BERLIN—Autologous hematopoietic stem cell transplantation (HSCT) could prevent disease activity and promote functional recovery in patients with aggressive multiple sclerosis (MS), according to a retrospective case series presented at ECTRIMS 2018.
A review article suggested that the likelihood of achieving no evidence of disease activity (NEDA) after two years of treatment ranges between 10% and 60% for traditional disease-modifying therapies. In comparison, between 70% and 90% of patients who receive HSCT achieve this outcome.
Like other highly effective therapies, HSCT has been considered to entail significant safety risks. When the European Society for Blood and Marrow Transplantation (EBMT) reviewed their data, however, they identified one death related to HSCT between 2012 and 2016. The estimated risk of death from HSCT is thus approximately 0.2%. “Mortality associated with transplantation has decreased so much that it is almost into the range of other standard disease-modifying therapies,” said Joyutpal Das, MBBS, a neuroscientist at Royal Hallamshire Hospital in Sheffield, United Kingdom.
EBMT recommended that neurologists consider HSCT for patients with highly active radiologic and clinical disease who have failed to respond to standard disease-modifying therapy. The treatment can be considered as first-line therapy for patients with exceptionally active disease who have become disabled, they added.
A Retrospective Case Series
To examine the efficacy of HSCT in this patient population, Dr. Das and colleagues conducted a retrospective case series of 20 patients with MS from five centers in various countries. The patients’ treating physicians decided that HSCT should be their first-line therapy. Dr. Das and colleagues used NEDA-3 (which includes relapses, disability progression, and MRI activity) as their primary outcome. Each patient underwent brain MRI during the first six months of treatment and at six- to 12-month intervals thereafter.
The case series included equal numbers of men and women. All patients had frequent relapses, incomplete recovery, and multiple gadolinium enhancing lesions on serial MRI scans. The lesions often affected the brainstem, cerebellum, and spine. Patients’ median age of diagnosis and median age of treatment were 28. The time between the first onset of symptoms and treatment was nine months, and that between diagnosis and treatment was five months. Patients’ median pretreatment Expanded Disability Status Scale (EDSS) score was 6.5. Median follow-up duration was 2.5 years.
EDSS Score Improved
Three patients had new lesions during the first six months of treatment, but no patients had new lesions on subsequent MRI scans. “It has been suggested … that if you want to use NEDA to measure efficacy, the patient should have rebaseline imaging after the initiation of treatment,” said Dr. Das. “If we use our six-month scan as rebaseline imaging, then we have no further disease activity on MRI scan.”
After treatment initiation, the median EDSS score decreased from 6.5 to 2. Patients’ median improvement on EDSS score was 2.5 points, which was statistically significant. Seven patients had an EDSS score improvement of 3 points or greater. EDSS score improved for all but one patient. The results suggest that HSCT induced rapid and sustained remission, said Dr. Das.
The investigators observed typical transplant-related toxicity in the population, and no patient died. One woman conceived and gave birth to a healthy baby, and one man fathered a healthy baby.