摘要：Abstract Introduction Management of phenylketonuria (PKU) is mainly achieved through strict dietary control that aims to limit the intake of phenylalanine (Phe). Adherence to this diet is burdensome due to the need for specially prepared low-Phe meals and regular monitoring of Phe concentrations. A {UK} cross-sectional study was conducted to identify the personal time and monetary burden associated with aspects of the {PKU} lifestyle for caregivers of children (aged < 18 years) living with PKU. Methods Caregivers of pediatric patients with {PKU} attending one of four specialist metabolic centers in the {UK} were invited to participate in a questionnaire-based survey that evaluated different aspects of {PKU} management that could potentially present out-of-pocket costs (OOPCs) or time burden. Medical clinicians/dieticians provided patient information on {PKU} severity and an assessment of blood Phe control. Results The survey was completed by 114 caregivers of 106 children having mild or moderate (n = 45; 39%) or classical (n = 60; 53%) {PKU} (severity data missing for n = 1), among whom 8 (8%) and 87 (82%) reported poorly controlled and controlled blood Phe status, respectively; Phe control data were missing for 11 children. Dietary management of {PKU} incurred a median time burden of > 19 h per week. {OOPCs} were incurred via attendance at {PKU} events, PKU-related equipment, and extra holiday expenditure. 21% of caregivers reduced their working hours (median 18.5 h/week) to care for their child, with a further 24% leaving their paid jobs completely. Discussion and conclusions Dietary management of {PKU} is associated with a considerable time burden for caregivers of pediatric patients with PKU. A personal financial burden also arises from {OOPCs} and lost earnings.
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