Autologous haematopoietic stem cell transplantation
Topic Status Complete
Autologous haematopoietic stem cell transplantation for previously treated, relapsing-remitting multiple sclerosis.
Outcome of the appraisal
The evidence supports the routine adoption of autologous haematopoietic stem cell transplantation (AHSCT) for people with relapsing-remitting multiple sclerosis (RRMS), in patients who have recurrence of symptoms despite previous treatment with disease modifying therapies (DMTs).
Why was this topic appraised?
Relapsing‑remitting multiple sclerosis (RRMS) is a condition that is characterised by a recurring pattern of disabling neurological symptoms. Over time, approximately two thirds of patients develop progressive disability and the diagnosis evolves into secondary progressive multiple sclerosis. Standard treatment for RRMS are immune system targeted disease modifying therapies (DMTs) that are most likely to be effective for the relapsing remitting phase of MS but are less effective as the disease progresses. Due to the lack of effective therapies for progressive MS, it is important 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. Based on the estimated number of people in Wales with RRMS, clinical experts estimate that there is a small cohort of people with inadequately controlled disease despite high efficacy DMTs who may be eligible for treatment with AHSCT. No AHSCT for patients with MS currently exists in Wales, although some patients from Wales have received AHSCT treatment in England through their involvement in research studies.
Plain language summary
Multiple sclerosis (MS) is condition of the central nervous system that can affect the brain, brain stem and spinal cord that gradually gets worse over time. MS causes inflammation that damages the cells of the central nervous system. People with MS can experience fatigue, movement disorders, visual problems, reduced mobility, bladder problems and mental health problems. There are several forms of disease modifying therapy (DMTs) medicines that are given to patients with MS.
Autologous Haematopoietic Stem Cell Transplantation (AHSCT) is a one-off procedure that aims to reset a patient’s immune system and so stop disease progression. It is a surgical procedure with risks of infection and is therefore proposed for patients who have disease that is progressing rapidly and that is not well controlled by DMTs. 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.
Health Technology Wales looked for evidence that Autologous Haematopoietic Stem Cell Transplantation (AHSCT) is an effective and cost effective treatment for previously treated, relapsing-remitting multiple sclerosis (RRMS). Overall, the evidence showed that AHSCT has the potential to improve the condition. Patients with RRMS who have AHSCT have a better quality of life than people who receive DMTs. AHSCT is also less expensive than DMTs.
The evidence supports the routine adoption of autologous haematopoietic stem cell transplantation (AHSCT) for patients with previously treated relapsing-remitting multiple sclerosis (RRMS) who have had symptom recurrence.