{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,11]],"date-time":"2026-05-11T11:15:56Z","timestamp":1778498156040,"version":"3.51.4"},"reference-count":35,"publisher":"Springer Science and Business Media LLC","issue":"1","license":[{"start":{"date-parts":[[2026,5,11]],"date-time":"2026-05-11T00:00:00Z","timestamp":1778457600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2026,5,11]],"date-time":"2026-05-11T00:00:00Z","timestamp":1778457600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"funder":[{"DOI":"10.13039\/501100009827","name":"Alexandria University","doi-asserted-by":"crossref","id":[{"id":"10.13039\/501100009827","id-type":"DOI","asserted-by":"crossref"}]}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Sci Rep"],"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>\n                    Severe aplastic anemia (SAA) in children is a life-threatening disease. While hematopoietic stem cell transplantation (HSCT) remains the standard of care, many SAA patients lack a suitable donor. Thrombopoietin receptor agonists (TPO-RAs) have emerged as adjuncts to immunosuppressive therapy (IST). The objective of this study was to report a multicenter experience of TPO-RAs use in treatment-na\u00efve children with SAA. We included 57 children, with a median age of 7\u00a0years (range 1.2\u201317), who received TPO-RA monotherapy (n\u2009=\u200913) or TPO-RA\u2009+\u2009cyclosporine A (CsA) (n\u2009=\u200944). Median hemoglobin, platelet counts, and absolute neutrophilic count all increased significantly in both groups (\n                    <jats:italic>p<\/jats:italic>\n                    \u2009&lt;\u20090.001). Overall, 68.4% of patients achieved remission, and none reported severe adverse events related to the TPO-RA. The rate of remission was higher in the TPO-RA monotherapy group (92.3%) vs TPO-RA\u2009+\u2009CsA (59.1%), (\n                    <jats:italic>p<\/jats:italic>\n                    \u2009=\u20090.074), with a shorter median time to remission (6 vs. 12\u00a0months;\n                    <jats:italic>p<\/jats:italic>\n                    \u2009=\u20090.025). Median observation period was 24\u00a0months (range 4\u2013108) with no significant difference between the two treatment groups. Treatment with TPO-RA monotherapy or TPO-RA\u2009+\u2009CsA resulted in clinically meaningful hematologic responses. These findings support prospective evaluation of TPO-RAs in frontline therapy for SAA when HSCT is not readily available, potentially changing management of pediatric SAA in specific contexts.\n                  <\/jats:p>","DOI":"10.1038\/s41598-026-51552-5","type":"journal-article","created":{"date-parts":[[2026,5,11]],"date-time":"2026-05-11T09:47:26Z","timestamp":1778492846000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Real-world use of thrombopoietic agents in treatment-na\u00efve children with severe aplastic anemia: a multicenter retrospective study"],"prefix":"10.1038","volume":"16","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-9849-2204","authenticated-orcid":false,"given":"Yasmine El","family":"Chazli","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Gehad","family":"Abdallah","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3562-7789","authenticated-orcid":false,"given":"Marwa","family":"Zakaria","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1835-1867","authenticated-orcid":false,"given":"Ahmad","family":"Darwish","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0009-2465","authenticated-orcid":false,"given":"Gehan","family":"Khalifa","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Tamer","family":"Hassan","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4460-1567","authenticated-orcid":false,"given":"Eslam","family":"Elhawary","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8465-3794","authenticated-orcid":false,"given":"Samar","family":"Elbahy","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0866-3688","authenticated-orcid":false,"given":"Mostafa","family":"Salama","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Abdulhakim","family":"Alrawas","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yasser","family":"Wali","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Maha","family":"Zeid","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2026,5,11]]},"reference":[{"key":"51552_CR1","doi-asserted-by":"publisher","first-page":"1643","DOI":"10.1056\/NEJMra1413485","volume":"379","author":"NS Young","year":"2018","unstructured":"Young, N. S. Aplastic anemia. N. Engl. J. Med. 379, 1643\u20131656. https:\/\/doi.org\/10.1056\/NEJMra1413485 (2018).","journal-title":"N. Engl. J. Med."},{"key":"51552_CR2","doi-asserted-by":"publisher","first-page":"187","DOI":"10.1111\/bjh.13853","volume":"172","author":"SB Killick","year":"2016","unstructured":"Killick, S. B. et al. Guidelines for the diagnosis and management of adult aplastic anaemia. Br. J. Haematol. 172, 187\u2013207. https:\/\/doi.org\/10.1111\/bjh.13853 (2016).","journal-title":"Br. J. Haematol."},{"key":"51552_CR3","doi-asserted-by":"publisher","first-page":"518","DOI":"10.3324\/haematol.11907","volume":"93","author":"E Montan\u00e9","year":"2008","unstructured":"Montan\u00e9, E. et al. Epidemiology of aplastic anemia: A prospective multicenter study. 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