Drug3rd line3 diseases
Rituximab
Anti-CD20 monoclonal antibody; depletes B cells
- Response rate
- Promising in refractory cases
- Onset
- Weeks–months
- Route
- IV 375 mg/m² x4 or 1000 mg x2
- Line
- 3rd
- IgM effect
- Reduces anti-C1q antibody production via B-cell depletion
- Evidence level
- amber
Used across diseases
| Disease | Response rate | Line | Evidence |
|---|---|---|---|
| Hypocomplementemic Urticarial Vasculitis Syndrome | Promising in refractory cases | 3rd | amber |
| Schnitzler Syndrome | Ineffective on inflammation | Not recommended | red |
| Waldenström's Macroglobulinemia | ~25-40% (monotherapy); higher in combinations | 1st | amber |
Evidence summary
B-cell depletion with rituximab reduces anti-C1q autoantibody production. Shown effective in refractory HUVS, especially with renal involvement. Long-term follow-up of pediatric DNASE1L3-mutant siblings showed rituximab was the most effective treatment. Emerging as a key option for severe/refractory disease.
Approved indications
Conditions for which Rituximab has regulatory approval (not specific to rare diseases covered here):
ANCA-Associated VasculitisRheumatoid ArthritisNon-Hodgkin LymphomaCLL
Drug identifiers
| DrugBank | DB00073 ↗ |
| ATC Code | L01FA01 |