Table 6.

Maintenance therapy for NMOSD [233,234]. GI: Gastrointestinal; UTI: urinary tract infection; URI: upper respiratory infection; PML: progressive multifocal leukoencephalopathy; DVT: deep venous thrombosis; TB: tuberculosis.

Medication Name	Mechanism of Action (MOA)	Dosage	Treatment Response	Side Effects
Azathioprine	Thiopurine antagonist of endogenous purines in DNA and RNA, interferes with lymphocyte proliferation	Initial: 2–3 mg/kg/day with concomitant prednisone (5–60 mg daily) for 6–12 months Maintenance: 2–3 mg/kg/day	Approximately 50/50 chance of preventing additional relapse	Nausea, diarrhea, rash, recurrent infections, leukopenia, transaminase elevation, increased risk of lymphoma
Cyclophosphamide	Cytotoxic alkylating agent, inhibits mitosis	Initial: 1000 mg every 2 months with associated steroid Maintenance: same as initial dosing	Specific treatment response unavailable – only recommended when other immunosuppressive therapies fail or are not available due to contradictory preliminary findings.	GI symptoms, hyponatremia, heart block, pancytopenia, opportunistic infections
Eculizumab	Binds to the complement protein C5 specifically, inhibiting its cleavage to C5a and C5b and subsequent generation of the terminal complement complex C5b-9	Standard dose: IV 600 mg weekly for four weeks, then IV 900 mg every two weeks	Specific treatment response unavailable at this time	Headache, increased risk of infection with encapsulated organisms, especially meningococcal infections
Methotrexate	Folic acid antagonist	Initial: start with 7.5 mg weekly with upward titration and concomitant prednisone (5–60 mg daily) Maintenance: 7.5–15 mg weekly with concurrent prednisone (5–10 mg daily for at least sixmonths)	Remission rates in up to 2/3 of subjects when used as monotherapy or in conjunction with corticosteroids	Pneumonitis, GI upset, cytopenia, hepatotoxicity
Mitoxatrone	Causes DNA cross-linking and strand breaks, interferes with DNA repair	Initial: 12 mg/m² for 3–6 months Maintenance: 6–12 mg/m² every 3 months	Remission in up to 70% of subjects when dosed appropriately	Nausea, transaminase elevation, leukopenia, hair loss, amenorrhea, minor infections including UTI and URI, rarely heart failure and acute leukemia
Mycophenolate mofetil	Inhibits inosine monophosphate dehydrogenase, impairs B- and T-cell synthesis	Initial: 1000–2000 mg daily with concurrent prednisone (5–60 mg daily) Maintenance: 1000–2000 mg	Approximately 60–75% achieve remission with fewer side effects and adverse effects	Photosensitivity, recurrent infections, headache, constipation, abdominal pain, leukopenia, PML is rare
Rituximab	Removal of B cells as antigen presenting cells and reduction in the CD20+ early plasmablast population generating anti-quaporin-4 antibodies	Initial: 1000 mg weekly for two weeks or 375 mg/m² weekly for four weeks Maintenance: 375 mg/m² or 1000 mg weekly for 2 weeks when CD19 count >1% on flow cytometry	Remission rates up to 83% were achieved with persistent B cell depletion	Sepsis, infections (Herpes zoster, UTIs, URIs), leukopenia, transaminase elevation, PML is rare
Tocilizumab	Directed against the IL-6 receptor reducing plasmablast survival, inhibiting AQP4 antibody production	Standard dose: 8 mg/kg every four weeks	Specific treatment response unavailable at this time	GI disturbance, fatigue, UTIs, neutropenia, leukopenia, elevation of cholesterol, transient mild transaminase elevation, DVT, TB reactivation