REPORT FROM ECTRIMS – BARCELONA, SPAIN – OCTOBER 7-10, 2015 – The MS-AHSCT Long-Term Outcomes Study Group has compiled data for MS patients undergoing autologous hematopoietic stem cell transplantation (HSCT) during the period 1995-2006 to determine long-term outcomes (Muraro et al. ECTRIMS 2015; abstract 1671). A total of 281 patients from 25 centres were evaluable. The median duration of follow-up was 6.6 years (range 0.2-16 years).
A majority of patients (78%) had primary-progressive MS. Median EDSS score prior to HSCT was 6.5 (range 1.5-9.0).
Overall survival at five years was 93%. The five-year probability of progression-free survival was 49%. Factors associated with progression post-transplant were progressive vs. relapsing disease (hazard ratio 1.68), more than two prior disease-modifying therapies (HR 1.61), and age >37 years (HR 1.40). Neurological improvement in the year following transplant occurred in 52% of relapsing patients and 31% of progressive patients.
The authors concluded that autologous HSCT outcomes are likely to be better if transplantation is performed earlier in the clinical course in patients with active inflammatory disease.
This accords with the results recently published for the phase II HALT-MS trial (Nash et al. JAMA Neurol 2015;72:159-169). In that study, 24 RRMS patients with breakthrough disease on treatment received high-dose immunosuppressant therapy (carmustine, etoposide, cytarabine, and melphalan, rabbit antithymocyte globulin) followed by autologous HSCT. Progression-free survival at three years was 90.9%. Overall event-free survival was 78.4%. Improvements were observed in neurologic disability, quality of life and functional scores.
Reviewer: Dr. Daniel Selchen, Head of Neurology, St. Michael’s Hospital, Toronto, Canada