Researchers from the US and Spain have developed novel patient- and observer-reported outcome questionnaires to evaluate the effects of cholestatic pruritus in children with cholestatic liver diseases such as Alagille syndrome and progressive familial intrahepatic cholestasis.

The new questionnaires, which the researchers called PRUCISION, can be useful in understanding the effect of new treatments on the symptoms of cholestatic liver diseases and their impact on patients.

Pruritus in cholestatic liver diseases like Alagille syndrome can be so intense that it can significantly impact patients’ daily functioning and substantially disturb their sleep.


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To develop new tools to evaluate the most impactful symptoms of cholestatic liver disease in children, a team of researchers led by Patrick Horn, MD, PhD, from Albireo Pharma in Boston, Massachusetts, conducted interviews with 5 doctors and 36 patients and their caregivers. They asked the participants about the frequency, severity, duration, and impact of disease symptoms.

Read more about the symptoms of Alagille syndrome

They found that pruritus was the most problematic symptom of the disease, and sleep disturbances including difficulty falling or staying asleep due to itchiness had the most significant impact on the children.

The questionnaires were then refined to improve comprehension and usability. The final questionnaires are in the form of an electronic diary to be completed twice a day, and response modules include pictorial, verbal, and numeric scales.

“These instruments can be used in clinical trials to evaluate the efficacy of novel treatments for children with cholestatic liver diseases,” the researchers concluded.

Alagille syndrome is a genetic disease that affects many organs and tissues, causing a wide range of symptoms including pruritus, jaundice, xanthomas, changes in the color of urine and feces, hepatomegaly, and splenomegaly. These symptoms often appear as early as the first year of life. 

Reference

Gwaltney C, Bean S, Venerus M, et al. Development of the patient- and observer-reported PRUCISION instruments to assess pruritus and sleep disturbance in pediatric patients with cholestatic liver diseases. Adv Ther. Published online September 6, 2022. doi:10.1007/s12325-022-02261-8