# Efficacy and safety of midostaurin in advanced systemic mastocytosis

Source: https://onco.cc/key-papers/paper-gotlib-midostaurin-advanced-systemic-mastocytosis-nejm-2016/  
OnCo record `paper-gotlib-midostaurin-advanced-systemic-mastocytosis-nejm-2016` (Key paper). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

The multi-kinase inhibitor midostaurin shrank the mast cell burden and reversed organ damage in six in ten patients with advanced systemic mastocytosis, becoming the first approved targeted drug for the disease.

## Summary

Open-label phase 2 study of 116 patients with advanced systemic mastocytosis (aggressive systemic mastocytosis, systemic mastocytosis with an associated haematological neoplasm, or mast cell leukaemia) treated with midostaurin 100 mg twice daily; 89 were evaluable for response.

The overall response rate was 60 percent with major responses in 45 percent, median overall survival 28.7 months and median progression-free survival 14.1 months; responses occurred regardless of KIT D816V status. Nausea, vomiting and cytopenias were the main toxicities. The FDA and EMA approved midostaurin for advanced systemic mastocytosis in 2017.

## Fields

- Kind: Key paper
- Last checked: 2026-09-18
- Journal: New England Journal of Medicine
- Year: 2016
- DOI: 10.1056/NEJMoa1513098
- Authors: Gotlib J, Kluin-Nelemans HC, George TI, et al.
- Findings: Overall response 60 percent, major response 45 percent.; Median overall survival 28.7 months; median progression-free survival 14.1 months.
- What it means: Midostaurin was the first effective targeted therapy for advanced systemic mastocytosis and remains an option, particularly where avapritinib is unsuitable or unavailable.
- Caveats: Single-arm study with response criteria that predate the current consensus.; Gastrointestinal toxicity limits tolerance in many patients.

## Sources

- N Engl J Med 2016: https://doi.org/10.1056/NEJMoa1513098
- PubMed: https://pubmed.ncbi.nlm.nih.gov/27355533/

## Connected records

- cancers: [Advanced systemic mastocytosis (aggressive SM, SM with an associated haematological neoplasm, mast cell leukaemia)](https://onco.cc/cancers/advanced-systemic-mastocytosis/)
- drugs: [Midostaurin](https://onco.cc/drugs/midostaurin/)
- journals: [New England Journal of Medicine](https://onco.cc/journals/nejm/)

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