Poor-prognosis relapsing-remitting patient treated by autologous hematopoietic stem-cell transplantation: 1 year follow-up.

Background: High-dose immunosuppressive therapy and autologous hematopoietic stem-cell transplantation (HDIT/AHSCT) may induce sustained remission in patients with autoimmune disease and is being tested as rescue therapy for multiple sclerosis (MS). In a previous clinical trial of HDIT/AHSCT for advanced progressive-type MS (median Expanded Disability Status Scale [EDSS] score 7.0), the estimated progression rate was 37% at 6 years. Because degenerative changes may contribute to loss of neurological function in progressive MS, HDIT/AHSCT in relapsing-remitting MS (RRMS) is currently being studied in a National Institutes of Health–sponsored clinical trial (HALT MS). Case Presentation: The case is a 27-year-old woman with RRMS for 8 years who continued to relapse on interferon ß-1b, interferon ß-1a, glatiramer acetate, methotrexate, and mitoxantrone. In the 12 months before enrollment, she had five relapses, and as many as 24 enhancing lesions were seen on a single magnetic resonance imaging (MRI) scan. At baseline, EDSS was 5.5, and she had 13 enhancing lesions. The most recent follow- up EDSS was 4.5. The patient is 1-year posttransplantation now and has not experienced further relapses or progression of disease nor developed new or enhancing MRI lesions. No unexpected or severe toxicity with HDIT/AHSCT was experienced. Neutrophil and platelet counts recovered by days 11 and 12, respectively. Discussion: This phase 2 study is being conducted in RRMS patients (EDSS 3.0–5.5 with =2 relapses on treatment and =1.0 EDSS worsening over past year) with HDIT/AHSCT with high-dose chemotherapy (BEAM), antithymocyte globulin, and T-cell depletion of hematopoietic cell grafts by CD34 selection. Twenty-five patients with an intended 5-year follow-up will be enrolled. To date, five patients have enrolled in the trial. Conclusions: HALT MS is the first clinical trial of HDIT/AHSCT in RRMS patients poorly responsive to conventional treatment. Thus far, patients have tolerated the treatment well, and early results suggest some neurological improvement, as illustrated by this 1-year follow-up of the first transplanted patient.