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[Clinical outcome 6 years after autologous hematopoietic stem cell transplantation in multiple sclerosis]
A Saiz1, Y Blanco, J Berenguer
1Servicio de Neurología, Hematología Hospital Clínic, Institut d'Investigació Biomèdica August Pi i Sunyer (IDIBAPS), Universitat de Barcelona, Barcelona. asaiz@clinic.ub.es
Insights
Autologous hematopoietic stem cell transplantation (AHSCT) may stabilize severe multiple sclerosis (MS) long-term. While not curative, this experimental treatment showed prolonged disease stabilization in patients with aggressive MS.
Area of Science:
- Neurology
- Immunology
- Stem Cell Research
Context:
- Severe multiple sclerosis (MS) presents significant disability.
- Autologous hematopoietic stem cell transplantation (AHSCT) is an experimental therapy for aggressive MS.
- Limited long-term data exists on AHSCT outcomes in MS.
Purpose:
- To evaluate the long-term clinical outcomes of AHSCT in patients with severe MS.
- To assess progression-free survival and disease activity after AHSCT.
- To identify any long-term complications associated with the AHSCT procedure.
Summary:
- This study followed 14 severe MS patients who underwent AHSCT (cyclophosphamide, carmustine, antithymocyte globulin, CD34+ selection).
- At a median 6-year follow-up, 6-year progression-free survival was 62.5%, with only 2 patients showing new T1 lesions.
- No significant long-term complications were observed, suggesting AHSCT can alter the disease course.
Impact:
- AHSCT offers a potential strategy for prolonged stabilization in aggressive multiple sclerosis.
- The findings suggest AHSCT can modify the natural history of severe MS.
- This research provides crucial long-term safety and efficacy data for AHSCT in MS treatment.
Introduction:
Autologous hematopoietic stem cell transplantation (AHSCT) remains as an experimental treatment for severe forms of multiple sclerosis (MS). We describe the clinical outcome of 14 patients included in a protocol of AHSCT after a median follow-up period of 6 years.
Methods:
14 patients (5 relapsing-remitting and 9 secondary progressive) with a median number of relapses in the year before of 3 (1-7), Expanded Disability Status Scale (EDSS) of 6 (4.5-6.5) and decile of the multiple Sclerosis Severity Store (MSSS) 9 (7-10) were included. The procedure included carmustine, cyclophosphamide, antithymocyte globulin and T-cell depletion by CD34+ selection.
Results:
The 4.5-year progression-free survival was 71%. The 6 year actuarial probability of progression-free survival was 62.5% and the disease activity-free survival of 7.1%. The median EDSS was 6 (4-8.5) and the MSSS 8 (5-10). Only 2 patients presented enhanced T1 lesions. No long-term complications related to the procedure were observed.
Conclusion:
AHSCT cannot be deemed a curative treatment but may cause prolonged stabilisation or change the aggressive course of the disease.
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