Original Title: Midostaurina asociada a quimioterapia estándar en leucemia mieloide aguda con mutación FLT3

Authors' objectives: To identify studies related to the beneficial and harmful effects of midostaurin and its relevant comparators for the treatment of adult patients with newly diagnosed AML who are fit for intensive chemotherapy.

Authors' results and conclusions: Based on the results of this assessment, midostaurin in combination with standard induction and consolidation chemotherapy is considered to be more effective than standard induction and consolidation chemotherapy alone. More uncertainty is related to the beneficial effects of midostaurin used in continuation therapy because of patient disposition in the trials assessed, leading to fewer patients receiving continuation therapy. Based on indirect comparison, there was insufficient evidence to determine whether midostaurin treatment was more beneficial than high-dose daunorubicin (90 mg/m2) used during induction in terms of OS. Serious limitations apply to this comparison. Patients over 60 years of age have not yet been studied in an RCT setting and the effect size of midostaurin on OS is unknown in this older population. However, it is the suitability of patients for intensive chemotherapy, rather than their age, which is the limiting factor to midostaurin use. The safety profile of treatment with midostaurin in combination with standard induction and consolidation chemotherapy is considered to be comparable to standard induction and consolidation chemotherapy. However, grade 3–4 exfoliative dermatitis and device-related infections occurred more frequently in patients receiving midostaurin. Furthermore, QTc prolongation has been observed in patients receiving midostaurin. Deaths during the study treatment and 30-day follow-up periods occurred more frequently in patients over 60 years of age compared with those who were younger.

Authors' identified further research: Further research is required on the effects of midostaurin in the older population. Health-related quality of life and disease-specific quality of life should be studied, because this evidence is currently lacking.

Project Status: Completed

Year Published: 2018

URL for published report: https://www.aetsa.org/publicacion/midostaurina-asociada-a-quimioterapia-estandar-en-leucemia-mieloide-aguda-con-mutacion-flt3/

Requestor: Consejería de Salud. Junta de Andalucía

English language abstract: An English language summary is available

Publication Type: Other

Country: Spain

Organisation Name: Andalusian Health Technology Assessment Area

Contact Address: Area de Evaluacion de Tecnologias Sanitarias Sanitarias de Andalucia (AETSA) Avda. Innovación, s/n Edificio Arena 1. Sevilla (Spain) Tel. +34 955 006 309

Contact Name: aetsa.csalud@juntadeandalucia.es

Contact Email: aetsa.csalud@juntadeandalucia.es

Copyright: <p>Andalusian Agency for Health Technology Assessment (AETSA)</p>