CYCLOPHOSPHAMIDE AND CAMPATH IN PATIENTS WITH SLE: PHASE I CLINICAL TRIAL

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This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Systemic lupus erythematosus (SLE) is a multi-system, inflammatory disorder characterized by the production of antibodies that react with many different self-antigens. Despite differences in presentation, manifestations, and clinical course, the common factor for patients with systemic lupus erythematosus is hyper-reactivity of T and B cells to endogenous stimuli. Currently, in developed countries, mortality from SLE is estimated at three per million person-years. A number of factors are associated with poor prognosis; these include the presence of renal disease causing an elevation in serum creatinine or urine protein excretion above normal, hypertension, diffuse proliferative glomerulonephritis on renal biopsy, chronic changes on biopsy, lung involvement, a high Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) score at the time of first lupus clinic visit, anemia, thrombocytopenia, and antibodies to phospholipids. This trial is designed to evaluate the safety and efficacy of treating systemic lupus erythematosus participants with high dose cyclophosphamide and CAMPATH-1H immune ablation, followed by rescue with peripheral stem cells. The primary outcome for this study is SLE remission at two years, which is defined according to the subject¿s baseline British Isles Lupus Assessment Group (BILAG) score within each category. Remission is defined as BILAG score of C or D in all categories that were initially A or B, BILAG score of D in all categories that were initially C, and a BILAG score of D or E in all categories that were initially D or E. The BILAG score will be evaluated six and 12 months post transplant, and then yearly for the duration of the study