Evidence map›Paper›PMID 40016445›Full record

Trial reportInternational journal of hematology2025

Association between gene mutations and outcomes in Japanese high-risk AML patients: a phase 1/2 study of NS-87/CPX-351.

Hideki Makishima, Taisuke Mikasa, Kento Isogaya, Toshihiro Miyamoto, Takuji Yamauchi, Akira Yokota, Masahiro Onozawa, Kiyoshi Ando, Yoshiaki Ogawa, Kensuke Usuki and 10 more

Abstract readClinical Trial, Phase IIClinical Trial, Phase I
PubMed Full text at PMC Publisher
In one paragraph

Trial report in International journal of hematology, 2025. 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

20 authors.

Hideki Makishima *Department of Hematology and Medical Oncology, Shinshu University School of Medicine, 3-1-1 Asahi, Matsumoto, 390-8621, Japan. makishimah@shinshu-u.ac.jp.ORCID http://orcid.org/0000-0001-5983-8578
Taisuke Mikasa *Clinical Development Department, Nippon Shinyaku Co., Ltd, Kyoto, Japan.
Kento IsogayaData Science Department, Nippon Shinyaku Co., Ltd, Kyoto, Japan.
Toshihiro MiyamotoDepartment of Hematology, Kanazawa University, Kanazawa, Japan.
Takuji YamauchiDepartment of Medicine and Biosystemic Science, Kyushu University Graduate School of Medical Sciences, Fukuoka, Japan.
Akira YokotaDepartment of Hematology, Chiba Aoba Municipal Hospital, Chiba, Japan.
Masahiro OnozawaDepartment of Hematology, Hokkaido University Hospital, Sapporo, Japan.
Kiyoshi AndoDepartment of Hematology and Oncology, Tokai University School of Medicine, Isehara, Japan.
Yoshiaki OgawaDepartment of Hematology and Oncology, Tokai University School of Medicine, Isehara, Japan.
Kensuke UsukiForth Department of Internal Medicine, Mizonokuchi Hospital, Teikyo University School of Medicine, Kawasaki, Japan.
Takahiro YamauchiDepartment of Hematology and Oncology, University of Fukui, Fukui, Japan.
Shuichi OtaDepartment of Hematology, Sapporo Hokuyu Hospital, Sapporo, Japan.
Satoru TakadaLeukemia Research Center, Saiseikai Maebashi Hospital, Maebashi, Japan.
Yasuyoshi MoritaDepartment of Hematology and Rheumatology, Kindai University Faculty of Medicine, Sayama, Japan.
Takayuki IshikawaDepartment of Hematology, Kobe City Medical Center General Hospital, Kobe, Japan.
Katsuto TakenakaDepartment of Hematology, Clinical Immunology and Infectious Disease, Ehime University Graduate School of Medicine, Toon, Japan.
Junya KurodaDivision of Hematology and Oncology, Department of Medicine, Kyoto Prefectural University of Medicine, Kyoto, Japan.
Naohiro SekiguchiDepartment of Hematology, National Hospital Organization Disaster Medical Center, Tachikawa, Japan.
Toshiro KawakitaDepartment of Hematology, NHO Kumamoto Medical Center, Kumamoto, Japan.
Yasushi MiyazakiDepartment of Hematology, Atomic Bomb Disease Institute, Nagasaki University, Nagasaki, Japan.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

This phase 1/2 study investigated the association between genetic characteristics and outcomes for NS-87/CPX-351 in Japanese patients with high-risk acute myeloid leukemia. Blood samples collected from 29 patients were analyzed using a 70-gene next-generation sequencing panel. The most frequently mutated genes were TP53 (44.8%), TET2 (24.1%), DNMT3A (13.8%), and NRAS (13.8%). The rates of composite complete remission (CRc; complete remission [CR] or CR with incomplete hematologic recovery [CRi]) were comparable between patients with and without mutations in TP53, TET2, DNMT3A, and NRAS (P = 0.571 for all). Notably, patients with TP53 mutations had a similar CRc rate (69.2% vs. 56.3%), but shorter overall survival (OS), event-free survival (EFS), and relapse-free survival (RFS) compared to patients with wild-type TP53 (median OS: 7.43 vs. 18.18 months; P = 0.108, median EFS: 2.43 vs. 6.28 months; P = 0.012, median RFS: 1.48 vs. 10.19 months; P = 0.012). In conclusion, no gene mutations directly associated with the efficacy of NS-87/CPX-351 were found. While NS-87/CPX-351 achieved remission even in patients with TP53 mutations, relapse risk was higher in these patients. Therefore, it is advisable to consider treatment strategies such as early transplantation after achieving remission with NS-87/CPX-351, especially in patients with TP53 mutations.

Indexed as

Leukemia, Myeloid, AcuteMutationAdultAgedAged, 80 and overDioxygenasesDNA-Binding ProteinsDNA (Cytosine-5-)-MethyltransferasesDNA Methyltransferase 3AEast Asian PeopleFemaleGTP PhosphohydrolasesHigh-Throughput Nucleotide SequencingHumansJapanMaleDioxygenasesDNA-Binding ProteinsDNA (Cytosine-5-)-MethyltransferasesDNA Methyltransferase 3ADNMT3A protein, humanGTP PhosphohydrolasesMembrane ProteinsNRAS protein, humanProto-Oncogene ProteinsTET2 protein, humanTP53 protein, humanTumor Suppressor Protein p53Acute myeloid leukemiaCPX-351NGSTP53 mutations

Identifiers

PMID40016445
PMCPMC12202506

What OpenQuestion holds

Textmetadata
LicenceCC BY
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 OpenQuestion graph.