Photo: Stethoscope

HSCT:  what to expect

HSCT is a hugely promising treatment for MS, but it is also very aggressive. This means that it comes with high risks and there are lots of factors to consider.

If you're considering having HSCT, we recommend you talk to your neurologist about whether it's the right treatment for you. You can also read the European Group for Blood and Marrow Transplantation (EBMT) guidelines for HSCT on the EBMT website.

Am I eligible for HSCT?

HSCT is available on the NHS for people who meet very specific medical criteria. These are based on the EBMT guidelines for HSCT, but may differ a little from centre to centre.

Because HSCT targets the immune system, you need to have evidence of active inflammation (new MRI lesions) for doctors to refer you for treatment on the NHS.

Right now, HSCT is  recommended on the NHS for people who have tried two different DMTs (including one of the more aggressive DMTs, like natalizumab or alemtuzumab) which have failed to control their MS.

People with early progressive MS may be considered if there are still signs of active inflammation (either relapses or lesions on an MRI.

> Read more about getting treatment for MS

What happens during HSCT?

infographic shows HSCT stages
Anyone being considered for HSCT will first be reviewed by the specialist team at the treatment centre.

The treatment begins with drugs to encourage HSCs to move out of your bone marrow and into your blood. This is known as mobilisation, and allows them to be collected. The symptoms of MS can get temporarily worse during this stage. Around 10 days later, when there are enough stem cells in your blood, they will be removed and stored for later in the procedure.

Next, chemotherapy is given as an infusion to wipe out the immune system. This part of the procedure can take several days. Chemotherapy may be myeloablative (completely wipes out the immune system) or non-myeloablative (partially wipes out the immune system). The side effects of chemotherapy include nausea and vomiting, so you may be given drugs to control this.

Finally, your stem cells are transplanted back into your blood by a drip to help regrow the immune system. This usually takes place a couple of days after the chemotherapy, once the drugs have cleared your system.

The stem cells start making new blood and immune cells within 10 and 30 days of the transplant. As your immune system isn't working yet, you are more likely to get infections during this period. You're usually put on antibiotics and transfusions to support you.

Anyone having HSCT also has to spend around a month in an isolation room while their immune system rebuilds. This isolation can be lonely and challenging.

HSCT is an aggressive treatment, so recovery can take some time. Typically, people need between 3 and 6 months to recover from HSCT. But for some people, it can take more than a year to fully recover.

What are the risks of HSCT?

HSCT has seen some very positive results, but it is an aggressive treatment. This means it comes with high short and long-term risks and complications. These include:

  • an increased, long-term risk of developing infections
  • an increased risk of developing cancer and autoimmune conditions, such as thyroiditis
  • early menopause
  • fertility problems.

The chemotherapy has its own side effects too. These include an increased risk of bleeding and bruising, fatigue, loss of appetite and hair loss.

For people with a higher level of disability before the transplant, chemotherapy can also do more harm than good. Chemotherapy treatments can lead to a further loss of mobility and worsened neurological function.

According to a European register, one or two in every 100 people (1.3%) having HSCT in clinical trials have died as a result of the treatment. But since 2005, the mortality rate has decreased to around 1 in 330 (0.3%).

What are the benefits of HSCT?

Clinical trials into HSCT have shown that HSCT is able to reduce relapses – mainly for people with relapsing MS. Some people experience more stable or improved symptoms, or an improved level of disability. But these improvements don’t always last.

For progressive MS, we’ve seen some encouraging results for people treated early on in their condition, and where there’s still evidence of inflammation (on an MRI, or through relapses). HSCT has been shown to slow clinical progression in a few people with early secondary progressive MS.

Unfortunately, HSCT has not been as effective for people with progressive MS who no longer show signs of inflammation and who have high levels of disability.

> Read more about HSCT clinical trials

Accessing HSCT

There are different routes to accessing HSCT for people in the UK, including through the NHS. We’re working to help people with MS understand these routes and to get access to the right treatment for them.

Through the NHS

HSCT is available on the NHS. At the moment, it’s only considered as a third line treatment for people in England who meet very specific medical criteria, including evidence of inflammation, such as active lesions on recent MRI scans. A third line treatment means it will only be considered for people who have tried two different DMTs (including one of the more aggressive DMTs) which have failed to control their MS. People in other nations who are eligible may be referred to England for HSCT, but you may need to speak to your neurologist about an individual funding request for the treatment. The main sites offering HSCT for MS in the UK are Sheffield and London (King’s College and Imperial College). The criteria vary slightly between sites, so it’s worth checking with your neurologist about which site would be best suited to your situation.

Going private

HSCT can be accessed privately at the centres in Sheffield and London. Again, the accessibility criteria varies between centres.

Going abroad

If you’re not eligible for HSCT on the NHS, it’s possible to get the treatment abroad. There are sites in Mexico, Russia and India that we know offer the treatment. This option is very expensive, and not always safe, as not all clinics work to the same safety standards as the UK. There is also no guarantee that the treatment will work. We would urge anyone considering going abroad for treatment to first talk their options through with their MS nurse or neurologist.

> Read more about having HSCT outside the NHS

HSCT access and the MS Society

We’re working alongside experts in the field to help ensure people with MS are able to access treatments that are right for them at the right time. This includes HSCT.

We are currently scoping potential sites of expertise where HSCT could be established. We have also been speaking to the National Institute for Health and Care Excellence (NICE) about when they plan to review HSCT for the NHS. And we’re working to clarify the access situation in Northern Ireland, Scotland and Wales.