Evidence map›Paper›PMID 40325238›Full record

ArticleAnnals of hematology2025

Successful treatment of multi-hit TP53-mutated myelodysplastic syndromes with erythroid predominance using allogeneic stem cell transplantation and ruxolitinib.

Seigi Oshima, Junya Kanda, June Takeda, Takashi Sakamoto, Chisaki Mizumoto, Kouhei Yamashita, Yasuhito Nannya, Seishi Ogawa, Akifumi Takaori-Kondo

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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 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

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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

1 citing paper in PubMed.

  1. 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

9 authors.

Seigi OshimaDepartment of Hematology, Graduate School of Medicine, Kyoto University, Kyoto, Japan.
Junya KandaDepartment of Hematology, Graduate School of Medicine, Kyoto University, Kyoto, Japan. jkanda16@kuhp.kyoto-u.ac.jp.
June TakedaDivision of Cancer Evolution, National Cancer Center Research Institute, Tokyo, Japan.
Takashi SakamotoDepartment of Hematology, Graduate School of Medicine, Kyoto University, Kyoto, Japan.
Chisaki MizumotoDepartment of Hematology, Graduate School of Medicine, Kyoto University, Kyoto, Japan.
Kouhei YamashitaDepartment of Hematology, Graduate School of Medicine, Kyoto University, Kyoto, Japan.
Yasuhito NannyaDepartment of Pathology and Tumor Biology, Graduate School of Medicine, Kyoto University, Kyoto, Japan.
Seishi OgawaDepartment of Pathology and Tumor Biology, Graduate School of Medicine, Kyoto University, Kyoto, Japan.
Akifumi Takaori-KondoDepartment of Hematology, Graduate School of Medicine, Kyoto University, Kyoto, Japan.

Funding

Japan Agency for Medical Research and Development (AMED) JP24ek0510046JSPS KAKENHI JP24K11515
6 · The paper itself

Abstract

TP53-mutated myelodysplastic syndrome (MDS) and acute erythroid leukemia (AEL) with complex karyotype have a very poor prognosis. The upregulation of the JAK-STAT pathway has been implicated in their pathogenesis, and inhibition of this pathway has shown promising disease control in preclinical models. Here, we report a case of refractory multi-hit TP53-mutated MDS with erythroid predominance on the verge of transitioning to AEL, which achieved hematological complete remission following allogeneic stem cell transplantation and ruxolitinib initiation. The patient exhibited chemoresistance to multiple regimens, including cytarabine with daunorubicin, high-dose cytarabine, and venetoclax with azacitidine. Despite the presence of residual disease post-transplant, complete remission was achieved two months after ruxolitinib initiation and tacrolimus tapering. At the 8-month follow-up, remission persists without evidence of relapse. This case highlights the potential of combining graft-versus-leukemia effects with ruxolitinib as a therapeutic strategy for TP53-mutated MDS/AEL. Further studies are warranted to evaluate the efficacy and safety of this strategy in a broader clinical setting.

Indexed as

Hematopoietic Stem Cell TransplantationMutationMyelodysplastic SyndromesPyrazolesTumor Suppressor Protein p53HumansNitrilesPyrimidinesTransplantation, HomologousNitrilesPyrazolesPyrimidinesruxolitinibTP53 protein, humanTumor Suppressor Protein p53Acute erythroid leukemiaAllogeneic stem cell transplantationComplex karyotypeMulti-hit TP53 mutationMyelodysplastic syndromesRuxolitinib

Identifiers

PMID40325238
PMCPMC12283891

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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 OpenQuestion graph.