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The aplastic anaemia working party deals with acquired and congenital forms of marrow failure. Quantitatively acquired idiopathic aplastic anaemia constitutes the most important disease.
The working party has, over the years, been successful in running clinical trials and conducting observational research projects using the EBMT database. A salient feature of the AAWP activity is that data are not only collected on patients receiving stem cell transplantation but also on patients receiving immunosuppressive treatment. This is therefore the only group within the EBMT that provides data not only on transplantation but also on the most important non-transplant treatment option. This has led to a series of comparative studies where outcome of stem cell transplantation has been compared to non-transplant approaches.
The working party has recently closed a large trial on immunosuppressive treatment studying the question of the value of adding growth factors to anti-thymocyte globulin and cyclosporine and also the value of early re-treatment in the case of non-response.
Results of this trial will be presented shortly. A new trial project is in preparation that will include a treatment guideline for first and second line treatment including transplantation from alternative donors.
The working party is interested in maintaining a high level of representation of cases with these rare diseases in the database and to have representation in all European countries. Centres are encouraged to not only fill out the MED-A and MED-B forms on patients receiving transplants but also on patients receiving other types of therapy. There is a short form available on the website for centres submitting non-transplant data without the data management capacity to fill out the complete MED-A and MED-B forms. The form is available for download from the AAWP web page.
Jakob Passweg
Chair, AAWP
session