I. Determine the response rate and 1-year event-free survival of patients with refractory
pemphigus treated with high-dose cyclophosphamide.
PROTOCOL OUTLINE: Patients receive cyclophosphamide IV on days 1-4 and filgrastim (G-CSF)
beginning on day 10 and continuing until blood counts recover.
Patients are followed monthly for 6 months, every 2 months for 6 months, every 4 months for
a year, and then annually thereafter.
PROTOCOL ENTRY CRITERIA:
Definitive diagnosis of pemphigus, including all of the following: Typical clinical
lesions Histologically proven by skin or mucous membrane biopsy Pemphigus IgG
autoantibodies in skin or mucous membrane by direct immunofluorescence (IF) Circulating
pemphigus IgG autoantibodies in serum by indirect IF
Dependence on high-dose corticosteroids
Persistent disease activity despite treatment with mycophenolate mofetil or azathioprine,
or inability to tolerate these drugs (for paraneoplastic pemphigus, persistent disease
activity despite treatment with cyclosporine)
No concurrent cytotoxic therapy
Performance status: Karnofsky 20-100%
Renal: Creatinine no greater than 2.5 mg/dL
Cardiovascular: LVEF at least 40%
Pulmonary: FVC, FEV1, or DLCO at least 50% predicted
Other: Not pregnant Fertile patients must use effective contraception during and for 9
months after study Not preterminal or moribund