Development of a Novel Tool to Assess the Impact of Itching in Pediatric Cholestasis

Binita M. Kamath (), Linda Abetz-Webb, Ciara Kennedy, Bonnie Hepburn, Martha Gauthier, Nathan Johnson, Sharon Medendorp, Alejandro Dorenbaum, Lora Todorova and Benjamin L. Shneider
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Binita M. Kamath: Hospital for Sick Children and The University of Toronto
Linda Abetz-Webb: Patient-Centred Outcomes Assessments Ltd
Ciara Kennedy: Amplyx Pharmaceuticals Inc.
Bonnie Hepburn: University of California, San Diego
Martha Gauthier: Endpoint Outcomes
Nathan Johnson: Endpoint Outcomes
Sharon Medendorp: Premier Research
Alejandro Dorenbaum: Stanford University
Lora Todorova: Shire International GmbH
Benjamin L. Shneider: Texas Children’s Hospital

The Patient: Patient-Centered Outcomes Research, 2018, vol. 11, issue 1, No 7, 69-82

Abstract: Abstract Objectives The aim was to develop a clinical outcome assessment (COA) for itching in children with cholestatic pruritus. Methods This prospective study aimed to enroll patients aged 4–30 years with Alagille syndrome (ALGS) or progressive familial intrahepatic cholestasis type 1 and caregivers of patients aged 5 months to 14 years. Eligible patients experienced itching during ≥3 of the 7 days before enrollment and had not undergone liver transplant or surgical interruption of the enterohepatic circulation. Open-ended qualitative interviews confirmed that itching was a primary concern for patients and caregivers. Diaries were modified and then evaluated by participants during cognitive debriefing. Interview results were reviewed by clinical, COA and statistical experts. Diary questions were revised following an interim analysis before finalizing the Itch Reported Outcome (ItchRO). Results Thirty-six interviews were analyzed, representing 25 families of patients with ALGS. Itching was reported spontaneously (without prompting by the interviewer) by ten of 12 patients with ALGS and 19 of 20 caregivers. Consequences of itching included skin damage (78%), mood changes (59%), and difficulties staying asleep (59%) or falling asleep (53%). Two versions of the ItchRO were developed: ItchRO(Patient) for self-completion by patients and ItchRO(Observer) for caregivers. The ItchRO diaries comprise a single scorable item to assess itch and are to be completed twice daily (morning and evening). Conclusions Itching was the most bothersome ALGS symptom reported by study participants. We have developed the ItchRO(Patient) and ItchRO(Observer) to assess itching in children with ALGS and other cholestatic liver diseases. These diaries are being validated for use in clinical trials.

Date: 2018
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DOI: 10.1007/s40271-017-0266-4

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