Outcomes in randomized RO+ LCH-III patients*
| RO+ Trial: Patients with RO involvement . | Arm A . | Arm B . | P value . |
|---|---|---|---|
| (RO+) | Without MTX | With MTX | |
| Randomized patients (n) | 115 | 120 | |
| Primary end point | |||
| Rsponse in RO at week 6/12 | |||
| Responders/evaluable pts. (%) | 74/105 (70) | 77/107 (72) | .811 |
| Secondary study end points | |||
| Response in RO at week 6 | |||
| NAD/AD better/evaluable pts., n (%) | 70/107 (65) | 72/109 (66) | .92 |
| NAD, n (%) | 21 (20) | 20 (18) | |
| AD better, n (%) | 49 (46) | 52 (48) | |
| AD intermediate, n (%) | 23 (21) | 21 (19) | |
| Worse, n (%) | 14 (13) | 16 (15) | |
| Survival | |||
| Deaths/patients | 14/112 | 19/115 | |
| 3-y pSU % ± SE | 88 ± 3 | 82 ± 4 | .36 |
| 5-y pSU % ± SE | 87 ± 3 | 82 ± 4 | |
| NAD in risk organs | 92/111 | 89/114 | |
| NAD at week 6 (cum. incidence ± SE) | 19 ± 4 | 17 ± 5 | |
| NAD at week 12 (cum. Incidence ± SE) | 34 ± 4 | 31 ± 4 | |
| 1-y cumulative incidence | 57 ± 5 | 63 ± 5 | .79 |
| Complete disease resolution | |||
| NAD/patients | 91/111 | 86/114 | |
| NAD at week 6 (cum. incidence ± SE) | 13 ± 3 | 9 ± 3 | |
| NAD at week 12 (cum. incidence ± SE) | 19 ± 4 | 19 ± 4 | |
| 1-y cumulative incidence % ± SE | 48 ± 5 | 48 ± 5 | |
| 5-y cumulative incidence % ± SE | 87 ± 4 | 85 ± 3 | .88 |
| Median time to disease resolution | 1 y | 1 y | |
| Reactivations after NAD/patients† | 23/90 | 21/83 | |
| 3-y cumulative incidence % ± SE | 25 ± 5 | 29 ± 6 | |
| 5-y cumulative incidence % ± SE | 25 ± 5 | 29 ± 6 | .98 |
| Toxicity, course 1 | |||
| Grade 3/4 Toxicity/evaluable pts n (%) | 33/109(30) | 52/112(46) | .02 |
| DI | |||
| At Dx/pts | 8/112 | 8/115 | |
| Developed DI/eval. pts | 10/104 | 9/107 | .87 |
| 3-y cumulative incidence % ± SE | 8 ± 3 | 9 ± 3 |
| RO+ Trial: Patients with RO involvement . | Arm A . | Arm B . | P value . |
|---|---|---|---|
| (RO+) | Without MTX | With MTX | |
| Randomized patients (n) | 115 | 120 | |
| Primary end point | |||
| Rsponse in RO at week 6/12 | |||
| Responders/evaluable pts. (%) | 74/105 (70) | 77/107 (72) | .811 |
| Secondary study end points | |||
| Response in RO at week 6 | |||
| NAD/AD better/evaluable pts., n (%) | 70/107 (65) | 72/109 (66) | .92 |
| NAD, n (%) | 21 (20) | 20 (18) | |
| AD better, n (%) | 49 (46) | 52 (48) | |
| AD intermediate, n (%) | 23 (21) | 21 (19) | |
| Worse, n (%) | 14 (13) | 16 (15) | |
| Survival | |||
| Deaths/patients | 14/112 | 19/115 | |
| 3-y pSU % ± SE | 88 ± 3 | 82 ± 4 | .36 |
| 5-y pSU % ± SE | 87 ± 3 | 82 ± 4 | |
| NAD in risk organs | 92/111 | 89/114 | |
| NAD at week 6 (cum. incidence ± SE) | 19 ± 4 | 17 ± 5 | |
| NAD at week 12 (cum. Incidence ± SE) | 34 ± 4 | 31 ± 4 | |
| 1-y cumulative incidence | 57 ± 5 | 63 ± 5 | .79 |
| Complete disease resolution | |||
| NAD/patients | 91/111 | 86/114 | |
| NAD at week 6 (cum. incidence ± SE) | 13 ± 3 | 9 ± 3 | |
| NAD at week 12 (cum. incidence ± SE) | 19 ± 4 | 19 ± 4 | |
| 1-y cumulative incidence % ± SE | 48 ± 5 | 48 ± 5 | |
| 5-y cumulative incidence % ± SE | 87 ± 4 | 85 ± 3 | .88 |
| Median time to disease resolution | 1 y | 1 y | |
| Reactivations after NAD/patients† | 23/90 | 21/83 | |
| 3-y cumulative incidence % ± SE | 25 ± 5 | 29 ± 6 | |
| 5-y cumulative incidence % ± SE | 25 ± 5 | 29 ± 6 | .98 |
| Toxicity, course 1 | |||
| Grade 3/4 Toxicity/evaluable pts n (%) | 33/109(30) | 52/112(46) | .02 |
| DI | |||
| At Dx/pts | 8/112 | 8/115 | |
| Developed DI/eval. pts | 10/104 | 9/107 | .87 |
| 3-y cumulative incidence % ± SE | 8 ± 3 | 9 ± 3 |