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Review
. 2024 Mar 13;13(6):1650.
doi: 10.3390/jcm13061650.

Scoring Abdominal Symptoms in People with Cystic Fibrosis

Harold Tabori  1   2   3 , Anton Barucha  1   3   4 , Carlos Zagoya  1 , Franziska Duckstein  1 , Gabor A Dunay  3   5 , Pauline Sadrieh  1   3 , Louise Polte  1   3 , Jochen G Mainz  1   3
Affiliations
Review

Scoring Abdominal Symptoms in People with Cystic Fibrosis

Harold Tabori et al. J Clin Med. .

Abstract

(1) Background: The introduction of highly effective CFTR-modulating therapies (HEMT) has changed the course of the disease for many people with Cystic Fibrosis (pwCF). Attention previously focused on life-threatening conditions of the respiratory system has broadened, bringing the involvement of the digestive system into the clinical and scientific focus. This emphasized the need for sensitive tools to capture and quantify changes in abdominal symptoms (AS), ideally applying patient-reported outcome measures (PROMs). (2) Methods: The present review focuses on studies addressing AS assessment deriving from the multi-organic abdominal involvement in pwCF. Among 5224 publications retrieved until Nov. 2022, 88 were eligible, and 39 were finally included. (3) Results: The review reveals that for a long time, especially before HEMT availability, AS in pwCF were assessed by single questions on abdominal complaints or non-validated questionnaires. PROMs focusing on quality of life (QOL) including a few GI-related questions were applied. Likewise, PROMs developed and partially validated for other non-CF GI pathologies, such as chronic inflammatory bowel diseases, irritable bowel syndrome, gastroesophageal reflux, constipation, or pancreatitis, were implemented. (4) Conclusions: Only lately, CF-specific GI-PROMs have been developed and validated following FDA guidelines, showing high sensitivity to changes and capturing marked and statistically significant reductions in the burden of AS achieved with HEMT implementation.

Keywords: CFAbd-Score; CFTR; PAC-QOL; PAC-SYM; PAGI-SYM; PedsQL-GI; gastrointestinal; modulator; patient-reported outcome measure (PROM).

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Conflict of interest statement

The authors declare no conflict of interest.

Figures

Figure 3
Figure 3
Reviewed studies in the course of the respective year of publication. Red dotted line indicates approval of the first CFTR-modulating therapy (ivacaftor). Box colours indicate applicability in the sense of CF specifity and quality of validation [5,23,26,27,28,29,30,31,32,33,34,35,36,37,39,40,41,42,43,44,45,46,47,48,49,50,51,52,53,54,55,56,57,58,59,60,61,62,63].
Figure 1
Figure 1
Multifactorial aspects contributing to the burden of gastrointestinal symptoms in pwCF MI = meconium ileus; DIOS: Distal Intestinal Obstruction Syndrome; PERT: Pancreatic Enzyme Replacement Therapy; PI: pancreatic insufficiency, PS: pancreatic sufficiency; GERD: Gastroesophageal Reflux Disease; CF: cystic fibrosis; CFLD: CF-associated Liver Disease, CFRD: CF-Related Diabetes, CFTR: CF Transmembrane Conductance Regulator. Figure adapted from [5].
Figure 2
Figure 2
Flow chart regarding literature search and the two-stage review process, whereby a total of 39 suitable articles were selected for full-text review.
See this image and copyright information in PMC

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References

    1. Cystic Fibrosis Mutation Database. [(accessed on 16 February 2023)]. Available online: http://www.genet.sickkids.on.ca/app.
    1. CFTR2 CFTR2 Variant List History. [(accessed on 16 February 2023)]. Available online: https://cftr2.org/
    1. Bacalhau M., Camargo M., Magalhaes-Ghiotto G.A.V., Drumond S., Castelletti C.H.M., Lopes-Pacheco M. Elexacaftor-Tezacaftor-Ivacaftor: A Life-Changing Triple Combination of CFTR Modulator Drugs for Cystic Fibrosis. Pharmaceuticals. 2023;16:410. doi: 10.3390/ph16030410. - DOI - PMC - PubMed
    1. Freedman S.D., Wilschanski M. Introduction. J. Cyst. Fibros. 2017;16((Suppl. 2)):S1. doi: 10.1016/j.jcf.2017.07.013. - DOI - PubMed
    1. Tabori H., Arnold C., Jaudszus A., Mentzel H.J., Renz D.M., Reinsch S., Lorenz M., Michl R., Gerber A., Lehmann T., et al. Abdominal symptoms in cystic fibrosis and their relation to genotype, history, clinical and laboratory findings. PLoS ONE. 2017;12:e0174463. doi: 10.1371/journal.pone.0174463. - DOI - PMC - PubMed