Changes in Self-Care, Treatment Adherence, and Renal Function in Children with Cystic Fibrosis

Document Type : original article

Authors

1 Assistant Professor of Pediatric Nephrology, Department of Pediatrics, School of Medicine, Bouali Hospital, Ardabil University of Medical Sciences, Ardabil, Iran.

2 Assistant Professor of Pediatrics Gastroenterology, Department of Pediatrics, School of Medicine, Bouali Hospital, Ardabil University of Medical Sciences, Ardabil, Iran.

3 Department of Pediatrics, School of Medicine, Bouali Hospital, Ardabil University of Medical Sciences, Ardabil, Iran.

4 Assistant Professor of Pediatric Pulmonology, Department of Pediatrics, School of Medicine, Bouali Hospital, Ardabil University of Medical Sciences, Ardabil, Iran.

5 Professor of Health Education and Promotion, School of Medicine, Ardabil University of Medical Sciences, Ardabil, Iran.

10.22038/jpp.2025.92029.5606

Abstract

Background: Cystic fibrosis (CF) is a chronic and progressive disease that requires continuous care and management. Self-care and treatment adherence play crucial roles in the effective management of these patients, particularly in children.  The present study aimed to assess the status of self-care, treatment adherence, and renal function indicators in children with CF, as well as to analyze their changes over a six-month period.
Methods: In this longitudinal study, 30 children with CF were evaluated on an outpatient basis at two time points: baseline and six months later. Data on nutritional self-care and treatment adherence were collected using validated questionnaires. Laboratory indicators including serum creatinine, blood urea nitrogen (BUN), estimated glomerular filtration rate (eGFR), blood glucose, and urinary protein-to-creatinine ratio were measured. Parametric statistical tests and linear mixed-effects regression models were applied for data analysis.
Results: The mean age was 10 years, with a mean body mass index of 15.5 kg/m², and the majority were male. The mean self-care score significantly declined over the six-month period (p = 0.024), while treatment adherence showed no significant change (p = 0.787). Serum creatinine (p = 0.019) and blood glucose (p = 0.009) levels increased significantly, whereas eGFR decreased (p = 0.003). Time was the only variable that had a significant effect on eGFR and creatinine (p < 0.05); self-care and treatment adherence scores had no significant impact. A significant negative correlation was observed between self-care and the urinary protein-to-creatinine ratio (r = -0.508, p < 0.01).
Conclusion: The observed decline in self-care and renal function among children with CF over time highlights the need for structured, multidimensional, and long-term interventions aimed at improving self-care and treatment adherence. Regular monitoring of renal function is also recommended for these patients.

