Diseases of the Esophagus (2017) 30, 1–9 DOI: 10.1093/dote/dox017 Original Article Development and pilot-testing of a condition-speciic instrument to assess the quality-of-life in children and adolescents born with esophageal atresia M. Dellenmark-Blom, 1, 2 K. Abrahamsson, 1 , 2 J. H. Quitmann, 3 R. Sommer, 3 S. Witt, 3 J. Dingemann, 4 S. Flieder, 4 L. J ¨ onsson, 2 V. Gatzinsky, 2 M. Bullinger, 3 B. M. Ure, 4 C. Dingemann, 4 J. E. Chaplin 1 1 Institute of Clinical Sciences, Department of Pediatrics, Queen Silvia Children’s Hospital, Gothenburg, Sweden, 2 Department of Pediatric Surgery, Queen Silvia Children’s Hospital, Gothenburg, Sweden, 3 Department of Med- ical Psychology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany, and 4 Center of Pediatric Surgery, Hannover Medical School and Bult Children’s Hospital, Hannover, Germany SUMMARY. The survival rate of children with esophageal atresia has today reached 95%. However, children are at risk of chronic morbidity related to esophageal and respiratory dysfunction, and associated anomalies. This study describes the pilot testing of a condition-speciic health-related quality-of-life instrument for children with esophageal atresia in Sweden and Germany, using a patient-derived development approach consistent with interna- tional guidelines. Following a literature review, standardized focus groups were conducted with 30 Swedish families of children with esophageal atresia aged 2–17 years. The results were used for item generation of two age-speciic pilot questionnaire versions. These were then translated from Swedish into German with considerations of linguistic and semantical perspectives. The 30-item pilot questionnaire for children aged 2–7 years was completed by 34 fam- ilies (parent report), and the 50-item pilot questionnaire for children aged 8–17 years was completed by 52 families (51 child report, 52 parent report), with an overall response rate of 96% in the total sample. Based on predeined psychometric criteria, poorly performing items were removed, resulting in an 18-item version with three domains (Eating, Physical health and treatment, Social isolation and stress,) for children aged 2–7 years and a 26-item ver- sion with four domains (Eating, Social relationships, Body perception, and Health and well-being) for children aged 8–17 years. Both versions demonstrated good internal consistency reliability and acceptable convergent and known- groups validity for the total scores. The study identiied speciic health-related quality-of-life domains for pediatric patients with esophageal atresia, highlighting issues that are important for follow-up care. After ield testing in a larger patient sample, this instrument can be used to enhance the evaluation of pediatric surgical care. KEY WORDS: children, esophageal atresia, patient-reported outcome, quality of life, rare condition. INTRODUCTION Esophageal atresia (EA), with or without a tracheoe- sophageal istula, is a rare congenital malformation that occurs in one in 2500–4500 live births. Survival rates now reach 95%; 1 however, following esophageal repair, long-term morbidity is common and children may suffer from dysphagia, gastroesophageal relux Address correspondence to: Michaela Dellenmark Blom, RN, MSc, Institute of Clinical Sciences, Department of Pediatrics, Gothenburg University and Department of Pediatric Surgery, 41685 Queen Silvia Children’s Hospital, Gothenburg, Sweden. Email: michaela.m.blom@vgregion.se Speciic author contributions: All authors made contributions to the design, and/or acquisition of data, and/or data analysis and interpretation. The irst and last author had the main responsibility for the data analysis and the irst author for drafting the article. All authors participated in revising it for important content and gave inal approval for submission. disease, 2 feeding dificulties, 3 and respiratory prob- lems. 4 Concomitant anomalies are present in 50% of the population. 1 Although the understanding of long- term medical outcomes in EA patients has advanced, knowledge of the child’s condition-speciic health- related quality of life (HRQOL) is sparse. Generic HRQOLpt instruments permit a comparison of out- comes to healthy references, while a condition-speciic questionnaire is more sensitive to the clinical char- acteristics 5 and enables an evaluation of healthcare needs and of the effect of disease and treatment from a patient perspective in a rare disease. 6 The study aim is to describe the results from a pilot test—including the initial validity and reliability—when using such a questionnaire for children with EA (the Esophageal Atresia Quality-Of-Life C  questionnaire, the EA- QOL C  questionnaire) in Sweden and Germany. C  The Authors 2017. Published by Oxford University Press on behalf of International Society for Diseases of the Esophagus. All rights reserved. For permissions, please e-mail: journals.permissions@oup.com 1 Downloaded from https://academic.oup.com/dote/article-abstract/30/7/1/3798661/Development-and-pilot-testing-of-a-condition by Bibliothekssystem Universitaet Hamburg user on 11 September 2017