ORIGINAL RESEARCH article
Front. Immunol.
Sec. Alloimmunity and Transplantation
Volume 16 - 2025 | doi: 10.3389/fimmu.2025.1583506
This article is part of the Research TopicAllo-HSCT: novel clinical applications and therapeutic strategies in adults and analysis of rare procedure complicationsView all articles
Successful Treatment of Transplant-Associated Thrombotic Microangiopathy with Iptacopan: A Non-adult Case Study
Provisionally accepted
- Shenzhen Hospital, Southern Medical University, Shenzhen, Guangdong Province, China
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Transplant-associated thrombotic microangiopathy (TA-TMA) is a severe complication following hematopoietic stem cell transplantation (HSCT), characterized by microangiopathic hemolytic anemia, thrombocytopenia, microthrombosis, and multi-organ dysfunction. Its mortality rates range from 50-90%, with higher rates observed in high-risk patients. The pathogenesis of TA-TMA involves abnormal activation of the complement system, particularly the alternative pathway, leading to endothelial injury and microthrombosis. We present a case of a 17-year-old male with high-risk TA-TMA who achieved a favorable outcome following oral administration of the factor B inhibitor Iptacopan. The patient exhibited significant improvements in laboratory markers, including a reduction in lactate dehydrogenase, urine protein/creatinine ratio, and C5b-9 levels, along with recovery of platelet counts and haptoglobin levels. This case highlights the potential efficacy of Iptacopan in managing TA-TMA, particularly in high-risk patients, and suggests that complement factor B inhibition may offer a promising therapeutic strategy for this challenging condition.
Keywords: iptacopan, complement, Allo-HSCT, TA-TMA, Factor B inhibitor
Received: 26 Feb 2025; Accepted: 21 Jul 2025.
Copyright: © 2025 Bao, Huang, Huang, Zhu and Sun. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence: Zhiqiang Sun, Shenzhen Hospital, Southern Medical University, Shenzhen, Guangdong Province, China
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