Long-term efficacy of autologous haematopoietic stem cell transplantation in multiple sclerosis at a single institution in China

Abstract

Autologous haematopoietic stem cell transplantation (AHSCT) is a promising treatment for multiple sclerosis (MS) patients who have not adequately responded to conventional therapies. We retrospectively evaluated the safety and long-term clinical outcome of AHSCT in MS patients in China. Twenty-five patients with various types of MS were treated with AHSCT. Peripheral blood stem cells were derived by leukapheresis after mobilized with granulocyte colony-stimulating factor. Then CD34+ cell selection of the graft was performed and anti-thymocyte globulin was given for T-cell depletion, with the conditioning regimen BEAM adopted and early and late toxicities recorded. Long-term responses were evaluated by the expanded disability status scale (EDSS), progression-free survival and gadolinium-enhanced magnetic resonance imaging scans. 10, 7 and 8 patients experienced neurological improvement, stabilization and progression, respectively. The median EDSS scores observed over 1-year follow-up after transplantation (5.5-7.0) were consistently lower than the baseline (8.0). The progression-free survival rate was 74, 65 and 48% at 3, 6 and 9 years post-transplant. 58% cases (7/12) had active lesions at baseline and all turned to inactive status in the years of follow-up. 25% cases (3/12) experienced progression after transplantation but had no active lesions in MRI over the whole follow-up period. 17% cases (2/12) without active lesions at baseline progressed active lesions in MRI. The major early toxicity resulted in fever and late toxicity caused transplantation-related mortality due to severe pneumonia and varicella-zoster virus hepatitis, respectively. AHSCT is a feasible treatment for severe MS and its long-term efficacy is favorable.

Hematopoietic Stem Cell Therapy for Multiple Sclerosis: Top 10 Lessons Learned

“…Other groups have also reported a sustained reduction in EDSS scores after HSCT. A reduction of the mean EDSS from a score of 3.5 before HSCT to 2.0 at 3 years after HSCT was seen among 90 patients in a cohort from Russia [58], whereas a reduction in the mean EDSS score by 3.1 was seen among 10 of 25 HSCT recipients in a study from China [59]. Although a few patients have had dramatic improvements after HSCT, most reports of improvement are modest (i.e., in the 0.5-1.0 range).…”

The role of immune cells, glia and neurons in white and gray matter pathology in multiple sclerosis

“…In fact, following HSC transplantation in patients with aggressive SP MS, a dramatic stabilization and/or improvement of the neurological condition has been observed over a follow-up period of four years (Fagius et al, 2009; Kimiskidis et al, 2008; Mancardi et al, 2005). Of note, HSC transplantation in non-aggressive SP MS patients only induced mild clinical improvements (Chen et al, 2012; Shevchenko et al, 2012). …”

Therapeutic approaches to disease modifying therapy for multiple sclerosis in adults: An Australian and New Zealand perspective Part 2 New and emerging therapies and their efficacy

“…There are a number of phase I/II and observational trials of AHSCT for MS [38][39][40][41][42][43]. Meta-analysis [44] and systematic review [45][46][47][48] of this therapy suggests a high level of efficacy in what are a group of patients for whom the outcome is generally poor [49].…”