Stem cell transplants to cure blood disorder

Stem cell transplants are to be offered to patients with Thalassaemia on the NHS.

The treatment could cure the long-term condition and help avoid life-long blood transfusions.

Thalassaemia affects the production of haemoglobin in the blood, leading to severe anaemia and debilitating tiredness, with patients needing to have blood transfusions every two to four weeks to survive.

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

The procedure – called allogeneic haematopoietic 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.

The treatment was previously only offered to children because of the potential risk of complications in adults. However, advances in transplant treatment including better medications to manage a patient before, during and after transplantation, mean the treatment is now being recommended for adults as we'll.

Professor Sir Stephen Powis, NHS England Medical Director, 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”.