Northwestern researchers report Phase III MS data for HSCT vs. continuing disease-modifying therapies

Researchers at Northwestern University and colleagues reported data from a Phase III trial in 110 patients with highly active relapsing-remitting multiple sclerosis (MS) showing that conditioning with cyclophosphamide and rabbit anti-thymocyte globulin followed by non-myeloablative hematopoietic stem cell transplantation (HSCT) significantly improved mean Expanded Disability Status Scale (EDSS) score compared with continuing treatment with disease-modifying therapies for MS (improvement of 1.1 points vs. worsening of 0.6 points, p<0.001). One patient in the HSCT arm relapsed during the first year of the trial vs. 39 patients in the control arm (p<0.001). At a mean follow-up of three years, HSCT led to a significantly lower rate of treatment failure vs. the control arm (6% vs. 60%, p<0.001).

Patients in the control arm who had a six-month confirmed increase in EDSS of ≥1 point despite at least one year of treatment were allowed to crossover to HSCT. In 30 patients in the control arm who failed treatment and crossed over to HSCT, mean EDSS improved by 2.6 points at one year post-transplant. Data were presented at the European Society for Blood and Marrow Transplantation (EBMT) meeting in Lisbon. Disease-modifying therapies used in the control arm included Tysabri natalizumab, Tecfidera dimethyl fumarate, Gilenya fingolimod, interferons, Copaxone glatiramer acetate and mitoxantrone. The trial enrolled patients who were on stable disease-modifying therapies for MS with ≥2 relapses in the previous 12 months...