Hematopoietic stem cell transplantation for multiple sclerosis. A retrospective multicenter study.

Area of Science:

• Neuroscience
• Immunology
• Hematology

Background:

• Autologous hematopoietic stem cell transplantation (HSCT) for multiple sclerosis (MS) is being investigated based on animal models and clinical observations.
• Early phase studies aimed to assess the feasibility and efficacy of HSCT in progressive MS patients.

Purpose of the Study:

• To evaluate the outcomes of autologous HSCT in a large cohort of patients with progressive multiple sclerosis.
• To determine the safety, efficacy, and long-term disease control associated with HSCT in MS management.

Main Methods:

• Eighty-five patients with progressive MS underwent autologous HSCT across 20 European centers.
• High-dose chemotherapy regimens were employed, with stem cell mobilization using cyclophosphamide and/or growth factors.
• Stem cell transplants were purged of lymphocytes in 52 patients, with a median follow-up of 16 months.

Main Results:

• A 3-year overall mortality risk of 10% was observed, primarily due to toxicity and infection.
• Twenty-one percent of patients experienced neurological improvement (EDSS score reduction ≥1 point).
• Confirmed progression-free survival at 3 years was 74%, with reduced disease activity on post-transplant MRI (8%).

Conclusions:

• Autologous HSCT demonstrates positive early results and feasibility for managing progressive MS.
• Significant mortality risks necessitate further research to optimize patient selection and reduce transplant-related complications.
• Multicenter data are crucial for planning future trials to mitigate HSCT-related mortality in MS patients.