Treatment responses and overall survival. (A) Duration of treatment and best overall response (n = 33*). (B) Kaplan-Meier estimate for overall survival (n = 33*). *One patient enrolled in dose-escalation phase was positive for the IDH1-D54N mutation by local testing and was not positive for the IDH1-R132 mutation by the companion diagnostic test; this patient was therefore excluded from the efficacy analyses. CRi, CR with incomplete hematologic recovery; CRp, CR with incomplete platelet recovery; HMA, hypomethylating agent; MDS, myelodysplastic syndrome; MLFS, morphologic leukemia-free state; MPN, myeloproliferative neoplasm; NA, not assessed; PD, progressive disease; PR, partial response; SD, stable disease.