Joint Topic: Enhancing the way we manage sickle cell disease ... Finally (202-2)

3:30 – 4:30 pm Thursday, October 28

Sickle cell disease (SCD) describes a group of inherited red blood cell disorders characterized by hemolytic anemia, frequent painful crises, infection, organ damage, and a shortened lifespan (Centers for Disease Control and Prevention [CDC], 2020).

SCD impacts 100,000 Americans; one in 365 African American babies and one in 16,300 Hispanic American babies are diagnosed with SCD at birth (CDC, 2020). Although the disease has been reviewed in the medical literature for almost 100 years, there are few proven treatment options for those with SCD. To date, the only curative option for SCD is a stem cell transplant, and until 2016, there was only one FDA-approved medication for symptom alleviation (Farooq et al., 2020). Now, in 2021, there are four FDA-approved medications, increasing hope for both patients and their caregivers. For many years, the mainstay of treatment for SCD has been hydroxyurea, which is approved for treatment of all types of SCD in people age 9 months and older but has proven most effective for treatment of HgbSS disease and HgbSBeta0 thalassemia. This option has the potential to improve some quality of life, but these patients deserve more. Despite treatment, many children with SCD will experience hospital admissions for frequent pain crises and infection, forcing them to miss out on school or other important life events and decreasing their overall quality of life. In the past 5 years, the FDA has approved three new treatment options, Endari (L-glutamine), Adakveo (crizanlizumab), and Oxbryta (voxeletor). These medications each have different mechanisms of action and are approved for patients of different ages. In order to ensure maximum benefit, it is essential for hematology providers to understand their use, educate those for whom they are suitable about their use, and secure access to these therapies.