Evidence map›Paper›PMID 36284811›Full record

ReviewCureus2022

Newly Discovered Cutting-Edge Triple Combination Cystic Fibrosis Therapy: A Systematic Review.

Sarah N Dawood, Ahmad M Rabih, Ahmad Niaj, Aishwarya Raman, Manish Uprety, Maria Jose Calero, Maria Resah B Villanueva, Narges Joshaghani, Nicole Villa, Omar Badla and 3 more

Open access · diamondAbstract readReview
In one paragraph

Review in Cureus, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
14citing papers in PubMed, 1 pooled it
3.5field-weighted citation impact, top 6% 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

14 citing papers in PubMed, 1 synthesis or guideline pooled it, 24 citations in OpenAlex.

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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 at 1 institution in 1 country.

Sarah N DawoodPediatrics, California Institute of Behavioral Neurosciences & Psychology, Fairfield, USA.
Ahmad M RabihInternal Medicine, California Institute of Behavioral Neurosciences & Psychology, Fairfield, USA.
Ahmad NiajInternal Medicine, California Institute of Behavioral Neurosciences & Psychology, Fairfield, USA.
Aishwarya RamanInternal Medicine, California Institute of Behavioral Neurosciences & Psychology, Fairfield, USA.
Manish UpretyInternal Medicine, California Institute of Behavioral Neurosciences & Psychology, Fairfield, USA.
Maria Jose CaleroObstetrics and Gynecology, California Institute of Behavioral Neurosciences & Psychology, Fairfield, USA.
Maria Resah B VillanuevaResearch, California Institute of Behavioral Neurosciences & Psychology, Fairfield, USA.
Narges JoshaghaniPsychiatry and Behavioral Sciences, California Institute of Behavioral Neurosciences & Psychology, Fairfield, USA.
Nicole VillaInternal Medicine, California Institute of Behavioral Neurosciences & Psychology, Fairfield, USA.
Omar BadlaGeneral Surgery, California Institute of Behavioral Neurosciences & Psychology, Fairfield, USA.
Raman GoitInternal Medicine, California Institute of Behavioral Neurosciences & Psychology, Fairfield, USA.
Samia E SaddikInternal Medicine, California Institute of Behavioral Neurosciences & Psychology, Fairfield, USA.
Lubna MohammedInternal Medicine, California Institute of Behavioral Neurosciences & Psychology, Fairfield, USA.
California Institute of Behavioral Neurosciences and Psychology (United States) · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

A cystic fibrosis (CF) transmembrane conductor regulator (CFTR) gene modulating triple therapy combining elexacaftor-tezacaftor-ivacaftor (Trikafta) has been recently discovered. Its approval by the Food and Drug Administration (FDA) in 2019 has expanded the target therapy group to individuals aged twelve and up with at least one Phe508del (phenylalanine 508 deletion) mutation in the CFTR gene. This systematic review aims to assess this combination therapy's safety and efficacy. Following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) 2020 guidelines, an in-depth search was performed. The search was done by utilizing databases such as PubMed Central (PMC), Google Scholar, and Science Direct for articles related to this topic. Studies published in the last five years in the English language were chosen preliminarily. Further eligibility criteria and quality assessment tools were employed to assess the risk of bias and finalize ten articles to be used in this review. The chosen articles constituted four randomized control trials (RCTs), four systematic reviews, and two narrative reviews. The last date for data collection was April 24, 2022. Based on the findings of this review, we concluded that by combining three CFTR modulators, this therapy had outperformed all the currently available medications in terms of improving pulmonary function, reducing exacerbations, and enhancing the quality of life of CF patients. In clinical trials, headache and rash were the most common side effects, and laboratory testing to assess liver function is suggested. Long-term safety and effectiveness must be confirmed by the continued review of real-life patient data. Studies done on triple therapy thus far have been promising. Unfortunately, a small proportion of the CF population remains ineligible for any form of CFTR modulator therapy owing to their type of genetic mutation, and this provides ground for further research in this field.

Indexed as

cftr gene mutationcftr modulating therapycftr modulatorcystic fibrosis (cf)cystic fibrosis triple therapyelexacaftorivacaftorphe508 mutationtezacaftortrikafta

Identifiers

PMID36284811
PMCPMC9583755
OpenAlexW4297903490

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.