{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,15]],"date-time":"2026-09-15T10:45:36Z","timestamp":1789469136797,"version":"build-2803163510"},"reference-count":28,"publisher":"Springer Science and Business Media LLC","issue":"9","license":[{"start":{"date-parts":[[2026,6,18]],"date-time":"2026-06-18T00:00:00Z","timestamp":1781740800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0"},{"start":{"date-parts":[[2026,8,28]],"date-time":"2026-08-28T00:00:00Z","timestamp":1787875200000},"content-version":"vor","delay-in-days":71,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0"}],"funder":[{"name":"CAMS Innovation Fund for Medical Sciences","award":["2021-I2M-C&T-B-082"],"award-info":[{"award-number":["2021-I2M-C&T-B-082"]}]},{"DOI":"10.13039\/501100001809","name":"National Natural Science Foundation of China","doi-asserted-by":"publisher","award":["82572140"],"award-info":[{"award-number":["82572140"]}],"id":[{"id":"10.13039\/501100001809","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Ann Hematol"],"DOI":"10.1007\/s00277-026-07088-9","type":"journal-article","created":{"date-parts":[[2026,6,18]],"date-time":"2026-06-18T19:41:38Z","timestamp":1781811698000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Safety, pharmacokinetics, and preliminary efficacy of hetrombopag in pediatric patients with aplastic anemia: A pilot study"],"prefix":"10.1007","volume":"105","author":[{"given":"Lixian","family":"Chang","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Beibei","family":"Zhao","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Min","family":"Ruan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Weiwang","family":"Li","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaoming","family":"Liu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tianfeng","family":"Liu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Li","family":"Zhang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yao","family":"Zou","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yumei","family":"Chen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaojuan","family":"Chen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wenyu","family":"Yang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaofan","family":"Zhu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2026,6,18]]},"reference":[{"issue":"1","key":"7088_CR1","doi-asserted-by":"publisher","first-page":"13","DOI":"10.1053\/j.seminhematol.2021.12.002","volume":"59","author":"V Giudice","year":"2022","unstructured":"Giudice V, Selleri C (2022) Aplastic anemia: pathophysiology. Semin Hematol 59(1):13\u201320. https:\/\/doi.org\/10.1053\/j.seminhematol.2021.12.002","journal-title":"Semin Hematol"},{"issue":"17","key":"7088_CR2","doi-asserted-by":"publisher","first-page":"1643","DOI":"10.1056\/NEJMra1413485","volume":"379","author":"NS Young","year":"2018","unstructured":"Young NS (2018) Aplastic anemia. N Engl J Med 379(17):1643\u20131656. https:\/\/doi.org\/10.1056\/NEJMra1413485","journal-title":"N Engl J Med"},{"issue":"4","key":"7088_CR3","doi-asserted-by":"publisher","first-page":"277","DOI":"10.1080\/17474086.2017.1302797","volume":"10","author":"S Kojima","year":"2017","unstructured":"Kojima S (2017) Why is the incidence of aplastic anemia higher in Asia? Expert Rev Hematol 10(4):277\u2013279. https:\/\/doi.org\/10.1080\/17474086.2017.1302797","journal-title":"Expert Rev Hematol"},{"issue":"12","key":"7088_CR4","doi-asserted-by":"publisher","first-page":"1155","DOI":"10.1016\/j.jtct.2024.09.017","volume":"30","author":"R Iftikhar","year":"2024","unstructured":"Iftikhar R, DeFilipp Z, DeZern AE, Pulsipher MA, Bejanyan N, Burroughs