Lancet·· 1 day agoPickScore81
Efficacy and safety of mitapivat in patients with sickle cell disease (RISE UP): a phase 3, double-blind, global, randomised, placebo-controlled trial.
In brief
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).
Why it matters
The placebo-controlled quantification of haemoglobin response, alongside the reported lack of significant difference in pain crises, helps gauge the benefit on the primary endpoints.
Source: Lancet · doi.org