In a study published at the 2023 American Society of Hematology Annual Meeting and Exposition, researchers found that the targeted therapy bezuclastinib may be a safe and effective treatment in reducing markers of burden of disease and symptom improvement in those with nonadvanced systemic mastocytosis. The phase 2 SUMMIT trial included 20 patients who were given either 100 mg or 200 mg of bezuclastinib or placebo. All patients had nonadvanced systemic mastocytosis and moderate-to-severe symptoms, and 75% of the patients had KIT D816V mutations. It was found that those who were treated with bezuclastinib had a 50% reduction in disease markers. Following 12 weeks of therapy, 63% of patients saw a decrease in symptoms. Zero percent of patients who received placebo saw significant improvement in overall symptoms, but in patients who transitioned from placebo to bezuclastinib, 67% achieved symptom improvement following 4 weeks. Researchers reported that adverse events were generally mild and reversible including change in hair color, nausea, and peripheral edema.
Reference: The ASCO Post Staff. Bezuclastinib May Reduce Symptoms in Patients With Nonadvanced Systemic Mastocytosis. The ASCO Post. Updated December 15, 2023. Accessed January 12, 2024. https://ascopost.com/news/december-2023/bezuclastinib-may-reduce-symptoms-in-patients-with-nonadvanced-systemic-mastocytosis/
