In a study appearing online in Neurology, the HALT-MS trial demonstrated that patients with active relapsing-remitting multiple sclerosis (RRMS) showed sustained remission out to 5 years following high dose immunosuppressive therapy combined with autologous hematopoietic cell transplantation (HDIT/HCT).
HALT-MS is a phase II clinical trial of HDIT/HCT for patients with RRMS who experienced relapses with loss of neurological function while on MS disease modifying therapy (DMT). The primary endpoint was event-free survival (EFS) defined as survival without death or disease activity from any one of: 1) loss of neurological function, 2) relapse, or 3) new lesions on MRI. Subjects were evaluated through 5 years after transplant. Toxicities were reported using the NCI Common Terminology Criteria for Adverse Events (AE).
Twenty-five subjects were evaluated for transplant and 24 patients underwent HDIT/HCT. Median follow-up was 62 months and EFS was 69.2%. Progression-free survival, clinical relapse-free survival and MRI activity-free survival were 91.3%, 86.9%, and 86.3% respectively. AE due to HDIT/HCT were consistent with expected toxicities and there were no significant late neurological adverse effects noted. Improvements were noted in neurological disability among all survivors.
The authors conclude that HDIT/HCT without maintenance therapy was effective for inducing long-term sustained remissions of active RRMS at 5 years.
For more information about the HALT-MS study and to view the data associated with publications, please see the HALT-MS study listing in ITN TrialShare.