{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,4,24]],"date-time":"2026-04-24T05:34:04Z","timestamp":1777008844570,"version":"3.51.4"},"reference-count":10,"publisher":"Frontiers Media SA","license":[{"start":{"date-parts":[[2026,4,24]],"date-time":"2026-04-24T00:00:00Z","timestamp":1776988800000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["frontiersin.org"],"crossmark-restriction":true},"short-container-title":["Front. Immunol."],"abstract":"<jats:p>Transplantation-associated thrombotic microangiopathy (TA-TMA) is a severe complication following hematopoietic stem cell transplantation, with its pathogenesis not yet fully understood and a lack of highly effective treatment options currently available. This article reports a case of a female patient with acute myeloid leukemia (AML) who was diagnosed with TA-TMA three months after a second allogeneic hematopoietic stem cell transplantation (allo-HSCT). After promptly discontinuing cyclosporine (CSA), and following ineffective treatments with anti-infective therapy, glucocorticoids, and plasma exchange, a short-course combination regimen\u2014defibrotide (10 days) and eculizumab (3 weeks)\u2014was administered. The TA-TMA-related indicators rapidly improved and returned to normal. During follow-up, the patient\u2019s primary disease remained in remission, and she remained free of TA-TMA recurrence for five months. At eight months after the second allo-HSCT, TA-TMA recurred due to concurrent pneumonia and sepsis. The same short-course combination regimen\u2014defibrotide (18 days) and eculizumab (3 weeks)\u2014was administered, and the patient showed improvement in clinical and laboratory parameters again. This study is the first to report the use of a short-course combination therapy with defibrotide and eculizumab for TA-TMA. The combination regimen demonstrated good efficacy and tolerability, offering a potential treatment option for refractory TA-TMA, though its broader applicability requires further research and validation.<\/jats:p>","DOI":"10.3389\/fimmu.2026.1771562","type":"journal-article","created":{"date-parts":[[2026,4,24]],"date-time":"2026-04-24T04:33:41Z","timestamp":1777005221000},"update-policy":"https:\/\/doi.org\/10.3389\/crossmark-policy","source":"Crossref","is-referenced-by-count":0,"title":["Case Report: Short-course defibrotide combined with eculizumab for TA-TMA"],"prefix":"10.3389","volume":"17","author":[{"given":"Yan","family":"Yan","sequence":"first","affiliation":[{"name":"Shanxi Bethune Hospital, Shanxi Academy of Medical Sciences, Third Hospital of Shanxi Medical University, Tongji Shanxi Hospital","place":["Taiyuan, China"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Xiaoning","family":"Li","sequence":"additional","affiliation":[{"name":"Shanxi Bethune Hospital, Shanxi Academy of Medical Sciences, Third Hospital of Shanxi Medical University, Tongji Shanxi Hospital","place":["Taiyuan, China"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Linyu","family":"Li","sequence":"additional","affiliation":[{"name":"Shanxi Bethune Hospital, Shanxi Academy of Medical Sciences, Third Hospital of Shanxi Medical University, Tongji Shanxi Hospital","place":["Taiyuan, China"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Shaoli","family":"Zhang","sequence":"additional","affiliation":[{"name":"Shanxi Bethune Hospital, Shanxi Academy of Medical Sciences, Third Hospital of Shanxi Medical University, Tongji Shanxi Hospital","place":["Taiyuan, China"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Liangming","family":"Ma","sequence":"additional","affiliation":[{"name":"Shanxi Bethune Hospital, Shanxi Academy of Medical Sciences, Third Hospital of Shanxi Medical University, Tongji Shanxi Hospital","place":["Taiyuan, China"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Lifang","family":"Huang","sequence":"additional","affiliation":[{"name":"Shanxi Bethune Hospital, Shanxi Academy of Medical Sciences, Third Hospital of Shanxi Medical University, Tongji Shanxi Hospital","place":["Taiyuan, China"]},{"name":"Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology","place":["Wuhan, China"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Weiwei","family":"Tian","sequence":"additional","affiliation":[{"name":"Shanxi Bethune Hospital, Shanxi Academy of Medical Sciences, Third Hospital of Shanxi Medical