Evidence map›Paper›PMID 39572468›Full record

Trial reportInternational journal of hematology2025

A phase 2 clinical trial of luspatercept in non-transfusion-dependent patients with myelodysplastic syndromes.

Hiroshi Kosugi, Tomoaki Fujisaki, Hiromi Iwasaki, Atsushi Shinagawa, Hiroatsu Iida, Tatsuro Jo, Shiro Kubonishi, Yasuyoshi Morita, Yasuhiro Nakashima, Koichi Onodera and 11 more

Abstract readClinical Trial, Phase IIMulticenter Study
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. Cited by 4 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
4citing 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

4 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Review
  3. Article
  4. Review
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

21 authors.

Hiroshi KosugiDepartment of Hematology, Ogaki Municipal Hospital, 4-86, Minaminokawacho, Ogaki, 503-8502, Japan. hkosugi@med.nagoya-u.ac.jp.ORCID http://orcid.org/0000-0003-3230-4837
Tomoaki FujisakiDepartment of Hematology, Matsuyama Red Cross Hospital, Matsuyama, Japan.
Hiromi IwasakiDepartment of Hematology, Clinical Research Institute, National Hospital Organization Kyushu Medical Center, Fukuoka, Japan.
Atsushi ShinagawaDepartment of Internal Medicine, Hitachi General Hospital, Hitachi, Japan.
Hiroatsu IidaDepartment of Hematology, National Hospital Organization Nagoya Medical Center, Nagoya, Japan.
Tatsuro JoDepartment of Hematology, Japanese Red Cross Nagasaki Genbaku Hospital, Nagasaki, Japan.
Shiro KubonishiDepartment of Hematology and Oncology, Japanese Red Cross Society Himeji Hospital, Himeji, Japan.
Yasuyoshi MoritaDepartment of Hematology and Rheumatology, Kindai University Hospital, Osakasayama, Japan.
Yasuhiro NakashimaDepartment of Hematology and Hematopoietic Cell Transplantation, Osaka Metropolitan University Hospital, Osaka, Japan.
Koichi OnoderaDepartment of Hematology, Tohoku University Hospital, Sendai, Japan.
Kenshi SuzukiDepartment of Hematology, Japanese Red Cross Medical Center, Tokyo, Japan.
Takahiro SuzukiDepartment of Hematology, Kitasato University Hospital, Sagamihara, Japan.
Yotaro TamaiDivision of Hematology, Shonan Kamakura General Hospital, Kamakura, Japan.
Kensuke UsukiDepartment of Hematology, NTT Medical Center Tokyo, Tokyo, Japan.
Akira YokotaDepartment of Hematology, Chiba Aoba Municipal Hospital, Chiba, Japan.
Hideyuki YonagaBristol Myers Squibb, Tokyo, Japan.
Jin HayakawaBristol Myers Squibb, Tokyo, Japan.
Shuichi MidorikawaBristol Myers Squibb, Tokyo, Japan.
Mitsufumi NishioBristol Myers Squibb, Tokyo, Japan.
Makoto SudaBristol Myers Squibb, Tokyo, Japan.
Kosei MatsueDivision of Hematology/Oncology, Department of Internal Medicine, Kameda Medical Center, Kamogawa, Japan.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Luspatercept has shown durable clinical efficacy for the treatment of anemia in transfusion-dependent patients with lower-risk myelodysplastic syndromes (LR-MDS). We report the results of a prespecified primary analysis of a phase 2 trial of luspatercept in non-transfusion-dependent (NTD) Japanese patients with anemia due to LR-MDS. Luspatercept (starting dose 1.0 mg/kg) was administered subcutaneously once every 3 weeks. The primary endpoint was the proportion of patients who achieved hematological improvement-erythroid (HI-E) response (≥ 1.5 g/dL increase in hemoglobin level for 8 weeks) without transfusions within the first 24 weeks of treatment. At the primary analysis data cutoff, 21 patients had been enrolled/treated; 17 and 10 patients had completed 24 and 48 weeks of treatment, respectively. HI-E response occurred within 24 weeks in 10 patients (47.6%; 95% confidence interval, 25.7-70.2; P < 0.0001), which was significantly higher than the predefined threshold (10%). By week 48, HI-E response occurred in 12 patients (57.1%) and 17 patients (81.0%) remained NTD. Luspatercept was well tolerated. Three patients (14.3%) had grade 3-4 treatment-related treatment-emergent adverse events. Luspatercept resulted in statistically and clinically significant improvements in hemoglobin levels, and may help delay the need for transfusions in NTD patients with LR-MDS.

Indexed as

Activin Receptors, Type IIAnemiaImmunoglobulin Fc FragmentsMyelodysplastic SyndromesRecombinant Fusion ProteinsAdultAgedAged, 80 and overBlood TransfusionFemaleHemoglobinsHumansMaleMiddle AgedTreatment OutcomeActivin Receptors, Type IIHemoglobinsImmunoglobulin Fc FragmentsluspaterceptRecombinant Fusion ProteinsAnemiaLow riskLuspaterceptMyelodysplastic syndromesNon-transfusion dependent

Identifiers

PMID39572468
PMCPMC11741997

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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.