Work

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

Year: 2025

Type: article

Abstract: 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.

Source: Annals of Hematology

DOI: 10.1007/s00277-025-06591-9

Authors: Jun Kong, Ze Tian, Dao-Xing Deng, Xiao-Dong Mo

Institutions: Peking University, Peking University People's Hospital

Language: English

Topic: Complement system in diseases

Subfield: Immunology

Field: Immunology and Microbiology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $4,590