Evidence map›Paper›PMID 33573995›Full record

Observational studyJournal of cystic fibrosis : official journal of the European Cystic Fibrosis Society2021

Measuring the impact of CFTR modulation on sweat chloride in cystic fibrosis: Rationale and design of the CHEC-SC study.

Edith T Zemanick, Michael W Konstan, Donald R VanDevanter, Steven M Rowe, J P Clancy, Katherine Odem-Davis, Michelle Skalland, Nicole Mayer-Hamblett

Open access · bronzeAbstract readMulticenter StudyObservational Study
In one paragraph

Observational study in Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.

0numbers the graph read from it
0cells of the map it votes in
9citing papers in PubMed
2.3field-weighted citation impact, top 11% 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

9 citing papers in PubMed, 22 citations in OpenAlex.

  1. Observational
  2. Article
  3. Heterogeneity of CFTR modulator-induced sweat chloride concentrations in people with cystic fibrosis.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2024
    Article
  4. Review
  5. Article
  6. Characterizing CFTR modulated sweat chloride response across the cf population: Initial results from the CHEC-SC study.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2023
    Article
  7. Observational
  8. Article
  9. New Therapies to Correct the Cystic Fibrosis Basic Defect.International journal of molecular sciences · 2021
    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.

Edith T ZemanickDepartment of Pediatrics, University of Colorado Anschutz Medical Campus, Aurora, CO 80045, USA. Electronic address: edith.zemanick@childrenscolorado.org.
Michael W KonstanDepartment of Pediatrics, Case Western Reserve University School of Medicine, Cleveland OH USA; Rainbow Babies and Children's Hospital, Cleveland OH USA.
Donald R VanDevanterDepartment of Pediatrics, Case Western Reserve University School of Medicine, Cleveland OH USA.
Steven M RoweDepartment of Medicine and the Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, Birmingham, AL USA.
J P ClancyCystic Fibrosis Foundation, Bethesda, MD USA.
Katherine Odem-DavisSeattle Children's Research Institute, Seattle, WA 98145, USA.
Michelle SkallandSeattle Children's Research Institute, Seattle, WA 98145, USA.
Nicole Mayer-HamblettSeattle Children's Research Institute, Seattle, WA 98145, USA; Department of Biostatistics and Pediatrics, University of Washington, Seattle, WA 98195, USA.
Children's Hospital Colorado · USCystic Fibrosis Foundation · USRainbow Babies & Children's Hospital · USUniversity of Alabama at Birmingham · USUniversity of Washington · USUniversity School · US

Funding

Transform Dissemination and Implementation Science in CTSA ProgramsUL1TR002319 · NCATS · UNIVERSITY OF WASHINGTON · PI John K. Amory · 2017 to 2026
$100.0M
National Dissemination of I-Corps@NCATS: Accelerating Translation through CommercializationUL1TR003096 · NCATS · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI GUTIERREZ, ORLANDO M, KIMBERLY, ROBERT P. · 2019 to 2023
$43.6M
Clinical and Translational Science Collaborative of ClevelandUL1TR002548 · NCATS · CASE WESTERN RESERVE UNIVERSITY · PI MCCOMSEY, GRACE A · 2018 to 2022
$35.5M
UAB CF Research and Translation Core CenterP30DK072482 · NIDDK · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI Brian R. Davis · 2007 to 2026
$23.0M
Translational Research Center to Expedite Novel Therapies in Cystic FibrosisP30DK089507 · NIDDK · SEATTLE CHILDREN'S HOSPITAL · PI Christopher Hooper Goss · 2010 to 2026
$21.4M
Project 3 - Personalized cystic fibrosis treatment strategies with synergistic therapies Identified in vitro.P01HL128192 · NHLBI · INDIANA UNIVERSITY INDIANAPOLIS · PI GASTON, BENJAMIN · 2016 to 2020
$12.2M
Translational Program in CFTR-Related Airway DiseasesR35HL135816 · NHLBI · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI ROWE, STEVEN MARK · 2017 to 2022
$6.3M
NCATS NIH HHS UL1 TR002319NCATS NIH HHS UL1 TR002548NCATS NIH HHS UL1 TR003096NHLBI NIH HHS P01 HL128192NHLBI NIH HHS R35 HL135816NIDDK NIH HHS P30 DK072482NIDDK NIH HHS P30 DK089507
6 · The paper itself

Abstract

backgroundThe Characterizing CFTR Modulated Changes in Sweat Chloride and their Association with Clinical Outcomes (CHEC-SC) study is a large epidemiologic study designed to determine the relationship between sweat chloride response and clinical outcomes in people with cystic fibrosis (CF) on commercially approved CFTR modulators. A challenge to study feasibility was capturing sweat chloride measurements before modulator initiation. We tested the hypothesis that historic sweat chloride approximated contemporary pre-modulator values to estimate CFTR modulator-induced changes, allowing a single-visit study design.

methodsGOAL and PROSPECT were multi-center prospective studies of individuals initiating ivacaftor or lumacaftor-ivacaftor. At enrollment, pre-modulator sweat chloride was measured and historic results recorded. Post-modulator sweat chloride was measured at 1, 3 and 6 months. For this analysis, differences between historic and pre-modulator sweat chloride were estimated. CFTR modulator-induced sweat chloride mean changes were compared using historic and pre-modulator sweat chloride.

resultsPaired historic and pre-modulator sweat chloride (n=406 participants) revealed a non-significant mean change of -1.0 mmol/L (95% CI: -2.71, 0.66) over an average of 17.2 years. Calculating sweat response to ivacaftor or lumacaftor-ivacaftor using historic or pre-modulator values resulted in similar estimates of modulator response. Based on these results, the CHEC-SC study was designed with a single, post-modulator sweat chloride measurement.

conclusionsHistoric sweat chloride values provide a reliable estimate of pre-modulator sweat chloride for people starting on modulator therapy. The CHEC-SC study anticipates capturing approximately 5,000 sweat chloride values, providing an unprecedented understanding of sweat chloride across the CF population in the era of CFTR modulators.

Indexed as

AdolescentAdultAgedAminophenolsAminopyridinesBenzodioxolesChildChloride Channel AgonistsChloridesCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorDrug CombinationsFemaleHumansMaleMiddle AgedAminophenolsAminopyridinesBenzodioxolesChloride Channel AgonistsChloridesCystic Fibrosis Transmembrane Conductance RegulatorDrug Combinationslumacaftor, ivacaftor drug combinationQuinolonesCFTR modulatorIvacaftorLumacaftor-ivacaftorSweat chlorideSweat test

Identifiers

PMID33573995
PMCPMC8349375
OpenAlexW3126766321

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.