Presenter:
Anhar Eldesouky, PharmD, lead author
Research Team:
Edward Pudim III, PharmD, MPH
Mark Tesell, PharmD, BCPS
Thomas C. Pomfret, PharmD, MPH, BCPS
Karen Stevens, PharmD
Caroline J. Alper, MD
Scott Bacon, PharmD
Karen Clements, ScD
Mckenzie McVeigh, PharmD, MS
Natalie Rodenberg, PharmD, BCPS
Diala Nicolas, PharmD, BCPS
Kimberly Lenz, PharmD, MBA, FAMCP
Sickle cell disease (SCD) is a group of inherited red blood cell (RBC) disorders affecting more than 100,000 individuals in the United States. Sickle cell disease is caused by a mutation in the beta-globin gene causing the production of sickle hemoglobin, resulting in the abnormal shaping of RBCs. SCD can cause blockages, anemia, and tissue damage with severe complications being strokes, acute chest syndrome, infections, and pain crises, which often may require emergency department (ED) visits and inpatient hospitalizations.
Treatment of SCD focuses on managing symptoms and preventing complications. Hydroxyurea (HU), a disease-modifying therapy, is the standard of care, while curative treatment includes bone marrow or stem cell transplants. Additional novel disease-modifying therapies (nDMTs) have become available in recent years, including crizanlizumab (Adakveo®), L-glutamine (Endari®), and voxelotor (Oxbryta®), which can be used either alone or in conjunction with HU. In 2023, two gene therapies were approved for the treatment of SCD. But there is limited real-world evidence comparing therapeutic outcomes associated with HU (with or without the use of nDMTs) in the Medicaid population.
This research compared the average number of ED visits and inpatient hospitalizations among members with SCD receiving HU with or without a nDMT, or no treatment, and to assess the impact of adherence to SCD medications on these outcomes within a state Medicaid population.
The team found that among the 1,088 participants in this evaluation, 68.9 percent either received no pharmacologic intervention or had less than 90 days of pharmacologic treatment in their claims history. Members who were adherent to treatment had fewer ED visits and inpatient hospitalizations on average when compared to those who were non-adherent to treatment across treatment groups.
The importance of initiation and adherence to SCD therapies presents an opportunity for collaboration between healthcare providers and payers to address underutilization of SCD therapies and barriers to treatment adherence.