Clinical outcome
| Outcome . | Prior corticosteroid untreated . | Prior corticosteroid treated . | Total . |
|---|---|---|---|
| RUX as frontline treatment, n | 34 | 18 | 52 |
| Favorable response with CR at day 28, n (%) | 15 (44.1) | 7 (38.9) | 22 (42.3) |
| Time to achieve CR, d | 21 (21-28) | 21 (14-28) | 21 (14-28) |
| Duration of CR response, mo (range) | 13.3 (9.0-17.5) | 9.5 (6.4-16.4) | 11.9 (6.4-17.5) |
| Unfavorable response requiring additional HLH treatments before day 28, n (%) | 19 (55.9) | 11 (61.1) | 30 (57.7) |
| No response, n (%) | 12 (35.3) | 4 (22.2) | 16 (28.9) |
| Progressive disease after PR,* n (%) | 5 (14.7) | 4 (22.2) | 9 (13.5) |
| Disease improvement, n (%) | 2 (5.9) | 3 (16.7) | 5 (15.4) |
| Additional treatment start day since first RUX administration (range) | 5 (3-12) | 7.5 (3-25) | 6 (3-25) |
| Death, n (%) | 0 | 0 | 0 |
| RUX + additional chemotherapy, n | 19 | 11 | 30 |
| Achieve CR at week 8, n (%) | 10 (52.6) | 6 (54.5) | 16 (53.3) |
| Refractory HLH, n (%) | 9 (47.4) | 5 (45.5) | 14 (46.7) |
| Salvage HSCT, n (% of refractory HLH) | 5 | 3 | 8 (57.1) |
| Alive, n (% of HSCT) | 3 | 2 | 5 (62.5) |
| Without HSCT, n (% of refractory HLH) | 3 | 3 | 6 (42.9) |
| Alive, n (% of without HSCT) | 0 | 1 | 1 (16.7) |
| Overall treatment, n | 34 | 18 | 52 |
| Achieve CR, n (%) | 25 (73.5) | 13 (72.2) | 38 (73.1) |
| Relapse, n (% of CR) | 2 | 1 | 3 (8.1) |
| Alive at last follow, n (%) | 29 (85.3) | 14 (77.8) | 43 (82.7) |
| Death before HSCT due to active HLH, n (%) | 3 (8.8) | 2 (11.1) | 5 (9.6) |
| Death after HSCT, n (%) | 2 (5.9) | 2 (11.1) | 4 (7.7) |
| 12-mo cumulative survival (±95% CI) | — | — | 86.4 ± 9.3 |
| Outcome . | Prior corticosteroid untreated . | Prior corticosteroid treated . | Total . |
|---|---|---|---|
| RUX as frontline treatment, n | 34 | 18 | 52 |
| Favorable response with CR at day 28, n (%) | 15 (44.1) | 7 (38.9) | 22 (42.3) |
| Time to achieve CR, d | 21 (21-28) | 21 (14-28) | 21 (14-28) |
| Duration of CR response, mo (range) | 13.3 (9.0-17.5) | 9.5 (6.4-16.4) | 11.9 (6.4-17.5) |
| Unfavorable response requiring additional HLH treatments before day 28, n (%) | 19 (55.9) | 11 (61.1) | 30 (57.7) |
| No response, n (%) | 12 (35.3) | 4 (22.2) | 16 (28.9) |
| Progressive disease after PR,* n (%) | 5 (14.7) | 4 (22.2) | 9 (13.5) |
| Disease improvement, n (%) | 2 (5.9) | 3 (16.7) | 5 (15.4) |
| Additional treatment start day since first RUX administration (range) | 5 (3-12) | 7.5 (3-25) | 6 (3-25) |
| Death, n (%) | 0 | 0 | 0 |
| RUX + additional chemotherapy, n | 19 | 11 | 30 |
| Achieve CR at week 8, n (%) | 10 (52.6) | 6 (54.5) | 16 (53.3) |
| Refractory HLH, n (%) | 9 (47.4) | 5 (45.5) | 14 (46.7) |
| Salvage HSCT, n (% of refractory HLH) | 5 | 3 | 8 (57.1) |
| Alive, n (% of HSCT) | 3 | 2 | 5 (62.5) |
| Without HSCT, n (% of refractory HLH) | 3 | 3 | 6 (42.9) |
| Alive, n (% of without HSCT) | 0 | 1 | 1 (16.7) |
| Overall treatment, n | 34 | 18 | 52 |
| Achieve CR, n (%) | 25 (73.5) | 13 (72.2) | 38 (73.1) |
| Relapse, n (% of CR) | 2 | 1 | 3 (8.1) |
| Alive at last follow, n (%) | 29 (85.3) | 14 (77.8) | 43 (82.7) |
| Death before HSCT due to active HLH, n (%) | 3 (8.8) | 2 (11.1) | 5 (9.6) |
| Death after HSCT, n (%) | 2 (5.9) | 2 (11.1) | 4 (7.7) |
| 12-mo cumulative survival (±95% CI) | — | — | 86.4 ± 9.3 |
HSCT, hematopoietic stem cell transplantation.
All patients achieving PR with RUX frontline therapy developed a progressive disease before day 28.