Midostaurin (Rydapt) for aggressive systemic mastocytosis

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Regimen in use at


FDA-recommended dose
Study Evidence Efficacy
Gotlib et al. 2016 (CPKC412D2201) Phase II (RT) ORR: 60% (95% CI 49 to 70)
Chandesris et al. 2016 (CEREMAST) Phase II (RT)

CPKC412D2201 included untreated and previously untreated patients.


28-day cycles


  1. CPKC412D2201: Gotlib J, Kluin-Nelemans HC, George TI, Akin C, Sotlar K, Hermine O, Awan FT, Hexner E, Mauro MJ, Sternberg DW, Villeneuve M, Huntsman Labed A, Stanek EJ, Hartmann K, Horny HP, Valent P, Reiter A. Efficacy and safety of midostaurin in advanced systemic mastocytosis. N Engl J Med. 2016 Jun 30;374(26):2530-41. link to original article contains verified protocol link to supplementary appendix PubMed Phase II Midostaurin in Aggressive Systemic Mastocytosis and Mast Cell Leukemia (NCT00233454) at ClinicalTrials.gov
  2. CEREMAST: Chandesris MO, Damaj G, Canioni D, Brouzes C, Lhermitte L, Hanssens K, Frenzel L, Cherquaoui Z, Durieu I, Durupt S, Gyan E, Beyne-Rauzy O, Launay D, Faure C, Hamidou M, Besnard S, Diouf M, Schiffmann A, Niault M, Jeandel PY, Ranta D, Gressin R, Chantepie S, Barete S, Dubreuil P, Bourget P, Lortholary O, Hermine O; CEREMAST Study Group. Midostaurin in advanced systemic mastocytosis. N Engl J Med. 2016 Jun 30;374(26):2605-7. link to original article PubMed