European Society for Immunodeficiencies

The ESID Bone Marrow Transplantation and Gene Therapy Working Party works very closely with the EBMT Inborn Errors Working Party (IEWP). The aim of the working party is to improve the outcome of transplant and gene therapy for severe congenital immunodeficiencies through:

1) enhanced interaction between centres involved in the application of BMT to treat severe congenital immunodeficiencies
2) undertaking retrospective collaborative studies to look at the outcome of transplant for these conditions
3) education of individuals involved in the care of severe immunodeficiencies
4) development of guidelines for treatment of severe congenital immunodeficiencies
5) the development and use of a common registry to document and analyse results of treatment

These conditions have a number of challenging aspects in common: their recognition and correct identification may be difficult, requiring special clinical experience and laboratory expertise. Patients are usually infants at high risk of rapidly developing life-threatening complications, particularly from infections. Their management requires a well-coordinated and structured approach and the prognosis can be extremely poor unless haematopoietic stem cell transplantation or more recently gene therapy can be applied in a timely and appropriate manner.

The rarity of the conditions is such that collection of meaningful data is only possible through collaboration between centres and the strength of the WP has been the excellent collaborative approach taken by European transplant groups. This has resulted in a number of major publications which have been extremely important in guiding management both in Europe and worldwide.

Members of the WP meet at least twice yearly, once at the main EBMT meeting in the spring and then a dedicated 2 day meeting in the Autumn. The autumn meeting provides an opportunity to discuss work in progress, decide on common protocol and guidelines for treatment, formulate prospective and retrospective studies, and exchange experience.