Evidence map›Paper›PMID 40593138›Full record

Trial reportAnnals of hematology2025

Predictive factors of romiplostim response in patients with refractory aplastic anemia: data from two clinical trials.

Jun Ho Jang, Kinuko Mitani, Yoshiaki Tomiyama, Koji Miyazaki, Koji Nagafuji, Kensuke Usuki, Nobuhiko Uoshima, Tomoaki Fujisaki, Hiroshi Kosugi, Itaru Matsumura and 13 more

2 registry-linked trialsAbstract readClinical Trial, Phase IIClinical Trial, Phase IIIMulticenter Study
In one paragraph

Trial report in Annals of hematology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 3 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
3citing 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.

NCT02094417 phase2completednot on this map

A Phase 2, Randomized, Open-Label, Parallel, Comparative, Dose-Finding Study to Evaluate the Efficacy and Safety of AMG531 in Aplastic Anemia Subjects With Thrombocytopenia Refractory to Immunosuppressive Therapy

TypeinterventionalSponsorKyowa Kirin Korea Co., Ltd.Ran2014 to 2017Enrolled35ConditionsAplastic AnemiaArmsAMG531
NCT02773290 phase2 / phase3completednot on this map

Phase 2/3 Study of Romiplostim(AMG531) in Subjects With Aplastic Anemia Refractory to or Ineligible for Immunosuppressive Therapy

TypeinterventionalSponsorKyowa Kirin Co., Ltd.Ran2016 to 2020Enrolled46ConditionsAplastic AnemiaArmsRomiplostim
3 · Its place in the literature

Who cites it

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

  1. Pooled it
  2. Article
  3. 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

23 authors.

Jun Ho Jang *Department of Hematology, Sungkyunkwan University Samsung Medical Center, Seoul, Republic of Korea.
Kinuko Mitani *Department of Hematology and Oncology, Dokkyo Medical University, Tochigi, Japan.
Yoshiaki TomiyamaDepartment of Blood Transfusion, Osaka University Hospital, Osaka, Japan.
Koji MiyazakiDepartment of Transfusion and Cell Transplantation, Kitasato University School of Medicine, Sagamihara, Japan.
Koji NagafujiDepartment of Hematology, Kurume University Hospital, Kurume, Japan.
Kensuke UsukiDepartment of Hematology, NTT Medical Center Tokyo, Tokyo, Japan.
Nobuhiko UoshimaDepartment of Hematology, Japanese Red Cross Kyoto Daini Hospital, Kyoto, Japan.
Tomoaki FujisakiDepartment of Internal Medicine, Matsuyama Red Cross Hospital, Matsuyama, Japan.
Hiroshi KosugiDepartment of Hematology, Ogaki Municipal Hospital, Ogaki, Japan.
Itaru MatsumuraDepartment of Hematology and Rheumatology, Faculty of Medicine, Kindai University, Osaka, Japan.
Ko SasakiDepartment of Hematology and Oncology, Dokkyo Medical University, Tochigi, Japan.
Masahiro KizakiDepartment of Hematology, Saitama Medical Center, Saitama Medical University, Saitama, Japan.
Masashi SawaDepartment of Hematology and Oncology, Anjo Kosei Hospital, Anjo, Japan.
Michihiro HidakaDepartment of Hematology, National Hospital Organization Kumamoto Medical Center, Kumamoto, Japan.
Naoki KobayashiDepartment of Hematology, Sapporo Hokuyu Hospital, Sapporo, Japan.
Satoshi IchikawaDepartment of Hematology, Tohoku University Hospital, Sendai, Japan.
Yuji YonemuraDepartment of Transfusion Medicine and Cell Therapy, Kumamoto University Hospital, Kumamoto, Japan.
Kenta MurotaniBiostatistics Center, Kurume University, Kurume, Japan.
Mami ShimizuKyowa Kirin Co., Ltd, Tokyo, Japan.
Akira MatsudaDepartment of Hemato-Oncology, Saitama Medical University International Medical Center, Saitama Medical University, Saitama, Japan.
Keiya OzawaDivision of Hematology, Jichi Medical University, Tochigi, Japan.
Shinji NakaoDivision of Hematology/Respiratory Medicine, Faculty of Medicine, Institute of Medical Pharmaceutical and Health Sciences, Kanazawa University, Kanazawa, Japan.
Jong Wook LeeDivision of Hematology-Oncology, Hanyang University Seoul Hospital, 222-1 Wangsimni-Ro, Seongdong-Gu, 04763, Seoul, Republic of Korea. jwlee@hyumc.com.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The thrombopoietin receptor agonist (TPO-RA) romiplostim has been shown to be efficacious in the treatment of aplastic anemia (AA) refractory to immunosuppressive therapy. However, there are few reports on predictors of TPO-RA efficacy. We analyzed patient clinical and baseline laboratory data from two clinical trials that enrolled patients with refractory AA, a phase 2 dose-finding study (NCT02094417) and a phase 2/3 study (NCT02773290), to determine predictors of romiplostim efficacy. Data from 66 patients (35 from the phase 2 dose-finding study and 31 from the phase 2/3 study), median age 47.0 (range 20-78) years, were analyzed. Of these, 51.5% (34/66) had non-severe AA, 86.4% (57/66) had received prior therapy with anti-thymocyte globulin plus cyclosporine, and 80.3% (53/66) were transfusion-dependent. At 27 weeks, no patients achieved a complete response, 57.4% (35/61) achieved a partial response, 49.0% (24/49) became red blood cell transfusion-independent, and 56.8% (21/37) became platelet transfusion-independent. Univariate logistic regression analysis identified duration from diagnosis (P = 0.040), baseline reticulocyte count (P < 0.001), and platelet count (P < 0.001) as predictors of response to romiplostim at 27 weeks. Receiver operating characteristic curve analysis identified a cutoff value of 30.77 × 10

Indexed as

Anemia, AplasticReceptors, FcRecombinant Fusion ProteinsThrombopoietinAdultAgedFemaleHumansMaleMiddle AgedReceptors, ThrombopoietinTreatment OutcomeYoung AdultReceptors, FcReceptors, ThrombopoietinRecombinant Fusion ProteinsromiplostimThrombopoietinAplastic anemiaHematological responsePredictorRefractoryReticulocyteRomiplostim

Identifiers

PMID40593138
PMCPMC12431918

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Registered trials

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.