Summary

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• Oxbryta (voxelotor) is indicated for the treatment of sickle cell disease in adults and pediatric patients 4 years of age and older.
• This summary is based on the review of five systematic review(s)/meta-analysis(es). ^[1-5]
• Voxelotor significantly increased hemoglobin levels (>1 g/dl from baseline) in patients with Sickle Cell Disease (SCD), as demonstrated in the HOPE trial, with no significant impact on reducing the frequency of vaso-occlusive crises (VOC). 4. l-glutamine and crizanlizumab were effective in reducing the frequency of VOC (p=0.0216 and p=0.02, respectively), while voxelotor was more effective in increasing hemoglobin levels but not in reducing VOC frequency.
• Combination therapy results indicated a synergistic effect of voxelotor with l-glutamine, an antagonistic effect with crizanlizumab, and an additive effect between l-glutamine and crizanlizumab.
• Voxelotor was generally well tolerated in both healthy volunteers and patients with SCD, with adverse effects incidence in the phase III HOPE trial comparable between voxelotor and placebo groups.
• Pain was the most common adverse event across all three therapies (voxelotor, l-glutamine, crizanlizumab). A theoretical concern exists that voxelotor might impair oxygen delivery due to its effect on hemoglobin oxygen affinity, warranting further evaluation. One patient with SCD, severe anemia, and a history of autoantibody-mediated hemolysis did not respond to voxelotor.
• There is no population types or subgroups information available in the reviewed studies.