HSCT Time

H

As ever I am not going to comment on this paper as it is bait for the Zealots but as as you can see the results are encouraging and they have reduced the death rate. It is highly un-optimised as you can see people underwent the procedure at EDSS9. Further evidence that relapsing disease is immune mediated

Long-Term Clinical Outcomes of Hematopoietic Stem Cell Transplantation in Multiple Sclerosis

Boffa G et al. Neurology,  10.1212/WNL.0000000000011461; DOI: 10.1212/WNL.0000000000011461

Objective: To determine whether autologous hematopoietic stem cell transplantation (aHSCT) is able to induce durable disease remission in people with multiple sclerosis (MS), we analyzed the long-term outcomes after transplant in a large cohort of MS patients.

Methods: To be included, a minimum data set (consisting of age, MS phenotype, EDSS at baseline, information on transplant technology and at least 1 follow-up visit after transplant) was required.

Results: 210 patients were included [relapsing-remitting (RR)MS=122(58%)]. Median baseline EDSS was 6(1-9), mean follow-up was 6.2(±5.0) years. Among RRMS patients, disability worsening-free survival (95%CI) was 85.5%(76.9-94.1%) at 5 years and 71.3%(57.8-84.8%) at 10 years. In patients with progressive MS, disability worsening-free survival was 71.0%(59.4-82.6%) and 57.2%(41.8-72.7%) at 5 and 10 years, respectively. In RRMS patients, EDSS significantly reduced after aHSCT [p=0.001; mean EDSS change per year -0.09 (95%CI=-0.15 to -0.04%)]. In RRMS patients, the use of the BEAM+ATG conditioning protocol was independently associated with a reduced risk of NEDA-3 failure [HR=0.27(0.14-0.50), p<0.001]. Three patients died within 100-days from aHSCT (1.4%); no deaths occurred in patients transplanted after 2007.

Conclusions: aHSCT prevents disability worsening in the majority of patients and induces durable improvement in disability in patients with RRMS. The BEAM+ATG conditioning protocol is associated with a more pronounced suppression of clinical relapses and MRI inflammatory activity.

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  • I can’t access the full paper. Do they break down the progressive ms subjects by subset (i.e. PPMS, active SPMS, inactive SPMS).

    The results stated for progressive patients overall seem very impressive, relative to what we see with the DMTs.

    Also, do they break down effectiveness based on EDSS at treatment outset?

    Thanks in advance for your answers…

    • Good questions. As a ppms patiënt i burned this ship a while ago. This makes me rethink. Need some numbers on non active ppms and HSCT.

  • Finally a statement on conditioning regime – although not really the truly interesting one (disease progression would be most interesting).

  • Maybe this has already been addressed but what is the effect on innate immune cells following HSCT and reconstitution? I usually think of lymphocyte depletion but is there macrophage and microglia depletion and reconstitution following HSCT? Also, would hematopoiesis have any effect on macrophage depletion and subsequent paramagnetic rims seen on 7T MRI associated with progressive MS? End of questions.

    • You remove your bone marrow so you stop producing new monocyts and neutrophils etc until they are generated from the bone marrow. Neutrophils last aboy 5 days and so you lack neutrophils after HSCT and this is why people die from infections after HSCT and o you have to careful until the neutrphils return typically within about 30days. Macrophages have a much slower turnover so it will take time for them to be replaced. Mouse studies suggest that microglia cells dont replace from the bone marrow, these take years to be replaced and maybe a reason why HSCT may noy have a majr impact on progression if the immune system in the brain doesn’t get replaced

  • I had EDSS 7. I had mine done in 2006. I’m 15 years going strong. I had mine in Chicago through a study. I also have a friend in Michigan I met right before we started the HSCT. So I thank Dr. Burt and staff. Dr. Burt talk to you as person. He knew his patients. The staff new you. It saved me!!

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