Scleroderma Treatment With Autologous Transplant (STAT) Study

Summary

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Description

PRIMARY OBJECTIVES: I. To evaluate the safety and potential efficacy of high-dose immunosuppressive therapy (HDIT) followed by autologous hematopoietic cell transplantation (HCT) (without cluster of differentiation [CD]34-selection) and maintenance therapy with mycophenolate mofetil (MMF) in systemi...

PRIMARY OBJECTIVES: I. To evaluate the safety and potential efficacy of high-dose immunosuppressive therapy (HDIT) followed by autologous hematopoietic cell transplantation (HCT) (without cluster of differentiation [CD]34-selection) and maintenance therapy with mycophenolate mofetil (MMF) in systemic scleroderma (SSc) patients by evaluating the effects on event-free survival (EFS) at 5 years post-transplant. SECONDARY OBJECTIVES: I. To evaluate safety of HDIT followed by autologous HCT as determined by regimen-related toxicities, infectious complications, treatment-related mortality, overall total mortality, and time to engraftment. II. To evaluate treatment effect on disease activation/progression. III. To evaluate health-related quality of life (HRQOL) using Short Form 36 (SF-36), the St. George's Respiratory Questionnaire (SGRQ), the modified scleroderma health assessment questionnaire (SHAQ), and PROMIS version (v) 1.0 measures. IV. To assess work productivity (Work Productivity Survey) and health care utilization (using University of California San-Diego [UCSD] healthcare utilization). OUTLINE: STEM CELL MOBILIZATION AND PREPARATION: Patients receive filgrastim subcutaneously (SC) on mobilization days 1-4 followed by apheresis until a target dose of CD34+ cells >= 2.5 x 10^6/kg are collected. Patients difficult to mobilize with filgrastim alone receive cyclophosphamide intravenously (IV) or *plerixafor subcutaneously (SC) on mobilization days 1-2 and filgrastim SC on mobilization days 5-7. HDIT CONDITIONING: Patients receive high-dose cyclophosphamide IV over 1-2 hours on days -5 to -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5. TRANSPLANTATION: Patients undergo autologous PBSCT on day 0. MAINTENANCE THERAPY: Beginning 2-3 months after transplant, patients receive mycophenolate mofetil orally (PO) twice daily (BID) for 2 years. NOTE: *Plerixafor is an alternative to the cyclophosphamide based mobilization. After completion of study treatment, patients are followed at 1 month, weeks 12 and 26, and then annually for 5 years.

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