Evidence map›Paper›PMID 27135461›Full record

ArticlePediatric blood & cancer2016

Thrombopoietin Receptor Agonist Use in Children: Data From the Pediatric ITP Consortium of North America ICON2 Study.

Cindy Neunert, Jenny Despotovic, Kristina Haley, Michele P Lambert, Kerri Nottage, Kristin Shimano, Carolyn Bennett, Robert Klaassen, Kimo Stine, Alexis Thompson and 5 more

Abstract readMulticenter Study
In one paragraph

Article in Pediatric blood & cancer, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 30 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
30citing papers in PubMed, 1 pooled it
–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

30 citing papers in PubMed, 1 synthesis or guideline pooled it.

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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

15 authors.

Cindy NeunertDivision of Pediatric Hematology/Oncology/Stem Cell Transplantation and New York Presbyterian Morgan Stanley Children's Hospital, Columbia University Medical Center, New York, New York.
Jenny DespotovicTexas Children's Hematology Center, Baylor College of Medicine, Houston, Texas.
Kristina HaleyDivision of Pediatric Hematology/Oncology, Oregon Health and Sciences University, Portland, Oregon.
Michele P LambertDepartment of Pediatrics and The Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania.
Kerri NottageSt. Jude Children's Research Hospital, Memphis, Tennessee.
Kristin ShimanoDivision of Pediatric Hematology/Oncology and Benioff Children's Hospital, University of California San Francisco, San Francisco, California.
Carolyn BennettAflac Cancer Center and Blood Disorders Service, Emory University School of Medicine and Children's Healthcare of Atlanta, Atlanta, Georgia.
Robert KlaassenDivision of Pediatric Hematology/Oncology, University of Ottawa, Ottawa, Ontario.
Kimo StineDivision of Pediatric Hematology/Oncology, University of Arkansas for Medical Sciences at Arkansas Children's Hospital, Little Rock, Arkansas.
Alexis ThompsonDivision of Hematology/Oncology, Feinberg School of Medicine, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois.
Yves PastoreCHU Ste-Justine, Montreal University, Montreal, Quebec, Canada.
Travis BrownDana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, Massachusetts.
Peter W ForbesClinical Research Center, Boston Children's Hospital, Boston, Massachusetts.
Rachael F GraceDana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, Massachusetts.
Pediatric ITP Consortium of North America (ICON)

Funding

Clinical Hematology Research Career Development Program (K12)K12HL087164 · NHLBI · BOSTON CHILDREN'S HOSPITAL · PI CANTOR, ALAN B. · 2006 to 2016
$3.9M
NHLBI NIH HHS K12 HL087164
6 · The paper itself

Abstract

backgroundData on second-line treatment options for pediatric patients with immune thrombocytopenia (ITP) are limited. Thrombopoietin receptor agonists (TPO-RA) provide a nonimmunosuppressive option for children who require an increased platelet count. PROCEDURE: We performed a multicenter retrospective study of pediatric ITP patients followed at ITP Consortium of North America (ICON) sites to characterize TPO-RA use.

resultsSeventy-nine children had a total of 87 treatments (28 eltrombopag, 43 romiplostim, and eight trialed on both). The majority had primary ITP (82%) and most (60.8%) had chronic ITP. However, 22% had persistent ITP and 18% had newly diagnosed ITP. During the first 3 months of treatment, 89% achieved a platelet count ≥ 50 × 10(9) /l (86% romiplostim, 81% eltrombopag, P = 0.26) at least once in the absence of rescue therapy. The average time to a response was 6.4 weeks for romiplostim and 7.0 weeks for eltrombopag (P = 0.83). Only 40% of patients demonstrated a stable response with consistent dosing over time. An intermittent response with constant dose titration was seen in 15%, and an initial response that waned to no response was seen in 13%. Significant adverse events were minimal with the exception of two patients with thrombotic events and one who developed a neutralizing antibody.

conclusionsOur results demonstrate that TPO-RA agents are being used in children with ITP of varying duration and severity. The response was similar to clinical trials, but the sustainability of response varied. Future studies need to focus on the ideal timing and rationale for these medications in pediatric patients.

Indexed as

AdolescentBenzoatesChildFemaleHumansHydrazinesMalePlatelet CountPurpura, Thrombocytopenic, IdiopathicPyrazolesReceptors, FcReceptors, ThrombopoietinRecombinant Fusion ProteinsRetrospective StudiesThrombopoietinBenzoateseltrombopagHydrazinesPyrazolesReceptors, FcReceptors, ThrombopoietinRecombinant Fusion ProteinsromiplostimThrombopoietinICONimmune thrombocytopeniapediatricsthrombopoietin receptor agoniststreatment

Identifiers

PMID27135461
PMCPMC5718620

What OpenQuestion holds

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Registered trials

None linked

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.