Mitapivat boosts haemoglobin but misses on pain crises in sickle cell disease phase 3
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The phase 3 RISE UP trial, a global, double-blind, randomised, placebo-controlled study published in The Lancet, tested mitapivat 100 mg twice daily in patients with sickle cell disease. Among 207 patients randomised 2:1 to mitapivat or placebo, haemoglobin response occurred in 56 of 138 patients versus 2 of 69 on placebo, an adjusted difference of 38% (95% CI 29–47; two-sided p<0·0001). The drug did not, however, significantly reduce pain crises, the central clinical burden of the disease. The results position mitapivat as an oral agent that improves haemoglobin, while its failure on pain outcomes leaves its ultimate place in sickle cell care unresolved.
Written by a model from the reports · updated 3 hours ago
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- LancetPickEfficacy and safety of mitapivat in patients with sickle cell disease (RISE UP): a phase 3, double-blind, global, randomised, placebo-controlled trial.
The phase 3 RISE UP trial showed that mitapivat 100 mg twice daily increased haemoglobin response in patients with sickle cell disease but did not significantly reduce pain crises. 207 patients were randomised 2:1 to mitapivat or placebo; haemoglobin response was 41% (56/138) versus 3% (2/69), an adjusted between-group difference of 38% (95% CI 29-47).
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