Summary

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• Midostaurin (Rydapt) is used for treating adults with newly identified acute myeloid leukemia (AML) that is FLT3 mutation positive, as well as for aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematological neoplasm (SM-AHN), or mast cell leukemia.
• Three studies were analyzed to gather information about this medication.
• In comparison to other tyrosine kinase inhibitors, midostaurin has demonstrated significant efficacy in lowering relapse rates by 65% and enhancing overall survival rates among AML patients, particularly those with FLT3 mutations.
• Although effective, there have been significant instances of grade 3 or higher adverse events associated with midostaurin; nonetheless, the advantages may surpass these risks since the incidence of serious adverse events was relatively low.
• Relative to other therapies such as tyrosine kinase inhibitors and hypomethylating agents used in maintenance therapy post-transplantation for AML, midostaurin's safety profile was found to be comparable in terms of grade 3/4 infections and graft versus host disease.
• Research is ongoing, particularly concerning older AML patients, indicating a need for more comprehensive data on how different demographics may react to FLT3 inhibitors like midostaurin.