Heuser, MichaelSmith, B DouglasFiedler, WalterSekeres, Mikkael AMontesinos, PauLeber, BrianMerchant, AkilPapayannidis, CristinaPerez-Simon, Jose AHoang, Caroline JO'Brien, ThomasMa, Weidong WendyZeremski, MirjanaO'Connell, AshleighChan, GeoffreyCortes, Jorge E2023-02-092023-02-092021-03-19Heuser M, Smith BD, Fiedler W, Sekeres MA, Montesinos P, Leber B, et al. Clinical benefit of glasdegib plus low-dose cytarabine in patients with de novo and secondary acute myeloid leukemia: long-term analysis of a phase II randomized trial. Ann Hematol. 2021 May;100(5):1181-1194. doi: 10.1007/s00277-021-04465-4. Epub 2021 Mar 19. Erratum in: Ann Hematol. 2021 Jul;100(7):1917-1918.http://hdl.handle.net/10668/17369This analysis from the phase II BRIGHT AML 1003 trial reports the long-term efficacy and safety of glasdegib + low-dose cytarabine (LDAC) in patients with acute myeloid leukemia ineligible for intensive chemotherapy. The multicenter, open-label study randomized (2:1) patients to receive glasdegib + LDAC (de novo, n = 38; secondary acute myeloid leukemia, n = 40) or LDAC alone (de novo, n = 18; secondary acute myeloid leukemia, n = 20). At the time of analysis, 90% of patients had died, with the longest follow-up since randomization 36 months. The combination of glasdegib and LDAC conferred superior overall survival (OS) versus LDAC alone; hazard ratio (HR) 0.495; (95% confidence interval [CI] 0.325-0.752); p = 0.0004; median OS was 8.3 versus 4.3 months. Improvement in OS was consistent across cytogenetic risk groups. In a post-hoc subgroup analysis, a survival trend with glasdegib + LDAC was observed in patients with de novo acute myeloid leukemia (HR 0.720; 95% CI 0.395-1.312; p = 0.14; median OS 6.6 vs 4.3 months) and secondary acute myeloid leukemia (HR 0.287; 95% CI 0.151-0.548; penAttribution 4.0 Internationalhttp://creativecommons.org/licenses/by/4.0/Acute myeloid leukemiaClinical trialGlasdegibSecondary acute myeloid leukemiaAgedAged, 80 and overAntineoplastic AgentsAntineoplastic Combined Chemotherapy ProtocolsBenzimidazolesCytarabineFemaleHumansLeukemia, Myeloid, AcuteMaleMiddle AgedNeoplasms, Second PrimaryPhenylurea CompoundsSurvival AnalysisTreatment Outcomeresearch article33740113open accessLeucemia mieloide agudaPacientesSobrevidaTiempoEficaciaCitogenéticaCitarabinaDosificaciónSeguridadQuimioterapia10.1007/s00277-021-04465-41432-0584PMC8043884https://link.springer.com/content/pdf/10.1007/s00277-021-04465-4.pdfhttps://www.ncbi.nlm.nih.gov/pmc/articles/PMC8043884/pdf