Table 2.
Characteristics and outcomes of patients enrolled to the RATIFY study or the ALLG NBCR aged 18–59 with FLT3 mutation receiving intensive chemotherapy#.
| Characteristics | RATIFY – EXP (n = 360) | RATIFY – PBO (N = 357) | NBCR (N = 121) |
|---|---|---|---|
| Age, years, median (range) | 47.1 (19.0–59.8) | 48.6 (18.0–60.9) | 44 (18–59) |
| Male gender (%) | 48.3 | 40.6 | 47.9 |
| White blood cell (x109/L), median (range) | 35.6 (0.6–421.8) | 33.0 (0.8–329.8) | 42.4 (1.3–349.2) |
| Cytogenetic risk according to ELN 2010, n (%)* | |||
| - Favorable | 16/269 (5.9) | 13/278 (4.7) | 10/113 (8.8) |
| - Intermediate | 231/269 (85.9) | 248/278 (89.2) | 100/113 (88.5) |
| - Adverse | 22/269 (8.2) | 17/278 (6.1) | 3/113 (2.7) |
| FLT3 mutation subtype, n (%) | |||
| - TKD | 81 (22.5) | 81 (22.7) | 19/93 (20.4) |
| - ITD allelic ratio <0.5 | 171 (47.5) | 170 (47.6) | 52 (43.0) |
| - ITD allelic ratio 0.5–0.7 | 19 (15.7) | ||
| - ITD allelic ratio >0.7 | 108 (30.0) | 106 (29.7) | 32 (26.4) |
| NPM1 mutation, n (%)† | 244/427 (57.1) | 57/101 (56.4) | |
| Treatment response, n (%) | |||
| - CR | 212 (58.9) | 191 (54.0) | 70/116 (60.3) |
| - Death | 16 (4.4) | 11 (3.1) | 2/116 (1.7) |
| Allogeneic HCT, n (%) | |||
| - All patients | 213 (59.2) | 196 (54.9) | 60 (49.6) |
| - In first remission | 101 (28.1) | 81 (22.7) | 56 (46.3) |
| OS, months, median (95% CI) | 74.7 (31.5–NR) | 25.6 (18.6–42.9) | 45.7 (29.3–NR) |
| OS at 4 years, % (95% CI)‡ | 51.4 | 44.3 | 43.2 (29.8–62.7) |
#N represents the total population; for individual variables, some data were not measured/reported, and the appropriate denominator is specified in these instances. Some percentages do not add up to 100 due to rounding.
*Modified to combine both normal karyotype and intermediate II categories into a single intermediate group.
†Data on NPM1 mutation were not separately reported for treatment arms (Döhner et al. Blood 2020).
‡95% CI not reported by the RATIFY study. Note median follow-up was 59 and 25 months in the RATIFY study and the NBCR cohort, respectively.
CR complete remission, CI confidence interval, ELN European LeukemiaNet, EXP experimental, HCT hematopoietic cell transplantation, ITD internal tandem duplication, NR not reached, OS overall survival, PBO placebo, TKD tyrosine kinase domain.