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

Development and Assessment of the Psychometric Properties of a Polycystic Liver Disease-specific Patient Reported Outcomes Questionnaire (PLD-Q).

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).

Study Overview

• Status: Completed
• Conditions: Polycystic Liver Disease
• Intervention / Treatment: Other: PLD-Q

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.

• Study Type: Observational
• Enrollment (Actual): 594

Contacts and Locations

Study Locations

• United States
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Mayo Clinic

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Patients with polycystic liver disease

Description

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

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Cross-Sectional

Number of groups / cohorts

5

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Polycystic liver disease patients; will receive PLD-Q, EORTC QLQ-30 symptoms subscale, EQ5D-VAS score and SF36	Other: PLD-Q; polycystic liver disease questionnaire
ADPKD group without PLD; will receive PLD-Q
Healthy controls; receive PLD-Q	Other: PLD-Q; polycystic liver disease questionnaire
PLD patient focus group; to discuss and improve PLD-Q
PLD clinical expert focus group; to discuss and improve PLD-Q

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Polycystic Liver Disease-specific patient reported outcomes questionnaire (PLD-Q) total score	total score on the PLD-Q	baseline to 6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The Short Form (36) Health Survey (SF36)		baseline to 6 months
European Organisation for Research and Treatment of Cancer Quality of life questionnaire (EORTC-QLQ-C30) symptom subscale		baseline to 6 months
Euroqol group 5 dimensions questionnaire visual analog score (EQ5D-VAS)		baseline to 6 months
Total liver volume		baseline
Total kidney volume	measured in patients with autosomal dominant polycystic kidney disease (ADPKD)	baseline

Collaborators and Investigators

• Sponsor: Mayo Clinic
• Investigators: Principal Investigator: Marie C Hogan, Mayo Clinic

Publications and helpful links

Helpful Links

• Mayo Clinic Clinical Trials

Study record dates

Study Major Dates

• Study Start (Actual): June 1, 2014
• Primary Completion (Actual): March 11, 2024
• Study Completion (Actual): March 11, 2024

Study Registration Dates

• First Submitted: June 20, 2014
• First Submitted That Met QC Criteria: June 20, 2014
• First Posted (Estimated): June 24, 2014

Study Record Updates

• Last Update Posted (Actual): September 19, 2024
• Last Update Submitted That Met QC Criteria: September 12, 2024
• Last Verified: September 1, 2024

More Information

Terms related to this study

• Keywords: quality of life; autosomal dominant polycystic kidney disease; autosomal dominant polycystic liver disease; patient reported outcome tool
• Additional Relevant MeSH Terms: Digestive System Diseases; Neoplasms; Pathological Conditions, Anatomical; Liver Diseases; Cysts
• Other Study ID Numbers: 14-003832