ArticleThe journal of pediatric pharmacology and therapeutics : JPPT : the official journal of PPAG2026
Trends and Outcomes of Voxelotor Treatment in Pediatric Patients With Sickle Cell Disease: A Retrospective Study.
Article in The journal of pediatric pharmacology and therapeutics : JPPT : the official journal of PPAG, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
objectiveSickle cell disease (SCD) is characterized by hemoglobin S polymerization, leading to anemia, pain, and organ damage. Voxelotor is a therapy for SCD that was withdrawn from worldwide markets in September 2024. We evaluated trends in voxelotor use and its impact on pediatric SCD patients before withdrawal.
methodsA retrospective study was conducted using patient records from 4 Children's Healthcare of Atlanta (CHOA) hospitals. Data on patient demographics, length of time on voxelotor, clinical markers, hospital usage, side effects, and discontinuation reasons were analyzed.
resultsTwo hundred forty-five patients were taking voxelotor between 2019 and 2024 outside of clinical trials. Of these, 188 (77%) patients were on voxelotor for at least 6 consecutive months, with an average of 25.0 months on treatment. The main reason for discontinuation was due to a side effect (47%) for patients on treatment for less than 6 months and market withdrawal (69%) for those taking 6 months or more. On average, patients had a significant increase in mean hemoglobin (0.5 g/dL, p < 0.001), a decrease in hemolysis markers, and a mean decrease in annualized emergency department visits. At the time of market withdrawal, 123 patients were actively taking voxelotor.
conclusionsOverall, patients had a positive hematologic and clinical response to voxelotor, with the majority remaining on treatment for at least 6 consecutive months. Despite market withdrawal due to safety and efficacy concerns, a subset of patients derived clinical benefits from voxelotor, highlighting the need for continued development of effective therapeutics for SCD.
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