HSCT for MS - Has it Come of Age?
Review from Dr Majid Kazmi
Consultant Haematologist
HSCT has been used for over 50 years in treatments of serious blood disorders such as leukaemia. An early observation was that in patients with a concurrent autoimmune illness the autoimmune disease often went into prolonged remission after HSCT. The reasons behind this were not fully understood for some time until we had a better understanding of the role of the immune system in both blood cancers and autoimmune disease.
In 1995 the first HSCT for multiple sclerosis alone was performed following successful animal experiments (using a mouse model with similar features to MS).
Despite early successes the main barrier to uptake of this technology was the associated risks in patients with advanced disability that were being put forward for this procedure when all else had failed to work. The early mortality rates approached 5-7%. For the neurology community this was unacceptable and the role of HSCT in MS followed a relatively niche course in a few centres with interested neurologists and haematologists.
Only in the last decade and particularly last 5 years has the evidence base for the success of HSCT in MS increased to the point where it has now become mainstream. Several high impact publications from around Europe, US and Canada have confirmed the durable responses seen in the majority of patients with MS receiving HSCT. With better patient selection the risk has also diminished and is now less than 1% in relapse -remitting MS (RRMS) with EDSS less than 4.5.
The largest randomised study to date comparing HSCT vs best available therapy in RRMS showed superiority of HSCT (MIST Trial- presented at EBMT 2018) over disease modifying therapies (DMT) including Tysabri. The follow-up remains short and the results are yet to be formally published but it has sparked significant interest from neurologists, patients and the Media.
HSCT for MS is commissioned by NHS England and is now in the draft Association of British Neurologists’ guidelines as an option for selected patients. It is also now routinely commissioned in Sweden. Several new prospective studies comparing HSCT against a DMT are underway or about to start and almost 1000 transplants for MS have been performed in Europe alone.
Exciting times lay ahead with the potential for a one-off treatment providing sustained response for patients with MS and for the first time significantly altering its natural history.
HSCT for MS has come of age at long last.
Consultant Haematologist
Guy’s & St Thomas’ NHS Foundation Trust
HCA London Bridge Private Care at Guys