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Potential cure for adults with immune system disorders

Bone marrow transplants (BMT) for patients with life threatening immune disorders, which up until now have only be carried out on children are safe and effective in treating adults, according to a new study. Historically the few adults treated with BMT have had poor outcomes, due in part to the impact of severe, recurrent infections over many years preceding the transplant.

The study reported outcomes from the world’s largest series of adult patients with inherited immunodeficiencies who had been treated with allogeneic bone marrow transplantation. Unexpectedly, the patients had a survival rate of 85% at three years post-transplant. These excellent outcomes were achieved with reduced intensity conditioning, no observation of early or late graft rejections, and importantly, resolution of infections and a re-establishment of good immune function was achieved.

Using bone marrow transplants to treat primary immunodeficiencies (PID) in adults could significantly change clinical practice.

A team of 21 scientists from UCL, UCLH and the Royal Free London carried out the study. Senior author and BRC supported researcher Professor Emma Morris said: “The results of our study show we can achieve comparable success in adult patients as that previously seen in much young patients and that bone marrow transplantation for adults is safe when delivered in a specialist centre. This should be considered as a potential cure for all adult PID patients with an appropriate donor and a sufficiently severe clinical picture”.

PIDs are a rare group of inherited disorders that are characterised by dysfunction of the adaptive or innate immune system, normally arising from genetic mutations in blood stem cells. Bone marrow transplants have been a major therapeutic option for paediatric patients since 1968, however scientific advances now pave the way for investigation and implementation of bone marrow transplants in adults.

To read the full paper please visit Blood Journal.