학술논문
Long-Term Clinical Outcomes of Hematopoietic Stem Cell Transplantation in Multiple Sclerosis
Document Type
Academic Journal
Author
Boffa, Giacomo; Massacesi, Luca; Inglese, Matilde; Mariottini, Alice; Capobianco, Marco; Lucia, Moiola; Amato, Maria Pia; Cottone, Salvatore; Gualandi, Francesca; De Gobbi, Marco; Greco, Raffaella; Scimè, Rosanna; Frau, Jessica; Zimatore, Giovanni Bosco; Bertolotto, Antonio; Comi, Giancarlo; Uccelli, Antonio; Signori, Alessio; Angelucci, Emanuele; Innocenti, Chiara; Ciceri, Fabio; Repice, Anna Maria; Sormani, Maria Pia; Saccardi, Riccardo; Mancardi, Gianluigi
Source
Neurology. Jan 20, 2021
Subject
Language
English
ISSN
0028-3878
Abstract
OBJECTIVE:: To determine whether autologous hematopoietic stem cell transplantation (aHSCT) is able to induce durable disease remission in people with multiple sclerosis (MS), we analyzed the long-term outcomes after transplant in a large cohort of MS patients. METHODS:: To be included, a minimum data set (consisting of age, MS phenotype, EDSS at baseline, information on transplant technology and at least 1 follow-up visit after transplant) was required. RESULTS:: 210 patients were included [relapsing-remitting (RR)MS=122(58%)]. Median baseline EDSS was 6(1-9), mean follow-up was 6.2(±5.0) years. Among RRMS patients, disability worsening-free survival (95%CI) was 85.5%(76.9-94.1%) at 5 years and 71.3%(57.8-84.8%) at 10 years. In patients with progressive MS, disability worsening-free survival was 71.0%(59.4-82.6%) and 57.2%(41.8-72.7%) at 5 and 10 years, respectively. In RRMS patients, EDSS significantly reduced after aHSCT [p=0.001; mean EDSS change per year -0.09 (95%CI=-0.15 to -0.04%)]. In RRMS patients, the use of the BEAM+ATG conditioning protocol was independently associated with a reduced risk of NEDA-3 failure [HR=0.27(0.14-0.50), p<0.001]. Three patients died within 100-days from aHSCT (1.4%); no deaths occurred in patients transplanted after 2007.Conclusions: aHSCT prevents disability worsening in the majority of patients and induces durable improvement in disability in patients with RRMS. The BEAM+ATG conditioning protocol is associated with a more pronounced suppression of clinical relapses and MRI inflammatory activity. CLASSIFICATION OF EVIDENCE:: This study provides Class IV evidence that for people with MS, aHSCT induces durable disease remission in most patients.