Evidence map›Paper›PMID 39780402›Full record

ArticlePediatric blood & cancer2025

Thrombopoietin Receptor Agonists for Thrombocytopenia in Pediatric Hematologic Malignancies.

Amanda E Marinoff, Allyson Thrall, Kathryn Aaronson, Benjamin S Braun, Maria Castellanos, Julia Chu, Michelle Hermiston, Benjamin J Huang, Anya Levinson, Erica Southworth and 3 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Pediatric blood & cancer, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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

2 citing papers in PubMed.

  1. Review
  2. 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

13 authors.

Amanda E MarinoffDepartment of Pediatrics, Benioff Children's Hospitals, University of California, San Francisco, California, USA.ORCID https://orcid.org/0000-0001-8600-6400
Allyson ThrallDepartment of Pediatrics, Benioff Children's Hospitals, University of California, San Francisco, California, USA.
Kathryn AaronsonDepartment of Pediatrics, Benioff Children's Hospitals, University of California, San Francisco, California, USA.
Benjamin S BraunDepartment of Pediatrics, Benioff Children's Hospitals, University of California, San Francisco, California, USA.
Maria CastellanosDepartment of Pediatrics, Benioff Children's Hospitals, University of California, San Francisco, California, USA.
Julia ChuDepartment of Pediatrics, Benioff Children's Hospitals, University of California, San Francisco, California, USA.
Michelle HermistonDepartment of Pediatrics, Benioff Children's Hospitals, University of California, San Francisco, California, USA.
Benjamin J HuangDepartment of Pediatrics, Benioff Children's Hospitals, University of California, San Francisco, California, USA.
Anya LevinsonDepartment of Pediatrics, Benioff Children's Hospitals, University of California, San Francisco, California, USA.
Erica SouthworthDepartment of Pediatrics, Benioff Children's Hospitals, University of California, San Francisco, California, USA.
Beth Apsel WingerDepartment of Pediatrics, Benioff Children's Hospitals, University of California, San Francisco, California, USA.ORCID https://orcid.org/0000-0002-6909-8072
Adam OlshenDepartment of Epidemiology and Biostatistics, University of California San Francisco, San Francisco, California, USA.
Elliot StieglitzDepartment of Pediatrics, Benioff Children's Hospitals, University of California, San Francisco, California, USA.ORCID https://orcid.org/0000-0001-7032-4623

Funding

Translational InformaticsP30CA082103 · NCI · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI Alan Ashworth · 1999 to 2026
$209.7M
Research Training in Childhood CancerT32CA128583 · NCI · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI Eric Alejandro Sweet-Cordero · 2007 to 2026
$6.0M
UCSF Paul Calabresi K12 Career Development Program in Clinical OncologyK12CA260225 · NCI · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI BERGSLAND, EMILY K, BIVONA, TREVER G · 2021 to 2025
$2.6M
Co-targeting BET Bromodomain Proteins and Aberrant Signaling in AML.K08CA256489 · NCI · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI HUANG, BENJAMIN · 2021 to 2025
$1.2M
NCI NIH HHS K08 CA256489NCI NIH HHS K12 CA260225NCI NIH HHS P30 CA082103NCI NIH HHS T32 CA128583
6 · The paper itself

Abstract

backgroundThrombopoietin receptor agonists (TPO-RAs) have demonstrated efficacy in treating clinically significant thrombocytopenia, including chemotherapy-induced thrombocytopenia in adults. However, data regarding their safety and efficacy in pediatric, adolescents, and young adult (AYA) patients with hematologic malignancies are limited.

methodsWe retrospectively identified 15 pediatric and AYA patients aged 25 years or younger with hematologic malignancies treated with a TPO-RA at UCSF Benioff Children's Hospitals between 2015 and 2023. Platelet counts and transfusion requirements were compared before and after TPO-RA therapy.

resultsThe median age at TPO-RA initiation was 16 years (range: 7-25 years). Nine patients (60%) had a history of bleeding or comorbidity that predisposed to severe bleeding risk. Eleven patients received romiplostim and four patients received eltrombopag. The median platelet count significantly increased from 24 × 10

conclusionTPO-RAs were effective in managing refractory thrombocytopenia in pediatric and young adult patients being treated for hematologic malignancies, with a favorable safety profile, even among patients with multiple comorbidities. These findings warrant further investigation through prospective clinical trials to confirm efficacy and establish clinical guidelines for this population.

Indexed as

BenzoatesHematologic NeoplasmsHydrazinesPyrazolesReceptors, FcReceptors, ThrombopoietinRecombinant Fusion ProteinsThrombocytopeniaThrombopoietinAdolescentAdultChildFemaleFollow-Up StudiesHumansMaleBenzoateseltrombopagHydrazinesPyrazolesReceptors, FcReceptors, ThrombopoietinRecombinant Fusion ProteinsromiplostimThrombopoietinadolescenteltrombopaghematologic malignanciesleukemiapediatricromiplostimthrombopoietin receptor agonistyoung adults

Identifiers

PMID39780402
PMCPMC12103215

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.