Summary

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• Rydapt (midostaurin) is indicated for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) that is FLT3 mutation positive as detected by an FDA-approved test, in combination with standard cytarabine and daunorubicin induction and cytarabine consolidation. It is also indicated for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN), or mast cell leukemia (MCL).
• This summary is based on the review of three systematic review(s)/meta-analysis(es). ^[1-3]
• Relapse-Free Survival (RFS) and Overall Survival (OS): Tyrosine kinase inhibitor (TKI) maintenance therapy, including midostaurin and sorafenib, significantly improved RFS (pooled RR 0.48, 95% CI 0.37-0.61, p < 0.001) and OS (pooled RR 0.48, 95% CI 0.36-0.64, p < 0.001) in FLT3-ITD-mutated AML patients post allogeneic stem-cell transplantation. Similar improvements were observed in the general AML post-transplant population, with maintenance therapy improving OS (HR 0.61, 95% CI 0.47-0.80) and RFS (HR 0.51, 95% CI 0.40-0.66).
• Event-Free Survival (EFS) and Complete Remission (CR): FLT3 inhibitors improved EFS (HR 0.85, 95% CI 0.77-0.94, p = 0.002) and CR (RR 1.11, 95% CI 1.00-1.22, p = 0.05) in AML patients.
• Safety Profile: Increased risk of graft-vs.-host disease (GVHD) with sorafenib, while no significant differences in non-relapse mortality or overall grade 3/4 adverse events were observed across different TKIs in the post-transplant setting. FLT3 inhibitors demonstrated a higher relative risk of grade 3 and above vascular, dermatological, respiratory, and hepatobiliary adverse events compared to controls.