LM, Kharfan-Dabaja MA, Arai S, Kassim A, Nakamura R, Saldana BJD, Aljurf M, Hamadani M, Carpenter PA, Antin JH (2024) Allogeneic hematopoietic cell transplantation for the treatment of severe aplastic anemia: evidence-based guidelines from the American Society for Transplantation and Cellular Therapy. Transplant Cell Ther 30(12):1155\u20131170. https:\/\/doi.org\/10.1016\/j.jtct.2024.09.017","journal-title":"Transplant Cell Ther"},{"issue":"3","key":"7088_CR5","doi-asserted-by":"publisher","first-page":"784","DOI":"10.1111\/bjh.19236","volume":"204","author":"A Kulasekararaj","year":"2024","unstructured":"Kulasekararaj A, Cavenagh J, Dokal I, Foukaneli T, Gandhi S, Garg M, Griffin M, Hillmen P, Ireland R, Killick S, Mansour S, Mufti G, Potter V, Snowden J, Stanworth S, Zuha R, Marsh J, Committee BSH (2024) Guidelines for the diagnosis and management of adult aplastic anaemia: a British Society for Haematology guideline. Br J Haematol 204(3):784\u2013804. https:\/\/doi.org\/10.1111\/bjh.19236","journal-title":"Br J Haematol"},{"key":"7088_CR6","doi-asserted-by":"publisher","DOI":"10.1016\/j.blre.2020.100772","volume":"47","author":"R Iftikhar","year":"2021","unstructured":"Iftikhar R, Chaudhry QUN, Anwer F, Neupane K, Rafae A, Mahmood SK, Ghafoor T, Shahbaz N, Khan MA, Khattak TA, Shamshad GU, Rehman J, Farhan M, Khan M, Ansar I, Ashraf R, Marsh J, Satti TM, Ahmed P (2021) Allogeneic hematopoietic stem cell transplantation in aplastic anemia: current indications and transplant strategies. Blood Rev 47:100772. https:\/\/doi.org\/10.1016\/j.blre.2020.100772","journal-title":"Blood Rev"},{"issue":"1","key":"7088_CR7","doi-asserted-by":"publisher","first-page":"21","DOI":"10.1053\/j.seminhematol.2022.01.002","volume":"59","author":"BA Patel","year":"2022","unstructured":"Patel BA, Townsley DM, Scheinberg P (2022) Immunosuppressive therapy in severe aplastic anemia. Semin Hematol 59(1):21\u201329. https:\/\/doi.org\/10.1053\/j.seminhematol.2022.01.002","journal-title":"Semin Hematol"},{"issue":"8","key":"7088_CR8","doi-asserted-by":"publisher","first-page":"1785","DOI":"10.1007\/s00277-022-04864-1","volume":"101","author":"F Zhou","year":"2022","unstructured":"Zhou F, Zhang F, Zhang L, Wu Q, Ma J, Zhao C, Wang L, Jie G, Zhang H, Zhang H, Wang S, Teng Q (2022) A multicentre trial of intensive immunosuppressive therapy combined with umbilical cord blood for the treatment of severe aplastic anaemia. Ann Hematol 101(8):1785\u20131794. https:\/\/doi.org\/10.1007\/s00277-022-04864-1","journal-title":"Ann Hematol"},{"key":"7088_CR9","doi-asserted-by":"publisher","DOI":"10.3389\/fimmu.2023.1197982","volume":"14","author":"Y Zhang","year":"2023","unstructured":"Zhang Y, He Y, Wang S, Sun J, Jia J, Gong Y, He G, Li J (2023) Transfusion-dependent non-severe aplastic anemia: characteristics and outcomes in the clinic. Front Immunol 14:1197982. https:\/\/doi.org\/10.3389\/fimmu.2023.1197982","journal-title":"Front Immunol"},{"issue":"12","key":"7088_CR10","doi-asserted-by":"publisher","first-page":"2479","DOI":"10.1038\/s41375-023-02047-z","volume":"37","author":"B Fattizzo","year":"2023","unstructured":"Fattizzo B, Gurnari C, Cassanello G, Bortolotti M, Awada H, Giammarco S, Consonni D, Sica S, Gandhi S, Trikha R, Large J, Salter S, Maciejewski JP, Barcellini W, Kulasekararaj AG (2023) Deciphering treatment patterns in non-severe\/moderate aplastic anemia: an international observational study. Leukemia 37(12):2479\u20132485. https:\/\/doi.org\/10.1038\/s41375-023-02047-z","journal-title":"Leukemia"},{"issue":"6","key":"7088_CR11","doi-asserted-by":"publisher","first-page":"954","DOI":"10.1111\/bjh.17403","volume":"194","author":"P