Evidence map›Paper›PMID 42816079›Full record

ArticleBMJ open2026

Efficacy and safety of risk-stratified chemotherapy with azacitidine for children with myeloid leukaemia with Down syndrome: protocol for a multicentre phase II trial (JCCG AML-D24).

Kentaro Nakashima, Daisuke Hasegawa, Jun Matsubayashi, Shiro Tanaka, Asahito Hama, Genki Yamato, Shota Kato, Mamoru Honda, Kozo Nagai, Hironori Goto and 14 more

Abstract readClinical Trial Protocol
PubMed Full text at PMC Publisher
In one paragraph

Article in BMJ open, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

24 authors.

Kentaro NakashimaDepartment of Pediatrics, Graduate School of Medical Sciences, Kyushu University, Fukuoka, Japan.ORCID http://orcid.org/0000-0002-4633-110X
Daisuke HasegawaDepartment of Pediatrics, St. Luke's International Hospital, Tokyo, Japan hasedai1313@gmail.com.
Jun MatsubayashiCenter for Clinical Research and Advanced Medicine, Shiga University of Medical Science, Otsu, Japan.ORCID http://orcid.org/0000-0003-4104-6833
Shiro TanakaCenter for Clinical and Translational Research, Kyushu University Hospital, Fukuoka, Japan.
Asahito HamaDepartment of Hematology and Oncology, Children's Medical Center, Japanese Red Cross Aichi Medical Center Nagoya First Hospital, Nagoya, Japan.
Genki YamatoDepartment of Pediatrics, Gunma University Graduate School of Medicine, Maebashi, Japan.
Shota KatoDepartment of Pediatrics, Graduate School of Medicine, The University of Tokyo, Tokyo, Japan.ORCID http://orcid.org/0000-0003-3656-2665
Mamoru HondaDepartment of Hematology/Oncology, Saitama Children's Medical Center, Saitama, Japan.
Kozo NagaiDepartment of Pediatrics, Ehime Prefectural Central Hospital, Matsuyama, Japan.
Hironori GotoDepartment of Pediatrics, Oita University Faculty Of Medicine, Oita, Japan.
Shin-Ichi TsujimotoDepartment of Pediatrics, Yokohama City University Graduate School of Medicine, Yokohama, Japan.
Takao DeguchiDivision of Cancer Immunodiagnostics, Children's Cancer Center, National Center for Child Health and Development, Tokyo, Japan.
Kaori NiwaDepartment of Pediatrics, Mie University Graduate School of Medicine, Tsu, Japan.
Hidefumi HiramatsuDepartment of Pediatrics, Graduate School of Medicine, Kyoto University, Kyoto, Japan.
Kiminori TeruiDepartment of Pediatrics, Hirosaki University Graduate School of Medicine, Hirosaki, Japan.
Tsutomu TokiDepartment of Pediatrics, Hirosaki University Graduate School of Medicine, Hirosaki, Japan.
Kenichi YoshidaDivision of Cancer Evolution, National Cancer Center Research Institute, Tokyo, Japan.
Tomoko KawaiDivision of Fetal Development, National Research Institute for Child Health and Development, Tokyo, Japan.
Akiko M SaitoClinical Research Center, NHO Nagoya Medical Center, Nagoya, Japan.
Hiroshi MoritakeDivision of Pediatrics, Faculty of Medicine, University of Miyazaki, Miyazaki, Japan.
Takashi TagaDepartment of Pediatrics, Shiga University of Medical Science, Otsu, Japan.
Yasuhiro OkamotoDepartment of Pediatrics, Kagoshima University Graduate School of Medical and Dental Sciences, Kagoshima, Japan.
Katsuyoshi KohDepartment of Hematology/Oncology, Saitama Children's Medical Center, Saitama, Japan.
Daisuke TomizawaDivision of Leukemia and Lymphoma, Children's Cancer Center, National Center for Child Health and Development, Tokyo, Japan.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionThe prognosis of myeloid leukaemia associated with Down syndrome (ML-DS) is excellent with reduced-intensity chemotherapy; however, that of refractory or relapsed ML-DS remains dismal. These contrasting outcomes highlight the importance of risk-stratified therapy, based on reliable prognostic factors, to prevent treatment-related mortality and relapse. In addition, novel therapeutic strategies capable of overcoming the poor prognosis of high-risk patients are eagerly anticipated. Based on the results of molecular analyses, in vitro functional assays and anecdotal case reports, azacitidine (AZA), a hypomethylating agent, is a promising candidate for novel therapeutic strategies for high-risk ML-DS. METHODS AND ANALYSIS: In this multicentre, single-arm phase II clinical trial, the Japan Children's Cancer Group AML-D24, we will stratify patients with newly diagnosed ML-DS according to age at diagnosis, ETHICS AND DISSEMINATION: This study was approved by the National Hospital Organization Review Board for Clinical Trials (Nagoya, Japan) on 12 May 2025 and was registered in the Japan Registry of Clinical Trials (https://jrct.mhlw.go.jp/latest-detail/jRCTs041250031). Written informed consent will be obtained from all patients and/or their guardians. The results of this study will be disseminated through publication in peer-reviewed journals and presentation at national and international scientific conferences. TRIAL REGISTRATION NUMBER: jRCTs041250031.

Indexed as

Antimetabolites, AntineoplasticAzacitidineDown SyndromeLeukemia, MyeloidLeukemia, Myeloid, AcuteAntineoplastic Combined Chemotherapy ProtocolsChildChild, PreschoolClinical Trials, Phase II as TopicFemaleGATA1 Transcription FactorHumansInfantJapanMaleMulticenter Studies as TopicAntimetabolites, AntineoplasticAzacitidineGATA1 protein, humanGATA1 Transcription FactorClinical ProtocolsLeukaemiaPaediatric oncology

Identifiers

PMID42816079
PMCPMC13629921

What Socratic holds

Textmetadata
Read underepoch 390

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.