Allogeneic stem cell transplantation compared to conservative management in adults with inborn errors of immunity
Summary of key findings
A collaboration with UCL (Institue of Immunity and transplantation) and medical teams in France matched adult patients with primary immunodeficiencies who had undergone a bone marrow transplant (BMT) to patients with the same disease/medical problems who had not undergone a transplant and looked at their outcomes. It included 20 CGD patients (who were each matched to a CGD patient who was not transplanted).
The findings were that although transplant carries a risk of mortality, if patients survive the transplant, then they remain healthy. In patients who did not have a transplant their disease caused progressive medical problems and ultimately morality from their disease. We also noticed that transplant outcomes were much better if the transplant was performed in an expert centre for primary immunodeficiencies (UCLH or Newcastle in the UK). Overall, the paper suggests that a bone marrow transplant although risky is beneficial in older patients with primary immunodeficiencies as it prevents the progressive decline seen in patients who do not undergo transplant.
The abstract from the paper can be found here Allogeneic stem cell transplantation compared to conservative management in adults with inborn errors of immunity - PubMed (nih.gov)
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