Autologous non-myeloablative hematopoietic stem cell transplant was found to be significantly better at reducing risks for disability in relapsing-remitting multiple sclerosis (RRMS) patients compared to disease-modifying drug (DMD) therapies, interim results of the MIST clinical trial show.
The results will be shared at the 2018 Annual Meeting of the American Academy of Neurology, in a presentation titled “Non-myeloablative hematopoietic stem cell transplantation (HSCT) is superior to disease modifying drug (DMD) treatment in highly active Relapsing Remitting Multiple Sclerosis (RRMS): interim results of the Multiple Sclerosis International Stem cell Transplant (MIST) Randomized Trial.”
Autologous non-myeloablative hematopoietic stem cell transplant (HSCT) previously was reported as a safe and effective therapy leading to long-term improvements in neurological disability in patients with RRMS.
This stem cell transplant strategy uses a patient’s own healthy bone marrow stem cells (aka autologous) together with a much less aggressive combination of chemotherapy and/or radiation (non-myeloablative) to prepare the patient for the transplant.
In the Phase 3 clinical trial MIST (NCT00273364), a team of researchers evaluated how non-myeloablative HSCT compares to continuous DMD therapy.
They randomized a group of 110 RRMS patients being treated with DMDs, but who relapsed at least twice in the year before randomization into two groups: one group (55 patients) received a chemotherapy drug (cyclophosphamide, sold under the brand name Cytoxan, among others) plus a suppressant of the immune system (to prevent HSCT rejection), followed by HSCT; the control group (55 patients) continued treatment with the most appropriate DMD prescribed by their neurologist.
Researchers analyzed the rate of treatment failure — defined as an increase of at least 1.0 point six sustained for at least 6 months, in the Expanded Disability Status Scale (EDSS).
“Patients on DMDs who failed after at least 1 year of treatment were allowed to crossover to HSCT,” researchers wrote.
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