Evidence map›Paper›PMID 39389908›Full record

Trial reportBritish journal of haematology2024

Phase II trial of imatinib mesylate in patients with PDGFRA/B-negative hypereosinophilic syndrome.

Dong Hyun Kim, Seokhyeon Kim, Seonyang Park, Ja Min Byun, Junshik Hong, Dong-Yeop Shin, Inho Kim, Soo Mee Bang, Jeong-Ok Lee, Ji Yun Lee and 6 more

Abstract readClinical Trial, Phase II
In one paragraph

Trial report in British journal of haematology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

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

3 citing papers in PubMed.

  1. Trial
  2. Review
  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

16 authors.

Dong Hyun KimDepartment of Internal Medicine, Seoul National University Hospital, Seoul, Korea.ORCID https://orcid.org/0000-0002-0369-5763
Seokhyeon KimCancer Research Institute, Seoul National University College of Medicine, Seoul, Korea.
Seonyang ParkDepartment of Internal Medicine, Inje University Haeundae Paik Hospital, Busan, Korea.
Ja Min ByunDepartment of Internal Medicine, Seoul National University Hospital, Seoul, Korea.ORCID https://orcid.org/0000-0003-1780-5553
Junshik HongDepartment of Internal Medicine, Seoul National University Hospital, Seoul, Korea.ORCID https://orcid.org/0000-0002-7829-397X
Dong-Yeop ShinDepartment of Internal Medicine, Seoul National University Hospital, Seoul, Korea.ORCID https://orcid.org/0000-0003-1753-8846
Inho KimDepartment of Internal Medicine, Seoul National University Hospital, Seoul, Korea.ORCID https://orcid.org/0000-0002-8785-6201
Soo Mee BangDepartment of Internal Medicine, Seoul National University Bundang Hospital, Seongnam, Korea.ORCID https://orcid.org/0000-0002-0938-3007
Jeong-Ok LeeDepartment of Internal Medicine, Seoul National University Bundang Hospital, Seongnam, Korea.
Ji Yun LeeDepartment of Internal Medicine, Seoul National University Bundang Hospital, Seongnam, Korea.
Sang-A KimDepartment of Internal Medicine, Seoul National University Bundang Hospital, Seongnam, Korea.ORCID https://orcid.org/0000-0002-8529-0232
Ki Hwan KimDepartment of Internal Medicine, Seoul National University Boramae Hospital, Seoul, Korea.
Yeun-Jun ChungDepartment of Microbiology, College of Medicine, The Catholic University of Korea, Seoul, Korea.
Seung-Hyun JungIntegrated Research Center for Genome Polymorphism, Precision Medicine Research Center, College of Medicine, The Catholic University of Korea, Seoul, Korea.
Youngil KohDepartment of Internal Medicine, Seoul National University Hospital, Seoul, Korea.ORCID https://orcid.org/0000-0002-4750-931X
Sung-Soo YoonDepartment of Internal Medicine, Seoul National University Hospital, Seoul, Korea.ORCID https://orcid.org/0000-0003-2591-7459

Funding

Korean Government (Ministry of Science, ICT, and Future Planning) NRF-2021R1A4A2001553National Research Foundation of Korea (NRF)
6 · The paper itself

Abstract

The role of imatinib in PDGFRA/B-negative hypereosinophilic syndromes (HES) is controversial because of the heterogeneity of HES and the scarcity of prospective studies. We conducted a phase II clinical trial to evaluate the efficacy of imatinib in PDGFRA/B-negative HES. Thirty-two patients were treated with imatinib (100-400 mg daily), and the molecular basis of their response was identified using whole-exome sequencing (WES) and whole-transcriptome sequencing (WTS). The haematological response rate was 46.9%, with a complete haematological response (CHR) rate of 18.8%. The median time to response was 1.5 months. Among the six patients who achieved CHR, five maintained it until the 24th cycle of imatinib and one lost response after 20 months. The median progression-free survival was 4.3 months. WES and WTS were conducted for 11 patients. The number of non-silent mutations did not differ between responders and non-responders. Nine differentially expressed genes, including SNORD15A, were downregulated in responders. STAT5B::RARA, PAK2::PIGX, and FIP1L1::CHIC2 fusions were identified in patients with sustained responses, and RNF130::BRAF and WNK1::KDM5A fusions were identified in non-responders. Imatinib, along with an appropriate biomarker, could be a promising option for PDGFRA/B-negative HES.

Indexed as

Hypereosinophilic SyndromeImatinib MesylateReceptor, Platelet-Derived Growth Factor alphaAdultAgedExome SequencingFemaleHumansMaleMiddle AgedMutationReceptor, Platelet-Derived Growth Factor betaTreatment OutcomeImatinib MesylatePDGFRB protein, humanReceptor, Platelet-Derived Growth Factor alphaReceptor, Platelet-Derived Growth Factor betahypereosinophilic syndromeimatinibPDGFRA/B‐negativewhole‐exome sequencingwhole‐transcriptome sequencing

Identifiers

PMID39389908
PMCPMC11637721

What OpenQuestion holds

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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.