Avapritinib effectively reduces indolent systemic mastocytosis severity: NEJM
Laboratory researcher, concept photo (stock image).
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A new study by Jason Gotlib and team showed that in individuals with indolent systemic mastocytosis (ISM), avapritinib was more effective than a placebo at lowering uncontrolled symptoms and mast-cell load. The findings of this study were published in NEJM Evidence.
The KIT D816V mutation causes the clonal mast-cell illness known as indolent systemic mastocytosis. In order to compare the effectiveness and safety of avapritinib vs placebo in patients with ISM receiving the best supportive care, this research was carried out.
Avapritinib 25 mg once a day (n=141) or placebo (n=71) were given out randomly to patients with moderate to severe ISM (total symptom score [TSS] of 28; scores range from 0 to 110, with higher values indicating more severe symptoms). The 14-day average of the patient-reported severity of 11 symptoms served as the main endpoint for measuring mean change in TSS. Reduced levels of serum tryptase, blood KIT D816V variant allele fraction, TSS (50% and 30%), bone marrow mast cells (50%), and quality-of-life indicators were among the secondary end objectives.
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