RARE Daily

NHS to Offer Stem Cell Transplants to Cure Life-limiting Blood Disorder

November 30, 2023

Rare Daily Staff

Patients in the United Kingdom with severe inherited blood disorders will be offered stem cell transplants on the U.K.’s National Health Service that could cure their condition and help avoid life-long blood transfusions.

Thalassemia is a rare disorder that affects the production of hemoglobin in the blood, leading to severe anemia and debilitating tiredness, with patients needing to have blood transfusions every two to four weeks to survive—which has a major impact on their quality of life.

Now, curative stem cell transplants will be funded by the NHS for the first time for adult patients with thalassemia following new guidance.

The procedure – called allogeneic hematopoietic stem cell transplant (Allo-HSCT) – involves replacing the bone marrow stem cells of a patient with ones from a matched sibling donor. Stem cells are given to the patient via an intravenous infusion which helps to re-establish healthy blood cell production.

Previously, this type of treatment was offered only to children because of the potential risk of complications among adults. But following advances in transplant treatment such as better medications to manage a patient before, during and after transplantation, new guidance from NHS England’s Clinical Priorities Advisory Group has recommended the treatment be made available for the first time to eligible thalassemia patients over the age of 18.

It is estimated that there are more than 600 adults across the U.K. who have transfusion-dependent thalassemia – a severe form of the condition, which was previously fatal in childhood, many of whom could be eligible for the stem cell transplant. Overall, there are 2,281 people with thalassemia registered on the National Haemoglobinopathy Registry in the United Kingdom, including 1,332 over the age of 18.

Thalassemia is more prevalent amongst Southern European, Middle Eastern, South American, Caribbean, Asian and Southeast Asian communities in the United Kingdom, and providing access to this treatment will play a role in reducing health inequalities for these populations.

“Expanding the availability of stem cell transplants to adults living with thalassemia is another vital step forward to help change the lives of those living with this deeply debilitating condition,” said Stephen Powis, medical director at NHS England. “Thalassemia 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.”

Earlier in 2023, the NHS became the first healthcare system in the world to provide blood group genotyping for people with thalassemia and sickle cell disease. This is a detailed DNA analysis of each patient’s blood group to match more accurately those in need of transfusions to donated blood.

The new program, 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.

Photo: Stephen Powis, medical director at NHS England

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