A preliminary study of nonmyeloablative Hematopoietic Stem Cell Transplantation (HSCT) has found it to be more effective than disease-modifying therapy (DMT) for patients with relapsing remitting multiple sclerosis (RRMS).
The study, published in the journal JAMA, compared nonmyeloablative HSCT with DMT in patients with highly-active RRMS between September 2005 and July 2016. One hundred and ten patients with RRMS, who had had at least two relapses while receiving DMT in the previous year and an Expanded Disability Status Scale (EDSS) score of 2.0 to 6.0 took part in the trial.
Patients were randomised to receive HSCT along with cyclophosphamide (200mg/kg) and antithymocyte globulin (6mg/kg), or DMT of higher efficacy or a different class than DMT taken during the previous year.
Of the 110 patients selected 103 remained in the trial, with 98 evaluated at one year and 23 evaluated yearly for five years. Disease progression occurred in three patients in the HSCT group and 34 patients in the DMT group. A median time to progression could not be calculated in the HSCT group because of too few events. In the DMT group it was 24 months to progression.
During the first year, mean EDSS scores decreased from 3.38 to 2.36 in the HSCT group and increased from 3.31 to 3.98 in the DMT group.
There were no deaths and no patients who received HSCT developed nonhematopoietic grade four toxicities, such as myocardial infarction, sepsis, or other disabling or potential life-threatening events.
Although the study shows positive findings it is still a small study. Researchers concluded that further research is needed to replicate these findings and to assess long-term outcomes and safety.
Source: MS-UK, 21/01/2019