Observational study finds almost half of patients progression-free at 5 years
by Kate Kneisel
Contributing Writer, MedPage Today
FEBRUARY 21, 2017
Autologous hematopoietic stem cell transplantation (AHSCT) was associated with long-term attenuation of disability progression in almost half of patients with aggressive, treatment-refractory multiple sclerosis (MS), researchers found.
The long-term observational study of 281 patients with predominantly progressive forms of MS found that 46% of the 239 evaluable patients were free from progression and overall survival was 93% at 5 years, Paolo Muraro, MD, of Imperial College London in England, and colleagues reported online in JAMA Neurology.
The profile of a patient who may benefit from AHSCT for treatment of aggressive MS includes being younger in age, with relapsing forms of MS, a low number of prior immunotherapies, and low or moderate but not severe level of established neurological disability, Muraro told MedPage Today.
“The study results suggest a long-term treatment effect of AHSCT and strengthen the case [presented in a 2012 position paper] for randomized controlled trials of AHSCT against standard care,” he added.
Patients in the multicenter study were followed for up to 16 years (mean 6.6 years). Three-quarters of patients had progressive forms of MS, mostly secondary progressive MS (66.2%). Transplant occurred a median of 81 months after MS diagnosis; 61% of patients had received at least 2 MS treatments before transplant. Median EDSS score was 6.5.