biostudies-literatureUnknown13(4)Janssen JTreatment results of AML in elderly patients are unsatisfactory. We hypothesized that addition of tosedostat, an aminopeptidase inhibitor, to intensive chemotherapy may improve outcome in this population. After establishing a safe dose in a run-in phase of the study in 22 patients, 231 eligible patients with AML above 65 years of age (median 70, range 66-81) were randomly assigned in this open label randomized Phase II study to receive standard chemotherapy (3+7) with or without tosedostat at the selected daily dose of 120 mg (<i>n</i> = 116), days 1-21. In the second cycle, patients received cytarabine 1000 mg/m² twice daily on days 1-6 with or without tosedostat. CR/CRi rates in the 2 arms were not significantly different (69% (95% C.I. 60-77%) vs 64% (55-73%), respectively). Cancers672https://www.ebi.ac.uk/biostudies/studies/S-EPMC7914531biostudies-literatureInferior Outcome of Addition of the Aminopeptidase Inhibitor Tosedostat to Standard Intensive Treatment for Elderly Patients with AML and High Risk MDS.PMC7914531Manz MNijziel MJie ASpertini OKuball JHoogendoorn Mvon dem Borne PGraux CLammeren-Venema DVGregor MHess UOosterveld MVelden WVVellenga EPabst TMaertens JStussi GSinnige HPoel MVBrouwer RJanssen JKlein STick LBargetzi MLowenberg BWesterweel PNorden YVKlift MVMoors IJongen-Lavrencic MDeeren DWeerdt OOssenkoppele GGjertsen BTBreems DEfthymiou AHeim DTerpstra WJaspers AVekemans MCChalandon YObbergh FVKooy MVMBiemond BLegdeur MCLoosdrecht AVfalseInferior Outcome of Addition of the Aminopeptidase Inhibitor Tosedostat to Standard Intensive Treatment for Elderly Patients with AML and High Risk MDS.Treatment results of AML in elderly patients are unsatisfactory. We hypothesized that addition of tosedostat, an aminopeptidase inhibitor, to intensive chemotherapy may improve outcome in this population. After establishing a safe dose in a run-in phase of the study in 22 patients, 231 eligible patients with AML above 65 years of age (median 70, range 66-81) were randomly assigned in this open label randomized Phase II study to receive standard chemotherapy (3+7) with or without tosedostat at the selected daily dose of 120 mg (<i>n</i> = 116), days 1-21. In the second cycle, patients received cytarabine 1000 mg/m² twice daily on days 1-6 with or without tosedostat. CR/CRi rates in the 2 arms were not significantly different (69% (95% C.I. 60-77%) vs 64% (55-73%), respectively). 2021-01-01T00:00:00Z2021 Feb2026-05-03T01:31:19.09Z2021-03-03T08:09:52ZS-EPMC79145313356239310.3390/cancers13040672