Scleroderma: Cyclophosphamide or Transplantation

Inclusion Criteria

• Severe systemic sclerosis (SSc) as defined by the American College of Rheumatology (ACR);
• SSc, including extensive skin and internal organ involvement involving either the lungs or the kidneys, that threatens participant's life; and
• Willingness to use accepted methods of contraception for at least 15 months after starting study treatment.

Exclusion Criteria

• Lung, heart, liver, or kidney impairment that would interfere with the study or compromise participant's survival;
• Active blood vessel dilation in the stomach (Active Gastric Antral Vascular Ectasia/GAVE, also known as "watermelon stomach"). Patients found to have this disorder at study screening can receive treatment outside the study and then be re-screened. For more information about this study criterion, refer to the study protocol.
• Previous treatment with cyclophosphamide, as defined by: a) prior IV cyclophosphamide administration for more than 6 months OR a total cumulative IV dose greater than 3 g/m^2; b) prior oral cyclophosphamide administration for more than 4 months, regardless of dose; or c) combination of prior oral and IV cyclophosphamide administration for more than 6 months, independent of dose.
• Steroid therapy at doses of greater than 10 mg/day, or more than 2 pulses for concurrent illnesses within prior 12 months;
• Unwillingness or inability to discontinue certain disease-modifying antirheumatic drugs (DMARDs) for the treatment of SSc;
• Presence of clinically significant rheumatic diseases other than scleroderma requiring significant immunosuppression;
• Any active uncontrolled infection that would interfere with high-dose therapy or pulse cyclophosphamide regimens:
• Hepatitis B virus infected
• Hepatitis C virus infected or
• HIV infected.
• Blood abnormalities;
• Diagnosis of cancer within 2 years prior to study entry. Participants with adequately treated squamous cell skin cancer, basal cell carcinoma, and carcinoma in situ are not excluded.
• Other comorbid illnesses with an estimated life expectancy of less than 5 years;
• Defective formation of bone marrow cells (myelodysplasia);
• Uncontrolled hypertension;
• History of hypersensitivity to murine or Escherichia coli (e.g., E. coli) proteins; History of noncompliance with prior medical care;
• History of substance abuse within 5 years prior to study entry; or
• Pregnancy.