Evidence map›Paper›PMID 41493719›Full record

Trial reportInternational journal of hematology2026

A phase I/II study of tagraxofusp in Japanese patients with blastic plasmacytoid dendritic cell neoplasm.

Akira Yokota, Wataru Munakata, Toru Kiguchi, Yoshiaki Ogawa, Masayuki Hino, Koji Kato, Masahiro Chiba, Daisuke Kawasaki, Kohei Wasa, Taisuke Mikasa and 4 more

Abstract readClinical Trial, Phase IClinical Trial, Phase IIMulticenter Study
In one paragraph

Trial report in International journal of hematology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed, 1 pooled it
–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 synthesis or guideline pooled it.

  1. Pooled it
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

14 authors.

Akira YokotaDepartment of Hematology, Chiba Aoba Municipal Hospital, 1273-2 Aoba-cho, Chuo-ku, Chiba, Chiba, 260-0852, Japan. taroyok@xb4.so-net.ne.jp.ORCID http://orcid.org/0000-0001-7488-1371
Wataru MunakataDepartment of Hematology, National Cancer Center Hospital, Chuo-ku, Tokyo, Japan.
Toru KiguchiDepartment of Diabetes, Endocrinology and Hematology, Dokkyo Medical University Saitama Medical Center, Koshigaya, Saitama, Japan.
Yoshiaki OgawaDepartment of Hematology and Oncology, Tokai University School of Medicine, Isehara, Kanagawa, Japan.
Masayuki HinoDepartment of Hematology, Graduate School of Medicine, Osaka Metropolitan University, Osaka, Osaka, Japan.
Koji KatoDepartment of Medicine and Biosystemic Science, Graduate School of Medical Sciences, Kyushu University, Fukuoka, Fukuoka, Japan.
Masahiro ChibaClinical Development Department, Nippon Shinyaku Co., Ltd., Kyoto, Kyoto, Japan.
Daisuke KawasakiData Science Department, Nippon Shinyaku Co., Ltd., Kyoto, Kyoto, Japan.
Kohei WasaClinical Development Department, Nippon Shinyaku Co., Ltd., Kyoto, Kyoto, Japan.
Taisuke MikasaClinical Development Department, Nippon Shinyaku Co., Ltd., Kyoto, Kyoto, Japan.
Kengo TakeuchiDivision of Pathology, the Cancer Institute, Japanese Foundation for Cancer Research, Koto-ku, Tokyo, Japan.
Koji IzutsuDepartment of Hematology, National Cancer Center Hospital, Chuo-ku, Tokyo, Japan.
Ritsuro SuzukiDepartment of Hematology, Shimane University Hospital, Izumo, Shimane, Japan.
Study Group

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Tagraxofusp (TAG), a first-in-class CD123-targeted therapy, is a recombinant fusion protein of human interleukin-3 conjugated to a truncated diphtheria toxin payload. Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is an aggressive orphan hematologic cancer with a poor prognosis, and is derived from plasmacytoid dendritic cells that overexpress interleukin-3 receptor subunit alpha (IL3RA or CD123). This open-label phase I/II study evaluated the efficacy and safety of TAG in 11 Japanese BPDCN patients, of whom seven were treatment-naïve (TN) and four had relapsed/refractory (R/R) disease. In the phase I portion, seven patients (five TN, two R/R) were treated, and no dose-limiting toxicity was observed, with 12 μg/kg/day tolerated. In the phase II portion, four patients (two TN, two R/R) were treated. Among the seven TN BPDCN patients, the rate of complete response (CR) + clinical CR (CRc: CR with minimal residual skin abnormality) was 57.1% (90% confidence interval [CI], 22.5-87.1), and the lower limit of the 90% CI (22.5%) exceeded the pre-specified threshold of 10%. Common adverse events included increased alanine aminotransferase (81.8%) and aspartate aminotransferase (72.7%), hypoalbuminemia, hypokalemia, and capillary leak syndrome (54.5%). The results indicate that TAG was effective and had a manageable safety profile in Japanese BPDCN patients.

Indexed as

Dendritic CellsHematologic NeoplasmsInterleukin-3Recombinant Fusion ProteinsAdultAgedBlastic Plasmacytoid Dendritic Cell NeoplasmEast Asian PeopleFemaleHumansInterleukin-3 Receptor alpha SubunitJapanMaleMiddle AgedTreatment OutcomeInterleukin-3Interleukin-3 Receptor alpha SubunitRecombinant Fusion ProteinstagraxofuspBlastic plasmacytoid dendritic cell neoplasmCD123SL-401Tagraxofusp

Identifiers

PMID41493719
PMCPMC13171773

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Registered trials

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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.