{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,18]],"date-time":"2026-08-18T00:45:38Z","timestamp":1787013938458,"version":"build-2736575974"},"reference-count":25,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2024,3,10]],"date-time":"2024-03-10T00:00:00Z","timestamp":1710028800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"DOI":"10.13039\/100004336","name":"Novartis","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004336","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Br J Haematol"],"published-print":{"date-parts":[[2024,5]]},"abstract":"<jats:title>Summary<\/jats:title>\n                  <jats:p>\n                    Diamond\u2013Blackfan anaemia (DBA) is a rare, inherited bone marrow failure syndrome with a ribosomal defect causing slowed globin chain production with normal haem synthesis, causing an overabundance of reactive iron\/haem and erythroid\u2010specific cellular toxicity. Eltrombopag, a non\u2010peptide thrombopoietin receptor agonist, is a potent intracellular iron chelator and induced a robust durable response in an\n                    <jats:italic>RPS19<\/jats:italic>\n                    \u2010mutated DBA patient on another trial. We hypothesized eltrombopag would improve RBC production in DBA patients. We conducted a single\u2010centre, single\u2010arm pilot study (NCT04269889) assessing safety and erythroid response of 6\u2009months of daily, fixed\u2010dose eltrombopag for DBA patients. Fifteen transfusion\u2010dependent (every 3\u20135\u2009weeks) patients (median age 18 [range 2\u201356]) were treated. One responder had sustained haemoglobin improvement and &gt;50% reduction in RBC transfusion frequency. Of note, 7\/15 (41%) patients required dose reductions or sustained discontinuation of eltrombopag due to asymptomatic thrombocytosis. Despite the low response rate, eltrombopag has now improved erythropoiesis in several patients with DBA with a favourable safety profile. Dosing restrictions due to thrombocytosis may cause insufficient iron chelation to decrease haem production and improve anaemia in most patients. Future work will focus on erythropoiesis dynamics in patients and use of haem synthesis inhibitors without an impact on other haematopoietic lineages.\n                  <\/jats:p>","DOI":"10.1111\/bjh.19357","type":"journal-article","created":{"date-parts":[[2024,3,11]],"date-time":"2024-03-11T01:58:49Z","timestamp":1710122329000},"page":"2077-2085","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":10,"title":["Treatment of refractory\/relapsed Diamond\u2013Blackfan anaemia with eltrombopag"],"prefix":"10.1111","volume":"204","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-2739-9712","authenticated-orcid":false,"given":"Brynn B.","family":"Duncan","sequence":"first","affiliation":[{"name":"Translational Stem Cell Biology Branch NHLBI  Bethesda Maryland USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jennifer L.","family":"Lotter","sequence":"additional","affiliation":[{"name":"Office of the Clinical 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