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 * Determine the rate of treatment-free remission in young patients with severe autoimmune enteropathy treated with high-dose cyclophosphamide. Secondary * Determine the toxic effects of this drug in these patients. 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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
SUPPORTIVE_CARE
Masking
NONE
Enrollment
3
Administered IV or subcutaneously once daily beginning on day 10 and continuing for 3 days or until blood counts recover
Administered IV over 1 hour on days 1-4
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Baltimore, Maryland, United States
Number of Participants With Treatment-free Remission at 1 Year After Study Completion
Number of participants off therapy 1 year after study completion without relapse.
Time frame: 1 year
Number of Participants Experiencing Intervention-related Adverse Events, as Defined by CTCAE at 1 Month
Time frame: 1 month
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