Inborn Errors Working Party (IEWP)

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Welcome to the Inborn Errors Working Party (IEWP)!

The ESID Inborn Errors 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 hematopoietic stem cell transplantation (HSCT) and gene therapy for inborn errors of immunity through:

1) enhanced interaction between centers involved in the application of these cellular therapies
2) undertaking retrospective and prospective collaborative studies to look at the outcome of HSCT for these conditions
3) education of individuals involved in the care of severe inborn errors of immunity
4) development and publication of HSCT guidelines for treatment of inborn errors of immunity
5) the collaborative use of registries to document and analyze 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 at high risk of rapidly developing life-threatening complications, particularly from infections or inflammation. 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 of different societies and registries. 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 3 day meeting in the autumn. The autumn meeting provides an opportunity to discuss work in progress, decide on common protocols and guidelines for treatment, formulate prospective and retrospective studies, and exchange experience. Members from outside Europe are welcome to contribute to the meeting and the group.