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NHS to offer stem cell transplants to patients living with rare blood disorder

The new treatment could cure adults living with thalassemia and help them avoid life-long blood transfusions

Hundreds of adults diagnosed with thalassaemia, severe inherited blood disorder, could benefit from stem cell transplants to cure their condition, under new guidelines by the NHS.

The procedure was previously only used as a treatment for children due to the potential risk of complications among adults. However, the guidance notes that following new advances in transplant treatment, including better medications to manage patients before, the treatment can be made available to eligible thalassaemia patients aged 18 or over.

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The stem cell transplant procedure, known as allogeneic haematopoietic stem cell transplant (Allo-HSCT), involves replacing the bone marrow stem cells of patients with ones from a matched sibling donor. Officials said this treatment would mean patients no longer require lifelong blood transfusions.

‘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 their quality of life,’ said Professor Sir Stephen Powis, NHS England medical director.

Thalassaemia is a rare disorder that affects the haemoglobin in the blood and requires patients to have blood transfusions every two to four weeks. Across the UK, more than 600 adults are estimated to be living with transfusion-dependant thalassaemia. On the National Haemoglobinopathy Registry alone, there are 2,281 people registered with the condition, 1,332 of whom are over the age of 18.

People diagnosed with thalassaemia have lauded this move. Kirthana Balachandran, a 20-year-old medical student from West London, was diagnosed with thalassaemia when she was three months old. Although she previously had the potential to receive a stem cell transplant as a child and teenager, no matched donors were found, and she still requires blood transfusions every three weeks and medication to manage her condition.

‘My condition affects me a lot,’ she said. ‘But 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,’ said Kirthana.

This news also comes after a significant achievement by NHS. Earlier this year, the NHS became the first healthcare system in the world to provide blood group genotyping for people with thalassaemia and sickle cell disease. The treatment, known as Casgevy, works by editing the faulty gene in a patient’s bone marrow stem cells so that the body produces functioning haemoglobin.

The landmark new programme, delivered in partnership by NHS England and NHS Blood and Transplant, will help ensure patients receive the best treatment for them, reducing the risk/impact of reactions to donor blood and the development of antibodies that attack the donor blood cells.