Table 2.
Efficacy and Safety of Mitoxantrone
| Overall n = 86 |
NMOSD-DN n = 20 |
NMOSD-AQP4+ n = 59 |
MOGAD n = 7 |
p Value | |
| Time from the first attack to MiTX (mo) | 0.084 | ||||
| Median (IQR) | 9.0 (2.0–52.0) | 19.0 (6.0–59.2) | 9.0 (2.0–47.5) | 2.0 (2.0–3.5) | |
| Total relapse count before MiTX, n (%) | 0.090 | ||||
| 1 | 31 (36.0) | 5 (25.0) | 20 (33.9) | 6 (85.7) | |
| 2 | 18 (20.9) | 4 (20.0) | 14 (23.7) | 0 (0.0) | |
| ≥3 | 37 (43.0) | 11 (55.0) | 25 (42.4) | 1 (14.3) | |
| MiTX from the first attack, n (%) | 31 (36.0) | 5 (25.0) | 20 (33.9) | 6 (85.7) | 0.015 |
| First endpoint, n (%) | |||||
| First relapse during the 96-wk follow-up | 25 (29.1) | 1 (5.0) | 23 (39.0) | 1 (14.3) | 0.007 |
| Treatment efficacya, n (%) | 0.121 | ||||
| Complete success | 61 (70.9) | 19 (95.0) | 36 (61.0) | 6 (85.7) | |
| Incomplete achievement | 6 (7.0) | 0 (0.0) | 6 (10.2) | 0 (0.0) | |
| Partial failure | 8 (9.3) | 0 (0.0) | 7 (11.9) | 1 (14.3) | |
| Complete failure | 11 (12.8) | 1 (5.0) | 10 (16.9) | 0 (0.0) | |
| Secondary endpoints | |||||
| Time to first relapse during the 96 wk of follow-up (wk) | 31.0 (18.0–58.0) | 18.0 (18.0–18.0) | 37.0 (16.5–58.5) | 18.0 (18.0–18.0) | 0.545 |
| ARR the year before MiTX (mean ± SD) | 0.85 ± 0.55 | 0.85 ± 0.40 | 0.87 ± 0.61 | 0.64 ± 0.38 | 0.414 |
| ARR T96 (mean, ±SD) | 0.32 ± 0.63 | 0.07 ± 0.31 | 0.41 ± 0.69 | 0.20 ± 0.52 | 0.020 |
| Reduction in ARR from T0 to T96 | 61.3 ± 77.1 | 93.1 ± 30.9 | 47.0 ± 87.8 | 86.9 ± 34.8 | 0.010 |
| EDSS T0 (mean, ±SD) | 4.95 ± 2.37 | 5.40 ± 2.36 | 5.06 ± 2.31 | 2.71 ± 1.91 | 0.028 |
| EDSS T96 (mean, ±SD) | 4.24 ± 2.55 | 4.58 ± 2.36 | 4.32 ± 2.56 | 2.64 ± 2.82 | 0.220 |
| EDSS severity at T0, n (%) | 0.556 | ||||
| Minor [0; 3.5] | 36 (41.9) | 8 (40.0) | 23 (39.0) | 5 (71.4) | |
| Moderate [4; 5.5] | 13 (15.1) | 3 (15.0) | 9 (15.3) | 1 (14.3) | |
| Severe [6; 10] | 37 (43.0) | 9 (45.0) | 27 (45.8) | 1 (14.3) | |
| EDSS severity at T96, n (%) | 0.835 | ||||
| Minor [0; 3.5] | 41 (47.7) | 9 (45.0) | 27 (45.8) | 5 (71.4) | |
| Moderate [4; 5.5] | 13 (15.1) | 3 (15.0) | 10 (16.9) | 0 (0.0) | |
| Severe [6; 10] | 32 (37.2) | 8 (40.0) | 22 (37.3) | 2 (28.6) | |
| First relapse during the entire follow-upb, n (%) | 36 (41.9) | 4 (20.0) | 31 (52.5) | 1 (14.3) | 0.010 |
| Severe secondary adverse events of MiTX, n (%) | >0.999 | ||||
| None | 83 (96.5) | 20 (100.0) | 56 (94.9) | 7 (100.0) | |
| Acute myeloid leukemia | 1 (1.2) | 0 (0.0) | 1 (1.7) | 0 (0.0) | |
| Severe neutropenia | 2 (2.3) | 0 (0.0) | 2 (3.4) | 0 (0.0) | |
| Death, n (%) | 6 (7.0) | 0 (0.0) | 6 (10.2) | 0 (0.0) | 0.468 |
| Cause of death, n (%) | >0.999 | ||||
| Consequence of NMOSD | 5 (83.3) | 0 (NA) | 5 (83.3) | 0 (NA) | |
| Acute myeloid leukemia | 1 (16.7) | 0 (NA) | 1 (16.7) | 0 (NA) |
Abbreviations: AQP4+ = positive antiaquaporin-4 antibody; ARR = annualized relapse rate; DN = double-seronegative; EDSS = Expanded Disability Status Scale; MiTX = mitoxantrone; MOGAD = myelin oligodendrocyte glycoprotein antibody–associated disease; NMOSD = neuromyelitis optica spectrum disorder; T0 = initiation of mitoxantrone; T96: at the 96th week of follow-up.
Complete success of MiTX: patients relapse-free at 96-wk follow-up; incomplete achievement: patients had a relapse while having a decrease in ARR at 96-wk follow-up; partial failure: patients had a relapse without modification in ARR at 96-wk follow-up; complete failure: patients had a relapse while having an increase in ARR at 96 wk of follow-up.
From January 1993 and December 2020.