Development and Assessment of The Polycystic Liver Disease Questionnaire (PLD-Q).

NCT ID: NCT02173080

Last Updated: 2024-09-19

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 594 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2014-06-30
• Study Completion Date: 2024-03-11

Brief Summary

This Pilot study will enable development & assessment of a Polycystic Liver Disease-specific patient reported outcomes questionnaire (PLD-Q). Polycystic liver disease (PLD) is characterized by the formation of numerous cysts in the liver, and can lead to severe symptomatic hepatomegaly. It is common in patients with autosomal dominant polycystic liver disease (ADPLD) and autosomal dominant polycystic kidney disease (ADPKD).

Detailed Description

As liver function is preserved, quality of life is the main outcome in PLD. Patient reported outcomes are increasingly utilized to assess treatment efficacy & can include any endpoint derived from patient reports & are usually assessed by questionnaire. Several existing generic questionnaires assess gastrointestinal symptoms, but include irrelevant items for polycystic liver disease (PLD) & do not examine relevant extra-abdominal symptoms. Also, a questionnaire that assesses the full range of PLD related problems is more likely to be responsive to changes after treatment initiation. Therefore, a tool that accurately detects changes in PLD symptoms is required.

The development and first steps of validation of this questionnaire are already completed in Netherlands. We will further develop this questionnaire for English speaking patients and validate it in Mayo Clinic cohorts.

The study contains three phases. Phase 1: pilot testing of the directly translated questionnaire (from dutch into english) in a small group of polycystic liver disease (PLD) patients. We will include both symptomatic & asymptomatic patients (i.e. from mild to severe PLD). Phase 2: further refinement of the questionnaire using the results of the pilot test and input of Mayo patient and clinician focus groups. Phase 3: Further validation in a large cohort of 200 PLD patients with (1) comparison with 200 controls without PLD, (2) cross-sectional correlation with total liver (TLV) & kidney volumes (TKV), (3) comparing ADPKD patients with & without PLD (5) against other validated questionnaires (e.g. SF36) & (5) development of a custom "Worry Questionnaire" examining disease-related worries & concerns. Finally, longitudinal analyses will be performed to assess PLD-Q sensitivity to change in symptoms. Statistical & psychometric analyses will be provided by Mayo Clinic quality of life Research group, & developed in accordance with Food and Drug Administration (FDA) guidance on patient-reported outcome measures in consultation with their Study Endpoints & Labeling Development Division.

Conditions

Polycystic Liver Disease

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: CROSS_SECTIONAL

Study Groups

Polycystic liver disease patients

will receive PLD-Q, EORTC QLQ-30 symptoms subscale, EQ5D-VAS score and SF36

PLD-Q

Intervention Type: OTHER

polycystic liver disease questionnaire

ADPKD group without PLD

will receive PLD-Q

No interventions assigned to this group

Healthy controls

receive PLD-Q

PLD-Q

Intervention Type: OTHER

polycystic liver disease questionnaire

PLD patient focus group

to discuss and improve PLD-Q

No interventions assigned to this group

PLD clinical expert focus group

to discuss and improve PLD-Q

No interventions assigned to this group

Interventions

PLD-Q

polycystic liver disease questionnaire

Intervention Type: OTHER

Eligibility Criteria

Inclusion Criteria

• Patients ≥ 18 year
• Polycystic liver, defined by > 20 liver cysts on imaging

Exclusion Criteria

• Patients unable to speak or read the English properly
• History of kidney or liver transplantation
• Dialysis
• Current use of experimental drugs (e.g. lanreotide, octreotide)
• Recent liver resection or major surgery
• Other comorbidities that can affect the outcome of the questionnaire, as deemed by the investigators

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Mayo Clinic

OTHER

Sponsor Role: lead

Responsible Party

Marie Hogan

PI

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Marie C Hogan

Role: PRINCIPAL_INVESTIGATOR

Mayo Clinic

Locations

Mayo Clinic

Rochester, Minnesota, United States

Countries

United States

Related Links

https://www.mayo.edu/research/clinical-trials

Mayo Clinic Clinical Trials

Other Identifiers

14-003832

Identifier Type: -

Identifier Source: org_study_id