Grace Kalmus, Carly Nesbitt, Jason Payne, Beatrice Gee, Amy Tang
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引用次数: 0
Abstract
Objective: Sickle 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 Voxelotor is a therapy for SCD that was withdrawn from worldwide markets in September 2024.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.
Methods: A 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.
Results: Two 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.
Conclusions: Overall, 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.
期刊介绍：
The Journal of Pediatric Pharmacology and Therapeutics is the official journal of the Pediatric Pharmacy Advocacy Group. JPPT is a peer-reviewed multi disciplinary journal that is devoted to promoting the safe and effective use of medications in infants and children. To this end, the journal publishes practical information for all practitioners who provide care to pediatric patients. Each issue includes review articles, original clinical investigations, case reports, editorials, and other information relevant to pediatric medication therapy. The Journal focuses all work on issues related to the practice of pediatric pharmacology and therapeutics. The scope of content includes pharmacotherapy, extemporaneous compounding, dosing, methods of medication administration, medication error prevention, and legislative issues. The Journal will contain original research, review articles, short subjects, case reports, clinical investigations, editorials, and news from such organizations as the Pediatric Pharmacy Advocacy Group, the FDA, the American Academy of Pediatrics, the American Society of Health-System Pharmacists, and so on.
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