Evidence map›Paper›PMID 38586424›Full record

ArticleHeliyon2024

Real-world effectiveness of elexacaftor/tezacaftor/ivacaftor on the burden of illness in adolescents and adults with cystic fibrosis.

Thomas Keens, Veena Hoffman, Ia Topuria, Ken Elder, Shannon Cerf, Kyra Mulder, Jon Roberts, Jerimiah Lysinger, Maria Del Carmen Reyes, Maria Berdella and 8 more

Abstract read
In one paragraph

Article in Heliyon, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.

0numbers the graph read from it
0cells of the map it votes in
5citing 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

5 citing papers in PubMed.

  1. Article
  2. The impact of elexacaftor-tezacaftor-ivacaftor on cardiometabolic risk factors: a systematic review.European respiratory review : an official journal of the European Respiratory Society · 2026
    Review
  3. Article
  4. Review
  5. Article
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

18 authors.

Thomas KeensChildren's Hospital of Los Angeles, Los Angeles, CA, USA.
Veena HoffmanOM1 Incorporated, Boston, MA, USA.
Ia TopuriaOM1 Incorporated, Boston, MA, USA.
Ken ElderOM1 Incorporated, Boston, MA, USA.
Shannon CerfOM1 Incorporated, Boston, MA, USA.
Kyra MulderOM1 Incorporated, Boston, MA, USA.
Jon RobertsDriscoll Children's Hospital, Corpus Christi, TX, USA.
Jerimiah LysingerBillings Clinic Hospital, Billings, MT, USA.
Maria Del Carmen ReyesChildren's Hospital of Los Angeles, Los Angeles, CA, USA.
Maria BerdellaNorthwell Health, New York, NY, USA.
Anne Marie CairnsMaine Medical Center, Portland, ME, USA.
Manu JainFeinberg School of Medicine, Northwestern University, Chicago, IL, USA.
Vaidyanathan GanapathyVertex Pharmaceuticals Incorporated, Boston, MA, USA.
Yiyue LouVertex Pharmaceuticals Incorporated, Boston, MA, USA.
Bassem MorcosVertex Pharmaceuticals Incorporated, Boston, MA, USA.
Chuntao WuVertex Pharmaceuticals Incorporated, Boston, MA, USA.
Laura SassChildren's Hospital of The King's Daughter, Norfolk, VA, USA.
VX19-CFD-003 Study Group

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background: Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) has been shown to be safe and efficacious in people with cystic fibrosis (CF) aged ≥2 years. Here, we describe results from an observational study assessing change in burden of illness following initiating ELX/TEZ/IVA in real-world settings. Methods: This US-based, multicenter, observational study used data from electronic medical records to evaluate real-world burden of illness before and after ELX/TEZ/IVA initiation in people with CF aged ≥12 years heterozygous for Results: Overall, 206 people with CF were enrolled (mean [SD] age 22.5 [11.1] years; 192 [93.2%] with Conclusions: ELX/TEZ/IVA treatment was associated with improved lung function, increased BMI, reduced frequency of PEx, and improved (i.e., reduced) HbA1c. These results confirm the broad clinical benefits of ELX/TEZ/IVA seen in clinical trials and show the potential for ELX/TEZ/IVA to improve markers of glucose metabolism.

Indexed as

Burden of diseaseCystic fibrosisElexacaftorIvacaftorReal-worldTezacaftor

Identifiers

PMID38586424
PMCPMC10998118

What OpenQuestion holds

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Registered trials

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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.