Our community were excited this week to hear about promising early results from the TIARA Trial taking place at King’s College Hospital and King’s College London, funded by The Aplastic Anaemia Trust and LifeArc.

Sam sits on the TIARA trial’s Steering Committee as a patient representative, alongside clinicians. His role is to bring the lay patient’s perspective and ensure the patient’s point of view is considered in the design of the study.

For example, Sam highlighted a need for additional budget for travel expenses – because participants need to travel into London numerous times, and may not feel safe on the Underground with a severely compromised immune system!

As a former AA patient himself, and as a Support Manager at The AAT, Sam knows very well the limitations of current treatments, and the hope that a brand new treatment could bring.

Currently, there are two standard first-line treatments for AA: immune-suppressive therapy, which uses drugs to dampen down abnormal immune responses (but this will only work in around two-thirds of patients); or a stem cell transplant, which is not a viable or safe treatment option for many people with AA. Those who fail to respond to treatment can experience significant worsening of their condition, and a reduction in quality of life as well as life expectancy.

Sam says, “AA is so rare with limited treatment options that haven’t really evolved very much – so the prospect of having a less intensive treatment with better success rates is really exciting – it could really make a difference for lots of people.”

Sam's experience with GvHD

Sam was first diagnosed with aplastic anemia when he was three years old. After treatment with immunosuppressant drugs, the condition seemed under control, but when he was 21 he discovered that he now had MDS and would need a bone marrow transplant after all. A transplant is the first line of treatment for children and young people diagnosed with severe aplastic anaemia in the UK.

The transplant was successful and saved Sam’s life. “I still have 100% chimerism, which means that my transplant worked perfectly. But the surrounding issues have meant I'm still always going into hospital, five years later.”

Sam has Graft Vs Host Disease GvHD, a common complication after transplant which occurs when transplanted donor cells recognize the recipient's tissues as foreign and attack them.

In some cases, milder GvHD can be a good thing after a transplant, but for others the effects can be debilitating.

Sam has chronic skin GvHD, which is painful and causes issues with his mobility. He has been receiving Extracorporeal Photopheresis (ECP) treatment for five years.

For GvHD treatment, Sam initially had to go into hospital for four hours for two consecutive days every two weeks. Five years on, this has reduced to two days every eight weeks.

After treatment, Sam often feels very tired and can need additional time off work.

Receiving treatment for such a long time after being cured by the transplant came as a shock to Sam. He remembers when he was preparing for transplant, his family were optimistic about having a curative treatment option. “I remember my dad joked, ‘blimey, I want a transplant as well - it sounds amazing!’”

Sam is glad he had the transplant and will always be grateful to his donor and medical team. But he feels that “when you hear stories about stem cell transplant, these elements are glossed over.”

The long recovery post-transplant

The length of time Sam’s been receiving GvHD treatment is unusual, and most people do not experience such long-term issues. But feeling unprepared for a long recovery is a common refrain: “I run our post-transplant AAT chat and I hear people say all the time ”they say my transplant has been a big success – but I still feel rubbish.”

“People often say they find the long recovery harder than the transplant itself and I agree.

The psychological and emotional side are a real challenge. After a transplant you want to move on, and get on with your life - because it’s curative! But then having ongoing treatment for so long because of side effects really takes its toll.”

Not available for everyone

Sam also works with people with AA who do not have access to curative treatment:

“Having a transplant worked for me, but I speak to lots of people for whom it isn’t an option. It’s disheartening when I speak to people with AA where the best treatment for them would be a transplant – but for whatever reason – comorbidities, age or not having the right match, they can’t have one.”

A patient’s ability to have a successful transplant can also vary according to their ethnicity. Anthony Nolan confirm that “Patients from minority ethnic backgrounds are more likely to have a rare or unique tissue type which can make it harder to find a genetically matched donor on the stem cell register. This may result in patients waiting longer for a transplant which can increase the risk of infection or their condition worsening.”

Exciting potential

It’s his experience of GvHD and understanding of these inequalities that make Sam so enthusiastic about the potential of the TIARA study.

“Obviously if you’re using your own cells – you're getting rid of that problem of having to find a donor. You’re also getting rid of that risk of Graft Vs Host, because I assume you could tolerate your own cells. I’m sure the new treatment would not be without other risks – but you can see why it’s so exciting for patients like me.”

The encouraging results so far have exceeded Sam’s expectations: “A Phase 1 trial like this is about patient safety. So to hear that some participants, even at this low dose, had a while where they were not reliant on blood transfusions feels huge.”