biostudies-literatureBrock KCancer Research UKBlood Cancer UK112https://www.ebi.ac.uk/biostudies/studies/S-EPMC5520236biostudies-literatureUnknown17(1)<h4>Background</h4>The Matchpoint trial aims to identify the optimal dose of ponatinib to give with conventional chemotherapy consisting of fludarabine, cytarabine and idarubicin to chronic myeloid leukaemia patients in blastic transformation phase. The dose should be both tolerable and efficacious. This paper describes our experience implementing EffTox in the Matchpoint trial.<h4>Methods</h4>EffTox is a Bayesian adaptive dose-finding trial design that jointly scrutinises binary efficacy and toxicity outcomes. We describe a nomenclature for succinctly describing outcomes in phase I/II dose-finding trials. We use dose-transition pathways, where doses are calculated for each feasible set of outcomes in future cohorts. We introduce the phenomenon of dose ambivalence, where EffTox can recommeBMC medical research methodologyImplementing the EffTox dose-finding design in the Matchpoint trial.PMC5520236140301595812045Siddique SBillingham LCopland MYap CBrock KSirovica MfalseImplementing the EffTox dose-finding design in the Matchpoint trial.<h4>Background</h4>The Matchpoint trial aims to identify the optimal dose of ponatinib to give with conventional chemotherapy consisting of fludarabine, cytarabine and idarubicin to chronic myeloid leukaemia patients in blastic transformation phase. The dose should be both tolerable and efficacious. This paper describes our experience implementing EffTox in the Matchpoint trial.<h4>Methods</h4>EffTox is a Bayesian adaptive dose-finding trial design that jointly scrutinises binary efficacy and toxicity outcomes. We describe a nomenclature for succinctly describing outcomes in phase I/II dose-finding trials. We use dose-transition pathways, where doses are calculated for each feasible set of outcomes in future cohorts. We introduce the phenomenon of dose ambivalence, where EffTox can recomme2017-01-01T00:00:00Z2017 Jul2025-04-26T01:38:50.97Z2019-03-27T02:51:09ZS-EPMC55202362872859410.1186/s12874-017-0381-x