Table 1.
Pharmacological therapies used for prevention of attacks in neuromyelitis optica spectrum disorders (NMOSD)
| Therapy | Regimen | Route | Comments |
|---|---|---|---|
| Prednisolone | Up to 1 mg/kg/day, usually 15–30 mg/day | Oral | Steroid side effects, taper after 1 year |
| Azathioprine | 2–3 mg/kg/day in 1 or 2 doses | Oral | First-line therapy, taper in, measure TPMT activity, target dose guided by ALC and MCV increase, liver toxicity |
| Rituximab | Various (250–2000 mg every 6–12 months; 4 weekly doses of 375 mg/m2) | IV | First-line therapy, B-cell count as biomarker |
| Mycophenolate mofetil | 1500–3000 mg/day in 2 doses | Oral | Taper in, target dose guided by ALC and trough blood concentration (1–2 μg/ml) |
| Methotrexate | 7.5–25.0 mg weekly | Oral | Substitute folic acid, liver toxicity |
| Ciclosporin A | 2–5 mg/kg/day in 2 doses | Oral | Nephrotoxic, target dose guided by trough blood concentration (70–100 ng/ml) |
| Tacrolimus | 1–6 mg/day in 2 doses | Oral | Nephrotoxic, target dose guided by trough blood concentration (5–10 ng/ml) |
| Mitoxantrone | 12 mg/m2 every 1–3 months | IV | Cardiac monitoring (LVEF), target dose guided by leukocyte count, total cumulative dose 100 mg/m2 |
| Tocilizumab | 8 mg/kg every 4 weeks | IV | Monitoring for infections, CRP no reliable biomarker for infection |
| Combination therapies | Usually prednisolone + immunosuppressant OR biological + immunsuppressant | IV or oral | Only few reports in NMOSD, monitoring for infections |
TPMT = thiopurine methyl transferase; ALC = absolute lymphocyte count; MCV = mean corpuscular volume; IV = intravenously; LVEF = left ventricular ejection fraction; CRP = C-reactive protein