Thalassaemia: NHS to offer stem cell transplants to adults who need transfusions

Adults who have transfusion dependent thalassaemia are being given access to stem cell transplantations on the NHS, a treatment previously available only to children.Advances in transplant treatment, such as better drugs to manage a patient before, during, and after transplantation, mean that allogeneic haematopoietic stem cell transplant (HSCT) has been recommended for eligible adults over 18 in new guidance from NHS England’s clinical priorities advisory group.Transfusion dependent thalassaemia is a severe form of the condition, which was previously fatal in childhood. Allogeneic HSCT involves replacing the bone marrow stem cells of a patient with those from a matched sibling donor.There are 2281 people with thalassaemia registered on the National Haemoglobinopathy Registry in the UK, including 1332 over the age of 18, and it is thought that about 600 people will be suitable for allogeneic HSCT.Stephen Powis, NHS England medical director, said, “Thalassaemia can be an incredibly painful condition with difficult…
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