Section 2 | SCD Evidence-Based Care: Therapies, Disease Prevention, Screening, Pain Management Consistent access to high-quality care profoundly affects outcomes for people living with SCD. For example, detection of affected individuals at birth and interventions, such as penicillin prophylaxis, vaccines, aggressive response to fever and administration of broad spectrum antibiotics (Brousseau et al., 2010; National Heart Lung and Blood Institute, 2002), blood transfusion protocols, introduction of medications to control condition symptoms, and Transcranial Doppler (TCD) screening (Section on Hematology/Oncology Committee on Genetics, 2002) have contributed to dramatic improvements in quality of life and life expectancy. However, some of these treatments require specialized clinical settings and not all patients have equal access to these types of care. Additionally, according to the Office of Minority Health, only approximately 1 in 4 people living with SCD receive the standard of care outlined in current National Lung Blood and Hemoglobin Institute EvidenceBased Management of Sickle Cell Disease (SCD) Expert Panel Report (Office of Minority Health, 2020), which provides standard primary guidelines for improving care.
Therapies The pathophysiology of SCD is well understood and development of medication has occurred over the last several years. The collective goal of available medications is two-fold: first, to improve quality of life for patients by reducing the incidence of common SCD-related complications, such as pain crises and acute chest syndrome; and, second, to reduce the associated emergency department visits and hospitalizations, which will lower overall costs of care (Wang et al., 2013). Below are medications that have been developed. Medication Hydroxyurea (HU, aka Siklos, Addmedica, Droxia) HU was approved by the U.S. Food and Drug Administration (FDA) for adults with SCD in 1998, but not for children until 2017. HU is a significant SCD therapy option. In SCD patients, it has been effective in reducing the frequency of pain crises and need for blood Sickle Cell Disease Treatment Demonstration Regional Collaboratives Program
transfusions in children 2 years and older (National Heart Lung and Blood Institute, 2014). In 2012, the Pediatric Hydroxyurea Phase 3 Clinical Trial (BABY HUG), registered with the National Institutes of Health (Jackson et al., 2020), was a Phase 3 multicenter, randomized, double-blind, placebocontrolled clinical trial of HU in infants (beginning at 9-18 months of age) who were living with sickle cell anemia. This study showed that HU was not associated with an increased risk of bacteremia or serious infection and was generally well tolerated by very young children living with sickle cell anemia. The findings of this study were recently reconfirmed by a 2019 study looking at children aged 5-12 months (Schuchard et al., 2019). However, despite the strong NHLBI recommendations, uptake has been inconsistent and below recommended levels. One recent study showed the proportion of SCD visits that included new or continued HU prescriptions increased from less than or equal to 8 percent before 2009 to just 33 percent in 2015 to 2017 (Su et al., 2019). In a recent review completed by Centers for Medicare and Medicaid Services (Center for Medicaid and CHIP Services Division of Quality and Health Outcomes, 2020), among CMS pediatric recipients living with SCD (≥21months, ≤ 20 years), 63 percent had no days of HU use in 2017, 21 percent had 1-180 days, and 16 percent had 181-365 days. Among CMS adult recipients (≥21 years, ≤75 years), 65 percent had no days of HU use in 2017, 25 percent had 1-180 days, and 10 percent had 181-365 days. Possible Factors for Low National HU Rates • Some providers do not understand or doubt the efficacy of treatment, warranting further education. Studies have shown providers, especially those treating adults, cited this as a reason for not prescribing HU (Brandow et al., 2010; Imegi, 2016; Lanzkron et al., 2008; Zumberg et al., 2005). In one survey conducted with community-based hematologist/ oncologists in two southeastern states (Zumberg et al., 2005), 4% of respondents indicated that doubts about the efficacy of HU were ‘‘very important’’ in their decision not to prescribe HU, while 36% rated this variable as ‘‘important.” • Provider belief that patient adherence to medications may be low (Brandow & Panepinto, 2010) • Provider concern about unsubstantiated side effects (e.g., risk of cancer) (Brandow & Panepinto, 2010; Imegi, 2016) • Lack of patient confidence in HU and need for improving shared decision-making between patient/family/caregiver and provider. Some
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