PMID- 37364374
OWN - NLM
STAT- MEDLINE
DCOM- 20230724
LR  - 20230724
IS  - 2211-0356 (Electronic)
IS  - 2211-0348 (Linking)
VI  - 76
DP  - 2023 Aug
TI  - Autologous hematopoietic stem cell transplantation of patients with aggressive 
      relapsing-remitting multiple sclerosis: Danish nation-wide experience.
PG  - 104829
LID - S2211-0348(23)00331-0 [pii]
LID - 10.1016/j.msard.2023.104829 [doi]
AB  - BACKGROUND: Autologous hematopoietic stem cell treatment (AHSCT) is considered an 
      effective treatment option for patients with aggressive relapsing-remitting 
      multiple sclerosis (RRMS). Still there are few randomized and controlled studies 
      of AHSCT to shed light on the safety and efficacy of the treatment, and therefore 
      experiences from single centers are important. AIM: To describe the Danish 
      experience with AHSCT regarding patient characteristics, safety, and efficacy. 
      METHOD: Nationwide retrospective single center study of patients with multiple 
      sclerosis (MS) treated with AHSCT. RESULTS: A total of 32 patients were treated 
      with AHSCT from May 2011 to May 2021. Seven were treated with carmustine, 
      etoposide, cytarabine arabinoside, and melphalan (BEAM) as well as antithymocyte 
      globulin (ATG). Twenty-five patients were treated with cyclophosphamide (CY) and 
      ATG. In the whole cohort, relapse-free survival (RFS) was 77% (95% CI: 64-94%), 
      worsening-free survival (WFS) was 79% (95% CI: 66-96%), MRI event-free survival 
      (MFS) was 93% (95% CI: 85-100%), and no evidence of disease (NEDA-3) was 69% (95% 
      CI: 54-89%) at the end of year two post-AHSCT. We had no treatment related 
      mortality and only few severe adverse events (AEs). CONCLUSION: AHSCT of patients 
      with aggressive RRMS was an effective and relatively safe treatment with few 
      serious AEs and no mortality in Danish patients.
CI  - Copyright (c) 2023 The Author(s). Published by Elsevier B.V. All rights reserved.
FAU - Jespersen, Freja
AU  - Jespersen F
AD  - Danish Multiple Sclerosis Center, Copenhagen University Hospital - 
      Rigshospitalet, Copenhagen, Denmark. Electronic address: 
      freja.johanne.krogsgaard.jespersen@regionh.dk.
FAU - Petersen, Soren Lykke
AU  - Petersen SL
AD  - Department of Hematology Copenhagen University Hospital - Rigshospitalet, 
      Copenhagen, Denmark.
FAU - Andersen, Pernille
AU  - Andersen P
AD  - Blood bank, Department of Clinical Immunology, Copenhagen University Hospital - 
      Rigshospitalet, Copenhagen, Denmark.
FAU - Sellebjerg, Finn
AU  - Sellebjerg F
AD  - Danish Multiple Sclerosis Center, Copenhagen University Hospital - 
      Rigshospitalet, Copenhagen, Denmark.
FAU - Magyari, Melinda
AU  - Magyari M
AD  - Danish Multiple Sclerosis Center, Copenhagen University Hospital - 
      Rigshospitalet, Copenhagen, Denmark.
FAU - Sorensen, Per Soelberg
AU  - Sorensen PS
AD  - Danish Multiple Sclerosis Center, Copenhagen University Hospital - 
      Rigshospitalet, Copenhagen, Denmark.
FAU - Blinkenberg, Morten
AU  - Blinkenberg M
AD  - Danish Multiple Sclerosis Center, Copenhagen University Hospital - 
      Rigshospitalet, Copenhagen, Denmark.
LA  - eng
PT  - Journal Article
DEP - 20230613
PL  - Netherlands
TA  - Mult Scler Relat Disord
JT  - Multiple sclerosis and related disorders
JID - 101580247
SB  - IM
MH  - Humans
MH  - *Multiple Sclerosis, Relapsing-Remitting/drug therapy
MH  - *Multiple Sclerosis/etiology
MH  - Retrospective Studies
MH  - Treatment Outcome
MH  - *Hematopoietic Stem Cell Transplantation
MH  - Denmark
OTO - NOTNLM
OT  - Autologous hematopoietic stem cell treatment
OT  - Multiple sclerosis
OT  - No evidence of disease
OT  - Relapsing remitting
COIS- Declaration of Competing Interest F. Jespersen has nothing to disclose. S. L. 
      Petersen has served on scientific advisory boards for Janssen-Cilag and Novartis. 
      P. Andersen has nothing to disclose. F. Sellebjerg has served on scientific 
      advisory boards for, served as consultant for, received support for congress 
      participation or received speaker honoraria from Alexion, Biogen, Bristol Myers 
      Squibb, H. Lundbeck A/S, Merck, Novartis, Roche and Sanofi Genzyme. 
      His-laboratory has received research support from Biogen, Merck, Novartis, Roche 
      and Sanofi Genzyme. M. Magyari has served in scientific advisory board for 
      Sanofi, Novartis, Merck, and has received honoraria for lecturing from Biogen, 
      Merck, Novartis, Roche, Genzyme, Bristol Myers Squibb. P. S. Sorensen has 
      received personal compensation for serving on scientific advisory boards, 
      steering committees, independent data monitoring committees or have received 
      speaker honoraria for Biogen, Merck, Novartis, TEVA and Celgene/BMS. M. 
      Blinkenberg reports personal fees from Biogen, Sanofi Genzyme, Biogen, Merck, 
      Novartis, Bristol-Myers Squibb, Roche and non-financial support from Biogen, 
      Roche and Sanofi Genzyme.
EDAT- 2023/06/27 01:07
MHDA- 2023/07/24 06:42
CRDT- 2023/06/26 18:03
PHST- 2023/04/01 00:00 [received]
PHST- 2023/05/28 00:00 [revised]
PHST- 2023/06/09 00:00 [accepted]
PHST- 2023/07/24 06:42 [medline]
PHST- 2023/06/27 01:07 [pubmed]
PHST- 2023/06/26 18:03 [entrez]
AID - S2211-0348(23)00331-0 [pii]
AID - 10.1016/j.msard.2023.104829 [doi]
PST - ppublish
SO  - Mult Scler Relat Disord. 2023 Aug;76:104829. doi: 10.1016/j.msard.2023.104829. 
      Epub 2023 Jun 13.