Table 3.
Clinical responses
| Enasidenib (n = 158) | CCR (n = 161) | |
|---|---|---|
| ORR,∗ n/N (%) | 64/158 (40.5) | 16/161 (9.9) |
| Enasidenib vs CCR: OR (95% CI); P value | 6.1 (3.3-11.1); P < .001 | |
| Time to response, d, median (IQR) | 92 (58-126) | 59 (39-134) |
| Duration of response (mo), median (95% CI) | 7.3 (5.6-11.1) | NE (2.5-NE) |
| CR rate, n (%) | 37 (23.4) | 6 (3.7) |
| Composite CR rate (CR+CRi+CRp), n (%) | 47 (29.7) | 10 (6.2) |
| CR+CRh rate, n (%) | 40 (25.3) | 8 (5.0) |
| Best response, n (%) | ||
| CR | 37 (23.4) | 6 (3.7) |
| CRi/CRp | 10 (6.3) | 4 (2.5) |
| PR | 7 (4.4) | 0 |
| MLFS | 10 (6.3) | 6 (3.7) |
| Stable disease | 64 (40.5) | 54 (33.5) |
| Disease progression | 13 (8.2) | 29 (18.0) |
| NE† | 17 (10.8) | 62 (38.5) |
| Any HI,‡ n (%) | 67 (42.4) | 18 (11.2) |
| HI–erythroid | 21 (13.3) | 9 (5.6) |
| HI–neutrophil | 57 (36.1) | 13 (8.1) |
| HI–platelet | 31 (19.6) | 7 (4.3) |
| TI,‡ n/N | ||
| RBC–TI, n/N (%) | 33/104 (31.7) | 9/97 (9.3) |
| Platelet–TI, n/N (%) | 26/88 (29.5) | 8/74 (10.8) |
CCR, conventional care regimen; CI, confidence interval; CR, complete remission; CRi, CR with incomplete hematologic recovery; CRh, CR with partial hematologic recovery; CRp, CR with incomplete platelet recovery; HI, hematologic improvement; IQR, interquartile range; IWG, International Working Group; MDS, myelodysplastic syndromes; MLFS, morphologic leukemia-free state; NE, not estimable; OR, odds ratio; ORR, overall response rate; PR, partial remission; RBC, red blood cell; TI, transfusion independence.
ORR included CR, CRi/CRp, PR, and MLFS, per IWG 2003 response criteria for AML. Response rates were compared for enasidenib vs CCR by OR from a logistic regression model and P value from a Cochrane-Mantel-Haenszel test.
No postbaseline marrow collected. Patients are considered nonresponders and included in the denominator for response assessments.
HI and TI were assessed according to IWG 2006 response criteria for MDS.21 HI was assessed among all randomized patients. RBC and platelet TI were assessed among patients who were transfusion-dependent at baseline.