{"entity":{"id":"paper-ratify-midostaurin-nejm-2017","kind":"paper","name":"RATIFY: midostaurin added to chemotherapy for acute myeloid leukaemia with a FLT3 mutation","aka":[],"tldr":"Adding the FLT3 inhibitor midostaurin to standard induction and consolidation chemotherapy, then as maintenance, lengthened survival in adults under 60 with FLT3-mutated acute myeloid leukaemia, the first targeted drug to do so.","summary":"Phase 3 placebo-controlled trial of 717 patients aged 18 to 59 with newly diagnosed FLT3-mutated AML (ITD or TKD) randomised to midostaurin or placebo with daunorubicin-cytarabine induction, high-dose cytarabine consolidation and a year of maintenance.\n\nMedian overall survival was 74.7 months with midostaurin against 25.6 months with placebo (hazard ratio 0.78), with benefit across FLT3 subtypes and in patients who went on to transplant.","asOf":"2026-09-17","links":[{"label":"N Engl J Med 2017","url":"https://doi.org/10.1056/NEJMoa1614359"},{"label":"PubMed","url":"https://pubmed.ncbi.nlm.nih.gov/28644114/"}],"tags":[],"related":[],"cancers":["aml-flt3"],"sections":[],"technologies":[],"targets":[],"drugs":["midostaurin"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["ratify"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":["nejm"],"dependsOn":[],"notes":[],"journal":"New England Journal of Medicine","year":2017,"doi":"10.1056/NEJMoa1614359","pmid":"28644114","authors":"Stone RM, Mandrekar SJ, Sanford BL, et al.","paperType":"rct","findings":["Median overall survival 74.7 vs 25.6 months; hazard ratio for death 0.78.","Four-year overall survival 51.4 percent vs 44.3 percent."],"whatItMeans":"FLT3 testing at diagnosis and a FLT3 inhibitor during chemotherapy became standard. Quizartinib (QuANTUM-First) is an alternative for FLT3-ITD, and the approach extended FLT3 inhibitors into maintenance and relapse.","caveats":["Patients over 60 were excluded.","The contribution of maintenance midostaurin could not be isolated."],"changedPractice":true,"participants":717},"route":"/key-papers/paper-ratify-midostaurin-nejm-2017/","neighbours":{"cancer":[{"id":"aml-flt3","kind":"cancer","name":"FLT3-mutated acute myeloid leukaemia","route":"/cancers/aml-flt3/"}],"drug":[{"id":"midostaurin","kind":"drug","name":"Midostaurin","route":"/drugs/midostaurin/"}],"trial":[{"id":"ratify","kind":"trial","name":"RATIFY (CALGB 10603)","route":"/trials/ratify/"}],"journal":[{"id":"nejm","kind":"journal","name":"New England Journal of Medicine","route":"/journals/nejm/"}]}}