#ResearchSpeak: fools rush in where angels fear to tread

AHSCT for children with MS; do the benefits justify the risks? #MSBlog #ResearchSpeak 

Some critics would state that resorting to HSCT to control MS in children is foolish. However, life sometimes rewards the brave. The paper below describes the use of autologous hematopoietic stem cell transplantation (aHSCT) in children with MS. They describe 21 children with MS from across Europe who underwent aHSCT. Please not all these children had very active and aggressive MS. It is quite remarkable that 100% had progression free survival. Is this too good to be true? I note that 2 of the children had relapses so not all children were NEDA. The question is what do you do post aHSCT to suppress disease activity? Could similar results have been achieved with natalizumab, or alemtuzumab, or rituximab/ocrelizumab with fewer side effects and lower risks? This is the real question that needs to be answered. I still think we need a randomised controlled trial to assess the risk:benefit of aHSCT compared to standard of care before we support the widespread use of aHSCT in pwMS, including children. That is why I am supportive of UK trial of AHSCT vs. Alemtuzumab. Do you agree? 

Burman et al. Autologous hematopoietic stem cell transplantation for pediatric multiple sclerosis: a registry-based study of the Autoimmune Diseases Working Party (ADWP) and Pediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT). Bone Marrow Transplant. 2017 Mar 20. doi: 10.1038/bmt.2017.40. 

Autologous hematopoietic stem cell transplantation (aHSCT) is a promising therapy for multiple sclerosis (MS), which has mainly been used in adults. The purpose of this study was to investigate efficacy and adverse events of aHSCT in the treatment of children with MS using data from the European Society for Blood and Marrow Transplantation registry. Twenty-one patients with a median follow-up time of 2.8 years could be identified. PFS at 3 years was 100%, 16 patients improved in expanded disability status scale score and only 2 patients experienced a clinical relapse. The procedure was generally well tolerated and only two instances of severe transplant-related toxicity were recorded. There was no treatment-related mortality, although one patient needed intensive care. aHSCT may be a therapeutic option for children with disease that does not respond to standard care.

Labels: , , ,