Dataset Information

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

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.

SUBMITTER: Kamath BM

PROVIDER: S-EPMC5766715 | biostudies-literature | 2018 Feb

REPOSITORIES: biostudies-literature

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Publications

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

Kamath Binita M BM Abetz-Webb Linda L Kennedy Ciara C Hepburn Bonnie B Gauthier Martha M Johnson Nathan N Medendorp Sharon S Dorenbaum Alejandro A Todorova Lora L Shneider Benjamin L BL

The patient 20180201 1

<h4>Objectives</h4>The aim was to develop a clinical outcome assessment (COA) for itching in children with cholestatic pruritus.<h4>Methods</h4>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 e ...[more]

PMID: 28710680

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Dataset Information

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

Objectives

Methods

Results

Conclusions

Publications

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

Similar Datasets

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