Abstract

The aim was to assess the cognitive dysfunction and physical disability after autologous hematopoietic stem cell transplantation (AHSCT), to explore the potential factors influencing disability regression after AHSCT and to estimate the safety of low-dose immunosuppressive therapy in highly active Multiple Sclerosis (MS) patients. In single-center prospective study patients who failed to conventional therapies for highly active relapsing MS underwent the AHSCT. The disability was followed up with Expanded Disability Status Scale and cognition with Brief International Cognitive Assessment for Multiple Sclerosis. Twenty four patients [18 (72.0%) female] underwent AHSCT. Two patients of 13 had one relapse during the first year and three patients—during the second year after AHSCT. Disability regression was found in 84.6% of patients. The scores of information processing speed and verbal learning were significantly higher at month 12 after AHSCT. The clinical variable that explained the disability regression at months 6 and 12 after AHSCT was the disability progression over 6 months before AHSCT. No transplant related-deaths were observed. Selective cognitive improvement was found after AHSCT in MS patients. The disability may be temporarily reversible after AHSCT in a significant proportion of highly active RMS patients if AHSCT is well-timed performed.

Highlights

  • The aim was to assess the cognitive dysfunction and physical disability after autologous hematopoietic stem cell transplantation (AHSCT), to explore the potential factors influencing disability regression after AHSCT and to estimate the safety of low-dose immunosuppressive therapy in highly active Multiple Sclerosis (MS) patients

  • Multiple Sclerosis (MS) is the most prevalent chronic autoimmune disease of the central nervous system (CNS) and is presumed to be the immune-mediated disease caused by autoreactive T c­ ells[1]

  • AHSCT ensures higher rates of disease activity (NEDA) than those achieved with any other disease-modifying treatments (DMT)

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Summary

Introduction

The aim was to assess the cognitive dysfunction and physical disability after autologous hematopoietic stem cell transplantation (AHSCT), to explore the potential factors influencing disability regression after AHSCT and to estimate the safety of low-dose immunosuppressive therapy in highly active Multiple Sclerosis (MS) patients. Despite advances in the current treatment of MS, some patients do not respond to available drugs and require other therapeutic strategies to control the disease activity and to prevent the progression of d­ isability[2,3,4]. During the last 25 years, severe autoimmune diseases including MS have been treated with immunosuppression and autologous hematopoietic stem cell transplantation (AHSCT)[5,6]. AHSCT is performed in patients with highly active disease course without adequate response to second line treatment, shorter disease duration and less d­ isability[20,21,22]. The benefit in progressive forms of the disease is more limited, patients with higher degrees of disability, or occasionally progressive disease course, can be considered if there is clear evidence of significant clinical and MRI disease a­ ctivity[19,23]

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