“Game changing” HSCT data is being reported in the media.
This is in response to data being presented at a meeting, which we commented on a few weeks ago.. I saw Roumen Balabanov last week and he wasn’t aware that the abstract below was published.
We made a post where the authors had complained about Social media as inaccurate, but I made the point that the authors are happy to take the benefits of Social Media.
This story is being circulated by Social Media.
If you want to read the abstract
SS2-8 – NON-MYELOABLATIVE HAEMATOPOIETIC STEM CELL TRANSPLANTATION VERSUS CONTINUED DISEASE MODIFYING THERAPIES (DMT) IN PATIENTS WITH HIGHLY ACTIVE RELAPSING REMITTING MULTIPLE SCLEROSIS (RRMS)
1Northwestern University, Division of Immunotherapy for Autoimmune Diseases, Chicago, IL, United States; 2Northwestern University, Department of Neurology, Chicago, IL, United States; 3Sheffield Teaching Hospitals NHS Foundation Trust, Department of Haematology, Sheffield, United Kingdom; 4Sheffield Teaching Hospitals NHS Foundation Trust, Department of Neurology, Sheffield, United Kingdom; 5University of São Paulo, Department of Internal Medicine, São Paulo, Brazil; 6University of Texas Health Science Center at Houston, Department of Neurology, Houston, TX, United States; 7Uppsala University, Department of Neuroscience, Uppsala, SwedenBackground: We previously reported (Journal of the American Medical Association, 2015) that non-myeloablative haematopoietic stem cell transplantation (HSCT) may be performed safely in patients with multiple sclerosis and is accompanied by long-term improvement in neurologic disability. We now report results on a randomized trial of non-myeloablative HSCT versus continued treatment with standard DMTs.
Methods: Patients on stable disease modifying therapy (DMT) with > 2 relapses in the previous 12 months were randomized (1:1) to treatment with either cyclophosphamide and rabbit anti-thymocyte globulin followed by haematopoietic stem cell infusion or to a control arm with continued treatment with standard DMTs. Evaluating neurologists scoring the Expanded Disability Status Scale (EDSS) were blinded to treatment arms. Patients in the control arm who had 6 month confirmed EDSS increase of > 1 point despite at least one year of treatment (defined as treatment failure) were allowed to crossover to HSCT.
Results: 110 patients were randomized, 55 to each arm. Three HSCT patients were withdrawn: two for failing enrollment criteria, one for recurrent infections occurring before transplant. Five control patients were withdrawn after soliciting transplants at other centers. All patients are at least one-year post enrollment. No deaths occurred and no CTC grade 4 non-hematopoietic toxicities occurred in the transplant arm. DMTs (number of patients) used in the control arm were: natalizumab (22), dimethyl fumarate (18), fingolimod (13) interferons (10), glatiramer acetate (8), mitoxantrone (5). During the first year after enrollment, one relapse occurred on the HSCT arm versus 39 on the DMT arm (P< 0.001). Mean EDSS improved from 3.5 to 2.4 after HSCT while it worsened from 3.3 to 3.9 on DMTs (P< 0.001). With a mean follow up of 3 years (range 1 to 5 years), treatment failure was 60% (30 of 50) for the control arm and 6% (3 of 52) for HSCT (P < 0.001). For the 30 patients who failed the control arm and crossed over to HSCT, by one year after HSCT, the mean EDSS improved from 5.2 to 2.6 (P< 0.001).
Conclusions: HSCT was statistically superior to continued DMTs in patients with RRMS with > 2 relapses a year
Clinical Trial Registry: NCT00273364, https://clinicaltrials.gov/ct2/show/NCT00273364
Conflict of interest: All the authors have nothing to discloseReporting of HSCT seems to attract the HSCT evangelists and dealing with this is rather trying, so please make points rather than sermons. Yes I can see it works very well. You have asked us to make comment so I have.This is a study where people were randomised to HSCT or other treatment and the people on HSCT did better. This is not surprising as HSCT is the ultimate immune reboot. However, I am surprised that people were offered DMF, interferons, glaterimer acetate as Natalizumab would be better than them. Surely we need to see highly active verses non-ablative HSCT for this type of analysis to be meaningful. Pitting highly-active treatment against low-hanging fruit is what pharma do to market their product. It is not going to convince those that need convincing.The other question is what is the optimum treatment schedule? In this study anti-thymocyte globulin is used to kill T cells. Does this say MS is a T cell problem. Alternatively is the cyclophophphamide doing the business.Lastly is the EDSS 5.2 to 2.6 a miraculous recovery or is this the effect of the natural resolution of a relapse. In the BBC film of a person making a startling recovery, I am led to believe they had had a relapse and so they were going to get better anyway. This is not the same as being permanently at EDSS 5.2 recovering to 2.6. That would be amazing