University, Tongji Shanxi Hospital","place":["Taiyuan, China"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jie","family":"Zhao","sequence":"additional","affiliation":[{"name":"Shanxi Bethune Hospital, Shanxi Academy of Medical Sciences, Third Hospital of Shanxi Medical University, Tongji Shanxi Hospital","place":["Taiyuan, China"]}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"1965","published-online":{"date-parts":[[2026,4,24]]},"reference":[{"key":"B1","doi-asserted-by":"publisher","DOI":"10.62116\/pnj.2025.51.4.169","article-title":"Chinese consensus on the diagnosis and management of transplant-associated thrombotic microangiopathy (2021)","volume":"42","author":"Xiaojun","year":"2021","journal-title":"Zhonghua Xue Ye Xue Za Zhi"},{"key":"B2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2011-02-321315","article-title":"Small vessels, big trouble in the kidneys and beyond: hematopoietic stem cell transplantation\u2013associated thrombotic microangiopathy","volume":"118","author":"Laskin","year":"2011","journal-title":"Blood"},{"key":"B3","doi-asserted-by":"publisher","DOI":"10.1016\/j.jtct.2022.11.015","article-title":"Harmonizing definitions for diagnostic criteria and prognostic assessment of transplantation-associated thrombotic microangiopathy: A report on behalf of the European Society for Blood and Marrow Transplantation, American Society for Transplantation and Cellular Therapy, Asia-Pacific Blood and Marrow Transplantation Group, and Center for International Blood and Marrow Transplant Research","volume":"29","author":"Schoettler","year":"2023","journal-title":"Transplant Cell Ther"},{"key":"B4","doi-asserted-by":"publisher","first-page":"191","DOI":"10.1016\/j.blre.2014.11.001","article-title":"A new paradigm: Diagnosis and management of HSCT-associated thrombotic microangiopathy as multi-system endothelial injury","volume":"29","author":"Jodele","year":"2015","journal-title":"Blood Rev"},{"key":"B5","doi-asserted-by":"publisher","DOI":"10.1182\/bloodadvances.2021004530","article-title":"A prognostic model\u00a0(BATAP) with external validation for patients with transplant-associated thrombotic microangiopathy","volume":"5","author":"Zhao","year":"2021","journal-title":"Blood Adv"},{"key":"B6","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2019004218","article-title":"Complement blockade for TA-TMA: lessons\u00a0learned from a large pediatric cohort treated with eculizumab","volume":"135","author":"Jodele","year":"2020","journal-title":"Blood"},{"key":"B7","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1016\/j.vph.2013.05.001","article-title":"Defibrotide: properties and clinical use\u00a0of an old\/new drug","volume":"59","author":"Pescador","year":"2013","journal-title":"Vascul Pharmacol"},{"key":"B8","doi-asserted-by":"publisher","DOI":"10.3389\/fimmu.2020.564647","article-title":"Efficacy and safety of eculizumab in the treatment of transplant-associated thrombotic microangiopathy: A systematic review and meta-analysis","volume":"11","author":"Zhang","year":"2020","journal-title":"Front Immunol"},{"key":"B9","doi-asserted-by":"publisher","DOI":"10.1038\/s41409-018-0256-8","article-title":"Use of defibrotide to treat adult patients with transplant-associated thrombotic microangiopathy","volume":"54","author":"Mart\u00ednez-Mu\u00f1oz","year":"2019","journal-title":"Bone Marrow Transplant"},{"key":"B10","doi-asserted-by":"publisher","DOI":"10.1038\/sj.bmt.1703879","article-title":"Endothelial damage caused by cytomegalovirus and human herpesvirus-6","volume":"31","author":"Takatsuka","year":"2003","journal-title":"Bone Marrow Transplant"}],"container-title":["Frontiers in Immunology"],"original-title":[],"link":[{"URL":"https:\/\/www.frontiersin.org\/articles\/10.3389\/fimmu.2026.1771562\/full","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,4,24]],"date-time":"2026-04-24T04:34:05Z","timestamp":1777005245000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.frontiersin.org\/articles\/10.3389\/fimmu.2026.1771562\/full"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,4,24]]},"references-count":10,"alternative-id":["10.3389\/fimmu.2026.1771562"],"URL":"https:\/\/doi.org\/10.3389\/fimmu.2026.1771562","relation":{},"ISSN":["1664-3224"],"issn-type":[{"value":"1664-3224","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,4,24]]},"article-number":"1771562"}}