Health Technology Wales published a report this week supporting the routine use of autologous haematopoietic stem cell transplantation (AHSCT) for people with relapsing remitting multiple sclerosis (RRMS), who have recurrent symptoms despite treatment with disease modifying drugs (DMDs). The MS Trust contributed evidence to the appraisal board, and we are pleased that the report recommends that people in Wales who fit the criteria for AHSCT should be able to access the treatment.
What is AHSCT?
The standard treatments for RRMS are disease modifying drugs (DMDs) that target the immune system. They are most likely to be effective for the relapsing remitting phase of MS but are less effective as the disease progresses. Due to the relative lack of effective therapies for progressive MS, doctors aim to treat RRMS early and aggressively so that disease progression can be delayed or halted.
Autologous haematopoietic stem cell transplantation (AHSCT) is an intensive, inpatient, one-off treatment that aims to reset a patient’s immune system. It is a surgical procedure with risks of infection and is therefore proposed for people who have disease that is progressing rapidly and that is not well controlled by DMDs. AHSCT consists of a number of complex steps and it may take several months to recover from the procedure. Because AHSCT is so complicated, only specialist units can carry it out.
Who can get AHSCT?
Based on the estimated number of people in Wales with RRMS, clinical experts estimate that there are currently less than 10 people with inadequately controlled disease despite high efficacy DMDs who may be eligible for treatment with AHSCT. However, the report recognises that demand for AHSCT may increase if the clinical trials that are currently under way show further benefit to other people with MS. There are currently no AHSCT centres in Wales for patients with MS, although some people from Wales have received AHSCT treatment in England through their involvement in research studies.
In England there are two accredited centres (Sheffield and London) where AHSCT treatment for MS can be obtained on the NHS. These centres operate under European guidelines for stem cell transplantation in autoimmune diseases. They accept referrals on behalf of people with severe and rapidly evolving MS. In practice, this means that you must have had 2 relapses or 1 relapse and MRI activity in the last year, or have rapidly worsening symptoms despite being treated with a disease modifying drug.
What does this report mean for people with MS?
Health Technology Wales looked at evidence submitted from clinical trials, patient organisations like the MS Trust, and personal accounts from people who had undergone AHSCT in the UK and elsewhere. The evidence showed that people with RRMS who have AHSCT have delayed progression of their MS and a better quality of life than people who receive DMDs. AHSCT is also less expensive than DMDs.
It is not clear what the next steps are for AHSCT in the UK. This report follows a similar one published by NHS Scotland in October 2019, which also called for more equal access to AHSCT for people with MS across the UK. This may lead to more specialist centres being set up to offer AHSCT, but there is no indication in the reports how soon that might be.
If you think that you fit the criteria for AHSCT, and you wish to be referred to a specialist centre for a consultation, you will need to speak to your current neurologist first.
- Bone Marrow Transplant 55, 283–306 (2020). Full article Autologous haematopoietic stem cell transplantation and other cellular therapy in multiple sclerosis and immune-mediated neurological diseases: updated guidelines and recommendations from the EBMT Autoimmune Diseases Working Party (ADWP) and the Joint Acc
- JAMA. 2019;321(2):165-174. doi:10.1001/jama.2018.18743 Full article Effect of Nonmyeloablative Hematopoietic Stem Cell Transplantation vs Continued Disease-Modifying Therapy on Disease Progression in Patients With Relapsing-Remitting Multiple Sclerosis A Randomized Clinical Trial
- Bone Marrow Transplantation volume 54, 933–942(2019) Guidance for people considering AHSCT General information for patients and carers considering haematopoietic stem cell transplantation (HSCT) for severe autoimmune diseases (ADs): A position statement from the EBMT Autoimmune Diseases Working Party (ADWP), the EBMT Nurses Group, the EBMT Pati
In this short animated film, we explain how AHSCT works.
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