CASE REPORT article
Front. Immunol.
Sec. Alloimmunity and Transplantation
This article is part of the Research TopicAllo-HSCT: novel clinical applications and therapeutic strategies in adults and analysis of rare procedure complicationsView all 7 articles
Efficacy Analysis of Iptacopan in a Patient with Thrombotic Microangiopathy after Allogeneic Hematopoietic Stem Cell Transplantation:a case report
Provisionally accepted
- The Third Affiliated Hospital of Soochow University, changzhou, China
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To investigate the efficacy of Iptacopan in transplantation-associated thrombotic microangiopathy (TA-TMA) after allogeneic hematopoietic stem cell transplantation (allo-HSCT),we report the case of a 43-year-old male with Acute Myeloid Leukemia, Myelodysplasia-Related (AML-MR, with IDH1 and STAG2 mutations) who developed TA-TMA after allo-HSCT. The patient had an initial partial response to the C5 inhibitor eculizumab, but the disease progressed. Oral Iptacopan (200 mg twice daily) was initiated on Day +36. The patient received a total of six sessions of therapeutic plasma exchange and two concurrent doses of defibrotide during the Iptacopan course. After 30 days of Iptacopan treatment, the patient exhibited a significant hematological response, evidenced by a reduction in LDH (758 U/L to 357 U/L), a rise in platelets (17 to 43×10⁹/L), and a drop in C5b-9 (305.32 to 153.70 ng/mL). This biochemical improvement coincided with key clinical outcomes: resolution of proteinuria and the achievement of sustained red blood cell transfusion independence after day +58 and platelet transfusion independence after day +66, marking a decisive turnaround in his TA-TMA course. Treatment with the novel oral complement inhibitor Iptacopan induced significant hematological and clinical responses in this TA-TMA patient, demonstrating its potential therapeutic efficacy and warranting further clinical investigation.
Keywords: Allo-HSCT, AML, Factor B inhibitor, iptacopan, TA-TMA
Received: 01 Nov 2025; Accepted: 16 Feb 2026.
Copyright: © 2026 zhao, guo, ge, wu, yue, Qiu, li, hua, Gu and Lin. 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:
Weiying Gu
Yan Lin
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