Pilot Study of Total Body Irradiation in Combination With Cyclophosphamide, Anti-thymocyte Globulin, and Autologous CD34-Selected Peripheral Blood Stem Cell Transplantation in Children With Refractory Autoimmune Disorders
Systemic Sclerosis, Systemic Lupus Erythematosus, Dermatomyositis
About this trial
This is an interventional treatment trial for Systemic Sclerosis focused on measuring arthritis & connective tissue diseases, dermatomyositis, immunologic disorders and infectious disorders, juvenile rheumatoid arthritis, rare disease, systemic lupus erythematosus, systemic sclerosis
Eligibility Criteria
PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Diagnosis of 1 of the following based on American College of Rheumatology (ACR) Criteria: Severe juvenile rheumatoid arthritis (systemic onset or polyarticular course) Juvenile systemic lupus erythematosus Systemic sclerosis Dermatomyositis Refractory to standard or aggressive therapy OR unacceptable toxicity from standard therapy Reasonable expectation of possible improvement as evidenced by a good potential for rehabilitation therapy and adequate social factors No serious CNS damage that would preclude significant functional recovery --Prior/Concurrent Therapy-- Chemotherapy: At least 4 weeks since prior methotrexate or cyclophosphamide Endocrine therapy: At least 4 weeks since prior intra-arterial steroids Juvenile rheumatoid arthritis patients should continue steroids without taper throughout mobilization and harvest of stem cells If receiving corticosteroids, must be continued without taper Other: At least 4 weeks since prior anti-inflammatory agents such as non-steroidal anti-inflammatory drugs (NSAIDs) or sulfasalazine At least 4 weeks since prior cyclosporine, tacrolimus, mycophenolate mofetil, azathioprine, penicillamine, or etanercept --Patient Characteristics-- Life expectancy: At least 30 days Hematopoietic: Absolute neutrophil count at least 1,000/mm3 OR Platelet count at least 100,000/mm3 No bone marrow aspirate or biopsy consistent with production defect (depletion of neutrophil precursors or megakaryocytes) No myelodysplasia Hepatic: Bilirubin no greater than 2.5 mg/dL AST no greater than 300 U/L on two sequential tests No severe liver dysfunction within past month No active hepatitis A, B, or C Renal: No end-stage glomerulonephritis or renal disease Creatinine clearance at least 40 mL/min Cardiovascular: No uncontrolled malignant arrhythmia No New York Heart Association class III or IV congestive heart failure Ejection fraction at least 50% Pulmonary: DLCO at least 45% (DLCO at least 70% for patients with pulmonary disease caused by documented processes other than primary autoimmune disorder, such as infectious pneumonia or aspiration pneumonia) No severe pulmonary hypertension (PAP greater than 50) without potential for significant improvement Other: No medical or psychosocial reasons that would make hematopoietic stem cell collection intolerable No increased anesthetic risks No fever higher than 39 degrees C No positive serology for toxoplasmosis No active life threatening infection not responsive to therapy No other disease or organ dysfunction that would limit survival No known hypersensitivity to murine or equine proteins No known primary immunodeficiency disease HIV negative
Sites / Locations
- Fred Hutchinson Cancer Research Center
Arms of the Study
Arm 1
Experimental
1
Participants will receive a stem cell transplant.