Treatment of autoimmune disease by intense immunosuppressive conditioning and autologous hematopoietic stem cell transplantation

Richard K. Burt*, Ann E. Traynor, Richard Pope, James Schroeder, Bruce Cohen, Karyn H. Karlin, Lorri Lobeck, Charles Goolsby, Philip Rowlings, Floyd A. Davis, Dusan Stefoski, Cass Terry, Carolyn Keever-Taylor, Steve Rosen, David Vesole, Maryanne Fishman, Mary Brush, Salim Mujias, Marcelo Villa, William H. Burns

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

197 Scopus citations


Multiple sclerosis, systemic lupus erythematosus, and rheumatoid arthritis are immune-mediated diseases that are responsive to suppression or modulation of the immune system. For patients with severe disease, immunosuppression may be intensified to the point of myelosuppression or hematopoietic ablation. Hematopoiesis and immunity may then be rapidly reconstituted by reinfusion of CD34+ progenitor cells. In 10 patients with these autoimmune diseases, autologous hematopoietic stem cells were collected from bone marrow or mobilized from peripheral blood with either granulocyte colony-stimulating factor (G-CSF) or cyclophosphamide and G-CSF. Stem cells were enriched ex vivo using CD34+ selection and reinfused after either myelosuppressive conditioning with cyclophosphamide (200 mg/kg), methylprednisolone (4 g) and antithymocyte globulin (ATG; 90 mg/kg) or myeloablative conditioning with total body irradiation (1,200 cGy), methylprednisolone (4 g), and cyclophosphamide (120 mg/kg). Six patients with multiple sclerosis, 2 with systemic lupus erythematosus, and 2 with rheumatoid arthritis have undergone hematopoietic stem cell transplantation. Mean time to engraftment of an absolute neutrophil count greater than 500/μL (0.5 x 109/L) and a nontransfused platelet count greater than 20,000/μL (20 x 109/L) occurred on day 10 and 14, respectively. Regimen-related nonhematopoietic toxicity was minimal. All patients improved and/or had stabilization of disease with a follow-up of 5 to 17 months (median, 11 months). We conclude that intense immunosuppressive conditioning and autologous T-cell-depleted hematopoietic transplantation was safely used to treat these 10 patients with severe autoimmune disease. Although durability of response is as yet unknown, all patients have demonstrated stabilization or improvement.

Original languageEnglish (US)
Pages (from-to)3505-3514
Number of pages10
Issue number10
StatePublished - Nov 15 1998

ASJC Scopus subject areas

  • Hematology
  • Biochemistry
  • Cell Biology
  • Immunology


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