Adherence to inhalatory treatment among children and adolescents with cystic fibrosis: a five-year follow-up study

Authors

DOI:

https://doi.org/10.11606/issn.2176-7262.rmrp.2025.215831

Keywords:

Cystic fibrosis, Adherence, Inhalation therapy, Lung function, Pulmonary exacerbation

Abstract

Introduction: Low adherence to drug treatment has negative effects on the quality of life and health of patients with cystic fibrosis (CF). Previous studies have reported a correlation between poor adherence to treatment and a decline in lung function, pulmonary exacerbations, and hospitalizations. Objective: To evaluate the long-term adherence to inhaled mucolytic treatment among patients with CF treated at João Paulo II Children's Hospital by collecting empty ampoules over a five-year period of follow-up and examining the relationship between the treatment adherence rate and clinical variables. Method: This retrospective analytical longitudinal study of adherence to drug treatment over a five-year period collected data regarding lung function (% of predicted FEV1), clinical signs of exacerbation, body mass index (BMI) Z-score, and dornase alfa medication possession rate (MPR) from the medical records. The MPR was calculated by dividing the number of ampoules returned by the number of prescribed ampoules of alpha dornase. Results: The MPR of dornase alfa indicated an increase in the adherence rate of the 26 patients assessed in this study. The MPR of dornase alfa exhibited no associations with signs of pulmonary exacerbation, use of oral antibiotics, or the BMI/Age Z-Score over the five-year period. Notably, the MPR regression model revealed that a 1% increase in FEV1 required a 0.5% increase in the MPR of dornase alfa. Thus, an improvement in FEV1 is directly related to increased medication use. Conclusion: Requesting the return of empty ampoules of dornase alfa improved adherence rates and lung function over time.

Downloads

Download data is not yet available.

Author Biographies

  • Evanirso da Silva Aquino , Hospital Infantil João Paulo II. Rede Fundação Hospitalar do Estado de Minas Gerais

    Fisioterapeuta 

  • Ana Carolina Pena, Hospital Infantil João Paulo II. Rede Fundação Hospitalar do Estado de Minas Gerais

    Residente Multiprofissional

  • Letícia Santos Ferreira do Nascimento, Hospital Infantil João Paulo II. Rede Fundação Hospitalar do Estado de Minas Gerais

    Residente Multiprofissional

     

  • Alberto Andrade Vergara, Hospital Infantil João Paulo II. Rede Fundação Hospitalar do Estado de Minas Gerais

    Coordenador do Ambulatório de Fibrose Cística

  • Diego Campos de Moura, Pontifícia Universidade Católica de Minas Gerais. Instituto de Ciências Biológicas e da Saúde

     Graduando em Fisioterapeuta

  • Cristiane Cenachi Coelho, Hospital Infantil João Paulo II. Rede Fundação Hospitalar do Estado de Minas Gerais

    Fisioterapeuta e Coordenadora do Programa de Residência Multiprofissional

References

O'Reilly R, Elphick HE. Development, clinical utility, and place of ivacaftor in the treatment of cystic fibrosis. Drug Des Devel Ther. 2013;7:929-37.

Dalcin PA, Rampn G, Pasin LR, Ramon GM, Abrahão CLO, Oliveira VZO. Adesão ao tratamento em pacientes com fibrose cística. J Bras Pneumol. 2007;(6):33.

Bregnballe V, Schiøtz PO, Boisen KA, Pressler T, Thastum M. Barriers to adherence in adolescents and young adults with cystic fibrosis: a questionnaire study in young patients and their parents. Patient Prefer Adherence. 2011;5:507-15.

Awad S, Williams DK, Berlinski A. Longitudinal evaluation of compressor/nebulizer performance. Respir Care [Internet]. 2014;59(7):1053-61.

Quittner AL, Zhang J, Marynchenko M, Chopra PA, Signorovitch J, Yushkina Y, Riekert KA. Pulmonary medication adherence and health-care use in cystic fibrosis. Chest. 2014;146(1):142-151.

Lask B. Non-adherence to treatment in cystic fibrosis. J R Soc Med. 1994;87 Suppl 21(Suppl 21):25-7

Dodd ME, Webb AK. Understanding non-compliance with treatment in adults with cystic fibrosis. J R Soc Med. 2000;93 Suppl 38(Suppl 38):2-8.

