Assessing gastro-intestinal related quality of life in cystic fibrosis: Validation of PedsQL GI in children and their parents


Autoři: Mieke Boon aff001;  Ine Claes aff001;  Trudy Havermans aff001;  Victoria Fornés-Ferrer aff002;  Joaquim Calvo-Lerma aff002;  Inês Asseiceira aff003;  Anna Bulfamante aff004;  María Garriga aff005;  Etna Masip aff002;  Sandra Woodcock aff006;  Sylvia Walet aff006;  Celeste Barreto aff003;  Carla Colombo aff004;  Paula Crespo aff002;  Els Van der Wiel aff006;  Jessie Hulst aff006;  Sandra Martinez-Barona aff002;  Rita Nobili aff004;  Luisa Pereira aff003;  Mar Ruperto aff005;  Saioa Vicente aff005;  Kris De Boeck aff001;  Carmen Ribes-Koninckx aff002
Působiště autorů: Cystic Fibrosis Center, Department of Pediatrics, University Hospital Gasthuisberg, Leuven, Belgium aff001;  Instituto de Investigación Sanitaria La Fe de Valencia, Spain aff002;  Associação para a Investigação e Desenvolvimento da Faculdade de Medicina, Lisbon, Portugal aff003;  Università degli Studi di Milano, Fondazione IRCCS Ca’ Granda, Ospedale Maggiore Policlinico, Milan, Italy aff004;  Hospital Universitario Ramón y Cajal, Madrid, Spain aff005;  Erasmus Medical Center, Sophia Children’s Hospital, Rotterdam, Netherlands aff006
Vyšlo v časopise: PLoS ONE 14(12)
Kategorie: Research Article
doi: 10.1371/journal.pone.0225004

Souhrn

Background

Most patients with cystic fibrosis (CF) suffer from pancreatic insufficiency, leading to fat malabsorption, malnutrition and abdominal discomfort. Until recently, no specific tool was available for assessing gastro-intestinal related quality of life (GI QOL) in patients with CF. As the Horizon2020 project MyCyFAPP aims to improve GI QOL by using a newly designed mobile application, a sensitive and reliable outcome measure was needed. We aimed to study the applicability of the existing child-specific Pediatric Quality of Life Inventory, Gastrointestinal Symptoms Scales and Module (PedsQL GI) in children with CF.

Methods

A multicenter, prospective observational study was performed in 6 European centers to validate the PedsQL GI in children with CF during 3 months.

Results

In total, 248 children and their parents were included. Within-patient variability of PedsQL GI was low (24.11), and there was reasonable agreement between children and parents (ICC 0.681). Nine of 14 subscales were informative (no ceiling effect). The PedsQL GI and the median scores for 4 subscales were significantly lower in patients compared to healthy controls. Positive associations were found between PedsQL GI and age (OR = 1.044, p = 0.004) and between PedsQL GI and BMI z-score (OR = 1.127, p = 0.036). PedsQL GI correlated with most CFQ-R subscales (r 0.268 to 0.623) and with a Visual Analogue Scale (r = 0.20).

Conclusions

PedsQL GI is a valid and applicable instrument to assess GI QOL in children with CF. Future research efforts should examine the responsiveness of the CF PedsQL GI to change in the context of clinical interventions and trials.

Klíčová slova:

Age groups – Constipation – Cystic fibrosis – Diarrhea – Pediatrics – Quality of life – Stomach – PERT


Zdroje

1. Orenti A, Zolin A, Naehrlich L, van Rens J et al. ECFSPR Annual Report 2016. https://www.ecfs.eu/sites/default/files/general-content-images/working-groups/ecfs-patient-registry/ECFSPR_Report2016_06062018.pdf

2. Smyth AR, Bell SC, Bojcin S, Bryon M, Duff A, Flume P et al. European Cystic Fibrosis Society Standards of Care: Best Practice guidelines. J Cyst Fibros 2014;13 Suppl 1:S23–42. doi: 10.1016/j.jcf.2014.03.010 24856775

3. Fieker A, Philpott J, Armand M. Enzyme replacement therapy for pancreatic insufficiency: present and future. Clin Exp Gastroenterol 2011;4:55–73. doi: 10.2147/CEG.S17634 21753892

4. Kerem E, Viviani L, Zolin A, MacNeill S, Hatziagorou E, Ellemunter H et al. Factors associated with FEV1 decline in cystic fibrosis: analysis of the ECFS patient registry. Eur Respir J 2014;43:125–33. doi: 10.1183/09031936.00166412 23598952