Scheinberg","year":"2021","unstructured":"Scheinberg P (2021) Acquired severe aplastic anaemia: how medical therapy evolved in the 20th and 21st centuries. Br J Haematol 194(6):954\u2013969. https:\/\/doi.org\/10.1111\/bjh.17403","journal-title":"Br J Haematol"},{"issue":"11","key":"7088_CR12","doi-asserted-by":"publisher","first-page":"1285","DOI":"10.1007\/s40265-021-01553-7","volume":"81","author":"J Gilreath","year":"2021","unstructured":"Gilreath J, Lo M, Bubalo J (2021) Thrombopoietin receptor agonists (TPO-RAs): drug class considerations for pharmacists. Drugs 81(11):1285\u20131305. https:\/\/doi.org\/10.1007\/s40265-021-01553-7","journal-title":"Drugs"},{"issue":"6","key":"7088_CR13","doi-asserted-by":"publisher","first-page":"1112","DOI":"10.3324\/haematol.2018.212845","volume":"104","author":"W Ghanima","year":"2019","unstructured":"Ghanima W, Cooper N, Rodeghiero F, Godeau B, Bussel JB (2019) Thrombopoietin receptor agonists: ten years later. Haematologica 104(6):1112\u20131123. https:\/\/doi.org\/10.3324\/haematol.2018.212845","journal-title":"Haematologica"},{"issue":"1","key":"7088_CR14","doi-asserted-by":"publisher","first-page":"11","DOI":"10.1056\/NEJMoa2109965","volume":"386","author":"R Peffault de Latour","year":"2022","unstructured":"Peffault de Latour R, Kulasekararaj A, Iacobelli S, Terwel SR, Cook R, Griffin M, Halkes CJM, Recher C, Barraco F, Forcade E, Vallejo JC, Drexler B, Mear JB, Smith AE, Angelucci E, Raymakers RAP, de Groot MR, Daguindau E, Nur E, Barcellini W, Russell NH, Terriou L, Iori AP, La Rocca U, Sureda A, Sanchez-Ortega S, Xicoy B, Jarque I, Cavenagh J, Sicre de Fontbrune F, Marotta S, Munir T, Tjon JML, Tavitian S, Praire A, Clement L, Rabian F, Marano L, Hill A, Palmisani E, Muus P, Cacace F, Frieri C, van Lint MT, Passweg JR, Marsh JCW, Socie G, Mufti GJ, Dufour C, Risitano AM, Severe Aplastic Anemia Working Party of the European Society for B, Marrow T (2022) Eltrombopag added to immunosuppression in severe aplastic anemia. N Engl J Med 386(1):11\u201323. https:\/\/doi.org\/10.1056\/NEJMoa2109965","journal-title":"N Engl J Med"},{"issue":"16","key":"7088_CR15","doi-asserted-by":"publisher","first-page":"1540","DOI":"10.1056\/NEJMoa1613878","volume":"376","author":"DM Townsley","year":"2017","unstructured":"Townsley DM, Scheinberg P, Winkler T, Desmond R, Dumitriu B, Rios O, Weinstein B, Valdez J, Lotter J, Feng X, Desierto M, Leuva H, Bevans M, Wu C, Larochelle A, Calvo KR, Dunbar CE, Young NS (2017) Eltrombopag added to standard immunosuppression for aplastic anemia. N Engl J Med 376(16):1540\u20131550. https:\/\/doi.org\/10.1056\/NEJMoa1613878","journal-title":"N Engl J Med"},{"issue":"1","key":"7088_CR16","doi-asserted-by":"publisher","DOI":"10.1186\/s40164-023-00377-3","volume":"12","author":"W Yang","year":"2023","unstructured":"Yang W, Zhao X, Liu X, Xiong Y, Fan H, Zhang L, Li J, Ye L, Zhou K, Li Y, Yang Y, Peng G, Jing L, Zhang F (2023) Hetrombopag plus porcine ATG and cyclosporine for the treatment of aplastic anaemia: early outcomes of a prospective pilot study. Exp Hematol Oncol 12(1):16. https:\/\/doi.org\/10.1186\/s40164-023-00377-3","journal-title":"Exp Hematol Oncol"},{"issue":"1","key":"7088_CR17","doi-asserted-by":"publisher","DOI":"10.1186\/s13643-024-02515-2","volume":"13","author":"Y Zhang","year":"2024","unstructured":"Zhang Y, Li J, Li X, Geng Q, Xie Y, Zhang G, Wei M, Ma Y (2024) Efficacy and safety of immunosuppressive therapy combined with eltrombopag for severe aplastic anemia: a systematic review and meta-analysis. Syst Rev 13(1):101. https:\/\/doi.org\/10.1186\/s13643-024-02515-2","journal-title":"Syst