Functional Stabilization of Worsening Multiple Sclerosis Patients Treated with High Dose Immunosuppression and Stem Cell Rescue.

TitleFunctional Stabilization of Worsening Multiple Sclerosis Patients Treated with High Dose Immunosuppression and Stem Cell Rescue.
Publication TypeJournal Article
AuthorsKraft GH, Bowen JD, Nash RA
JournalArchives of Physical Medicine and Rehabilitation

Objective: To test the hypothesis that intense immunosuppression could prevent the progression of sever multiple sclerosis (MS) refractory to conventional medications Design: Phas I and II clinical trial. Setting: Transplant centers at several US medical centers. Participants: 26 relapsing or remitting secondary progressive or primary progressive MS patients, ages 18 to 60 years,who had failed conventional medications (interferon beta-1a [Avonex, Betaseron], glatiramer [Copaxone], methotrexate, cyclophosphamide,azothiaprine, steroids, intravenous immunoglobin), andhwo had progressed at least 1 Kurtzke Expanded Disability Status Scale (EDSS) point over that from the previous 12 months. Interventions: Granulocyte colony-stimulating factor (G-CSF) was given to the patients, followed by harvest of blood stem cells. Stem cells were then frozen at -180ºC. Following this, high-dose total body irradiation, cyclophosphamide, and anti-thymocytic globulin (HDIT) were given to suppress immune function. Finally, the stem cells were transplanted back (SCT) to reconstitute naïve immune systems. Main Outcome Measures: EDSS, Scripps Neurological Rating Scale, Amulation Index, 9-Hole Peg Test, Paced Auditory Serial Addition Test (PASAT), and magnetic resonance imaging (MRI) of brain. Results: With a mean follow-up period of 15.1 months (range 6-45 months), we found the following: on the EDSS, 77% were stable or showed slight improvement. There was no change in median Ambulation Index score (n=9) or in median Scripps rating (n=20). On the 9-Hole Peg Test (n=17), 53% were stable or improved; on the PASAT (n=13); 70% were stable or improved. For MRI(n=26); only 4 subjects had enhancement on posttreatment images. For adverse events, there was 1 death and several manageable transplant-related disorders. Conclusion: HDIT and SCT appear to stabilize the majority of severe MS patients who have failed conventional treatments. Further follow-up and phase III trial will be done.

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