5. Stephenson AL, Mannik LA, Walsh S, Brotherwood M, Robert R, Darling PB et al. Longitudinal trends in nutritional status and the relation between lung function and BMI in cystic fibrosis: a population-based cohort study. Am J Clin Nutr. 2013;97:872–7. doi: 10.3945/ajcn.112.051409 23388659

6. Calvo-Lerma J, Martinez-Barona S, Masip E, Fornés V, Ribes-Koninckx C Pancreatic enzyme replacement therapy in cystic fibrosis: dose, variability and coefficient of fat absorption. Rev Esp Enferm Dig 2017;109:684–689. doi: 10.17235/reed.2017.4951/2017 28747058

7. Turck D, Braegger CP, Colombo C, Declercq D, Morton A, Pancheva R et al. ESPEN-ESPGHAN-ECFS guidelines on nutrition care for infants, children, and adults with cystic fibrosis. Clin Nutr. 2016;35:557–77. doi: 10.1016/j.clnu.2016.03.004 27068495

8. Tabori H, Arnold C, Jaudszus A, Mentzel HJ, Renz DM, Reinsch S 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 eCollection 2017. 28472055

9. Singh VK, Schwarzenberg SJ, Pancreatic insufficiency in Cystic Fibrosis J Cyst Fibros. 2017;16 Suppl 2:S70–S78. doi: 10.1016/j.jcf.2017.06.011 28986019

10. Calvo-Lerma J, Martinez-Jimenez CP, Lázaro-Ramos JP, Andrés A, Crespo-Escobar P, Stav E et al. Innovative approach for self-management and social welfare of children with cystic fibrosis in Europe: development, validation and implementation of an mHealth tool (MyCyFAPP). BMJ Open 2017;7: e014931. doi: 10.1136/bmjopen-2016-014931 28302638

11. Calvo-Lerma J, Fornés-Ferrer V, Peinado I, Heredia A, Ribes-Koninckx C, Andrés A.https://www.ncbi.nlm.nih.gov/pubmed/30794618 PLoS One. 2019;14(2):e0212459. doi: 10.1371/journal.pone.0212459 eCollection 2019. 30794618

12. Calvo-Lerma J, Hulst J, Boon M, Colombo C, Masip E, Ruperto M et al. Clinical validation of an evidence-based method to adjust Pancreatic Enzyme Replacement Therapy through a prospective interventional study in paediatric patients with Cystic Fibrosis. PLoS One. 2019;14(3):e0213216. doi: 10.1371/journal.pone.0213216 eCollection 2019. 30861039

13. Royce FH, Carl JC. Health-related quality of life in cystic fibrosis. Curr Opin Pediatr. 2011;23:535–40. doi: 10.1097/MOP.0b013e32834a7829 21900781

14. Abbott J, Hart A, Havermans T, Matossian A, Goldbeck L, Barreto C et al. Measuring health-related quality of life in clinical trials in cystic fibrosis. J Cyst Fibros. 2011;10 Suppl 2:S82–5. doi: 10.1016/S1569-1993(11)60013-1

15. Quittner AL, Buu A, Messer MA, Modi AC, Watrous M. Development and validation of The Cystic Fibrosis Questionnaire in the United States: a health-related quality-of-life measure for cystic fibrosis. Chest. 2005;128:2347–54. doi: 10.1378/chest.128.4.2347 16236893

16. Quittner AL, Sawicki GS, McMullen A, Rasouliyan L, Pasta DJ, Yegin A et al. Psychometric evaluation of the Cystic Fibrosis Questionnaire-Revised in a national sample. Qual Life Res. 2012;21:1267–78. doi: 10.1007/s11136-011-0036-z 21993695

17. Alpern AN, Brumback LC, Ratjen F, Rosenfeld M, Davis SD, Quittner AL. Initial evaluation of the Parent Cystic Fibrosis Questionnaire—Revised (CFQ-R) in infants and young children. J Cyst Fibros. 2015;14: 403–11. doi: 10.1016/j.jcf.2014.11.002 25443473

18. Retsch-Bogaert GZ, Quittner AL, Gibson RL, Oermann CM, McCoy KS, Montgomery AB et al. Efficacy and safety of inhaled aztreonam lysine for airway pseudomonas in cystic fibrosis. Chest. 2009;135:1223–1232. doi: 10.1378/chest.08-1421 19420195

19. Wainwright CE, Quittner AL, Geller DE, Nakamura C, Wooldridge JL, Gibson RL et al. Aztreonam for inhalation solution (AZLI) in patients with cystic fibrosis, mild lung impairment, and P. aeruginosa. J Cyst Fibros. 2011;10:234–42. doi: 10.1016/j.jcf.2011.02.007 21441078