Rev"},{"issue":"13","key":"7088_CR18","doi-asserted-by":"publisher","first-page":"1581","DOI":"10.1007\/s40265-021-01575-1","volume":"81","author":"YY Syed","year":"2021","unstructured":"Syed YY (2021) Hetrombopag: first approval. Drugs 81(13):1581\u20131585. https:\/\/doi.org\/10.1007\/s40265-021-01575-1","journal-title":"Drugs"},{"key":"7088_CR19","doi-asserted-by":"publisher","DOI":"10.1177\/20406207221085197","volume":"13","author":"G Peng","year":"2022","unstructured":"Peng G, He G, Chang H, Gao S, Liu X, Chen T, Li P, Han B, Miao M, Ge Z, Ge X, Li F, Li Y, Wang S, Wang Y, Shen Y, Zhang T, Zou J, Zhang F (2022) A multicenter phase II study on the efficacy and safety of Hetrombopag in patients with severe aplastic anemia refractory to immunosuppressive therapy. Ther Adv Hematol 13:20406207221085197. https:\/\/doi.org\/10.1177\/20406207221085197","journal-title":"Ther Adv Hematol"},{"issue":"8","key":"7088_CR20","doi-asserted-by":"publisher","first-page":"932","DOI":"10.1002\/jcph.2430","volume":"64","author":"W Zhang","year":"2024","unstructured":"Zhang W, Chang LX, Zhao BB, Zheng Y, Shan DD, Tang BH, Yang F, Zhou Y, Hao GX, Zhang YH, van den Anker J, Zhu XF, Zhang L, Zhao W (2024) Efficacy, safety, and population pharmacokinetics of Eltrombopag in children with different severities of aplastic anemia. J Clin Pharmacol 64(8):932\u2013943. https:\/\/doi.org\/10.1002\/jcph.2430","journal-title":"J Clin Pharmacol"},{"issue":"7","key":"7088_CR21","doi-asserted-by":"publisher","first-page":"633","DOI":"10.1080\/08880018.2021.1895924","volume":"38","author":"M Fang","year":"2021","unstructured":"Fang M, Song H, Zhang J, Li S, Shen D, Tang Y (2021) Efficacy and safety of immunosuppressive therapy with or without eltrombopag in pediatric patients with acquired aplastic anemia: A Chinese retrospective study. Pediatr Hematol Oncol 38(7):633\u2013646. https:\/\/doi.org\/10.1080\/08880018.2021.1895924","journal-title":"Pediatr Hematol Oncol"},{"issue":"7","key":"7088_CR22","doi-asserted-by":"publisher","first-page":"647","DOI":"10.1080\/08880018.2021.1900475","volume":"38","author":"M Jie","year":"2021","unstructured":"Jie M, Fu L, Li S, He Y, Yao J, Cheng X, Zhang L, Zheng J, Zhang R, Wu R (2021) Efficacy and safety of eltrombopag in the first-line therapy of severe aplastic anemia in children. Pediatr Hematol Oncol 38(7):647\u2013657. https:\/\/doi.org\/10.1080\/08880018.2021.1900475","journal-title":"Pediatr Hematol Oncol"},{"issue":"6","key":"7088_CR23","doi-asserted-by":"publisher","first-page":"655","DOI":"10.1016\/j.pedneo.2021.07.002","volume":"62","author":"MY Su","year":"2021","unstructured":"Su MY, Chang HH, Chou SW, Lu MY, Yang YL, Lin DT, Lin KH, Jou ST (2021) Role of eltrombopag in severe aplastic anemia treatment in children. Pediatr Neonatol 62(6):655\u2013657. https:\/\/doi.org\/10.1016\/j.pedneo.2021.07.002","journal-title":"Pediatr Neonatol"},{"issue":"6","key":"7088_CR24","doi-asserted-by":"publisher","first-page":"953","DOI":"10.1182\/bloodadvances.2021006716","volume":"7","author":"O Goronkova","year":"2023","unstructured":"Goronkova O, Novichkova G, Salimova T, Kalinina I, Baidildina D, Petrova U, Antonova K, Sadovskaya M, Suntsova E, Evseev D, Matveev V, Venyov D, Khachatryan L, Litvinov D, Pshonkin A, Ovsyannikova G, Kotskaya N, Gobadze D, Olshanskaya Y, Popov A, Raykina E, Mironenko O, Voronin K, Purbueva B, Boichenko E, Dinikina Y, Guseynova E, Sherstnev D, Kalinina E, Mezentsev S, Streneva O, Yudina N, Plaksina O, Erega E, Maschan M, Maschan A (2023) Efficacy of combined immunosuppression with or without eltrombopag in children with newly diagnosed aplastic anemia. Blood Adv 7(6):953\u2013962. https:\/\/doi.org\/10.1182\/bloodadvances.2021006716","journal-title":"Blood