EDUCATION EVENTS
November 1-2, 2024Birmingham A1F Education Day Birmingham, AL
For more information about Alpha-1 Education Days, please visit www.alpha1.org
UPCOMING EVENTS
September 2024
Alpha-1 Riding for a Reason
September 27-19, 2024 Escape to the Cape Bike Trek
October 5, 2024
Nationwide
Cape Cod, MA
Alpha-1 500 Charity Race VIRTUAL
October 5, 2024 Gulf Coast Bike Trek for Alpha-1 Clearwater, FL
November 2024
Alpha-1 Awareness Month & Virtual Walks
(Please visit our website to find a walk near you)
Nationwide
November 16, 2024Utah Rocks Alpha-1 Spanish Fork, UT
For more information about Alpha-1 Support Group Meetings and A1F Education Days, please visit www.alpha1.org
