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Table 3 Therapeutic options for long-term relapse prevention in NMOSD

From: Neuromyelitis optica spectrum disorders: from pathophysiology to therapeutic strategies

Drug Study design Study phase/ClinicalTrials.gov Identifier (status 01/2021) Number of patients (randomization) NMOSD serostatus Free of relapses/relapse reduction (RR) Follow-up Disability (EDSS stabilization or improvement) Safety concerns
Azathioprine (AZA) Retrospective studies Off-label (including meta-analysis) 977 AQP4-ab + and - 34–61% 18–47 months Up to 69% during 5 year GI, infections, Hepatotoxicity
Bone marrow suppression
Malignancy
Micophenolate mofetil (MMF) Retrospective studies Off-label (including meta-analysis) 799 AQP4-ab + and - 46–73% 20–27 months Up to 90% during 5 year GI, infections, Hepatotoxicity
Bone marrow suppression
Teratogenicity
Rituximab (RTX) Retrospective studies Off-label (including meta-analysis) 577 AQP4-ab + and - 62.9 (52–88%) 24–60 months Up to 97% during 5 year
Dif EDSS: -1.16
Mucocutaneous reactions
HBV reactivation
Severe hypogammaglobulinemia
Multi-center, randomized, double-blind, placebo-controlled trial (Japan) Phase 2,3 study
UMIN000013453
38 (1:1) AQP4-ab + 100% 72 weeks Change in EDSS (− 0.26 vs. − 0.32)
NSD
NSD compared with placebo
Tocilizumab (TCZ) Single-center randomized, open-label, parallel-group study (TANGO; China) Phase 2,3 study
comparing TCZ vs AZA
NCT03350633
118 (1:1) AQP4-ab + and - TCZ: 92%
AZA: 68%
RR: NA
48 weeks
48 weeks
OR = 0.34 Pneumonia (3%), herpes zoster virus (2%), deep vein thrombosis (2%), basal ganglia haemorrhage (2%)
Single-center, open-label trial (China) Phase 1 and 2 study as monotherapy (completed)
NCT03062579
10 AQP4-ab + and -* NA 1 year NA NA
Eculizumab Multicenter, randomized, placebo-controlled, time-to-event trial (PREVENT trial) Phase 3 study (add-on therapy)
NCT01892345
143 (2:1) AQP4-ab + 96.1%
RR:93.1%
96 weeks NSD Increased risk of meningococcal infection. All patients had to receive vaccination 2 weeks prior to first dose
Inebilizumab Multi-center, randomized, double-blind, placebo-controlled (N-Momentum) Phase 2/3 study
NCT02200770
231 (3:1) AQP4-ab + and - 87.6%
RR: 73% (77.3% in AQP4-ab+)
28 weeks OR = 0.37 NSD compared to placebo
Satralizumab Multicenter, randomized, placebo-controlled, time-to-event trial (SakuraSky) Phase 3 study (add-on therapy)
NCT02028884
83 (1:1) AQP4-ab + and - AQP4+ 91.5%
AQP4-: 56.3%
RR:62% (79% in AQP4-ab+)
96 weeks NA NSD compared to placebo
Multicenter, randomized, placebo-controlled, time-to-event trial (SakuraStar) Phase 3 study (monotherapy)
NCT02073279
95 (2:1) AQP4-ab + and - AQP4+ 76.5%
AQP4-: 63.3%
RR:55% (74% in AQP4-ab+)
96 weeks NA NSD compared to placebo
Telitacicept Randomized, placebo-controlled (China) Phase 3 study (active, recruiting)
NCT03330418
118 (estimated enrollment) AQP4-ab + NA Time frame: 144 weeks NA NA
Ravulizumab Multicenter, open-label, external placebo-controlled Phase 3 study (active, recruiting)
NCT04201262
55 (estimated enrollment) AQP4-ab +* NA Time frame: 2 years NA NA
Bortezomib Single-center, Open Label Trial (USA) Phase 2 add-on study (completed)
NCT02893111
5 AQP4-ab+* NA Time frame: 1 year NA NA
Cetirizine Single-center, Open Label Trial (USA) Phase 2 add-on study (completed)
NCT02865018
16 AQP4-ab+ (with ON or TM)** NA Time frame: 1 year NA NA
BAT4406F Single-center, Open Label Trial (China) Phase 1 dose-escalation study (not yet recruiting)
NCT04146285
48 (estimated enrollment) AQP4-ab + and -* NA NA NA NA
SHR1459 Single-center, Open Label Trial (China) Phase 2 study (not yet recruiting)
NCT04670770
10 (estimated enrollment) AQP4-ab +* NA Time frame: 52 weeks NA NA
Hematopoietic Stem Cell Transplantation Single Group Assignment; Open Label study (USA) Phase 1, 2 study
(completed)
NCT00787722
13 (12 completed the study) AQP4-ab+ (n=12) and unknown (n=1) 80% 5 years at baseline: 4.4
at FU: 3.3
1/13 died due to unrelated complications from SLE
Neutropenic fever (5/13), Hypophosphatemia (9/13) Infections (1/13)
  1. AQP4-ab + positive aquaporin-4 antibodies, NMOSD neuromyelitis optica spectrum disorders, EDSS Expanded disability Status Scale, NSD no statistical differences, USA United States of America, NA not available, ON optic neuritis, TM transverse myelitis, SLE systemic lupus erythematosus, GI gastrointestinal
  2. *Participants must have AQP4-ab + and a diagnosis of NMOSD as defined by the 2015 International Consensus Diagnostic Criteria. ** Diagnosis of NMOSD as defined by the 2006 Revised NMO Diagnostic Criteria