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

Study Status: Terminated
Recruit Status: Unknown status
Study Type: Observational

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

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 Summary:

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 &am
Sponsor: Mayo Clinic

Current Primary Outcome: Polycystic Liver Disease-specific patient reported outcomes questionnaire (PLD-Q) total score [ Time Frame: baseline to 6 months ]

total score on the PLD-Q


Original Primary Outcome: Same as current

Current Secondary Outcome:

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


Original Secondary Outcome: Same as current

Information By: Mayo Clinic

Dates:
Date Received: June 20, 2014
Date Started: June 2014
Date Completion: June 2016
Last Updated: April 6, 2015
Last Verified: April 2015