Abstract
Abstract 2020
Background
Autologous haematopoietic stem cell transplantation (AHSCT) is a promising treatment of multiple sclerosis (MS) who had not adequately responded to conventional therapies. We retrospectively evaluated the safety and long-term clinical outcome of AHSCT in MS patients in China.
Methods
Twenty-five patients with various types of MS were treated with AHSCT. Peripheral blood stem cells were obtained by leukapheresis after mobilization with granulocyte colony stimulating factor. Then CD34+ cell selection of the graft was performed and anti-thymocyte globulin was given for T-cell depletion. Conditioning regimen was BEAM. Early and late toxicities were also recorded. Long-term outcomes were evaluated by the expanded disability status scale, progression free survival and gadolinium-enhancing magnatic resonance imaging (MRI) scans.
Results
10, 7 and 8 patients experienced neurological improvement, stabilization and progression, respectively. The median EDSS scores at every follow-up year 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 follow-up years. 25% cases (3/12) experienced progression after transplantation but had no active lesions in MRI in the whole follow-up period. 17% cases (2/12) without active lesions at baseline progressed and appeared active lesions in MRI.
Conclusion
AHSCT is a feasible treatment for severe MS and its long-term efficacy is favorable. However, more random and controlled clinical trials are required to fully assess long-term efficacy of AHSCT for MS.
Disclosures:
No relevant conflicts of interest to declare.
Long-term efficacy and safety of fingolimod in Japanese patients with relapsing multiple sclerosis: 3-year results of the phase 2 extension study
