Rationale and study protocol of ACQUIRE, a prospective, observational study measuring quality of life, treatment preference and treatment satisfaction of autosomal dominant polycystic kidney disease (ADPKD) patients in Europe

Dominique Joly, Jennifer Quinn, Stella Mokiou, Karl O'Reilly, Joaquín Sánchez-Covisa, Jing Wang-Silvanto, Helen Doll, Dominique Joly, Jennifer Quinn, Stella Mokiou, Karl O'Reilly, Joaquín Sánchez-Covisa, Jing Wang-Silvanto, Helen Doll

Abstract

Background: Autosomal dominant polycystic kidney disease (ADPKD) is considered the most common inherited renal disease. Patient-Reported Outcomes (PROs) and patient experience in ADPKD are difficult to quantify and have not been well studied, particularly in the early stages of the disease. There is evidence to suggest that early-stage ADPKD patients have a lower Health-Related Quality of Life (HRQoL) than the general population due to the signs and symptoms of early-stage ADPKD. However, no research has been carried out on the HRQoL of early-stage ADPKD patients using validated ADPKD-specific PRO measures. Additionally, a new disease progression delaying treatment option has recently emerged for ADPKD. Patient preference for this treatment and unmet treatment needs have not yet been investigated.

Methods: The ACQUIRE study is a prospective, observational study investigating the influence of early-stage ADPKD-related symptoms and treatments on PROs. It aims to collect real-world data on patient demographics, treatment patterns, clinical outcomes, and PROs such as HRQoL, treatment satisfaction and treatment preference in early-stage ADPKD. Adult ADPKD patients in stages 1-3 of chronic kidney disease (CKD) with evidence of rapidly progressing disease are being recruited from seven European countries. At baseline and every 3 months, for a follow-up period of 18 months, general and disease-specific questionnaires are completed remotely to capture patients' own assessment of their overall and ADPKD-related HRQoL. A Discrete Choice Experiment (DCE) is also used to investigate the value patients place on different attributes of hypothetical treatment options (e.g. treatment outcomes, side effects) and the role each attribute plays in determining overall patient treatment preference.

Discussion: The results of this study will highlight the real-world effects of ADPKD-related challenges on PROs including HRQoL, treatment experience and satisfaction; and help physicians gain greater insight into likely disease outcomes based on early-stage patient symptoms and patients' experience with treatment. Data captured by the DCE may inform ADPKD treatment decision-making from a patient perspective. The DCE will also provide insights into which patients are more likely to perceive benefit from treatments based on the value and trade-offs they place on specific treatment attributes.

Trial registration: NCT02848521 . Protocol Number/Version: 156-303-00096/Final.

Keywords: ADPKD; Aquaresis; Discrete choice experiment (DCE); Observational; Quality of life; Tolvaptan.

Conflict of interest statement

DJ: DJ received honoraria from Otsuka (from lectures and for participating in Advisory Boards) and is the principal investigator for studies conducted by Otsuka and Ipsen.

JQ: employee of BioMarin Pharmaceutical Inc.

JYL: employee of Otsuka Pharmaceutical Europe Ltd.

SM: employee of Otsuka Pharmaceutical Europe Ltd.

KO: employee of Otsuka Pharmaceutical Europe Ltd.

JSC: employee of Otsuka Pharmaceutical Europe Ltd.

JWS: employee by Otsuka Pharmaceutical Europe Ltd.

HD: employee of Clinical Outcomes Solutions.

Figures

Fig. 1
Fig. 1
Study Design. ADPKD: autosomal dominant polycystic kidney disease; ADPKD-IS: ADPKD Impact Scale; ADKPD-PDS: ADPKD-Pain and Discomfort Scale; ADPKD-UIS: ADPKD-Urinary Impact Scale; CKD: chronic kidney disease; DCE: discrete choice experiment; SF-12: 12-item Short Form Health Survey; TSQM-9: Abbreviated Treatment Satisfaction Questionnaire for Medication
Fig. 2
Fig. 2
Example DCE Question. ADPKD: autosomal dominant polycystic kidney disease; ESRD: end stage renal disease

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