Kidney Week

Abstract: SA-PO498

Identifying Patient-Important Outcomes in Polycystic Kidney Disease: An International Nominal Group Technique Study

Session Information

• ADPKD: Clinical Studies
  October 27, 2018 | Location: Exhibit Hall, San Diego Convention Center
  Abstract Time: 10:00 AM - 12:00 PM

Category: Genetic Diseases of the Kidney

• 1001 Genetic Diseases of the Kidney: Cystic

Authors

• Cho, Yeoung Jee, Princess Alexandra Hospital, Brisbane, Queensland, Australia

Yeoung Jee Cho,

• Gutman, Talia M., Centre for Kidney Research, Bondi Junction, New South Wales, Australia

Talia M. Gutman,

• Rangan, Gopi, Westmead Institute for Medical Research, The University of Sydney, Sydney, New South Wales, Australia

Gopi Rangan,

• Craig, Jonathan C., University of Sydney/Children's Hospital, Sydney, New South Wales, Australia

Jonathan C. Craig,

• Viecelli, Andrea K., Princess Alexandra Hospital, Brisbane, Queensland, Australia

Andrea K. Viecelli,

• Geneste, Claire, University Francois Rabelais, Tours, France

Claire Geneste,

• Kim, Yaerim, Seoul National University Hospital, Seoul, Korea (the Republic of)

Yaerim Kim,

• Ahn, Curie, Seoul National University Hospital, Seoul, Korea (the Republic of)

Curie Ahn,

• Kim, Hyunsuk, Chuncheon Sacred Heart Hospital, Chuncheon, Gangwon-Do, Korea (the Republic of)

Hyunsuk Kim,

• Johnson, David W., Princess Alexandra Hospital, Brisbane, Queensland, Australia

David W. Johnson,

• Tong, Allison, The University of Sydney, Seven Hills, New South Wales, Australia

Allison Tong,

Group or Team Name

• on behalf of SONG-PKD Steering Committee

Background

Patients with autosomal dominant polycystic kidney disease (ADPKD) are at increased risk of premature mortality, morbidities, and complications, which severely impair quality of life. However, patient-centered outcomes are not consistently reported intrials in ADPKD, which can limit shared decision-making. We aimed to identify outcomes important to patients and caregivers and the reasons for their priorities.

Methods

Patients with ADPKD and their caregivers were purposively selected from eight centers across Australia, France and Republic of Korea. Participants identified ranked and discussed outcomes for trials in ADPKD. We calculated an importance score (0-1) for each outcome and conducted thematic analyses.

Results

Across 17 groups, 154 participants (121 patients, 33 caregivers) aged 19 to 78 (mean 54.5 years) identified 55 oucomes. The 10 highest ranked outcomes were: kidney function (mean importance score 0.36), end stage kidney disease (0.32), survival (0.21), cyst size/growth (0.20), cyst pain/bleeding (0.18), blood pressure (0.17), ability to work (0.16), cerebral aneurysm/stroke (0.14), mobility/physical function (0.12), and fatigue (0.12). Three themes were identified: threatening sembalance of normality, inability to control, and making sense of the diverse risks.

Conclusion

For patients with ADPKD and their caregivers, kidney function, delayed progression to end stage kidney disease and survival were the highest priorities, and were focused on achieving normality, and maintaining control over health and lifestyle. Implementing these patient-important outcomes may improve the meaning and relevance of trials to inform clinical care in ADPKD.

Mean importance score by patient status

Funding

• Private Foundation Support