Evidence map›Paper›PMID 42257533›Full record

ArticleClinical and translational science2026

Eltrombopag Combined With Cyclosporine A in the Treatment of Pediatric Patients With SAA: Establishing the Range for Eltrombopag Concentrations.

Fashuang Li, Na Li, Xiaoling Tang, Tilong Huang, Linbo Li, Huiying Li, Danyang Ren, Caixia Tu, Dan Li

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Article in Clinical and translational science, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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1 · What the graph read from it

What it found

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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

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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

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4 · The record

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5 · Who and what money

Authors and funding

9 authors.

Fashuang LiPharmacy Department, Kunming Children's Hospital, Kunming, China.ORCID 0000-0002-1167-3943
Na LiPharmacy Department, Yunnan Provincial Hospital of Infectious Disease, Kunming, China.ORCID 0009-0001-6328-8174
Xiaoling TangOrthopedics Department, Kunming First People's Hospital, Kunming, China.
Tilong HuangHematology Department, Kunming Children's Hospital, Kunming, China.
Linbo LiPharmacy Department, Kunming Children's Hospital, Kunming, China.
Huiying LiPharmacy Department, Kunming Children's Hospital, Kunming, China.ORCID 0000-0002-1920-5722
Danyang RenPharmacy Department, Kunming Children's Hospital, Kunming, China.
Caixia TuPharmacy Department, Kunming Children's Hospital, Kunming, China.
Dan LiPharmacy Department, Kunming Children's Hospital, Kunming, China.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Eltrombopag (ELT), as an oral thrombopoietin receptor agonist, in combination with cyclosporine A (CsA) provides a novel therapeutic option for pediatric severe aplastic anemia (SAA). However, current clinical protocols predominantly adopt fixed-dose regimens without adequate consideration of pediatric pharmacokinetic characteristics and drug-drug interactions, resulting in interindividual variability in efficacy and safety, as well as a lack of standardized therapeutic drug monitoring criteria for personalized treatment. This retrospective study analyzed treatment data from 83 pediatric patients with SAA (diagnosed at Kunming Children's Hospital between January 2020 and May 2025) to evaluate dose-concentration, concentration-efficacy, and concentration-adverse effects (ADRs) relationships, aiming to establish ELT concentration thresholds and reference ranges associated with therapeutic efficacy and ADRs. Results demonstrated a significant positive correlation between dosage and concentration (Pearson analysis). The study identified an efficacy threshold of 2.5 μg/mL (AUC = 0.85) and an ADRs threshold of 4.0 μg/mL (AUC = 0.82). Kaplan-Meier analysis revealed significant differences in time-to-response and time-to-adverse events among different concentration groups (4.0 μg/mL). The study confirms that ELT concentrations are closely associated with clinical efficacy and ADRs, and the established reference range of 2.5-4.0 μg/mL can serve as a basis for individualized pediatric treatment.

Indexed as

Anemia, AplasticBenzoatesCyclosporineHydrazinesPyrazolesAdolescentChildChild, PreschoolDose-Response Relationship, DrugDrug InteractionsDrug MonitoringDrug Therapy, CombinationFemaleHumansImmunosuppressive AgentsMaleBenzoatesCyclosporineeltrombopagHydrazinesImmunosuppressive AgentsMPL protein, humanPyrazolesReceptors, Thrombopoietinchildrencyclosporineeltrombopagsevere aplastic anemiatherapeutic drug monitoringtrough concentration

Identifiers

PMID42257533
PMCPMC13244668

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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.