HbS polymerization inhibitors remain a valuable therapeutic option for adult patients with SCD with limited therapeutic alternatives. 2. The market withdrawal highlights the urgent need for continued research, regulatory support, and access to safe and effective treatments. On September 25, 2024, Pfizer announced the voluntary withdrawal of voxelotor due to an imbalance in vaso-occlusive crises (VOCs) and fatal events. Medical records of 31 sickle cell disease (SCD) patients (October 2021 to September 2024) (age (median) 33 years, range 21 to 55; homozygous SCD (HbS/S) 83.9% (26/31), 48 week prior VOC rate 1.8) were analyzed. At week 24, 60% of the patients (N= 12/20) met the primary EP (increase in hemoglobin concentration by ≥1 g/dL). Sustained reduction in hemolytic markers was evident in all patients (total serum bilirubin p=0.027, N= 19; absolute reticulocyte count p=0.137, N= 14; lactate dehydrogenase p=0.268, N= 20). At week 24 the mean VOC rate had decreased from 0.9 to 0.5 (p=0.111), at week 48, from 1.8 to 1.0 (p=0.168). Treatment-related adverse events (TEAEs) were observed in 48.4% of the patients; most were self-limited and grade 1 to 2. The observed results are consistent with those reported in the HOPE Trial, supporting the therapeutic efficacy of voxelotor in adult SCD patients. However, a high rate of TEASs was observed, underscoring the need to carefully balance clinical benefits with safety considerations, especially in newly approved medications.
Poppenborg et al. (Wed,) studied this question.
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