TY - JOUR
T1 - Long-term clinical outcomes of hematopoietic stem cell transplantation in multiple sclerosis
AU - Boffa, Giacomo
AU - Massacesi, Luca
AU - Inglese, Matilde
AU - Mariottini, Alice
AU - Capobianco, Marco
AU - Moiola, Lucia
AU - Amato, Maria Pia
AU - Cottone, Salvatore
AU - Gualandi, Francesca
AU - de Gobbi, Marco
AU - Greco, Raffaella
AU - Scimè, Rosanna
AU - Frau, Jessica
AU - Zimatore, Giovanni Bosco
AU - Bertolotto, Antonio
AU - Comi, Giancarlo
AU - Uccelli, Antonio
AU - Signori, Alessio
AU - Angelucci, Emanuele
AU - Innocenti, Chiara
AU - Ciceri, Fabio
AU - Repice, Anna Maria
AU - Sormani, Maria Pia
AU - Saccardi, Riccardo
AU - Mancardi, Gianluigi
N1 - Publisher Copyright:
Copyright © 2021 American Academy of Neurology
PY - 2021/2/23
Y1 - 2021/2/23
N2 - 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 transplantation in a large cohort of patients with MS. Methods To be included, a minimum dataset (consisting of age, MS phenotype, Expanded Disability Status Scale [EDSS] score at baseline, information on transplantation technology, and at least 1 follow-up visit after transplantation) was required. Results Two hundred ten patients were included (relapsing-remitting [RR] MS 122 [58%]). Median baseline EDSS score was 6 (1–9); mean follow-up was 6.2 (±5.0) years. Among patients with RRMS, disability worsening–free survival (95% confidence interval [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 patients with RRMS, EDSS significantly reduced after aHSCT (p = 0.001; mean EDSS change per year −0.09 [95% CI −0.15% to −0.04%]). In patients with RRMS, the use of the BCNU+Etoposide+Ara-C+Melphalan (BEAM) + anti-thymocyte globulin (ATG) conditioning protocol was independently associated with a reduced risk of no evidence of disease activity 3 failure (hazard ratio 0.27 [95% CI 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.
AB - 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 transplantation in a large cohort of patients with MS. Methods To be included, a minimum dataset (consisting of age, MS phenotype, Expanded Disability Status Scale [EDSS] score at baseline, information on transplantation technology, and at least 1 follow-up visit after transplantation) was required. Results Two hundred ten patients were included (relapsing-remitting [RR] MS 122 [58%]). Median baseline EDSS score was 6 (1–9); mean follow-up was 6.2 (±5.0) years. Among patients with RRMS, disability worsening–free survival (95% confidence interval [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 patients with RRMS, EDSS significantly reduced after aHSCT (p = 0.001; mean EDSS change per year −0.09 [95% CI −0.15% to −0.04%]). In patients with RRMS, the use of the BCNU+Etoposide+Ara-C+Melphalan (BEAM) + anti-thymocyte globulin (ATG) conditioning protocol was independently associated with a reduced risk of no evidence of disease activity 3 failure (hazard ratio 0.27 [95% CI 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.
UR - http://www.scopus.com/inward/record.url?scp=85104825189&partnerID=8YFLogxK
U2 - 10.1212/WNL.0000000000011461
DO - 10.1212/WNL.0000000000011461
M3 - Article
C2 - 33472915
AN - SCOPUS:85104825189
SN - 0028-3878
VL - 96
SP - E1215-E1226
JO - Neurology
JF - Neurology
IS - 8
ER -