Evidence map›Paper›PMID 41016978›Full record

ArticleAnnals of hematology2025

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

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

Abstract readCase Reports
PubMed Full text at PMC Publisher
In one paragraph

Article in Annals of hematology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

2 citing papers in PubMed.

  1. Article
  2. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

4 authors.

Jun Kong *Peking 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.
Ze Tian *Peking 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.
Dao-Xing Deng *Peking 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.
Xiao-Dong MoPeking 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. mxd453@163.com.

Funding

the National Key Research and Development Program of China 2022YFA1103300the Natural Science Foundation of Beijing Z230016
6 · The paper itself

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.

Indexed as

AdamantaneHematopoietic Stem Cell TransplantationThrombotic MicroangiopathiesAdultBenzoatesFemaleHumansIndolesLeukemia, Myeloid, AcuteMalePiperidinesPrecursor T-Cell Lymphoblastic Leukemia-LymphomaAdamantaneBenzoatesIndolesiptacopanPiperidinesHematopoietic stem cell transplantationIptacopanThrombotic microangiopathy

Identifiers

PMID41016978
PMCPMC12619809

What Socratic holds

Textmetadata
LicenceCC BY-NC-ND
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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.