Best clinical response, treatment duration, and survival. (A) Number and proportion of best response (modified criteria) per patient by cohort. Includes all patients with a best response of at least PR. Bars are overlaid with the geometric mean of steady-state AUC_0-6 (ng × h/mL) for each cohort. The ORR for the study was 19 (21%) of 90 and CRc rate is 10 (11%) of 90. (B) Best percentage change in blasts is shown for individual patients, and bars are blue gradient–filled with steady-state AUC_0-6. Gray-filled bars indicate that AUC_0-6 was not determined. Patients who were successfully bridged to transplantation are shown with solid triangles. Best response by modified criteria is indicated at the top of each bar. (C) Treatment duration is represented by a gray line drawn from C1D1 to the day of last known dose. If last dose day is unknown, gray line extends to the day of study discontinuation or database lock. Response assessments by modified criteria are shown with color-coded boxes. Empty boxes indicate that a patient was not assessable (NA). The day of disease progression or death (end PFS), whichever came first, is marked with a red "×." In part 1 of the study, 2 patients were successfully bridged to transplantation (07_002 [2000 mg] and 07_004 [5000 mg]), discontinued the study drug to undergo HSCT, and did not resume pexidartinib treatment. In part 2, 4 patients were successfully bridged to transplantation. Of these, 2 patients (03_009 and 06_013) resumed maintenance treatment with pexidartinib, as provided for by protocol amendment 7, but later discontinued treatment because of an AE or voluntary withdrawal from the study. One patient (05_017) resumed treatment with pexidartinib and was still on treatment at database lock, and 1 patient (06_022) did not resume study treatment. (D) OS stratified by response (≥PR). PD, progressive disease; SD, stable disease.