RESEARCH presented at the 66th American Society of Hematology Annual Meeting highlights the significant underuse of disease-modifying therapies for sickle cell disease, despite their established benefits in reducing pain and preventing organ damage.
Researchers analysed electronic health records from 22,793 patients with sickle cell disease across 14 USA. sites using the ASH Research Collaborative Sickle Cell Disease Data Hub. The study spanned from 2015–2023 and included patients of all genotypes. Disease-modifying therapy use was defined as treatment with hydroxyurea, voxelotor, or L-glutamine for at least 90 days within a year, or six or more transfusions in a year. The study aimed to assess prescribing trends and identify potential disparities in therapy utilisation.
Only 24% of patients received at least one disease-modifying therapy, and just 3% were prescribed two or more therapies in combination. Among those with hemoglobin SS genotype, the most severe form of the disease, 35% received treatment. Patients who were prescribed disease-modifying therapies were generally younger (22.6 vs 24.6 years), had higher rates of emergency department visits (2.7 vs 1.4 per year), and had lower haemoglobin levels (8.8 vs 9.9 g/dL), indicating that therapy use was concentrated in those with more severe disease. Geographic disparities were also observed, with utilisation rates highest in the Northeast (33%) and lowest in the Southwest (15.4%). Between 2015–2023, overall therapy use rose modestly from 7.2% to 19.8%, primarily driven by increased hydroxyurea prescriptions (5.5% to 16.5%), while newer therapies saw minimal uptake.
These findings suggest that a significant proportion of patients with sickle cell disease are not receiving therapies that could improve their health outcomes. Further research is needed to identify barriers to prescribing and ensure broader access to effective treatments.
Ada Enesco, EMJ
Reference
Niss O et al. Disease-modifying therapies remain underused in SCD despite safety and efficacy. Abstract 2312. American Society of Hematology (ASH) Annual Meeting, 6-9 December, 2025.
