Evidence map›Paper›PMID 41214865›Full record

ArticlePediatric blood & cancer2026

Real-World Use of Avatrombopag in Children and Adolescents With Immune Thrombocytopenia.

Emily M Harris, Caitlin Montcrieff, Candelaria O'Farrell, Praharsha Konde, Michele P Lambert, Amanda B Grimes, Rachael F Grace

Abstract readMulticenter Study
In one paragraph

Article in Pediatric blood & cancer, 2026. 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

7 authors.

Emily M HarrisDana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA.ORCID 0000-0001-8583-6939
Caitlin MontcrieffDana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA.
Candelaria O'FarrellDepartment of Pediatrics, Division of Hematology/Oncology, Baylor College of Medicine, Houston, Texas, USA.
Praharsha KondeDivision of Hematology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.ORCID 0009-0002-7022-2891
Michele P LambertDivision of Hematology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.
Amanda B GrimesDepartment of Pediatrics, Division of Hematology/Oncology, Baylor College of Medicine, Houston, Texas, USA.
Rachael F GraceDana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA.ORCID 0000-0001-7302-0449

Funding

PATHOPHYSIOLOGY OF HUMAN BLOOD CELLST32HL007574 · NHLBI · CHILDREN'S HOSPITAL BOSTON · PI Daniel Evan Bauer · 1985 to 2026
$18.4M
NHLBI NIH HHS T32 HL007574
6 · The paper itself

Abstract

backgroundAvatrombopag, a second-generation thrombopoietin receptor agonist (TPO-RA), is approved for treatment of chronic immune thrombocytopenia (ITP) in adults and has been studied in a Phase 3 trial in pediatric ITP. The reported efficacy, oral administration, safety profile, and lack of dietary restrictions are appealing treatment characteristics for children. Data on real-world use in children are limited.

methodsThis multicenter cohort study included patients diagnosed with ITP at age < 21 years treated with avatrombopag. Clinical features were collected from electronic medical records. Overall platelet response was defined as platelet count ≥ 30 × 10

resultsA total of 58 pediatric patients were treated with avatrombopag. Median time from diagnosis to treatment initiation was 4.6 years, with a median of 3 (range 0-11) prior treatments. Overall platelet response was 89.3% with similar response in primary and secondary ITP. Most patients achieved a response on the initial dose. Response rates did not differ by age at treatment initiation. Median time to platelet response was 13.5 days. High-grade bleeding scores were lower after initiation of treatment. Most patients (62%) switched from another TPO-RA to avatrombopag. Response to prior TPO-RA was associated with higher likelihood of response to avatrombopag (OR 15.00, 95% CI 1.824-187.7, p = 0.017). Of 12 patients with no response to prior TPO-RA, 8 responded to avatrombopag. Adverse effects were rare.

conclusionsIn this real-world study, avatrombopag was highly effective at both improving platelet counts and reducing bleeding in children with ITP, even in patients with secondary ITP or who had failed other TPO-RAs.

Indexed as

Purpura, Thrombocytopenic, IdiopathicReceptors, ThrombopoietinThiazolesThiophenesAdolescentChildChild, PreschoolFemaleFollow-Up StudiesHumansInfantMalePlatelet CountPrognosisYoung AdultavatrombopagReceptors, ThrombopoietinThiazolesThiophenesavatrombopagimmune thrombocytopeniapediatricthrombopoietin receptor agonisttreatment

Identifiers

PMID41214865
PMCPMC12921557

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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.