MS patients are in sustained remission after stem cell transplantation, small study findsBMJ 2014; 349 doi: https://doi.org/10.1136/bmj.g7832 (Published 30 December 2014) Cite this as: BMJ 2014;349:g7832
Most patients with relapsing remitting multiple sclerosis (MS) who are taking part in a small study of high dose immunosuppressive therapy followed by transplantation of their own haematopoietic stem cells have experienced sustained remission for three years, a study has found.
The ongoing study includes 25 MS patients who had experienced relapses with loss of neurologic function while on disease modifying therapies during the 18 months before recruitment. They were selected from 36 patients who were screened by referral centres.
The researchers hypothesised that controlling inflammation in early relapsing remitting MS might provide prolonged remission with the potential to reverse neurologic dysfunction. To achieve this they treated patients with high dose immunosuppressive therapy followed by autologous haematopoietic cell transplantation to remove disease-causing immune cells and repopulate patients’ immune systems with healthy cells. They reported their latest results in JAMA Neurology.1
The results showed that over three quarters (78.4% (90% confidence interval 60.1% to 89.0%)) of the 24 patients who received the full treatment programme had no MS disease activity—defined as clinical relapse, new lesions, or loss of neurologic function, at three years. Most of the patients (90.9% (73.7% to 97.1%)) were free from any disease progression. Patients also showed improvements in neurologic disability, quality of life, and functional scores.
The study authors concluded that this treatment approach “may represent a potential therapeutic option for patients with MS in [whom] conventional immunotherapy fails.” They said that the high event-free survival rate at three years compared favourably with a study of natalizumab in which 37% of patients were free from disease activity at two years.
An editorial2 accompanying the study report said that the findings “leave little doubt that high dose immunotherapy is able to substantially suppress inflammatory disease activity in MS.” But the authors cautioned, “Clinical or radiologic evidence of relapse trumps immunologic evidence of immune reset, and this study raises concern that those endpoints have not been adequately achieved.”
They concluded, “The jury is still out regarding the appropriateness and indication of HCT [haematopoietic cell transplantation] for MS.”
Cite this as: BMJ 2014;349:g7832