Fangfang Ge1, Hong Lin1, Zhuyi Li1, Ting Chang2. 1. Department of Neurology, Tangdu Hospital, the Fourth Military Medical University, Xi'an, Shaanxi Province, People's Republic of China. 2. Department of Neurology, Tangdu Hospital, the Fourth Military Medical University, Xi'an, Shaanxi Province, People's Republic of China. changting1981@163.com.
Abstract
BACKGROUND: Autologous hematopoietic stem-cell transplantation (AHSCT) has been utilized as a treatment option for multiple sclerosis (MS) since 1995. However, this procedure has not been widely implemented in clinical practice owing to its mortality risk. Here, we conduct a meta-analysis to evaluate the long-term efficacy and safety of AHSCT in MS treatment, aiming to optimize the benefit/risk ratio of this therapeutic strategy. METHODS: We searched the PubMed Web site and clinicaltrials.gov databases. The efficacy endpoints were progression-free survival (PFS) and disease activity-free survival. The safety outcomes were transplant-related mortality (TRM) and overall deaths. RESULTS: Eighteen eligible studies with a total of 732 participants were enrolled. The PFS was 75% (95% CI, 0.69-0.81), and the estimate of disease activity-free survival was 61% with 48-month follow-up. Subgroups analysis showed that low- and intermediate-intensity regimens were associated with higher PFS 80%. Relapsing remitting MS (RRMS) benefited more from AHSCT than other MS subtypes with PFS 85%. Patients with Gd+ lesions at baseline MRI responded better to AHSCT with PFS 77%. The estimate of TRM was 1.34% (95% CI, 0.39-2.30), and the overall mortality was 3.58%. TRM was significantly higher in high-intensity regimen studies (3.13%) and in older studies (1.93%) performed before 2006. CONCLUSIONS: This meta-analysis provides evidences that AHSCT can induce long-term remissions for MS patients with a high degree of safety. We indicate low- and intermediate-intensity regimens and RRMS patients with the presence of Gd+ lesions at baseline MRI can obtain the optimal benefit/risk ratio from AHSCT.
BACKGROUND: Autologous hematopoietic stem-cell transplantation (AHSCT) has been utilized as a treatment option for multiple sclerosis (MS) since 1995. However, this procedure has not been widely implemented in clinical practice owing to its mortality risk. Here, we conduct a meta-analysis to evaluate the long-term efficacy and safety of AHSCT in MS treatment, aiming to optimize the benefit/risk ratio of this therapeutic strategy. METHODS: We searched the PubMed Web site and clinicaltrials.gov databases. The efficacy endpoints were progression-free survival (PFS) and disease activity-free survival. The safety outcomes were transplant-related mortality (TRM) and overall deaths. RESULTS: Eighteen eligible studies with a total of 732 participants were enrolled. The PFS was 75% (95% CI, 0.69-0.81), and the estimate of disease activity-free survival was 61% with 48-month follow-up. Subgroups analysis showed that low- and intermediate-intensity regimens were associated with higher PFS 80%. Relapsing remitting MS (RRMS) benefited more from AHSCT than other MS subtypes with PFS 85%. Patients with Gd+ lesions at baseline MRI responded better to AHSCT with PFS 77%. The estimate of TRM was 1.34% (95% CI, 0.39-2.30), and the overall mortality was 3.58%. TRM was significantly higher in high-intensity regimen studies (3.13%) and in older studies (1.93%) performed before 2006. CONCLUSIONS: This meta-analysis provides evidences that AHSCT can induce long-term remissions for MS patients with a high degree of safety. We indicate low- and intermediate-intensity regimens and RRMS patients with the presence of Gd+ lesions at baseline MRI can obtain the optimal benefit/risk ratio from AHSCT.
Authors: Azza Ismail; Basil Sharrack; Riccardo Saccardi; John J Moore; John A Snowden Journal: Curr Opin Support Palliat Care Date: 2019-12 Impact factor: 2.302
Authors: Valentin von Niederhäusern; Josefine Ruder; Marie Ghraichy; Ilijas Jelcic; Antonia Maria Müller; Urs Schanz; Roland Martin; Johannes Trück Journal: Neurol Neuroimmunol Neuroinflamm Date: 2022-10-13