Please find a continually updated list of IEWP studies
If you want to contribute to these studies please contact [email protected] for details.
Ongoing studies (last updated 24.4.24):
| study subject | status |
| FHLH2/3 | recruiting |
| TTC7A | recruiting |
| ARPC1B | recruiting |
| C1Q deficiency | recruiting |
| NFkB2 | recruiting |
| osteopetrosis | in preparation |
| long-term outcome of Chediak-Higashi | in preparation |
| Cartilage hair hypoplasia | in preparation |
| NBS | recruiting |
| IL10R | recruiting |
| CARD9 | recruiting |
| CVID | recruiting |
Recent publications :
(last updated 24.4.24)
JAK inhibitor treatment for inborn errors of JAK/STAT signaling: An ESID/EBMT-IEWP retrospective study. J Allergy Clin Immunol, 2024
Busulfan-fludarabine- or treosulfan-fludarabine-based conditioning before allogeneic HSCT from matched sibling donors in paediatric patients with sickle cell disease: A study on behalf of the EBMT Paediatric Diseases and Inborn Errors Working Parties. Br J Haematol, 2024
Hypomorphic RAG deficiency: impact of disease burden on survival and thymic recovery argues for early diagnosis and HSCT. Blood, 2023
Predicting Patient Death after Allogeneic Stem Cell Transplantation for Inborn Errors Using Machine Learning (PREPAD): A European Society for Blood and Marrow Transplantation Inborn Errors Working Party Study. Transplant Cell Ther, 2023
Decision making on HSCT in patients with hemoglobinopathies; an EBMT Pediatric Diseases Working Party and Inborn Errors Working Party scenario-based survey on physicians’ perspectives. Bone Marrow Transplant., 2023
Outcome of haematopoietic cell transplantation in children with lysosomal acid lipase deficiency: a study on behalf of the EBMT Inborn Errors Working Party. Bone Marrow Transplant., 2023
Use of eculizumab in children with allogeneic haematopoietic stem cell transplantation associated thrombotic microangiopathy – a multicentre retrospective PDWP and IEWP EBMT study. Bone Marrow Transplant., 2023
Outcomes of HLA-mismatched hematopoietic stem cell transplantation (HSCT) with CD3+TCRαβ/CD19 depleted PBSC or in vivo T-cell depletion with post-transplant cyclophosphamide for inborn errors of immunity (IEI): an analysis by the EBMT IEWP (in press, Blood)