20. Ronit A, Gelpi M, Argentiero J, Mathiesen I, Nielsen SD, Pressler T et al. Electronic applications for the CFQ-R scoring. Respir Res. 2017;30:18:108. doi: 10.1186/s12931-017-0592-z 28558706

21. Varni JW, Kay MT, Limbers CA, Franciosi JP, Pohl JF. PedsQL gastrointestinal symptoms module item development: qualitative methods. J Pediatr Gastroenterol Nutr. 2012;54:664–71. doi: 10.1097/MPG.0b013e31823c9b88 22008958

22. Varni JW, Bendo CB, Denham J, Shulman RJ, Self MM, Neigut DA et al. PedsQL Gastrointestinal Symptoms Scales and Gastrointestinal Worry Scales in pediatric patients with functional and organic gastrointestinal diseases in comparison to healthy controls. Qual Life Res. 2015;24:363–78. doi: 10.1007/s11136-014-0781-x 25148757

23. Varni JW, Franciosi JP, Shulman RJ, Saeed S, Nurko S, Neigut DA et al. PedsQL gastrointestinal symptoms scales and gastrointestinal worry scales in pediatric patients with inflammatory bowel disease in comparison with healthy controls. Inflamm Bowel Dis. 2015;21:1115–24. doi: 10.1097/MIB.0000000000000351 25793327

24. Varni JW, Bendo CB, Denham J, Shulman RJ, Self MM, Neigut DA et al. PedsQL gastrointestinal symptoms module: feasibility, reliability, and validity. J Pediatr Gastroenterol Nutr. 2014;59:347–55. doi: 10.1097/MPG.0000000000000414 24806837

25. Quanjer PH, Stanojevic S, Cole TJ, Baur X, Hall GL, Culver BH et al. Multi-ethnic reference values for spirometry for the 3–95 year age range: the global lung function 2012 equations. Eur Respir J 2012;40:1324–43. doi: 10.1183/09031936.00080312 22743675

26. Kuczmarski RJ, Ogden CL, Guo SS, Grummer-Strawn LM, Flegal KM, Mei Z et al. 2000 CDC Growth Charts for the United States: methods and development. Vital Health Stat 11. 2002;(246):1–190. 12043359

27. Kline P. (2000). The handbook of psychological testing (2nd ed.). London: Routledge, p 13

28. Koo TK, Li MY. A Guideline of Selecting and Reporting Intraclass Correlation Coefficients for Reliability Research. J Chiropr Med. 2016;15:155–163. doi: 10.1016/j.jcm.2016.02.012 27330520

29. Rowbotham NJ, Smith S, Leighton PA, Rayner OC, Gathercole K, Elliott ZC et al. The top 10 research priorities in cystic fibrosis developed by a partnership between people with CF and healthcare providers. Thorax. 2018;73:388–390. doi: 10.1136/thoraxjnl-2017-210473 28778919

30. Tabori H, Jaudszus A, Arnold C, Mentzel HJ, Lorenz M, Michl RK et al. Relation of Ultrasound Findings and Abdominal Symptoms obtained with the CFAbd-Score in Cystic Fibrosis Patients. Sci Rep. 2017;7:17465. doi: 10.1038/s41598-017-17302-4 29234058

31. Jaudszus A, Zeman E, Jans T, Pfeifer E, Tabori H, Arnold C et al. Validity and Reliability of a Novel Multimodal Questionnaire for the Assessment of Abdominal Symptoms in People with Cystic Fibrosis (CFAbd-Score). Patient. 2019 doi: 10.1007/s40271-019-00361-2 30887269

32. Varni JW, Bendo CB, Shulman RJ, Self MM, Nurko S, Franciosi JP et al. Interpretability of the PedsQL Gastrointestinal Symptoms Scales and Gastrointestinal Worry Scales in Pediatric Patients with functional and organic gastrointestinal disease. J Pediatr Psychol. 2015;40:591–601. doi: 10.1093/jpepsy/jsv005 25682210

33. Varni JW, Shulman RJ, Self MM, Nurko S, Saps M, Saeed SA et al. Symptom profiles in patients with irritable bowel syndrome or functional abdominal pain compared with healthy controls. J Pediatr Gastroenterol Nutr. 2015;61:323–9. doi: 10.1097/MPG.0000000000000795 26020482

34. Calvo-Lerma J, Hulst JM, Asseiceira I, Claes I, Garriga M, Colombo C et al. Nutritional status, nutrient intake and use of enzyme supplements in paediatric patients with Cystic Fibrosis; a European multicentre study with reference to current guidelines. J Cyst Fibr 2017;16:510–518. doi: 10.1016/j.jcf.2017.03.005 28320633


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PLOS One


2019 Číslo 12