Background: There are gaps in our understanding of the impact of conditions that affect sex development, such as DSD and CAH, on the parent and patient.
Aims: The project aimed to explore whether patient reported outcomes (PRO), assessed by standardised questionnaires, could be integrated within routine paediatric endocrine clinic visits.
Methods: Previous validated questionnaires were used to develop a parent Self-Report questionnaire containing eight domains (40 items) for caregivers of children <7 years. Domains were derived from a validated DSD-specific measure and a brief screening measure for adult depression and anxiety. A parallel parent Proxy-Report questionnaire containing eight domains (30 items) and assessing the childs adaptation was also developed. Each subscale was scored as the raw score and presented as a standard deviation score (SDS) relative to a DSD or healthy population depending on subscale. Parents of 31 children were approached and 24 (12 - conditions affecting sex development, 12 - other endocrine conditions) completed the forms. 27 parents completed the parent Self-Report questionnaire and 15 parents the Proxy-Report.
Results: Both questionnaires were found to be acceptable for routine use in clinic and took less than 10 minutes to complete. On the parent Self-Report scale, the median (range) SDS score for Future Concerns was −0.35 (−1.87, 1.04) and 1.04 (−2.00, 1.73) for mothers of children with conditions affecting sex development compared to other endocrine conditions, respectively (P=0.02). Talking to Others, Stigma and Surgery also had a lower score in parents of children with a condition affecting sex development but these were not significantly different from the other group. Although both groups of parents had mainly positive SDS on the Proxy-Report, parents of these children tended to score lower than those of children with other endocrine conditions on the majority of the subscales.
Conclusions: Parents of children with conditions affecting sex development reported greater levels of stress, particularly relating to future concerns. These questionnaires can be used as a routine health care evaluation and their use may allow greater targeting of support as well as the development of clinical benchmarks.
22 - 24 Nov 2017
British Society for Paediatric Endocrinology and Diabetes