Presentation (CTI)

OBJECTIVES: To assess health resource utilization (HRU) in adolescents and adults with phenylketonuria (PKU) compared with a matched non-PKU group from the general population.
METHODS: A retrospective study was conducted using de-identified HealthVerity claims data in the United States from 24 May 2018 to 31 March 2023. Adolescents aged 12-17 years and adults aged 18+ years with a record of PKU and with at least 12-months of continuous insurance enrolment (i.e., medical and pharmacy) were eligible for analysis. The analysis included 405 adolescents and 1721 adults with PKU, and a random sample of 1746 adolescents and 6536 adult non-PKU controls matched 1:5 based on birth year, gender, state, and payer type. Annualized average HRU for the study period was calculated and differences between groups were tested using 95% confidence intervals (CI) of mean differences (MD).
RESULTS: Annualized HRU for adolescents with PKU was significantly higher than for non-PKU controls: total visits (MD: 31.5 [CI: 24.9-38.1]), medical visits (MD: 26.4 [CI: 19.8-33.0]), outpatient visits (MD: 3.9 [CI: 2.3-5.5]), other visits (MD: 28.7 [CI: 20.1-37.3]), and pharmacy visits (MD: 7.8 [CI: 6.5- 9.1]). Similarly, annualized average HRU for adults with PKU was significantly higher than for non-PKU controls: total visits (MD: 33.7 [CI: 29.8-37.6]), medical visits (MD: 28.3 [CI: 24.4-32.2]), outpatient visits (MD: 9.1 [CI: 7.2-11.0]), other visits (MD: 24.5 [CI: 19.4-29.6]) and pharmacy visits (MD: 8.5 [CI:7.5-9.5]). Although the number of inpatient visits was not different, the length of stay (days) for those hospitalized was significantly longer for individuals with PKU compared with non-PKU controls (MD: 37.8 [CI: 28.0-47.6]).
CONCLUSIONS: HRU among both adolescents and adults with PKU was higher compared with matched non-PKU controls, highlighting the significant health economic burden of PKU on the healthcare system.