Evidence map›Paper›PMID 40003259›Full record

ArticleChildren (Basel, Switzerland)2025

Clinical Outcomes in Patients with Cystic Fibrosis Receiving CFTR Modulators: A Comparison of Childhood Versus Adolescent Initiation.

Eman A Toraih, Hassan A Malik, Rahib K Islam, Humza A Pirzadah, Ahmed Abdelmaksoud, Rami M Elshazli, Paul Antwi Boasiako, Shehab Ahmed Alenazi, Angelique Dabel, Jessan A Jishu and 3 more

Abstract read
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In one paragraph

Article in Children (Basel, Switzerland), 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

13 authors.

Eman A ToraihDepartment of Surgery, School of Medicine, Tulane University, New Orleans, LA 70112, USA.ORCID 0000-0001-9267-3787
Hassan A MalikLouisiana State University Health Sciences Center, New Orleans School of Medicine, New Orleans, LA 70112, USA.ORCID 0009-0007-2031-8517
Rahib K IslamLouisiana State University Health Sciences Center, New Orleans School of Medicine, New Orleans, LA 70112, USA.
Humza A PirzadahLouisiana State University Health Sciences Center, New Orleans School of Medicine, New Orleans, LA 70112, USA.ORCID 0009-0009-6815-1896
Ahmed AbdelmaksoudDepartment of Surgery, School of Medicine, Tulane University, New Orleans, LA 70112, USA.
Rami M ElshazliDepartment of Surgery, School of Medicine, Tulane University, New Orleans, LA 70112, USA.ORCID 0000-0002-3381-2641
Paul Antwi BoasiakoCollege of Health Professions, SUNY Upstate Medical University, New York, NY 13210, USA.
Shehab Ahmed AlenaziDepartment of Pediatrics, Faculty of Medicine, Northern Border University, Arar 91431, Saudi Arabia.
Angelique DabelCollege of Health Professions, SUNY Upstate Medical University, New York, NY 13210, USA.
Jessan A JishuSchool of Medicine, Tulane University, New Orleans, LA 70112, USA.ORCID 0009-0008-3436-8889
Bandar T AleneziDepartment of Pharmacology, Faculty of Medicine, Northern Border University, Arar 91431, Saudi Arabia.ORCID 0009-0006-2758-5581
Hani AiashSUNY Upstate Medical University, New York, NY 13210, USA.ORCID 0000-0001-9191-7479
Manal S FawzyCenter for Health Research, Northern Border University, Arar 91431, Saudi Arabia.ORCID 0000-0003-1252-8403

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

BACKGROUND/

objectivesCystic fibrosis (CF) is a life-limiting genetic disorder affecting multiple organ systems. This study compared clinical outcomes, hospitalization rates, and survival between children and adolescents with CF who received CFTR modulator therapies (ivacaftor, lumacaftor, tezacaftor, and elexacaftor).

methodsA retrospective cohort study was conducted using data from the TriNetX global collaborative network. Patients with CF aged 2-12 years (children) and 13-18 years (adolescents) who received CFTR modulator therapies were included. The propensity score matching balanced baseline characteristics between the two age groups.

resultsAfter propensity score matching, 946 patients per group were analyzed. The incidence of respiratory failure (3.81% vs. 1.06%,

conclusionsCF patients who initiated CFTR modulator therapies during adolescence had a higher incidence of respiratory failure, respiratory infections, hospitalization rates, and healthcare resource utilization compared to those who started therapy in childhood, despite similar mortality rates. These findings highlight the importance of the early initiation of CFTR modulator therapies.

Indexed as

genetic inheritanceglobal research networkhealthcare burdenmulticentertargeted therapy

Identifiers

PMID40003259
PMCPMC11854606

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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.