Summary

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• This summary is based on the review of three randomized controlled trial(s). ^[1-3]
• Mitapivat led to significant improvements in markers of iron overload and ineffective erythropoiesis in non-regularly transfused adults with pyruvate kinase deficiency, including reductions in liver iron concentration (M/M arm: -2.0 mg Fe/g dry weight, P/M arm: -1.8 mg Fe/g dry weight by week 96).
• In regularly transfused adults, mitapivat reduced the transfusion burden by at least 33% in 37% of participants in the ACTIVATE (a specific clinical trial)-T trial.
• Hemoglobin levels increased significantly in 40% of non-regularly transfused adults treated with mitapivat, with no response in the placebo group, alongside improvements in secondary endpoints like markers of hemolysis and patient-reported outcomes.
• Common adverse events included increased alanine aminotransferase (37%), headache (37%), increased aspartate aminotransferase (19%), fatigue (19%), and nausea (19%). Two grade 3 treatment-emergent adverse events were related to mitapivat: joint swelling (4%) and increased aspartate aminotransferase (4%). No treatment-related deaths were reported.
• Grade 3 or higher adverse events occurred in 25% of patients in the mitapivat group and 13% in the placebo group. No new safety signals were identified.
• There is no population types or subgroups information available in the reviewed documents.