Highlights from

EAN 2019

European Academy of Neurology

Oslo 29 June - 2 July 2019

Autologous hematopoietic stem cell transplantation

Autologous hematopoietic stem cell transplantation (aHSCT) is a promising treatment for aggressive refractory relapsing-remitting MS, as was illustrated in a workshop; it may however be effective in the progressive phase as well. Stabilisation of disability progression was observed in 75% of the patients and normalisation of brain atrophy in 35%, respectively [1].

A focused workshop was dedicated to aHSCT in relapsing-remitting MS. A meta-analysis of HSCT-studies from 1995 to 2016 showed that transplant-related mortality had dropped substantially over 20 years [2]. Patients with aggressive RRMS who have not yet accumulated a high level of disability, had the most favourable benefit-risk profile for this treatment. The pooled proportion of patients with NEDA was 67% after 5 years and almost 50% after 10 years. At the same workshop results from the phase 3 MIST trial were presented. In this randomised trial, nonmyeloablative HSCT treatment was compared to standard disease-modifying therapy (DMT) [3]. Patients had had at least 2 relapses while receiving DMT in the prior year, and an EDSS score of 2.0 to 6.0. HSCT resulted in prolonged time to disease progression compared to DMT; 78.5% of patients in the HSCT group were without signs of disease activity at 5 years of follow-up.

A study presented by Dr Alice Mariottini (Careggi University Hospital, Italy), included 20 patients with a median age at aHSCT of 37 years, median disease duration of 11.5 years, and median EDSS of 6.0; 15 were females. EDSS worsened in 14 patients (70%) at year 5 of follow-up. Also, persistent interruption of disability progression following a single-step EDSS deterioration was seen in 9/14 patients. The progressive course of disability was not altered in the remaining 5/14 patients. The only baseline feature correlating with clinical outcome was annualised brain volume change. In patients who progressed, annualised brain volume change was non-significantly higher (with a median of -0.45) compared to those who showed EDSS stabilisation (median -0.14). The authors speculate that the absence of a correlation between brain volume change and disability suggests a possible major role of spinal cord involvement in disability progression. They also conclude that interruption of disability progression could be a good outcome measure of treatment efficacy in progressive MS.

  1. Mariottini A, et al. EAN 2019, O4109.
  2. Sormani RP, et al. Neurology. 2017;88(22):2115-22.
  3. Burt RK, et al. JAMA. 2019;321(2):165-74.

The content and interpretation of these conference highlights are the views and comments of the speakers/authors.