Cyclophosphamide in Treating Young Patients With Severe Autoimmune Enteropathy
Condition: Diarrhea; Gastrointestinal Complications; Unspecified Childhood Solid Tumor, Protocol Specific
MeSH terms: Diarrhea
Intervention: filgrastim; cyclophosphamide
Intervention description: 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
Intervention MeSH terms: Cyclophosphamide
Phase: Phase 2
Study type: Interventional
Primary purpose: Supportive Care
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 3.0
Sex: All
Minimum age: 1 Year
Maximum age: 21 Years
Healthy volunteers: No
Keywords: unspecified childhood solid tumor, protocol specific; gastrointestinal complications; diarrhea

Summary:

      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.
    

Eligibility criteria:

        DISEASE CHARACTERISTICS:

          -  Diagnosis of severe autoimmune enteropathy

               -  Condition is resistant to conventional therapy

          -  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:

               -  Cyclosporine resulting in ≥ 1 whole blood level of > 200 ng/mL

               -  Tacrolimus resulting in ≥ 1 whole blood level of 5 ng/mL

          -  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

          -  Lansky 60-100%

        Life expectancy

          -  Not specified

        Hematopoietic

          -  Not specified

        Hepatic

          -  Not specified

        Renal

          -  Not specified

        Cardiovascular

          -  Ejection fraction ≥ 40% OR shortening fraction ≥ 20%

        Pulmonary

          -  FVC or FEV_1 ≥ 50% of predicted (for patients > 8 years of age)

          -  No clinically abnormal pulmonary function or abnormal pulse oximetry (for patients ≤ 8
             years of age)

        Other

          -  Not pregnant

          -  Negative pregnancy test

          -  Fertile patients must use effective contraception during and for at least 9 months
             after completion of study treatment

          -  No known chromosomal abnormality

        PRIOR CONCURRENT THERAPY:

        Biologic therapy

          -  No immunizations for at least 6 months after completion of study treatment

        Endocrine therapy

          -  See Disease Characteristics

          -  At least 5 days since prior corticosteroids

          -  No concurrent dexamethasone as an anti-emetic

        Other

          -  At least 5 days since other prior immunosuppressive medications (e.g., tacrolimus or
             cyclosporine)