Evidence map›Paper›PMID 36949472›Full record

ArticleTrials2023

BEAT CF pulmonary exacerbations core protocol for evaluating the management of pulmonary exacerbations in people with cystic fibrosis.

Andre Schultz, Charlie McLeod, Scott Berry, Julie Marsh, Anne McKenzie, Mitch Messer, Jamie Wood, Ben Saville, Adam Jaffe, Sarath Ranganathan and 4 more

Open access · goldFull text readClinical Trial Protocol
PubMed Full text at PMC Publisher
In one paragraph

Article in Trials, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed, 1 pooled it
1.3field-weighted citation impact, top 19% 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

1 citing paper in PubMed, 1 synthesis or guideline pooled it, 5 citations in OpenAlex.

  1. Pooled it
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

14 authors at 9 institutions in 2 countries.

Andre SchultzWal-yan Respiratory Research Centre, Telethon Kids Institute, University of Western Australia, Nedlands, Australia.
Charlie McLeodWesfarmers Centre of Vaccines and Infectious Diseases, Telethon Kids Institute, Nedlands, Australia. charlie.mcleod@health.wa.gov.au.ORCID http://orcid.org/0000-0002-3197-7493
Scott BerryBerry Consultants, LLC, Austin, TX, USA.
Julie MarshWesfarmers Centre of Vaccines and Infectious Diseases, Telethon Kids Institute, Nedlands, Australia.
Anne McKenzieSchool of Population and Global Health, University of Western Australia, Nedlands, Australia.
Mitch MesserTelethon Kids CONNECT, Telethon Kids Institute, Nedlands, Australia.
Jamie WoodDepartment of Rehabilitation and Human Performance, Abilities Research Centre, Icahn School of Medicine, Mount Sinai, New York, USA.
Ben SavilleBerry Consultants, LLC, Austin, TX, USA.
Adam JaffeDiscipline of Paediatrics and Child Health, Faculty of Medicine and Health, University of New South Wales, Kensington, Australia.
Sarath RanganathanDepartment of Paediatrics, University of Melbourne, Parkville, Australia.
Steve StickWal-yan Respiratory Research Centre, Telethon Kids Institute, University of Western Australia, Nedlands, Australia.
Peter WarkImmune Health Program, Hunter Medical Research Institute, University of Newcastle, Callaghan, Australia.
Steve WebbAustralian and New Zealand Intensive Care Research Centre, Monash University, Clayton, Australia.
Tom SnellingSydney School of Public Health, The University of Sydney, Sydney, Australia.
The Kids Research Institute Australia · AUBerry & Associates (United States) · USIcahn School of Medicine at Mount Sinai · USJohn Hunter Hospital · AURoyal Children's Hospital · AUSt John of God Subiaco Hospital · AUThe University of Sydney · AUThe University of Western Australia · AUUNSW Sydney · AU

Funding

MRFF GNT1152376
6 · The paper itself

Abstract

backgroundCystic fibrosis (CF) is a rare, inherited, life-limiting condition predominantly affecting the lungs, for which there is no cure. The disease is characterized by recurrent pulmonary exacerbations (PEx), which are thought to drive progressive lung damage. Management of these episodes is complex and generally involves multiple interventions targeting different aspects of disease. The emergence of innovative trials and use of Bayesian statistical methods has created renewed opportunities for studying heterogeneous populations in rare diseases. Here, we present the protocol for the BEAT CF PEx cohort, a prospective, multi-site, perpetual, platform enrolling adults and children with CF. The BEAT CF PEx cohort will be used to evaluate the comparative effectiveness of interventions for the treatment of PEx requiring intensive therapy (PERITs), with a primary focus on short-term improvements in lung function. This will be achieved through the conduct of cohort-nested studies, including adaptive clinical trials, within the BEAT CF PEx cohort. This protocol will outline key features of the BEAT CF PEx cohort, including the design, implementation, data collection and management, governance and analysis, and dissemination of results.

methodsThis platform will be conducted across multiple sites, commencing with CF treatment centers in Australia. People of all ages with a clinical diagnosis of CF will be eligible to participate, except those who have previously received a lung transplant. Data including demographic and clinical information, treatment details, and outcomes (including safety, microbiology, and patient-reported outcome measures including quality of life scores) will be systematically collected and securely stored via a digital centralized trial management system (CTMS). The primary endpoint is the absolute change in the percentage predicted forced expiratory volume in 1 s (ppFEV DISCUSSION: The BEAT CF PEx cohort will report clinical, treatment, and outcome data for PEx among people with CF and is intended to serve as a core (master) protocol for future nested, interventional trials evaluating treatment(s) for these episodes. The protocols for nested sub-studies are beyond the scope of this document and will be reported separately.

trial registrationANZCTR BEAT CF Platform - ACTRN12621000638831. Registration date: Sept. 26, 2022.

Indexed as

Cystic FibrosisAdaptive Clinical Trials as TopicAdultAnti-Bacterial AgentsBayes TheoremChildForced Expiratory VolumeHumansLungMulticenter Studies as TopicProspective StudiesQuality of LifeAnti-Bacterial AgentsClinical trialCohortCystic fibrosisExacerbationsFEV1Lung functionManagementPlatformTreatment

Identifiers

PMID36949472
PMCPMC10031862
OpenAlexW4360600869

What OpenQuestion holds

Textfull text, public
LicenceCC BY
measurements read23
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.