Pemphigus
Conditions
Keywords
immunologic disorders and infectious disorders, pemphigus, rare disease
Brief summary
OBJECTIVES: I. Determine the response rate and 1-year event-free survival of patients with refractory pemphigus treated with high-dose cyclophosphamide.
Detailed description
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.
Interventions
DRUGcyclophosphamide
DRUGfilgrastim
Sponsors
Johns Hopkins University
Study design
Primary purpose
TREATMENT
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
No
Inclusion criteria
PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- 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) --Prior/Concurrent Therapy-- No concurrent cytotoxic therapy --Patient Characteristics-- 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
Countries
United States
Outcome results
None listed