The NHS is to offer stem cell transplants to adults with thalassaemia for the first time, it has been announced.
The treatment could benefit more than 600 people in the UK who have transfusion-dependent disease. In total 1,332 adults are registered as having thalassaemia.
Stem cell transplants have been available to children but until now it was thought the risks to adults outweighed the potential benefits. NHS England said improvements in treatment, including additional medications, have made adult transplants viable.
Medical director Professor Sir Stephen Powis said: “Expanding the availability of stem cell transplants to adults living with thalassaemia is another vital step forward to help change the lives of those living with this deeply debilitating condition.
“Thalassaemia can be an incredibly painful condition with difficult symptoms for patients as well as the impact on their heart, liver and bones, and it’s fantastic that offering this evidence-based curative stem cell treatment can now offer new hope to help significantly improve patients’ quality of life.”
Romaine Maharaj, executive director of the UK Thalassaemia Society, said: “We celebrate the long-awaited approval of Allo-HSCT for adults with transfusion dependent thalassaemia. This remarkable milestone offers hope to adults with donor matches who were previously excluded from accessing a curative option.
“While it is a huge step in the right direction and a monumental win for thalassaemia, we also eagerly await the much-needed approval for gene therapies. Having both curative options available will grant more patients the chance to live transfusion-independent lives, enhancing both their quality of life and life expectancy.”
One of the beneficiaries could be Kirthana Balachandran, a 20-year-old medical student from West London. She was unable to find a matching donor as a child but is now hoping to continue the search.
She said: “When I was younger, we looked for a donor from my family, but no one was a match and the possibility just faded away. With today’s news, if we were able to find a donor, that would be an amazing possibility, as it could really change my life. I would never need to go for a blood transfusion again for my condition and I wouldn’t have to worry about the side effects from transfusions or my health in future either.
“It’s amazing that they’ve made this treatment option available for adults, because it means we can still have a chance of finding a donor, even at an older age.”
She added: “Sometimes I have muscle pain, back pain and I can even feel breathless or have palpitations when I walk uphill. It just depends on your haemoglobin level. When it’s low, I feel so tired and it can affect me at the most inconvenient times, like when I have exams. But when my levels are good, I don’t really feel any symptoms at all.”
Source: NHS England
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