Conway, S. P., Pond, M. N., Hamnett, T., & Watson, A. (1996). Compliance with treatment in adult patients with cystic fibrosis. Thorax. 1996;51(1), 29–33.

Eakin MN, Bilderback A, Boyle MP, Mogayzel PJ, Riekert KA. Longitudinal association between medication adherence and lung health in people with cystic fibrosis. J Cyst Fibros. 2011;10(4):258-64.

Ribeiro JD, Ribeiro MÂG de O, Ribeiro AF. Controvérsias na fibrose cística: do pediatra ao especialista. J Pediatr (Rio J) [Internet]. 2002;78:171-86.

Azevedo MFM, Guimarães JA, Pessoa VLMP. Adesão ao tratamento medicamentoso em adolescentes com fibrose cística. Boletim Informativo Geum, 2017;8(3):1-7.

Obreli Neto PR, Baldoni AO, Guidoni CM, Bergamini D, Hernandes KC, Luz RT, Silva FB, Silva RO, Pereira LRL, Cuman RKN. Métodos de avaliação de adesão a farmacoterapia. Rev Bras Farm. 2012;93(4):403-410.

Obreli-Neto PR, Prado MF, Vieira JC, Fachini FC, Pelloso SM, Marcon SS, et al. Fatores interferentes na taxa de adesão à farmacoterapia em idosos atendidos na rede pública de saúde do Município de Salto Grande – SP, Brasil. Rev Ciênc Farm Básica Apl. 2010;31(5790):229-33.

Castro MCS, Firmida MC. O tratamento na fibrose cística e suas complicações. Revista do Hospital Universitário Pedro Ernesto. 2011;10(4):82-108.

Fuchs H, Borowitz D, Christiansen D, Morris E, Nash M, Ramsey B, et al. Effect of aerosolized recombinant human DNase on exacerbations of respiratory symptoms and on pulmonary function in patients with cystic fibrosis. N Engl J Med. 1994;331(10):637-42.

Liang KY & Zeger SL. Longitudinal data analysis using generalized linear models. Biometrika. 1986;73(1):13-22.

Newsome SJ, Daniel RM, Carr SB, Bilton D, Keogh RH. Investigating the effects of long-term dornase alfa use on lung function using registry data. J Cyst Fibros. 2019;18(1):110-117

White H, Shaw N, Denman S, Pollard K, Wynne S, Peckham DG. Variation in lung function as a marker of adherence to oral and inhaled medication in cystic fibrosis. Eur Respir J. 2017;49(3):1600987.

Shakkottai A, Kidwell KM, Nasr SZ. A Five-Year Retrospective Analysis of Adherence in Cystic Fibrosis. Pediatr Pulmonol. 2015;50(12):1224-9.

McKone EF, Jackson AD, Fletcher G, Kirwan L. Dornase alfa and rate of lung function decline in European patients with cystic fibrosis: A retrospective registry cohort study. J Cyst Fibros. 2021;20(3):552-554.

Dentice R, Elkins M. Timing of dornase alfa inhalation for cystic fibrosis. Cochrane Database Syst Rev. 2018: 2;11:

Reynolds D, Kollef M. The Epidemiology and Pathogenesis and Treatment of Pseudomonas aeruginosa Infections: An Update. Drugs. 2021;81(18):2117-2131.

Bergeron C, Cantin AM. New Therapies to Correct the Cystic Fibrosis Basic Defect. Int J Mol Sci. 2021;22(12):6193.

Bower JK, Volkova N, Ahluwalia N, Sahota G, Xuan F, Chin A, Weinstock TG, Ostrenga J, Elbert A. Real-world safety and effectiveness of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis: Interim results of a long-term registry-based study. J Cyst Fibros. 2023;22(4):730-737.

Published

2025-10-01

Issue

Section

Original Articles

How to Cite

1.
Aquino E da S, Pena AC, Nascimento LSF do, Vergara AA, Moura DC de, Coelho CC. Adherence to inhalatory treatment among children and adolescents with cystic fibrosis: a five-year follow-up study. Medicina (Ribeirão Preto) [Internet]. 2025 Oct. 1 [cited 2026 Jan. 10];58(1):e-215831. Available from: https://revistas.usp.br/rmrp/article/view/215831

Funding data