DeAngelo DJ, et al. – A research work was set up to assess the efficacy and safety of midostaurin in patients with advanced systemic mastocytosis (SM). In view of the data obtained, midostaurin was active and tolerable for this group of patients.

• A phase II trial of midostaurin 100 mg twice daily, administered as 28-day cycles, in 26 patients (ASM, n=3; SM-AHN, n= 17; MCL, n=6) with at least one sign of organ damage, was performed.
• The overall response rate was 69% (major/partial response: 50/19%) with clinical benefit in all advanced SM variants, during the first 12 cycles.
• 2 patients achieved a complete remission of their SM, with ongoing therapy.
• This study reported a 50% reduction in bone marrow mast cell burden and serum tryptase level in 68% and 46% of patients by midostaurin.
• For MCL patients, median overall survival for the entire cohort was 40 months, and 18.5 months.
• With antiemetics, low-grade gastrointestinal side effects were common and manageable.
• Asymptomatic hyperlipasemia (15%) and anemia (12%) were the most frequent grade 3/4 nonhematologic and hematologic toxicities.
• There were no unexpected toxicities emerged, with median follow-up of 10 years.