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Real-world application of autologous hematopoietic stem cell transplantation in 507 patients with multiple sclerosis.
- Source :
-
Journal of neurology [J Neurol] 2022 May; Vol. 269 (5), pp. 2513-2526. Date of Electronic Publication: 2021 Oct 11. - Publication Year :
- 2022
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Abstract
- Objective: To investigate the results of real-world application of non-myeloablative autologous HSCT for multiple sclerosis (MS).<br />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.<br />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.<br />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.<br /> (© 2021. The Author(s), under exclusive licence to Springer-Verlag GmbH Germany.)
Details
- Language :
- English
- ISSN :
- 1432-1459
- Volume :
- 269
- Issue :
- 5
- Database :
- MEDLINE
- Journal :
- Journal of neurology
- Publication Type :
- Academic Journal
- Accession number :
- 34633525
- Full Text :
- https://doi.org/10.1007/s00415-021-10820-2