Challenging symptom profiles of life-limiting conditions in children: A survey of care professionals and families

Abstract

This paper reports on data from the initial phase of a UK-wide study exploring life-limiting conditions (LLCs) in childrenwhere, because of their rarity, little is understood regarding the significant symptom challenges of families and careprofessionals who support them. In this initial phase, care professionals (n¼43) and families (n¼16) completed a surveyto identify which rare LLCs present symptom challenges and which individual symptoms prove difficult to manage.Findings led to the prioritization of mucopolysaccharidoses, Batten Disease and leukodystrophy. Care professionals'rationales for selecting these conditions included the presence of symptoms that are very difficult to manage, a requirementfor additional clinical evidence to support symptom management, and the progressive nature of these diseaseswhere symptoms frequently change and are difficult to predict. Families identified symptoms such as behavioural difficulties,visual impairments and communication impairments as those being most difficult to manage and having mostimpact on quality of life for children and families.