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Treatment of transplantation-associated thrombotic microangiopathy with iptacopan: two cases report

Jun Kong1, Ze Tian1, Dao-Xing Deng1

  • 1Peking University People's Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Beijing Key Laboratory of Cell and Gene Therapy for Hematologic Malignancies, Peking University, Beijing, China.

Annals of Hematology
|September 28, 2025
PubMed

Transplantation-associated thrombotic microangiopathy (TA-TMA) is a life-threatening complication of hematopoietic stem cell transplantation (HSCT). With mortality rates up to 90% without timely treatment, effective management remains a challenge. Complement dysregulation plays a central role in TA-TMA pathogenesis. We reported two TA-TMA cases successfully treated with iptacopan, a factor B inhibitor targeting the alternative pathway. A 40-year-old male with early T-cell precursor acute lymphoblastic leukemia developed early-onset TA-TMA post-HSCT, and a 41-year-old female with acute myeloid leukemia arising from previous myelodysplastic syndrome developed delayed-onset TA-TMA after allo-HSCT. Iptacopan administration led to significant clinical and biochemical recovery in both two patients. These two cases highlight iptacopan's potential as an effective therapy for TA-TMA.

Keywords:
Hematopoietic stem cell transplantationIptacopanThrombotic microangiopathy

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