Evidence map›Paper›PMID 39730862›Full record

ArticleInternational journal of hematology2025

Adjunctive effects of eltrombopag on immunosuppressive therapy for childhood aplastic anemia.

Katsuhide Eguchi, Masataka Ishimura, Shouichi Ohga, Saori Endo, Shoji Saito, Sachiyo Kamimura, Dai Keino, Shota Kato, Yoshihiro Azuma, Atsushi Watanabe and 7 more

Abstract read
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In one paragraph

Article in International journal 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

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

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

17 authors.

Katsuhide EguchiDepartment of Pediatrics, Graduate School of Medical Sciences, Kyushu University, 3-1-1 Maidashi, Higashi-ku, Fukuoka, 812-8582, Japan.
Masataka IshimuraDepartment of Pediatrics, Graduate School of Medical Sciences, Kyushu University, 3-1-1 Maidashi, Higashi-ku, Fukuoka, 812-8582, Japan. ischii-pediatr@kyudai.jp.ORCID http://orcid.org/0000-0002-0735-6566
Shouichi OhgaDepartment of Pediatrics, Graduate School of Medical Sciences, Kyushu University, 3-1-1 Maidashi, Higashi-ku, Fukuoka, 812-8582, Japan.
Saori EndoDepartment of Pediatrics, Gifu University Graduate School of Medicine, Gifu, Japan.
Shoji SaitoDepartment of Pediatrics, Shinshu University School of Medicine, Matsumoto, Japan.
Sachiyo KamimuraDivision of Pediatrics, Faculty of Medicine, University of Miyazaki, Miyazaki, Japan.
Dai KeinoDivision of Hematology/Oncology, Kanagawa Children's Medical Center, Yokohama, Japan.
Shota KatoDepartment of Pediatrics, Graduate School of Medicine, The University of Tokyo, Tokyo, Japan.
Yoshihiro AzumaDepartment of Pediatrics, Yamaguchi University Graduate School of Medicine, Yamaguchi, Japan.
Atsushi WatanabeFaculty of Medicine, Department of Pediatrics, University of Yamanashi, Yamanashi, Japan.
Akiko InoueDepartment of Pediatrics, Osaka Medical and Pharmaceutical University, Osaka, Japan.
Takeshi HigaDivision of Pediatric Hematology/Oncology, Okinawa Prefectural Nanbu Medical Center and Children's Medical Center, Okinawa, Japan.
Shuichi OzonoDepartment of Pediatrics and Child Health, Kurume University School of Medicine, Kurume, Japan.
Naoto FujitaDepartment of Pediatrics, Hiroshima Red Cross Hospital & Atomic-Bomb Survivors Hospital, Hiroshima, Japan.
Kenichiro WatanabeDepartment of Hematology and Oncology, Shizuoka Children's Hospital, Shizuoka, Japan.
Yoshiyuki TakahashiDepartment of Pediatrics, Nagoya University Graduate School of Medicine, Nagoya, Japan.
Japan Childhood Aplastic Anemia Study Group

Funding

the Health and Labor Sciences Research grants 22FC1004
6 · The paper itself

Abstract

Eltrombopag is used with first-line immunosuppressive therapy for adult aplastic anemia, although its practical utility in childhood remains unclear. We retrospectively analyzed the outcomes of pediatric patients who received eltrombopag in Japan. Of the 27 eligible patients, 23 (85%) were previously treated, and 15 (56%) had severe or very-severe disease. Seventeen (63%) received eltrombopag with or after rabbit anti-thymocyte globulin plus cyclosporin-A. Within the first year of eltrombopag therapy, 12 patients showed a good or partial response, 15 showed no response, and 8 non-responders successfully underwent hematopoietic cell transplantation. Within the first 3 months after eltrombopag therapy, all but one of the transfusion-dependent responders became transfusion-independent. At 12 months, 6 of 12 responders were disease-free off-treatment. The one-year overall response rate was higher for severe or very-severe than non-severe cases (p = 0.006). Multivariable analysis showed that very-severe disease at the start of eltrombopag therapy was a predictor of being disease-free off-treatment (p = 0.03). No cytogenetic abnormalities developed, but myelofibrosis occurred 4 months after eltrombopag therapy in one non-responder with very-severe disease. The first 3 months' response to adjunctive eltrombopag may guide to the safe and effective use for the cure of disease, although prospective trials are needed to determine its long-term effects.

Indexed as

Anemia, AplasticBenzoatesHydrazinesImmunosuppressive AgentsPyrazolesAdolescentAntilymphocyte SerumChildChild, PreschoolCyclosporineDrug Therapy, CombinationFemaleHematopoietic Stem Cell TransplantationHumansInfantMaleAntilymphocyte SerumBenzoatesCyclosporineeltrombopagHydrazinesImmunosuppressive AgentsPyrazolesAnti-thymocyte globulinBone marrow failureCyclosporin-AHematopoietic cell transplantationThrombopoietin

Identifiers

PMID39730862

What OpenQuestion holds

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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 OpenQuestion graph.