2 min read

ID: 4956

Short Link: https://gregory-ms.com/articles/4956/

Discovery Date: 11 October 2021, 23:06:14 UTC

Published Date: 2021-12-19 04:33:45.748000

Source: PubMed

Link: https://pubmed.ncbi.nlm.nih.gov/34633525/?utm_source=Other&utm_medium=rss&utm_campaign=pubmed-2&utm_content=10guX6I3SqrbUeeLKSTD6FCRM44ewnrN2MKKTQLLPMHB4xNsZU&fc=20210216052009&ff=20211011190614&v=2.1

Manual Selection: true

Machine Learning Gaussian Naive Bayes Model: true


J Neurol. 2021 Oct 11. doi: 10.1007/s00415-021-10820-2. Online ahead of print.


OBJECTIVE: To investigate the results of real-world application of non-myeloablative autologous HSCT for multiple sclerosis (MS).

METHODS: Between July 2003 and October 2019 at a single center (Northwestern University), 414 patients with relapsing remitting MS (RRMS) and 93 patients with newly diagnosed secondary progressive MS (SPMS) underwent non-myeloablative HSCT.

RESULTS: There was one treatment-related death (0.19%) due to hospital-acquired legionella pneumonia, and one patient developed neutropenic bacteremia (Klebsiella pneumonia) without sepsis. Overall 5-year survival was 98.8%. Post HSCT secondary autoimmune diseases (2nd ADs) were idiopathic thrombocytopenia (ITP) and hypo or hyperthyroidism. ITP was highest with alemtuzumab (14%) and 0 to 2.8% for the non-alemtuzumab regimens. After HSCT, 16 patients developed hypothyroidism (3.5%) and 15 developed hyperthyroidism / Grave's disease (3.3%). Relapse free survival (RFS) at 5 years for RRMS and SPMS was 80.1% and 98.1%, respectively, while progression free survival (PFS) at 4 years for RRMS and SPMS was 95% versus 66%, respectively. For patients with RRMS, the EDSS significantly improved (p < 0.0001) at each follow-up from a pre-HSCT mean of 3.87 to 2.51, 2.50, 2.41, 2.33, and 2.19 at 1, 2, 3, 4, and 5 years, respectively. For SPMS, the EDSS improved significantly only at 1 year but not thereafter. For SPMS, the mean baseline EDSS of 5.09 changed post-HSCT to 4.85 (p = 0.04), 4.88 (p = 0.2), 4.92 (p = .27), 4.72 (p = 0.07), and 4.2 (p = 0.21) at 1, 2, 3, 4, 5 years, respectively.

CONCLUSION: In patients with RRMS, autologous non-myeloablative HSCT is an effective one-time therapy, while HSCT appears of less benefit for newly diagnosed SPMS.

PMID:34633525 | DOI:10.1007/s00415-021-10820-2

Noun Phrases in Title

  • Real-world application
  • autologous hematopoietic stem cell transplantation
  • 507 patients
  • multiple sclerosis
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