Autologous hematopoietic stem cell transplantation for multiple sclerosis: A current perspective

The most effective treatment at halting inflammation in patients with highly active multiple sclerosis (MS) is immune ablation followed by autologous hematopoietic stem cell transplantation (AHSCT). Better patient selection and supportive management, as well as advances in conditioning regimens have resulted in improved safety with AHSCT. However, which comorbidities or prior therapies increase the risks associated with AHSCT still need to be determined. In addition, there is still debate as to which AHSCT conditioning regimen offers the best balance of long-term efficacy and safety. New studies comparing AHSCT with highly effective disease-modifying therapies will help to inform on the ideal placement of AHSCT in the treatment algorithm. Currently, many centers are experienced and use AHSCT to treat select patients with MS, contributing to ongoing registries and clinical trials which will help answer these questions.

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Can multiple sclerosis be cured? A case of highly active relapsing multiple sclerosis treated with autologous hematopoietic stem-cell transplantation 13 years ago

Multiple Sclerosis and Related Disorders ◽

10.1016/j.msard.2020.102253 ◽

2020 ◽

Vol 44 ◽

pp. 102253 ◽

Cited By ~ 1

Author(s):

Jan Lycke ◽

Markus Axelsson

Keyword(s):

Multiple Sclerosis ◽

Stem Cell ◽

Hematopoietic Stem Cell Transplantation ◽

Stem Cell Transplantation ◽

Cell Transplantation ◽

Hematopoietic Stem Cell ◽

Hematopoietic Stem ◽

Highly Active ◽

Relapsing Multiple Sclerosis

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Low intensity lympho-ablative regimen followed by autologous hematopoietic stem cell transplantation in severe forms of multiple sclerosis: A MRI-based clinical study

Multiple Sclerosis Journal ◽

10.1177/1352458514564484 ◽

2015 ◽

Vol 21 (11) ◽

pp. 1423-1430 ◽

Cited By ~ 29

Author(s):

Daniela Curro’ ◽

Luisa Vuolo ◽

Francesca Gualandi ◽

Andrea Bacigalupo ◽

Luca Roccatagliata ◽

...

Keyword(s):

Multiple Sclerosis ◽

Stem Cell ◽

Hematopoietic Stem Cell Transplantation ◽

Stem Cell Transplantation ◽

Cell Transplantation ◽

Hematopoietic Stem Cell ◽

Conditioning Regimen ◽

Complete Suppression ◽

Hematopoietic Stem ◽

Low Intensity

Background: Autologous hematopoietic stem cell transplantation (AHSCT) has been successfully used to treat aggressive forms of multiple sclerosis (MS) that are unresponsive to approved therapies. In the last years, in view of the risk of mortality related to the procedure, the utilization of low-intensity conditioning regimens has been considered. Objective: To report magnetic resonance imaging (MRI) and clinical data in a small cohort of patients treated with a low-intensity lympho-ablative regimen, followed by AHSCT. Methods: Seven patients affected by relapsing–remitting MS (RRMS) underwent AHSCT, with cyclophosphamide 120 mg/kg in 2 days as the conditioning regimen; and were then followed with serial MRI evaluations until 36 months, with clinical evaluations until 60 months. Results: The mean number of gadolinium (Gd)-enhancing lesions significantly decreased after treatment, but a complete suppression of inflammatory activity was not obtained. No deaths occurred, but every patient developed adverse events, although not severe. After 5 years of follow-up, two patients remained stable, one patient markedly improved and four patients had a mild progression of the disease. Only one patient experienced a relapse after treatment. Conclusion: A low-intensity conditioning regimen with AHSCT has a profound effect on MRI inflammation and relapses, but is not able to completely abrogate MRI activity and disease progression of aggressive RRMS.

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