The NHS has announced that it will offer stem cell transplants that could cure patients living with thalassaemia, a severe and life-limiting inherited blood disorder.

Funded for the first time by the NHS, curative stem cell transplants will be eligible for patients living with the condition following new guidance from NHS England’s Clinical Advisory Group.

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.

The 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.

The procedure was previously only used as a treatment for children due to the potential risk of complications among adults.

However, thanks to new advances in transplant treatment, including better medications to manage patients before, during and after transplantation, the new guidance has recommended that the Allo-HSCT treatment be made available to eligible thalassaemia patients aged 18 or over.

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.

Furthermore, the disorder is more prevalent among Southern European, Middle Eastern, South American, Caribbean, Asian and South East Asian communities in the UK.

Providing access to Allo-HSCT treatment will play a significant role in reducing health inequalities for these populations.

“This remarkable milestone offers hope to adults with donor matches who were previously excluded from accessing a curative option,” said Romaine Maharaj, executive director, UK Thalassaemia Society.

Professor 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.”

In May, the NHS became the first healthcare system globally to introduce genetic genotyping blood-matching tests for transfusion-based treatments, including thalassaemia and sickle cell disease.

The new programme was delivered in partnership with NHS England and NHS Blood and Transplant to reduce the painful side effects of blood transfusion treatments for patients.

https://www.pmlive.com/pharma_news/nhs_to_offer_stem_cell_transplants_to_patients_with_thalassaemia_1504377