Monday, 27 March 2017

#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? 



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.

4 comments:

  1. I don't think you would find many rms patients who would not want this trial done. Whilst no neurologist in their right mind would put me in that trial; I have peers with ms who, I feel, really deserve this trial.

    From memory you have previously put a grant through for this but it was rejected? What reasons were given?

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    1. What value and limits do you place on your quality of life and EDSS?
      This treatment has been most effective in halting not repairing damage.

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  2. I think the question that needs asking is what about the risks and side effects (Especially) long term of DMDs?
    What are the long term NEDAs of said DMDs, surely that data exists?
    Surely aHSCT mortality risk of c0.5% or lower can be compared to PML risk of Tysabri (similar)?
    My comments are in general not re children with MS

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  3. I would rather take a risk and maybe die fast vs having my life slowly taken away from me due to increasing disability, kognitive decline, fatigue. I know here in Norway pwMS are in the high range of divorce statistics, suicide statistics, and the biggest group of young people who are disability pensioners. Life with MS is not easy, even in the happiest country in the world. Can any of the DMDs aquire NEDA?

    I recently listened to an interview with the first person they did HSCT on in Sweden, she's had NEDA for 12 years now. She will always have this ghost of the MS returning back to her life hanging over her, but in the meanwhile she got to have a normal life, she got to have a family and kids, she got to have a job. Would she have had the same opportunities on a DMD?

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