Autologous haematopoietic stem cell transplant for patients with highly active relapsing remitting multiple sclerosis not responding to high efficacy disease modifying therapies

Recommendation for NHSScotland

Where patients understand and are willing to accept the demands, risks and uncertainties of treatment, autologous haematopoietic stem cell transplant (AHSCT) should be considered as a treatment option for patients with relapsing-remitting multiple sclerosis (RRMS) who have evidence of significant inflammatory disease activity that has not responded to adequate treatment with licensed high-efficacy disease modifying therapies (DMTs).

The evidence for efficacy and safety of AHSCT in patients with RRMS is from a collection of single-arm observational studies and one randomised controlled trial that has limitations in terms of its applicability to current standard of care in Scotland. Robust cost-effectiveness analysis is not available.

There should be equity of access across Scotland to the procedure and to appropriate follow-up.

Haematological centres offering AHSCT should have multi-disciplinary expertise in the management of multiple sclerosis, clear protocols for patient selection, and be appropriately accredited.

Enrolment of patients into clinical trials is encouraged wherever possible and outcomes of all procedures undertaken should be submitted to relevant audits/registries. Consideration should be given to developing Scottish national audit.

NHSScotland is required to consider the Scottish Health Technologies Group (SHTG) advice.

What were we asked to look at?

We were asked to look at the evidence surrounding autologous haematopoietic stem cell transplant (AHSCT) for patients with highly active relapsing remitting multiple sclerosis (RRMS) not responding to high-efficacy disease-modifying therapies (DMTs).

The annual incidence rate of multiple sclerosis in Scotland is 8.64 cases per 100,000 people, which represents a high incidence relative to other countries. Of the total cases, around 85% of patients have the relapsing remitting form of the disease.

A number of high-efficacy drug treatments have been approved for the treatment of relapsing remitting multiple sclerosis (for example ocrelizumab, alemtuzumab, natalizumab) but these therapies do not always work. For patients with highly active disease not responding to disease-modifying therapies, AHSCT may have a role in reducing disease progression.

Why is this important?

The annual incidence rate of multiple sclerosis in Scotland is 8.64 cases per 100,000 people, which represents a high incidence relative to other countries. Of the total cases, around 85% of patients have the relapsing remitting form of the disease.

A number of high-efficacy drug treatments have been approved for the treatment of relapsing remitting multiple sclerosis (for example ocrelizumab, alemtuzumab, natalizumab) but these therapies do not always work. For patients with highly active disease not responding to disease-modifying therapies, AHSCT may have a role in reducing disease progression.

Referred by

Strategic Planning and Clinical Priorities Team, Planning and Quality Division, Scottish Government