scholarly journals Autologous Hematopoietic Cell Transplant for Patients With Multiple Sclerosis

2021 ◽  
Vol 1 (8) ◽  
Author(s):  
Khai Tran ◽  
Hannah Loshak

Evidence from 2 randomized controlled trials and 4 retrospective studies with limited methodological quality suggests that treatment with autologous hematopoietic stem cell transplantation was associated with significant improvement in clinical outcomes (e.g., disease progression, clinical relapse), MRI outcomes, the composite outcome “No Evidence of Disease Activity,” and quality of life compared to disease-modifying therapies. Treatment with autologous hematopoietic stem cell transplantation was associated with no treatment-related mortality or life-threatening complications including progressive multifocal leukoencephalopathy. However, autologous hematopoietic stem cell transplantation was associated with expected short-term adverse events including febrile neutropenia, organ infections, sepsis, and viral reactivations; and long-term adverse events including the development of new autoimmune diseases, mainly thyroid disease. Both identified guidelines recommend the use of autologous hematopoietic stem cell transplantation as standard of care for the treatment of highly active relapsing-remitting multiple sclerosis patients refractory to disease-modifying therapies and suggest that the treatment may be appropriate for progressive forms of multiple sclerosis with an active inflammatory component. No cost-effectiveness studies were identified.

2021 ◽  
Vol 1 (7) ◽  
Author(s):  
Sinwan Basharat ◽  
Hannah Loshak

Autologous hematopoietic stem cell transplantation is an emerging health technology for treating multiple sclerosis, in particular for relapse-remitting forms of the disease. In 2019, transplant societies in the US and Europe both indicated the procedure may be provided as a standard of care for a subset of people with multiple sclerosis based on age and clinical classification criteria. The Canadian Multiple Sclerosis Working Group indicated in their 2020 treatment optimization recommendations that autologous hematopoietic stem cell transplantation may be considered for younger people (aged 18 to 31) who are early in their treatment course. The procedure is offered in 2 provinces, Alberta and Ontario, as an experimental treatment. Results from a phase III randomized controlled trial, a systematic review of single-arm trials, and retrospective analyses from European transplant registries provides evidence that the procedure may provide effective disease control for patients who show high disease activity despite receiving disease-modifying therapies. Evidence also shows that the safety profile of the procedure has been improving over the past 25 years. Although cost-effectiveness studies are not yet available, the one-time procedure may have important economic implications and may have the potential to provide cost-savings to health systems, as it may reduce the need for ongoing disease-modifying therapies that may be required for patients throughout their lives. At least 3 additional phase III randomized controlled trials are ongoing and aim to provide a stronger evidence base to inform optimal treatment regimens and appropriate eligibility criteria. As results from these studies develop, this horizon scan aims to provide health care stakeholders in Canada with an early overview of the technology and existing evidence, while highlighting considerations related to health equity, the need for multidisciplinary care, and transplant centre infrastructure that would be important if there is to be wider use across Canada should emerging evidence demonstrate value.


2012 ◽  
Vol 40 (11) ◽  
pp. 892-898 ◽  
Author(s):  
Jury L. Shevchenko ◽  
Alexey N. Kuznetsov ◽  
Tatyana I. Ionova ◽  
Vladimir Y. Melnichenko ◽  
Denis A. Fedorenko ◽  
...  

2019 ◽  
Vol 77 (8) ◽  
pp. 531-535 ◽  
Author(s):  
Elizabeth R. Comini-Frota ◽  
Bruna C. C. Marques ◽  
Caio Torres ◽  
Karoline M. S. Cohen ◽  
Eduardo Carvalho Miranda

ABSTRACT Multiple sclerosis (MS) is an immune-mediated disease of the central nervous system. Its treatment has focused on inflammation control as early as possible to avoid disability. Autologous hematopoietic stem cell transplantation (AHSCT) has been used for treating MS since 1996, with recent decisive results regarding benefits in long-term efficacy. Five patients followed up at an MS center in Belo Horizonte, Brazil, who had relapsing-remitting MS with high disease activity, underwent AHSCT between 2009 and 2011. They were evaluated clinically, with magnetic resonance imaging, and by the EDSS every six months after transplantation, up to July 2018. The patients were four women and one man, with ages ranging from 25-50 years, and time since disease onset ranging from 4-17 years at the time of the procedure. Four patients improved, one patient was stabilized, and all patients were free of disease activity after 5-9 years. Through improving patient selection and decreasing the time from disease onset, AHSCT could stop epitope spreading and disease progression. Despite multiple other therapeutic choices being approved for relapsing-remitting MS, AHSCT continues to be a treatment to consider for aggressive MS disease.


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