BackgroundEvidence supporting personalised treatment for asthma based on an individual’s genetics is mounting. The views of children and young people (CYP), parents and healthcare professionals (HCPs) about this evolution of clinical care are not known.MethodsA pilot prospective questionnaire-based study was undertaken of CYP with asthma, their parents and HCPs at a secondary/tertiary children’s hospital in the UK.ResultsFifty-nine questionnaires were distributed and 50 returned (response rate 84.7%), comprising 26 CYP (10 were 5–11 years, 11 were 12–15 years and 5 were 16–18 years old), 13 parents and 11 HCPs. For all types of data, personal information was ranked as the ‘most important’ (n=19, 47.5%) and ‘most private’ (n=16, 40%), but with considerable variation across groups. Within health data, allergies were rated as ‘most important’ (n=12, 30.8%), and mental health records the ‘most private’ (n=21, 53.8%), again with variation across groups. A ‘personalised genetic asthma plan’ was acceptable to the majority overall (n=40, 80.0%). With regard to sharing CYP’s genetic data, 23 (46%) of participants were happy for unconditional sharing between HCPs, and 23 (46%) agreed to sharing solely in relation to the CYP’s asthma management. Forty-two (84.0%) of participants felt CYP should be informed about genetic data being shared, and the majority felt this should commence by 12 years of age.ConclusionThe use of genetic information to guide management of asthma in CYP is largely acceptable to CYP, parents/guardians and HCPs. However, there are key differences between the opinions of CYP, parents and HCPs.

AimHydrocortisone is used in Congenital Adrenal Hyperplasia (CAH) as a long-term replacement therapy. Accurate dosing, patient acceptability, and ease of administration of the available dosage forms are vital as treatment is life-long.1Prior to the introduction of a licensed immediate-release formulation the use of a variety of unlicensed oral hydrocortisone preparations was widespread. This project aimed to explore patient/parent acceptability of oral hydrocortisone preparations in the real-world setting. This included assessment of preferences and the reasons for discontinuation if more than one formulation had been used.MethodThis clinical audit was registered within the Trust. Two e-surveys were developed by a multi-disciplinary team (MDT) using Microsoft Forms, one for parents of children <8 years and the other for parents and children ≥8 years. With permission, both e-surveys included the validated Pediatric Oral Medicines Acceptability Questionnaire for caregivers (POMAQ-C) and the POMAQ-P was optional for patients ≥8 years old.2Most questions utilised the Likert rating scale, with 5 being positive and 1 being negative. The form was piloted with one family, then parents were contacted by the clinical team and if happy to take part, a member of the project team contacted them with the survey link details. Inclusion criteria: Patients with CAH aged 6 months to 17 years (inclusive) taking an oral form of hydrocortisone. Exclusion criteria: non-UK residents, non-English speaking, non-classical CAH patients, and/or not taking an oral form of hydrocortisone.Results33 eligible patients were identified. The results below represent the findings from the first 8 families. Patients were aged between 1 to 17 (mean 7.7) years. Three (37.5%) were taking hydrocortisone tablets, one (12.5%) was taking Alkindi® granules, and four (50%) hydrocortisone liquid. The mean score for parent-rated overall acceptability of tablets, granules, and liquid preparations was 4.3, 4, and 4.75, respectively. Parent-rated mean acceptability score for their child was 4.67, 3, and 5, respectively for the different types of formulations. One patient had moved from liquid hydrocortisone to tablets due to problems obtaining prescriptions and transporting/refrigerating the product when not at home. Another had moved from granules to liquid as the parent found it ‘difficult to give to a baby’ and reported issues obtaining a prescription.ConclusionAssessment of the acceptability of medicines for children in a real-world setting is possible and allows for parents/carers and patients to provide practical feedback on available treatment options. To date, parental feedback received indicates a slight preference for liquids over tablet and granule hydrocortisone formulations, although data is currently limited.ReferencesBoulos N. Formulation matters: safe oral hydrocortisone use in children.The Pharmaceutical JournalDecember 2021;No 7956;307(7956).Turner-Bowker DM, An Haack K, Krohe M,et al. Development and content validation of the pediatric oral medicines acceptability questionnaires (P-OMAQ): patient-reported and caregiver-reported outcome measures.Journal of Patient-Reported Outcomes2020;4(1).