{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T06:17:49Z","timestamp":1790835469192,"version":"4.1.0"},"reference-count":18,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2024,8,19]],"date-time":"2024-08-19T00:00:00Z","timestamp":1724025600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"},{"start":{"date-parts":[[2024,8,19]],"date-time":"2024-08-19T00:00:00Z","timestamp":1724025600000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Br J Haematol"],"published-print":{"date-parts":[[2026,1]]},"abstract":"<jats:title>Summary<\/jats:title>\n                  <jats:p>Modalities and timing of haematopoietic stem cell transplant (HSCT) in patients with GATA2 deficiency are still subject to debate. On June 2022, 67 patients (median age 20.6\u2009years) underwent a first allogeneic HSCT among 21 centres. Indications for HSCT were myelodysplastic syndrome (MDS) \u22645% blasts \u00b1 immunodeficiency (66%), MDS &gt;5% blasts (15%), acute myeloid leukaemia (19%). Conditioning regimen was myeloablative in 85% and anti\u2010thymocyte globulins were used in 67%. The cumulative incidence (CInc) of acute graft versus host disease (GvHD) grade II\u2013IV and III\u2013IV at day 100 were 42% and 13%, and CInc of chronic and extensive chronic GvHD at 2\u2009years were 42% and 23%. CInc of relapses was 3% and 11% at 1 and 5\u2009years. Overall survival (OS) at 1 and 5\u2009years was 83% and 72% (median follow\u2010up 5.6\u2009years). The factors associated with worse OS in multivariable analysis were the year of HSCT, a history of excess blasts before transplant and peripheral blood stem cell (PBSC) grafts. Age at HSCT, non\u2010myeloablative conditioning and PBSC grafts were associated with increased non\u2010relapse mortality. In conclusion, bone marrow monitoring to identify clonal evolution and perform HSCT before the appearance of excess blast is mandatory.<\/jats:p>","DOI":"10.1111\/bjh.19691","type":"journal-article","created":{"date-parts":[[2024,8,19]],"date-time":"2024-08-19T20:19:00Z","timestamp":1724098740000},"page":"250-258","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":10,"title":["Long\u2010term outcome after allogeneic stem cell transplantation for\n                    <scp>GATA2<\/scp>\n                    deficiency: An analysis of 67 adults and children from France and Belgium"],"prefix":"10.1111","volume":"208","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-2000-1556","authenticated-orcid":false,"given":"Flore","family":"Sicre de Fontbrune","sequence":"first","affiliation":[{"name":"H\u00e9matologie Greffe, H\u00f4pital Saint\u2010Louis APHP  Paris 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