Evidence map›Paper›PMID 40910281›Full record

Trial reportPharmacotherapy2025

A Multicenter, Open-Label Study to Assess the Safety of Nebulized Tissue Plasminogen Activator for the Acute Treatment of Pediatric Plastic Bronchitis: The PLATyPuS Trial.

Kathleen A Stringer, David Goldberg, Sharon Chen, Philip Thrush, Eric M Graham, Adam Lubert, Jeffrey Myers, Laura McLellan, Thomas Flott, Samya Nasr and 1 more

Registry-linked trialAbstract readMulticenter StudyClinical Trial, Phase II
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Pharmacotherapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02315898 (Safety and Efficacy of Inhaled Tissue Plasminogen Activator), which is not on this map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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.

NCT02315898 phase2completednot on this map

Safety and Efficacy of Inhaled Tissue Plasminogen Activator (tPA) for the Acute Treatment of Pediatric Plastic Bronchitis

TypeinterventionalSponsorUniversity of MichiganRan2018 to 2023Enrolled40ConditionsPlastic Bronchitis, Protein-Losing Enteropathies, HealthyArmsTreatment-inhaled tPA
3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Review
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

11 authors.

Kathleen A StringerDepartment of Clinical Pharmacy and the NMR Metabolomics Laboratory, College of Pharmacy, University of Michigan, Ann Arbor, Michigan, USA.ORCID 0000-0003-0238-7774
David GoldbergDivision of Pediatric Cardiology, Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.ORCID 0000-0002-4194-2067
Sharon ChenDivision of Pediatric Cardiology, Department of Pediatrics, Lucile Packard Children's Hospital, Stanford University, Palo Alto, California, USA.ORCID 0000-0002-3705-258X
Philip ThrushDivision of Pediatric Cardiology, Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA.
Eric M GrahamDivision of Pediatric Cardiology, Department of Pediatrics, Medical University of South Carolina, Charleston, South Carolina, USA.ORCID 0009-0007-8103-2723
Adam LubertDivision of Pediatric Cardiology, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.ORCID 0000-0001-6401-9980
Jeffrey MyersDepartment of Pathology, Lung and Thoracic Pathology, University of Michigan Health System, Ann Arbor, Michigan, USA.
Laura McLellanDepartment of Clinical Pharmacy and the NMR Metabolomics Laboratory, College of Pharmacy, University of Michigan, Ann Arbor, Michigan, USA.
Thomas FlottDepartment of Clinical Pharmacy and the NMR Metabolomics Laboratory, College of Pharmacy, University of Michigan, Ann Arbor, Michigan, USA.ORCID 0000-0002-3962-6014
Samya NasrDivision of Pediatric Pulmonology, Department of Pediatrics, University of Michigan Health System, Ann Arbor, Michigan, USA.ORCID 0000-0002-4124-316X
Kurt R SchumacherDivision of Pediatric Cardiology, Department of Pediatrics, University of Michigan Health System, Ann Arbor, Michigan, USA.ORCID 0000-0001-8659-736X

Funding

The Vanderbilt Institute for Clinical and Translational Research (VICTR)UL1TR000445 · NCATS · VANDERBILT UNIVERSITY MEDICAL CENTER · PI BERNARD, GORDON RAPHAEL · 2012 to 2016
$41.4M
Translational Metabolomics in Critical CareR35GM136312 · NIGMS · UNIVERSITY OF MICHIGAN AT ANN ARBOR · PI KATHLEEN A STRINGER · 2020 to 2026
$2.8M
Daniel Foy Plastic Bronchitis FundFDA HHS R01 FD005393Michigan Institute for Clinical & Health Research (MICHR)NCATS NIH HHS UL1 TR000445NIGMS NIH HHS R35 GM136312NIH HHS NIH/NCATS UL1 TR000445NIH HHS NIH/NIGMSR35GM136312U.S. Food and Drug Administration R01FD005393
6 · The paper itself

Abstract

introductionPediatric plastic bronchitis (PB) is a rare complication of surgically palliated congenital heart disease (CHD). Fibrin casts obstruct airways and can cause respiratory distress. There are no therapeutics approved by the United States Food and Drug Administration to treat PB, but inhaled tissue plasminogen activator (tPA) has been anecdotally used to relieve symptoms. We conducted a phase II open-label clinical trial to test the safety of inhaled tPA in pediatric PB.

methodsPatients with an acute exacerbation of PB requiring hospitalization were enrolled to test the safety of an inhaled tPA regimen (5 mg every 6 h). The primary end point was to assess the safety and tolerability of repeated doses of nebulized, inhaled tPA in pediatric patients with acute PB. Safety parameters consisted of clinical laboratories to assess bleeding, which were measured prior to, during, and after tPA treatment. To benchmark efficacy using spirometry and oxygen saturation, children with Fontan-palliated CHD without a history of PB, with and without protein losing enteropathy (PLE), and healthy children were enrolled in a control arm that did not receive tPA.

resultsOf the 10 patients with PB screened for enrollment, eight qualified for immediate treatment with inhaled tPA. A total of 29 non-PB participants (PLE, n = 8 [10-18 yo]; CHD, n = 9 [8-17 yo]; and healthy, n = 12 [7-16 yo]) were enrolled. There were no differences in pretreatment clinical blood laboratory values of hemostasis and those during and after treatment with the study drug (primary safety outcome). However, there were four episodes of self-limiting epistaxis related to the study drug. Inhaled tPA statistically improved oxygen saturation although this was moderate and likely not clinically significant; inhaled tPA did not alter spirometry values.

conclusionIn this small, phase II study, repeated doses of inhaled tPA in patients with an acute exacerbation of PB did not result in disrupted systemic coagulation or hematological homeostasis or serious bleeding. However, patients should be monitored for localized bleeding. Larger, randomized trials are needed to provide more comprehensive assessments of bleeding risk and to further assess efficacy.

trial registrationClinicalTrials.gov identifier: NCT02315898.

Indexed as

BronchitisFibrinolytic AgentsTissue Plasminogen ActivatorAcute DiseaseAdministration, InhalationAdolescentChildChild, PreschoolFemaleHeart Defects, CongenitalHumansMaleNebulizers and VaporizersTreatment OutcomeFibrinolytic AgentsTissue Plasminogen ActivatorBrasfield scorecongenital heart diseaseFontan physiologymetabolomicsoxygen saturationspirometry

Identifiers

PMID40910281
PMCPMC12530010

What Socratic 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 Socratic graph.