Keywords


  1. Dickinson KM, Collaco JM. Cystic Fibrosis. Pediatrics in review. 2021;42(2):55-67.
  2. Varkki SD, Aaron R, Chapla A, Danda S, Medhi P, Rani NJ, et al. CFTR mutations and phenotypic correlations in people with cystic fibrosis: a retrospective study from a single centre in south India. The Lancet Regional Health-Southeast Asia. 2024 Aug 1;27.
  3. Hart M, Kumar M, Goswami HB, Harris WT, Skopelja-Gardner S, Swiatecka-Urban A. Cystic fibrosis–related kidney disease—emerging morbidity and disease modifier. Pediatric Nephrology. 2025 Mar 17:1-0.
  4. Ni Q, Chen X, Zhang P, Yang L, Lu Y, Xiao F, et al. Systematic estimation of cystic fibrosis prevalence in Chinese and genetic spectrum comparison to Caucasians. Orphanet journal of rare diseases. 2022 Mar 21;17(1):129.
  5. Hammoudeh S, Aqel S, Mukthar F, Chandra P, Janahi IA. A systematic review of the epidemiology of cystic fibrosis in arab countries: an update. Clinical Epidemiology and Global Health. 2024 Jul 1;28:101697.
  6. Harvey C, Weldon S, Elborn S, Downey DG, Taggart C. The effect of CFTR modulators on airway infection in cystic fibrosis. International Journal of Molecular Sciences. 2022 Mar 23;23(7):3513.
  7. Lopes-Pacheco M. CFTR modulators: the changing face of cystic fibrosis in the era of precision medicine. Frontiers in pharmacology. 2020 Feb 21;10:1662.
  8. Hosseini Nami A, Kabiri M, Zafarghandi Motlagh F, Shirzadeh T, Fakhari N, Karimi A, et al. Genetic attributes of Iranian cystic fibrosis patients: the diagnostic efficiency of CFTR mutations in over a decade. Frontiers in Genetics. 2023 May 18;14:1140034.
  9. Bresnick K, Arteaga-Solis E, Millar SJ, Laird G, LeCamus C. Burden of cystic fibrosis in children< 12 years of age prior to the introduction of CFTR modulator therapies. BMJ open respiratory research. 2021 Dec 2;8(1).
  10. Shahin W, Badr A, Rabie W, Ahmed R, Mohsen M, El-Falaki M. Early renal involvement in children with cystic fibrosis.52(suppl 62):PA4627.2018.
  11. Karami H, Rafati S, Shiri MS, Mouseli A, Salari H, Ghanbarnejad A, et al. Societal Economic Burden of Cystic Fibrosis in Iran: A Cost-of-Illness Study. Journal of Health Economics and Outcomes Research. 2025 Sep 9;12(2):116.
  12. Bonfim BS, Melo Filho VM, Fontenelle FM, Souza EL. Treatment adherence among children and adolescents in a cystic fibrosis reference center. Revista Paulista de Pediatria. 2020 May 22;38:e2018338.
  13. Panahishokouh M, Kasaeian A, Jafarrangraz SS, Amini S. Assessment of Medication Adherence and Related Factors in Children and Adolescents with Cystic Fibrosis: A Pharmacist-Led Study. Journal of Reports in Pharmaceutical Sciences. 2025;13(13).
  14. Wildman MJ, O’Cathain A, Maguire C, Arden MA, Hutchings M, Bradley J, et al. Self-management intervention to reduce pulmonary exacerbations by supporting treatment adherence in adults with cystic fibrosis: a randomised controlled trial. Thorax. 2022 May 1;77(5):461-9.
  15. Moharamzad Y, Saadat H, Shahraki BN, Rai A, Saadat Z, Aerab-Sheibani H, et al. Validation of the Persian version of the 8-item Morisky Medication Adherence Scale (MMAS-8) in Iranian hypertensive patients. Global journal of health science. 2014 Dec 31;7(4):173.
  16. Goodfellow NA, Hawwa AF, Reid AJ, Horne R, Shields MD, McElnay JC. Adherence to treatment in children and adolescents with cystic fibrosis: a cross-sectional, multi-method study investigating the influence of beliefs about treatment and parental depressive symptoms. BMC pulmonary medicine. 2015 Apr 26;15(1):43.
  17. 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 preference and adherence. 2011 Oct 11:507-15.
  18. Rego TD, de Moraes JR, Cabral IE, de Souza TV. Self-care deficits reported by school children with cystic fibrosis. Journal of Pediatric Nursing. 2024 Jul 1;77:e335-42.
  19. Grossoehme DH, Szczesniak RD, Britton LL, Siracusa CM, Quittner AL, Chini BA, et al. Adherence determinants in cystic fibrosis: cluster analysis of parental psychosocial, religious, and/or spiritual factors. Annals of the American Thoracic Society. 2015 Jun;12(6):838-46.
  20. Shakkottai A, Kidwell KM, Townsend M, Nasr SZ. A five‐year retrospective analysis of adherence in cystic fibrosis. Pediatric Pulmonology. 2015 Dec;50(12):1224-9.
  21. Narayanan S, Mainz JG, Gala S, Tabori H, Grossoehme D. Adherence to therapies in cystic fibrosis: a targeted literature review. Expert review of respiratory medicine. 2017 Feb 1;11(2):129-45.
  22. Faint NR, Staton JM, Stick SM, Foster JM, Schultz A. Investigating self‐efficacy, disease knowledge and adherence to treatment in adolescents with cystic fibrosis. Journal of Paediatrics and Child Health. 2017 May;53(5):488-93.
  23. Bishay LC, Sawicki GS. Strategies to optimize treatment adherence in adolescent patients with cystic fibrosis. Adolescent health, medicine and therapeutics. 2016 Oct 21:117-24.
  24. Ng DK, Pierce CB. Kidney disease progression in children and young adults with pediatric CKD: epidemiologic perspectives and clinical applications. InSeminars in nephrology 2021 Sep 1 (Vol. 41, No. 5, pp. 405-415). WB Saunders.
  25. Prestidge C, Chilvers MA, Davidson AG, Cho E, McMahon V, White CT. Renal function in pediatric cystic fibrosis patients in the first decade of life. Pediatric Nephrology. 2011 Apr;26(4):605-12.
  26. Rosner GM, Goswami HB, Sessions K, Mendyka LK, Kerin B, Vlasac I, et al. Lung-kidney axis in cystic fibrosis: Early urinary markers of kidney injury correlate with neutrophil activation and worse lung function. Journal of Cystic Fibrosis. 2025 Jan 3.
  27. Sawicki GS, Heller KS, Demars N, Robinson WM. Motivating adherence among adolescents with cystic fibrosis: youth and parent perspectives. Pediatric pulmonology. 2015 Feb;50(2):127-36.