Table 2.

Investigator-reported efficacy outcomes and survival in patients with R/R AML

R/R AML
Enasidenib, 100 mg/d (n = 214)All doses (N = 280)
ORR, % (n/N) [95% CI]* 38.8 (83/214) [32.2%-45.7%] 39.6 (111/280) [33.9%-45.6%] 
CR + CRi/CRp rate, % (n/N) 29.0 (62/214) 27.9 (78/280) 
Best response   
 CR, n (%) [CR rate 95% CI] 42 (19.6) [14.5-25.6] 53 (18.9) [14.5-24.0] 
 CRi/CRp, n (%) 20 (9.3) 25 (8.9) 
 PR, n (%) 9 (4.2) 17 (6.1) 
 MLFS, n (%) 12 (5.6) 16 (5.7) 
 SD, n (%) 98 (45.8) 122 (43.6) 
 PD, n (%) 19 (8.9) 26 (9.3) 
 Not evaluable, n (%) 3 (1.4) 4 (1.4) 
Time to first response, median (range), mo 1.9 (0.5-9.4) 1.9 (0.5-9.4) 
Duration of response, median (95% CI), mo 5.6 (3.8-7.4) 5.6 (4.6-6.5) 
Time to best response, median (range), mo 3.7 (0.6-14.7) 3.7 (0.5-14.7) 
Time to CR, median (range), mo 3.7 (0.7-14.7) 3.8 (0.5-14.7) 
OS, median (95% CI), mo 8.8 (7.7-9.6) 8.8 (7.8-9.9) 
EFS, median (95% CI), mo§ 4.7 (3.7-5.6) 4.6 (3.7-5.6) 
R/R AML
Enasidenib, 100 mg/d (n = 214)All doses (N = 280)
ORR, % (n/N) [95% CI]* 38.8 (83/214) [32.2%-45.7%] 39.6 (111/280) [33.9%-45.6%] 
CR + CRi/CRp rate, % (n/N) 29.0 (62/214) 27.9 (78/280) 
Best response   
 CR, n (%) [CR rate 95% CI] 42 (19.6) [14.5-25.6] 53 (18.9) [14.5-24.0] 
 CRi/CRp, n (%) 20 (9.3) 25 (8.9) 
 PR, n (%) 9 (4.2) 17 (6.1) 
 MLFS, n (%) 12 (5.6) 16 (5.7) 
 SD, n (%) 98 (45.8) 122 (43.6) 
 PD, n (%) 19 (8.9) 26 (9.3) 
 Not evaluable, n (%) 3 (1.4) 4 (1.4) 
Time to first response, median (range), mo 1.9 (0.5-9.4) 1.9 (0.5-9.4) 
Duration of response, median (95% CI), mo 5.6 (3.8-7.4) 5.6 (4.6-6.5) 
Time to best response, median (range), mo 3.7 (0.6-14.7) 3.7 (0.5-14.7) 
Time to CR, median (range), mo 3.7 (0.7-14.7) 3.8 (0.5-14.7) 
OS, median (95% CI), mo 8.8 (7.7-9.6) 8.8 (7.8-9.9) 
EFS, median (95% CI), mo§ 4.7 (3.7-5.6) 4.6 (3.7-5.6) 
*

Responses were evaluated by study investigators and classified according to the 2003 revised IWG criteria for AML.16  ORR consists of CR, CRi, CRp, PR, and MLFS.

SD was defined as failure to achieve a response but not meeting criteria for disease progression for >8 consecutive weeks.

For patients with 5% to 66% bone marrow blasts at nadir, a >50% increase in bone marrow blast count percentage from the nadir with percentage ≥ 20%; and for patients with ≥67% bone marrow blasts at nadir, a doubling of the nadir absolute peripheral blood blast count with a final absolute peripheral blood blast count >10 × 109/L

§

Date of first documented response to date of relapse, disease progression, or death.