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Development of a phase III trial of hematopoietic stem cell transplantation for systemic lupus erythematosus

Burt RK, Marmont A, Arnold R, Heipe F, Firestein GS, Carrier E, Hahn B, Barr W, Oyama Y, Snowden J, Kalunian K, Traynor A. Bone Marrow Transplant. 2003 Aug;32 Suppl 1:S49-51.

Abstract

At Northwestern University, a phase I/II trial of hematopoietic stem cell transplant (HSCT) for systemic lupus erythematosus (SLE) has shown promising results. A phase III HSCT trial is being developed to confirm efficacy of HSCT vs continuing the currently accepted standard of care, intravenous pulse cyclophosphamide.

Main

Taken together all patients with systemic lupus erythematosus have a mortality of approximately 1% per year. High-risk patients are at greater risk with a 20% 1-year, 35% 5-year and 45% 10-year mortality. Patients at high risk for lethal complications may be identified by renal disease, hypertension, lung involvement, anemia, thrombocytopenia, and antibodies to phospholipids, or active disease demonstrated by a high disease activity index score despite therapy.

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