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Poster - 46
Development of a patient-reported outcome measure for symptoms of respiratory disease in esophageal atresia: experiences reported in young children
M Dellenmark-Blom*, J Bennett**, R Micalizzi**, K Woods**, L Cardoni**, L Cole***, L Frain**, A Mohamed**, J Yasuda****, P Ngo****, A Widenmann*****, G Slater*****, B Zendejas**
*Department of Pediatric Surgery, Queen Silvia Children’s Hospital, Sahlgrenska University Hospital, Gothenburg, Sweden Institute of Clinical Sciences, Department of Pediatrics, University of Gothenburg, Gothenburg, Sweden
**Boston Children's Hospital, Department of Surgery, Esophageal and Airway Treatment Center, Boston, USA
***Department of Surgery, Boston Children's Hospital, Boston, MA, USA
****Division of Gastroenterology, Hepatology and Nutrition, Boston Children’s Hospital, Boston, MA, USA
*****EAT (Esophageal Atresia Global Support Groups), Sommerrainstr. 61, 70374 Stuttgart, Germany
Purpose: A patient-reported outcome measure (PROM) should reflect items reported as important to patients and parents in their own words. Currently, no such PROM for symptoms of respiratory disease in children with esophageal atresia (EA) is reported in the literature. This study aimed to identify parental perspectives on respiratory symptoms in young EA children to inform PROM development.
Method: Following PROM development standards, this project included:
● A literature review of existing PROMs.
● Analysis of 10 focus groups (FGs) with families of children with EA (n=30) in Sweden.
● Input from a multidisciplinary committee, including representatives from an international EA support group.
This formed the development of a standardized FG manual. To explore in-depth parental perspective, this was applied in five new FGs with 22 parents of EA children (aged 0–7) from a U.S. tertiary surgical center. Discussions were audio-recorded, transcribed and analyzed using predefined symptom definitions and content analysis. (Ethical approval P00048407)
Results: The five FGs generated 167 parent statements, consolidated into 86 unique expressions, categorized as:
● Breathing difficulties (n=22)
● Mucus problems (n=21)
● Prone to respiratory infections (n=20)
● Cough (n=14)
● Breathing sounds (n=6)
● Reduced energy (n=3)
These symptoms experiences occurred during:
● Respiratory infections (n=29)
● Feeding (n=22)
● Baseline conditions (n=18)
● Exertion (n=17)
● Sleep (n=13)
Symptoms were linked to parental distress (e.g., worry, fear, frustration) and child distress (e.g., struggle, crying, sleep disturbance).
Conclusion: The parent-reported symptoms expressions and distress highlight a significant burden and provide a strong foundation for PROM development in young EA children. Ongoing research will ensure this PROM is reliable for use across childhood.