High-Dose Cyclophosphamide for the Treatment of Severe Autoimmune Enteropathy
High-Dose Cyclophosphamide for the Treatment of Severe Autoimmune Enteropathy
RATIONALE: Cyclophosphamide may help control the symptoms of autoimmune enteropathy .
PURPOSE: This phase II trial is studying how well cyclophosphamide works in treating young patients with severe autoimmune enteropathy.
OBJECTIVES:
Primary
Secondary
OUTLINE: Patients receive cyclophosphamide IV over 1 hour on days 1-4. Patients then receive filgrastim (G-CSF) IV or subcutaneously once daily beginning on day 10 and continuing for 3 days or until blood counts recover.
After completion of study treatment, patients are followed periodically for up to 1½ years.
PROJECTED ACCRUAL: A total of 7-11 patients will be accrued for this study.
DISEASE CHARACTERISTICS:
Diagnosis of severe autoimmune enteropathy
Histologic evidence of severe villous atrophy with intense lymphocytic infiltrate of the lamina propria by small intestinal biopsy within the past 3 months
Disease failed to respond after ≥ 2 months of corticosteroid therapy at a dose of ≥ 0.5 mg/kg/day or ≥ 40 mg/day for patients > 20 kg AND 1 of the following therapies:
At least 50% estimated caloric needs provided by parenteral nutrition
History of intractable diarrhea, defined as frequent watery stools for > 3 months that does not respond to dietary restriction
No celiac disease, defined by a history of positive antiendomysial antibody or tissue transglutaminase antibody
No primary immunodeficiency or x-linked autoimmunity-allergy dysregulation
PATIENT CHARACTERISTICS:
Performance status
Life expectancy
Hematopoietic
Hepatic
Renal
Cardiovascular
Pulmonary
Other
PRIOR CONCURRENT THERAPY:
Biologic therapy
Endocrine therapy
Other