Autologous hematopoietic stem cell transplantation for multiple sclerosis: A current perspective

Bose, Gauruv; Thebault, Simon; Rush, Carolina; Atkins, Harold L.; Freedman, Mark S.

doi:10.1177/1352458520917936

Cited by 10 publications

(16 citation statements)

References 44 publications

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“…It is the largest and only phase III RCT with AHSCT for the treatment of MS to have been completed to date. 18 The trial enrolled 110 participants (n = 55 in each arm) who were randomized to receive either AHSCT (non-myeloablative regimen) or standard DMTs available at the time of the study. The authors of the MIST trial described significantly better outcomes in disability progression, rate of relapses, and quality of life in the AHSCT intervention arm compared to the DMT arm.…”

Section: Summary Of the Evidencementioning

confidence: 99%

“…20,40 Similar to most cell transplant procedures, there is risk of respiratory and urinary tract infections, gastrointestinal damage, and organ damage. 18 Intermediate-intensity regimens using BEAM or CP are considered to have less severe adverse effects than high-intensity regimens that use BU, which may increase the risk of severe liver toxicity, requiring intensive care support. 18,40 Late adverse events, reported 6 months or more following the procedure, include a 2% risk of malignancies and a 5% to 26% risk of secondary autoimmune dysfunction, in particular thyroid disease, as reported by various single-arm and retrospective studies.…”

Section: Safetymentioning

confidence: 99%

“…[14][15][16][17] Autologous hematopoietic stem cell transplantation (AHSCT), also referred to autologous hematopoietic cell transplantation, is a well-established immune reconstitution therapy for many blood cancers and is an emerging therapy for treating people with MS and other autoimmune disorders. 18,19 The procedure has the potential to provide a therapeutic option for people with aggressive or highly active RRMS for whom drug-based DMTs have had limited effect in disease control. AHSCT is not currently offered as part of routine care and protocols about the procedure are still be refined in research trials.…”

Section: Introductionmentioning

confidence: 99%

“…As the therapy is intended to be a one-time procedure (compared to ongoing DMTs), AHSCT may alleviate the need for DMTs that may cause adverse events and incur recurring health care costs. 18 In particular, given that MS onset tends to be in one's 30s, if the safety and effectiveness of AHSCT can be wellestablished, it has the potential to improve the quality of life and reduce the treatment-related side effects and financial cost associated with DMTs over a longer time period. 20 Although research about the type and dosing of regimens and eligibility criteria for AHSCT is emerging, guidelines from Canada, the US, and Europe recommend that AHSCT may be offered as a treatment option for a subset of patients with MS who qualify based on their age and clinical presentation.…”

Section: Introductionmentioning

confidence: 99%

“…19 European transplant guidelines recommend only using BEAM-ATG or CP-ATG (most common), while in Canada, the BU-CP-ATG regimen is the primary regimen used. 18,19 Research trials directly comparing the safety and effectiveness of different regimens are currently lacking but trials are underway to determine the optimal dosing for individual regimens. 20 Step 4.…”

Section: Introductionmentioning

confidence: 99%

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Autologous Hematopoietic Stem Cell Transplantation for the Treatment of Multiple Sclerosis

Basharat¹,

Loshak²

2021

cjht

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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.

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Section: Summary Of the Evidencementioning

confidence: 99%