Evidence map›Paper›PMID 39266929›Full record

ArticlePulmonary therapy2024

A Retrospective, Longitudinal Registry Study on the Long-Term Durability of Ivacaftor Treatment in People with Cystic Fibrosis.

Christian Merlo, Teja Thorat, Lisa J McGarry, Christina V Scirica, Maral DerSarkissian, Catherine Nguyen, Yuqian M Gu, Aruna Muthukumar, Joe Healy, Jaime L Rubin and 1 more

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PubMed Full text at PMC Publisher
In one paragraph

Article in Pulmonary therapy, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing 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.

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

6 citing papers in PubMed.

  1. Trial
  2. Article
  3. Review
  4. Review
  5. Review
  6. 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.

Christian MerloDivision of Pulmonary and Critical Care Medicine, Johns Hopkins University School of Medicine, 1830 E. Monument Street 5 Floor, Baltimore, MD, 21205, USA. cmerlo@jhmi.edu.ORCID http://orcid.org/0000-0002-9283-3928
Teja ThoratVertex Pharmaceuticals Incorporated, Boston, MA, USA.ORCID http://orcid.org/0000-0003-2856-752X
Lisa J McGarryVertex Pharmaceuticals Incorporated, Boston, MA, USA.ORCID http://orcid.org/0000-0003-3454-9184
Christina V SciricaVertex Pharmaceuticals Incorporated, Boston, MA, USA.
Maral DerSarkissianAnalysis Group, Inc., Los Angeles, CA, USA.
Catherine NguyenAnalysis Group, Inc., Los Angeles, CA, USA.
Yuqian M GuAnalysis Group, Inc., Los Angeles, CA, USA.
Aruna MuthukumarAnalysis Group, Inc., Los Angeles, CA, USA.
Joe HealyVertex Pharmaceuticals Incorporated, Boston, MA, USA.
Jaime L RubinVertex Pharmaceuticals Incorporated, Boston, MA, USA.
M Alan BrookhartDuke University, Durham, NC, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionIvacaftor (IVA) has been shown to change the trajectory of cystic fibrosis (CF) disease progression by slowing the rate of lung function decline in clinical studies. Long-term real-world data help to confirm the durability of this response.

methodsThis non-interventional, longitudinal study used data from the US CF Foundation Patient Registry to describe the annualized rate of change in lung function in people with CF receiving IVA. The IVA-treated cohort included people with CF aged ≥ 6 years who had ≥ 1 CF transmembrane conductance regulator (CFTR)-gating mutation and initiated IVA between 31 January 2012 and 31 December 2018. An age-matched comparator cohort included people with CF heterozygous for the F508del-CFTR mutation and a minimal function mutation (R117H excluded) and had not received CFTR modulator therapy. Baseline characteristics were balanced using standardized mortality ratio (SMR) weights computed from estimated propensity scores. The annualized rate of change in percent predicted forced expiratory volume in 1 s (ppFEV

resultsIn the 5-year follow-up period, 548 people were in the IVA-treated and 541 in the comparator cohorts after SMR weighting. The annualized rate of change in ppFEV

conclusionIVA showed a durable clinical benefit by slowing the rate of lung function decline over 5 years. Results support a sustained and consistent impact of IVA on lung function trajectory in people with CF. Word count: 300 (limit: 300 words).

Indexed as

CFTR modulatorCystic fibrosisDisease trajectoryIvacaftorLongitudinal studyLung function declineppFEV1Registry study

Identifiers

PMID39266929
PMCPMC11573995

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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.