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

TitlePoor-prognosis relapsing-remitting patient treated by autologous hematopoietic stem-cell transplantation: 1 year follow-up.
Publication TypeJournal Article
2008
AuthorsWundes A, Kraft GH, Bowen JD, Openshaw H, Forman S, Frohman E, Griffith L, Hutton G, Muraro P, Popat U, Racke M, Sayre P, Stüve O, Nash RA
JournalInternational Journal of MS Care
Volume10
IssueS1
Pagination54-55

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.

http://www.ijmsc.org/doi/pdf/10.7224/1537-2073-10.S1.i

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