JAMA: clinical trials show that nonmyeloablative hematopoietic stem cell transplantation is promising in the treatment of relapsing remitting multiple sclerosis

October 27, 2019, Non myeloablative HSCT represents a potential and effective method to slow or prevent relapsing remitting multiple sclerosis, but it is not clear whether it is better than disease modifying therapy (DMT) Picture from JAMA, 2019, DOI: 10.1001/jama.2018.18743 In a new clinical study, to compare the effect of nonmyeloablative autogenous HSCT and DMT on disease progression, researchers from the United States, Sweden, the United Kingdom and Brazil conducted a randomized clinical trial (clinical trial registration number: NCT00273364) The results showed that compared with DMT, non myeloablative HSCT significantly prolonged the time of disease progression The related research results were recently published in the Journal of JAMA The title of the paper is "effect of nonmyeloablative hematopoietic stem cell transformation vs continuous release modifying therapy on release progress in patients with releasing multiple sciences" Between September 20, 2005 and July 7, 2016, the authors recruited 110 relapsing remitting multiple sclerosis patients in four centers in the United States, Europe and South America: at least two relapses after DMT treatment in the previous year; expanded disability status scale, EDSS, with a score range of 0 to 10, 10 being the most severe neurological disability) was 2.0 to 6.0 The authors randomly assigned these patients The last follow-up occurred in January 2018, and database locking occurred in February 2018 These patients were randomly assigned to receive HSCT combined with cyclophosphamide (200 mg / kg) and antithymocyte globulin (6 mg / kg) (n = 55, HSCT treatment group) or DMT treatment (n = 55, DMT treatment group), where the efficacy of DMT was higher than that of the previous year, or different from that of the previous year The primary end point of this clinical trial was determined to be an increase of 1.0 point or more in the EDSS score (the minimum change value of clinical significance is 0.5) in two assessments at six months after treatment for at least one year, and the difference between disease progression was estimated by hazard ratio (HR) Of the 110 randomly assigned patients (73 [66%] women; average age 36 [SD, 8.6] years), 103 continued to participate in the clinical trial, of whom 98 were assessed in one year and 23 in five years (median follow-up 2 years; average 2.8 years) Three patients in the HSCT group and 34 patients in the DMT group developed disease progression Due to the small number of events, the HSCT treatment group was unable to calculate the median time of progression The median progression time of DMT group was 24 months (interquartile interval 18-48 months) (risk ratio 0.07; 95% CI 0.02-0.24; P < 0.001) In the first year, the average EDSS score of HSCT group decreased from 3.38 to 2.36, while the average EDSS score of DMT group increased from 3.31 to 3.98 (the average difference between groups was - 1.7; 95% CI was - 2.03 to - 1.29; P < 0.001) No patient died, and no non hematopoietic level 4 toxic events (such as myocardial infarction, septicemia or other disabling or potentially life-threatening events) occurred in patients receiving HSCT It can be seen from the above that in this preliminary clinical study on relapsing remitting multiple sclerosis, non myeloablative HSCT treatment is superior to DMT treatment, and compared with DMT treatment, it leads to significantly longer disease progression time Further research is needed to replicate these findings and assess long-term results and safety (bio Com) reference: 1 Richard K Burt et al Effect of nonmyeloablative hematopoietic stem cell transformation vs continuing release modifying therapy on release progress in patients with releasing releasing multiple sciences JAMA, 2019, DOI: 10.1001/jama.2018.18743