Adv"},{"issue":"9","key":"7088_CR25","doi-asserted-by":"publisher","first-page":"2328","DOI":"10.1038\/s41375-022-01636-8","volume":"36","author":"EM Groarke","year":"2022","unstructured":"Groarke EM, Patel BA, Shalhoub R, Gutierrez-Rodrigues F, Desai P, Leuva H, Zaimoku Y, Paton C, Spitofsky N, Lotter J, Rios O, Childs RW, Young DJ, Dulau-Florea A, Dunbar CE, Calvo KR, Wu CO, Young NS (2022) Predictors of clonal evolution and myeloid neoplasia following immunosuppressive therapy in severe aplastic anemia. Leukemia 36(9):2328\u20132337. https:\/\/doi.org\/10.1038\/s41375-022-01636-8","journal-title":"Leukemia"},{"issue":"Supplement 1","key":"7088_CR26","doi-asserted-by":"publisher","first-page":"304","DOI":"10.1182\/blood-2024-206377","volume":"144","author":"F Zhang","year":"2024","unstructured":"Zhang F, Zhao X, Chang H, Han B, Gao S, Miao M, Hu J, Lin Y, Liu X, Zhang L, Jin C, Li Y, Lin S, Jia J, Wang Y, Chang C, Ge Z, He G, Xia R, Fu X, Li Y, Liu H, Sun K, Wang S, Xia L, Zhang W, Li R, Xiong J, Zhai W (2024) Hetrombopag added to immunosuppressive therapy in first-line treatment of severe aplastic anemia: A randomized, double-blind, placebo-controlled phase 3 trial. Blood 144(Supplement 1):304\u2013304. https:\/\/doi.org\/10.1182\/blood-2024-206377","journal-title":"Blood"},{"issue":"3","key":"7088_CR27","doi-asserted-by":"publisher","first-page":"605","DOI":"10.1111\/bjh.17232","volume":"192","author":"EM Groarke","year":"2021","unstructured":"Groarke EM, Patel BA, Gutierrez-Rodrigues F, Rios O, Lotter J, Baldoni D, St Pierre A, Shalhoub R, Wu CO, Townsley DM, Young NS (2021) Eltrombopag added to immunosuppression for children with treatment\u2010na\u00efve severe aplastic anaemia. Br J Haematol 192(3):605\u2013614. https:\/\/doi.org\/10.1111\/bjh.17232","journal-title":"Br J Haematol"},{"key":"7088_CR28","doi-asserted-by":"publisher","DOI":"10.3389\/fped.2023.1149718","volume":"11","author":"MM Marrapodi","year":"2023","unstructured":"Marrapodi MM, Mascolo A, Roberti D, Martino MD, Rafaniello C, Riccardi C, Rossi F (2023) The efficacy and the safety of eltrombopag in pediatric patients with severe aplastic anemia: A systematic review. Front Pediatr 11:1149718. https:\/\/doi.org\/10.3389\/fped.2023.1149718","journal-title":"Front Pediatr"}],"container-title":["Annals of Hematology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s00277-026-07088-9","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s00277-026-07088-9.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s00277-026-07088-9.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,9,15]],"date-time":"2026-09-15T09:43:08Z","timestamp":1789465388000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s00277-026-07088-9"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,6,18]]},"references-count":28,"journal-issue":{"issue":"9","published-online":{"date-parts":[[2026,9]]}},"alternative-id":["7088"],"URL":"https:\/\/doi.org\/10.1007\/s00277-026-07088-9","relation":{},"ISSN":["1432-0584"],"issn-type":[{"value":"1432-0584","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,6,18]]},"assertion":[{"value":"30 October 2025","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"22 May 2026","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"18 June 2026","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"The authors declare no conflicts of interest.","order":1,"name":"Ethics","label":"Disclosures","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"The authors declare no competing interests.","order":2,"name":"Ethics","label":"Competing interests","group":{"name":"EthicsHeading","label":"Declarations"}}],"article-number":"396"}}