Evidence map›Paper›PMID 42481135›Full record

Trial reportBritish journal of haematology2026

Avatrombopag for persistent and chronic immune thrombocytopenia in children: A post hoc analysis of clinical characteristics of response from a phase 3b clinical trial.

Rachael F Grace, Göksel Leblebisatan, Yesim Aydinok, Sule Unal, John Grainger, Amanda B Grimes, Michele P Lambert, Jessica Zhang, Brian D Jamieson, Faizan Sattar and 3 more

Registry-linked trialAbstract readClinical Trial, Phase IIIRandomized Controlled TrialMulticenter Study
PubMed Publisher
In one paragraph

Trial report in British journal of haematology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04516967 (Randomized, Double-blind, Placebo-controlled Study With Open-label Extension Phase to Evaluate the Efficacy and Safety of Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia for ≥6 Months), which is not on this map. Not yet cited in PubMed.

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

NCT04516967 phase3completednot on this map

Randomized, Double-blind, Placebo-controlled Study With Open-label Extension Phase to Evaluate the Efficacy and Safety of Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia for ≥6 Months

TypeinterventionalSponsorSobi, Inc.Ran2021 to 2025Enrolled75ConditionsImmune ThrombocytopeniaArmsAvatrombopag, Placebo
3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

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.

Rachael F GraceDepartment of Pediatrics, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA.ORCID https://orcid.org/0000-0001-7302-0449
Göksel LeblebisatanDepartment of Pediatric Hematology, Çukurova University Medical Faculty, Adana, Turkey.ORCID https://orcid.org/0000-0003-3435-757X
Yesim AydinokDepartment of Pediatric Hematology and Oncology, Ege University School of Medicine, Izmir, Turkey.ORCID https://orcid.org/0000-0001-6194-6826
Sule UnalDepartment of Pediatric Hematology, Faculty of Medicine, Hacettepe University, Ankara, Turkey.ORCID https://orcid.org/0000-0002-3842-8788
John GraingerDepartment of Haematology, Royal Manchester Children's Hospital, Manchester, UK.
Amanda B GrimesTexas Children's Cancer and Hematology Center, Texas Children's Hospital, Houston, Texas, USA.ORCID https://orcid.org/0000-0003-2842-9266
Michele P LambertChildren's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.ORCID https://orcid.org/0000-0003-0439-402X
Jessica ZhangSobi, Inc., Morrisville, North Carolina, USA.
Brian D JamiesonSobi, Inc., Morrisville, North Carolina, USA.ORCID https://orcid.org/0009-0000-3926-8117
Faizan SattarSobi, Inc., Morrisville, North Carolina, USA.ORCID https://orcid.org/0009-0006-5550-5729
Scott KolodnySobi, Inc., Morrisville, North Carolina, USA.ORCID https://orcid.org/0009-0008-7793-8722
Michael VredenburgSobi, Inc., Morrisville, North Carolina, USA.ORCID https://orcid.org/0000-0001-7691-8027
AVA‐PED‐301 Study Group

Funding

Sobi, Inc.
6 · The paper itself

Abstract

Avatrombopag treatment resulted in significantly higher platelet response rates in patients when compared with placebo in a phase 3b study of patients with paediatric immune thrombocytopenia (ITP) (NCT04516967). These post hoc analyses of the 12-week core phase further assessed the efficacy of avatrombopag, considering the influence of baseline characteristics and protocol-defined dose adjustments on the achievement and sustainability of response. The cumulative percentage of patients who achieved a platelet response (2 consecutive platelet counts ≥50 × 10

Indexed as

HydrazinesPurpura, Thrombocytopenic, IdiopathicThiazolesThiazolidinesAdolescentChildChild, PreschoolChronic DiseaseFemaleHumansInfantMalePlatelet CountThiophenesTreatment OutcomeavatrombopagHydrazinesThiazolesThiazolidinesThiophenesavatrombopagpaediatric immune thrombocytopeniaplatelet responserefractory immune thrombocytopeniathrombopoietin receptor agonist

Identifiers

PMID42481135

What OpenQuestion holds

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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.