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A Pilot Study of High-Dose Immunosuppression Followed by Infusion of CD34-Selected Autologous or Syngeneic Peripheral Blood Stem Cells for Treatment of Refractory Autoimmune Disorders

Phase 1
2 Years
18 Years
Not Enrolling
Systemic Sclerosis, Systemic Lupus Erythematosus, Dermatomyositis, Juvenile Rheumatoid Arthritis, Autoimmune Diseases

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Trial Information

A Pilot Study of High-Dose Immunosuppression Followed by Infusion of CD34-Selected Autologous or Syngeneic Peripheral Blood Stem Cells for Treatment of Refractory Autoimmune Disorders

PROTOCOL OUTLINE: This is a multicenter study. Patients receive filgrastim (G-CSF)
subcutaneously daily until peripheral blood stem cell (PBSC) collection is completed. CD34+
cells are separated from the rest of the PBSCs.

Patients undergo total body irradiation twice daily on days -5 and -4. Patients receive
anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5 and cyclophosphamide IV on days
-3 and -2. CD34-selected PBSCs are reinfused on day 0. Patients receive G-CSF IV daily
beginning on day 0 and continuing until blood counts recover.

Patients are followed annually for 5 years and then every 5 years thereafter.

Inclusion 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

- 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


- 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


- No medical or psychosocial reasons that would make hematopoietic stem cell collection

- 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

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:


Outcome Time Frame:

Annually for 5 years and then every 5 years thereafter

Safety Issue:


Principal Investigator

Ann Woolfrey

Investigator Role:

Study Chair

Investigator Affiliation:

Fred Hutchinson Cancer Research Center


United States: Food and Drug Administration

Study ID:




Start Date:

November 2000

Completion Date:

May 2011

Related Keywords:

  • Systemic Sclerosis
  • Systemic Lupus Erythematosus
  • Dermatomyositis
  • Juvenile Rheumatoid Arthritis
  • Autoimmune Diseases
  • arthritis & connective tissue diseases
  • dermatomyositis
  • immunologic disorders and infectious disorders
  • juvenile rheumatoid arthritis
  • rare disease
  • systemic lupus erythematosus
  • systemic sclerosis
  • Arthritis
  • Arthritis, Rheumatoid
  • Autoimmune Diseases
  • Dermatomyositis
  • Lupus Erythematosus, Systemic
  • Scleroderma, Systemic
  • Scleroderma, Diffuse
  • Sclerosis
  • Arthritis, Juvenile Rheumatoid



Fred Hutchinson Cancer Research Center Seattle, Washington  98109