Evidence map›Paper›PMID 26321218›Full record

Trial reportJournal of cystic fibrosis : official journal of the European Cystic Fibrosis Society2016

Inhaled alpha1-proteinase inhibitor therapy in patients with cystic fibrosis.

Amit Gaggar, Junliang Chen, James F Chmiel, Henry L Dorkin, Patrick A Flume, Rhonda Griffin, David Nichols, Scott H Donaldson

Abstract readMulticenter StudyRandomized Controlled Trial
In one paragraph

Trial report in Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 34 papers.

0numbers the graph read from it
0cells of the map it votes in
34citing papers in PubMed
4.8field-weighted citation impact, top 5% of its field
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

34 citing papers in PubMed, 53 citations in OpenAlex.

  1. Article
  2. Review
  3. Article
  4. Review
  5. Review
  6. Making real progress in the world of bronchiectasis.Journal of thoracic disease · 2025
    Article
  7. Article
  8. Review
  9. Neutrophil serine proteases in cystic fibrosis: role in disease pathogenesis and rationale as a therapeutic target.European respiratory review : an official journal of the European Respiratory Society · 2024
    Review
  10. Review
  11. Nebulizers effectiveness on pulmonary delivery of alpha-1 antitrypsin.Drug delivery and translational research · 2023
    Article
  12. Review
  13. Article
  14. The future of inhalation therapy in chronic obstructive pulmonary disease.Current research in pharmacology and drug discovery · 2022
    Review
  15. Review
  16. Review
  17. Review
  18. Alpha-1 antitrypsin for cystic fibrosis complicated by severe cytokinemic COVID-19.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2021
    Article
  19. Review
  20. 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

8 authors at 6 institutions in 1 country.

Amit GaggarUniversity of Alabama at Birmingham, Birmingham, AL, United States.
Junliang ChenClinical Development, Bioscience Industrial Group, Grifols Inc., Research Triangle Park, NC, United States.
James F ChmielDivision of Pediatric Pulmonology, Case Western Reserve, University School of Medicine and Rainbow Babies and Children's Hospital, Cleveland, OH, United States.
Henry L DorkinHarvard Medical School, Boston, MA, United States.
Patrick A FlumeMedical University of South Carolina, Charleston, SC, United States.
Rhonda GriffinClinical Development, Bioscience Industrial Group, Grifols Inc., Research Triangle Park, NC, United States. Electronic address: Rhonda.Griffin@grifols.com.
David NicholsNational Jewish Health, Denver, CO, United States.
Scott H DonaldsonUniversity of North Carolina at Chapel Hill School of Medicine, Chapel Hill, NC, United States.
Harvard University · USMedical University of South Carolina · USRainbow Babies & Children's Hospital · USUniversity of Alabama at Birmingham · USUniversity of Colorado Denver · USUniversity of North Carolina at Chapel Hill · US

Funding

Re-Entry Supplement: Investigation of Oral Microbial Enzymes for the Detection and Treatment of Periodontal DiseaseUL1TR002489 · NCATS · UNIV OF NORTH CAROLINA CHAPEL HILL · PI BUSE, JOHN BERNARD, SHAHEEN, NICHOLAS J · 2018 to 2022
$48.6M
North Carolina Translational & Clinical Sciences Institute (NC TraCS)UL1TR001111 · NCATS · UNIV OF NORTH CAROLINA CHAPEL HILL · PI BUSE, JOHN BERNARD, CAREY, TIMOTHY S · 2013 to 2017
$43.9M
UAB CF Research and Translation Core CenterP30DK072482 · NIDDK · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI David M. Bedwell · 2007 to 2026
$23.0M
South Carolina Clinical & Translational Research Institute (SCTR)UL1TR000062 · NCATS · MEDICAL UNIVERSITY OF SOUTH CAROLINA · PI BRADY, KATHLEEN T. · 2012 to 2014
$9.4M
NCATS NIH HHS 1UL1TR001111NCATS NIH HHS UL1 TR000062NCATS NIH HHS UL1 TR001111NCATS NIH HHS UL1 TR002489NCATS NIH HHS ULT TR000062NIDDK NIH HHS L30 DK069164NIDDK NIH HHS P30 DK072482
6 · The paper itself

Abstract

backgroundInhaled alpha1-proteinase inhibitor (PI) is known to reduce neutrophil elastase burden in some patients with CF. This phase 2a study was designed to test inhaled Alpha-1 HC, a new aerosolized alpha1-PI formulation, in CF patients.

methodsWe performed a randomized, double-blind, placebo-controlled study and evaluated the safety of 100 or 200mg of inhaled Alpha-1 HC once daily for 3 weeks in subjects with CF. Thirty adult subjects were randomized in a 2:1 ratio to receive Alpha-1 HC or placebo.

resultsDrug delivery was confirmed by a dose-dependent increase in the sputum alpha1-PI. Seven (20.0%) of the 35 adverse events in the 100-mg dose group, 3 (13.0%) of 23 in the 200-mg dose group, and 4 (14.3%) of 28 in the placebo group were drug-related in these subjects. One serious adverse event occurred in 1 subject within each group.

conclusionsAlpha-1 HC inhalation was safe and well tolerated.

Indexed as

Administration, InhalationAdultalpha 1-AntitrypsinCystic FibrosisDose-Response Relationship, DrugDouble-Blind MethodFemaleFollow-Up StudiesHumansLeukocyte ElastaseMaleSerine Proteinase InhibitorsSputumTime FactorsTreatment Outcomealpha 1-AntitrypsinLeukocyte ElastaseSerine Proteinase InhibitorsAlpha-1 antitrypsinAlpha-1 HCCystic fibrosisProlastin

Identifiers

PMID26321218
PMCPMC4993024
OpenAlexW1586387535

What OpenQuestion holds

Textmetadata
LicenceTDM
Read underepoch 390

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.