HALT-MS - Intense immunosuppression and stem cell transplantation for relapsing-remitting multiple sclerosis.

TitleHALT-MS - Intense immunosuppression and stem cell transplantation for relapsing-remitting multiple sclerosis.
Publication TypeJournal Article
AuthorsHutton G, Nash RA, Wundes A, Stüve O, Sayre P, Racke M, Popat U, Openshaw H, Muraro P, Kraft GH, Griffith L, Frohman E, Bowen JD, Forman S
JournalInternational Journal of MS Care

Objective: To determine whether high-dose immunosuppressive therapy followed by autologous hematopoietic stem cell transplantation (HDIT/AHSCT) induces sustained remissions and prevents loss of neurologic function in active relapsing-remitting multiple sclerosis (RRMS). Background: HDIT/AHSCT has been assessed as a possible new therapeutic strategy in severe forms of MS. Small uncontrolled studies showed that about 60% to 70% of treated patients do not worsen in the follow-up period of at least 3 years. Responses were best in those with earlier disease stages. Since degenerative changes may contribute to loss of neurologic function in progressive MS, HDIT/AHSCT is being studied in RRMS. Methods: HALT-MS is an ongoing phase 2 clinical trial of HDIT/AHSCT with high-dose chemotherapy (BEAM) and antithymocyte globulin (ATG) in active RRMS (EDSS score, 3.0–5.5; =2 relapses on treatment and EDSS score worsening over past year). This is followed by transplantation with autologous CD34+-selected cells from granulocyte colonystimulating factor (G-CSF) mobilized blood. The sample size is 25 patients, with a 5-year follow-up. Results: Nine RRMS patients underwent HDIT/AHSCT (mean EDSS score, 4.3; median 4.3 × 106 CD34+ cells/kg, purity 95.2%). Hematologic recovery occurred 11 to 13 days post-transplant, and patients were discharged to the outpatient setting. During the median follow-up of 11.5 months (range, 1–23 months), there were no further relapses and EDSS score was stable in two subjects, improved up to 2 points in three subjects, and worse by 0.5 point in one subject. Brain magnetic resonance imaging did not reveal any new or enhancing lesions. Post-transplantation complications included pseudo–graft-vs-host disease (n = 1), pseudo- relapse (n = 1), and methicillin-resistant Staphylococcus aureus infection (n = 1), but no other unexpected or severe toxicity. Six more patients have met eligibility and are pretransplant. Conclusion: These early results for HALT-MS in patients with active RRMS poorly responsive to conventional treatment are encouraging, with suggestion of disease stabilization and neurologic improvement. The first nine patients tolerated treatment well. Additional clinical and radiologic follow- up and mechanistic studies are ongoing.


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