Inborn Errors Working Party (IEWP)
News & Events
Clinical outcome and quality of life in patients with ARPC1B deficiency managed conservatively or with hematopoietic stem cell transplantation
we are promoting a collaborative study to assess clinical outcome and quality of life in patients with ARPC1B deficiency managed conservatively or with hematopoietic stem cell transplantation
Brief rationale of the project:
ARPC1B deficiency results in a combined immunodeficiency, characterized by recurrent infections, allergic manifestations, and platelet abnormalities with bleeding tendency.
Although the only curative therapy currently available is haematopoietic stem cell transplantation (HSCT) there is a lack of data comparing the clinical outcome and quality of life of patients undergoing transplantation or treated conservatively.
In the Details.pdf attached you can find more informations about the project.
If you would like to participate and receive the Clinical Report Form (CRF) or have any questions about the study, please contact us at:
Compiled by: Daniela Sperl, Markus Seidel
The authors and ESID exclude any liability for the content or its correct reproduction in terms of errors or mistakes as well as typos that may have occurred when taking notes.
The Inborn Errors Working Party met for its annual meeting in Graz, Austria at a wonderful venue in the old city called Alte Universität. The program contained cutting-edge scientific advances in the treatment of inborn errors of immunity, metabolism and hematopoiesis. More than 120 participants from all over Europe and as far away as Australia engaged in interactive discussions.
This year, the annual meeting of the Inborn Errors Working Party (IEWP) will be happening in Paris, France, September 23-25th 2022 as a face-to-face event.
Due to the ongoing COVID19 pandemic, we announce that The 3rd International WAS symposium will be postponed to June 17th, 2022 in Munich, Germany. This event will enable researchers and clinicians to meet, present and discuss their work in the area of WAS and WASp research. Abstract submission remains open! More details about the event are available at www.was2022.org
Following the cancelation of WAS2020 due to the COVID19 pandemic, we are happy to announce that The 3rd International WAS symposium will take place on Friday Mars 18th, 2022 in Munich, Germany. This event will precede the EBMT2022 and will enable researchers and clinicians to meet, present and discuss their work in the area of WAS and WASp research. More details about the event are available at www.was2022.org