Development of the Patient- and Observer-Reported PRUCISION Instruments to Assess Pruritus and Sleep Disturbance in Pediatric Patients with Cholestatic Liver Diseases

Bile, a greenish liquid that is made in the liver, is released into the gut to help digest food. In cholestatic liver disease (CLD), bile flow is interrupted, and bile can build up in the body. One potential effect of this buildup is pruritus, or itchiness of the skin, which can be so intense that it interferes with daily activities. In this study, interviews were done with doctors, patients, and their caregivers to develop new tools to evaluate the most impactful symptoms of CLD in children. After interviewing five doctors and 36 patients and caregivers, two questionnaires called PRUCISION were developed and refined. During this process, participants were first asked about the frequency, severity, duration, and impact of their or their child’s symptoms; pruritus was identified as the most common and disruptive symptom associated with CLD, even interfering with sleep. Then, the wording of the questionnaires was modified to make them easier to understand, particularly for younger children. The researchers also had patients do a card-sorting task to ensure that they understood the picture-based responses used in the questionnaires. Finally, more details were added to the instructions for caregivers to more clearly define scratching behaviors. In summary, the questionnaires developed in this study include the perspective of the patient or their caregiver and may be useful to see if new treatments can impact the most prominent symptoms and impacts associated with CLD.