Drug3rd line
Cyclophosphamide
Alkylating agent; potent immunosuppression, depletes B and T cells
- Response rate
- Effective for severe organ involvement
- Onset
- Weeks
- Route
- IV pulse or oral
- Line
- 3rd
- IgM effect
- Reduces anti-C1q antibody production
- Evidence level
- amber
Evidence summary
Reserved for severe HUVS with major organ involvement (crescentic GN, severe vasculitis). Worm et al. demonstrated efficacy of cyclophosphamide-dexamethasone pulse therapy in 2 refractory patients. Often used as induction therapy before transitioning to mycophenolate or azathioprine for maintenance.
Approved indications
Conditions for which Cyclophosphamide has regulatory approval (not specific to rare diseases covered here):
Lupus NephritisANCA-Associated VasculitisVarious Malignancies
Drug identifiers
| DrugBank | DB00531 ↗ |
| ATC Code | L01AA01 |