ABSTRACT
Introduction: Autologous haematopoietic stem cell transplantation (auto-HSCT) has been performed for severe multiple sclerosis (MS) refractory to standard therapy with increasing frequency worldwide. However, experience in Asia employing this modality in MS has been limited. In this review, we explored the pathophysiology of autoimmunity and the underlying rationale for auto-HSCT in treating autoimmune diseases including MS, as well as existing published pre-clinical and clinical data. We aimed thereby to better understand the utility of treating MS with auto-HSCT and the feasibility of this procedure in Singapore. Methods: A Medline search was performed with the terms “haematopoietic stem cell transplantation”, “multiple sclerosis” and “autoimmune diseases” from 1996 to 2005. Both original papers and review articles were considered. Main Findings: The majority of publications were from Europe or the United States and most clinical series from single centres had relatively small numbers of patients. Worldwide, the number of patients reported has been less than 300 since 1997. Existing data support the feasibility and promise of this procedure and ongoing Phase III trials may serve to confirm this initial experience. Conclusion: Pre-clinical and early clinical data support the rationale for immunoablative therapy for autoimmune disorders. Auto-HSCT for severe MS is a feasible procedure and can be safely performed in centres with experience managing HSCT patients.
Multiple sclerosis (MS) is the most common cause of chronic neurological disability in young adults. It is characterised by a variable disease course with multiple sites of inflammation and demyelination within the central nervous system (CNS). Studies of its natural history suggest that patients may also have a reduced life expectancy1 and about 50% of patients will require walking aids within at least 15 years of disease onset.2-4 As the disease is widely accepted to be of autoimmune aetiology, the mainstays of therapy have been immunosuppressive or immunomodulatory agents. These may alter the rate of progression of disease but none have so far been curative.5 In this review, we discuss the increasing use of haematopoietic stem cell transplantation (HSCT) over the last decade for the treatment of several autoimmune diseases, including MS.
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