Midostaurin (Rydapt) in all diseases

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Acute myeloid leukemia

Source: Midostaurin (Rydapt) maintenance therapy for acute myeloid leukemia (AML)

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Regimen

Study Evidence Efficacy
Stone et al. 2017 (RATIFY) Non-randomized portion of RCT CR rate: 59% after induction

Preceding treatment

Chemotherapy

28-day cycle for up to 13 cycles (1 year)

References

  1. RATIFY: Stone RM, Mandrekar SJ, Sanford BL, Laumann K, Geyer S, Bloomfield CD, Thiede C, Prior TW, Döhner K, Marcucci G, Lo-Coco F, Klisovic RB, Wei A, Sierra J, Sanz MA, Brandwein JM, de Witte T, Niederwieser D, Appelbaum FR, Medeiros BC, Tallman MS, Krauter J, Schlenk RF, Ganser A, Serve H, Ehninger G, Amadori S, Larson RA, Döhner H. Midostaurin plus chemotherapy for acute myeloid leukemia with a FLT3 mutation. N Engl J Med. 2017 Aug 3;377(5):454-464. Epub 2017 Jun 23. link to original article link to PMC article contains verified protocol PubMed

Systemic mastocytosis

Source: Midostaurin (Rydapt) for aggressive systemic mastocytosis

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Regimen

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.

Chemotherapy

28-day cycles

References

  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