HSCT in London

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You can all read this. I am not going to comment the results look good, the 2.5% mortality rate not so good

Autologous Haematopoietic Stem Cell Transplantation in Active Multiple Sclerosis: a Real-world Case Series.Nicholas RS, Rhone EE, Mariottini A, Silber E, Malik O, Singh-Curry V, Turner B, Scalfari A, Ciccarelli O, Sormani MP, Olavarria E, Mehra V, Gabriel I, Kazmi MA, Muraro P; London Group on Autologous Hematopoietic Stem Cell Transplantation for Multiple Sclerosis.Neurology. 2021 Jul 12:10.1212/WNL.0000000000012449. doi: 10.1212/WNL.0000000000012449. Online ahead of print.

Objective: to examine outcomes in people with multiple sclerosis (PwMS) treated with autologous hematopoietic stem cell transplantation (AHSCT) in a real-world setting.

Methods: retrospective cohort study on PwMS treated with AHSCT at two centers in London, UK, consecutively between 2012 and 2019 who had ≥ 6 months of follow-up or died at any time. Primary outcomes were survival free of MS relapses, MRI new lesions and worsening of expanded disability status scale (EDSS). Adverse events rates were also examined.

Results: the cohort includes 120 PwMS; 52% had progressive MS (primary or secondary) and 48% had relapsing-remitting MS (RRMS). At baseline, the median expanded disability status scale (EDSS) was 6.0; 90% of the evaluable cases showed MRI activity in the 12 months preceding AHSCT. Median follow-up after AHSCT was 21 months (range 6-85). MS relapse-free survival was 93% at 2 years and 87% at 4 years after AHSCT. No new MRI lesions were detected in 90% of subjects at 2 years and 85% at 4 years. EDSS progression-free survival (PFS) was 75% at 2 years and 65% at 4 years. EBV reactivation and monoclonal paraproteinemia were associated with worse PFS. There were 3 transplant-related deaths within 100 days (2.5%), all following fluid overload and cardiac or respiratory failure.

Conclusions: efficacy outcomes of AHSCT in this real-world cohort are similar to those reported in more stringently selected clinical trial populations, although the risks may be higher.

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11 comments

  • I think that including people without relapses/new lesions prior to HSCT into the “relapse free”, “MS lesions free” post HSCT may be a bit confusing and boosting the outcome.

    I also wonder what do they call the EBV reactivation, I thought that none of the currently available treatments can get rid of the EBV, and it will reproduce with time if not interrupted aby any drugs. Did the EBNA levels drop directly after the treatment, or how was the EBV reactivation measured?

    Cheers,

  • How much of the high mortality has to do with the fact that average EDSS at time of treatment was 6.0 (=probably older with more comorbidities). I bet the stats are much better for young, otherwise healthy people with minimal disability. Even in the worst case scenario, I would personally have no problem with risking a 2.5% chance of dying in order to have the maximum possible impact on preventing brain and spinal cord damage. The brain is what makes us who we are. Brain damage is just another, worse kind of death.

    As always, every MS patient should be allowed to decide for themselves whether the risks are worth it and not denied HSCT on the basis of not having sufficiently “active” disease.

  • Not sure from the abstract – do the figures for progressive patients add up as promisingly as for RR subjects? If so, I think I would probably be happy to run the risk.

  • If the average EDSS was 6 that implies some candidates were higher and unable to walk so how were they included? Advanced secondary is usually refused.

  • Blimey! Why is the mortality rate so high in London? That’s about ten times higher than the mortality rates in Russia or Mexico HSCT facilities.

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