Patient-Reported Outcomes Measures, Polycystic Kidney Disease Burden, and Outcomes in Autosomal Dominant Polycystic Kidney Disease
Rationale & Objective: Using OVERTURE (NCT01430494) study data on patient-perceived health, health care utilization, and productivity in autosomal dominant polycystic kidney disease (ADPKD), this research was conducted to characterize the burden of illness in patients with ADPKD and assess w...
| Main Authors: | , , , |
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| Format: | Article |
| Language: | English |
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Elsevier
2024-01-01
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| Series: | Kidney Medicine |
| Subjects: | |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S2590059523001735 |
| _version_ | 1797385177637322752 |
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| author | Dorothee Oberdhan Aaron Yarlas Jakob B. Bjorner Holly Krasa |
| author_facet | Dorothee Oberdhan Aaron Yarlas Jakob B. Bjorner Holly Krasa |
| author_sort | Dorothee Oberdhan |
| collection | DOAJ |
| description | Rationale & Objective: Using OVERTURE (NCT01430494) study data on patient-perceived health, health care utilization, and productivity in autosomal dominant polycystic kidney disease (ADPKD), this research was conducted to characterize the burden of illness in patients with ADPKD and assess whether patient-reported outcome (PRO) assessment scores predict clinical and health-economic outcomes. Study Design: Data were analyzed from a prospective, observational study. Setting & Participants: The study cohort comprised 3,409 individuals with ADPKD in 20 countries who were aged 12-78 years and were in chronic kidney disease (CKD) stages G1-G5 and Mayo risk subclasses 1A-1E. Predictors: Scores on PRO instruments, including disease-specific assessments [ADPKD-Impact Scale (ADPKD-IS), and ADPKD-Urinary Impact Scale (ADPKD-UIS)] and generic measures were assessed. Outcomes: Clinical variables [eg, height-adjusted total kidney volume (htTKV), estimated glomerular filtration rate (eGFR), and abdominal girth] and health-economic outcomes were assessed. Analytical Approach: Associations among variables were evaluated using Spearman correlations, logistic regression, and generalized linear mixed effects repeated measures models. Results: Baseline CKD stage and Mayo risk classification showed little correlation with baseline PRO scores; however, scores on disease-specific instruments and measures of physical functioning were worse at more severe CKD stages. PRO scores predicted hospitalizations and sick days at 6-18 months, with strongest associations noted for the ADPKD-IS. PRO scores were not associated with htTKV and eGFR, but worse PRO scores were associated with greater abdominal girth. Poor baseline ADPKD-IS scores were positively associated with occurrence of ADPKD-related symptoms up to 18 months, including kidney pain (OR, 5.30; 95% CI, 2.75-10.24), hematuria (OR, 4.58; 95% CI, 1.99-10.53), and urinary tract infection (OR, 4.41; 95% CI, 1.93-10.11; P < 0.001 for all). Limitations: A limitation of the study was the maximum 18 months of follow-up available to assess outcomes. Conclusions: PRO scores predicted clinical and health-economic outcomes, such as hospitalization and absence from work, underscoring the importance of quality of life assessment of individuals with ADPKD. Plain-Language Summary: Patient-reported outcomes (PROs) are increasingly recognized as important parameters for assessing the clinical and humanistic burden of autosomal dominant polycystic kidney disease (ADPKD). We analyzed data from the observational OVERTURE study to better characterize disease impact on quality of life and determine whether patient-perceived burden might predict outcomes. Scores on PRO assessment instruments predicted hospitalizations and sick days at 6-18 months, with associations strongest for the disease-specific ADPKD-Impact Scale. Compared to patients who rated their health-related quality of life as good, those with poor baseline scores were significantly more likely to report ADPKD-related signs and symptoms up to 18 months of follow-up. These findings support using disease-specific PRO assessment instruments to assess and predict the impact of ADPKD. |
| first_indexed | 2024-03-08T21:50:23Z |
| format | Article |
| id | doaj.art-d3d9dece2cf94dd5b58e2532ab9b978e |
| institution | Directory Open Access Journal |
| issn | 2590-0595 |
| language | English |
| last_indexed | 2024-03-08T21:50:23Z |
| publishDate | 2024-01-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Kidney Medicine |
| spelling | doaj.art-d3d9dece2cf94dd5b58e2532ab9b978e2023-12-20T07:35:30ZengElsevierKidney Medicine2590-05952024-01-0161100755Patient-Reported Outcomes Measures, Polycystic Kidney Disease Burden, and Outcomes in Autosomal Dominant Polycystic Kidney DiseaseDorothee Oberdhan0Aaron Yarlas1Jakob B. Bjorner2Holly Krasa3Otsuka Pharmaceutical Development & Commercialization, Inc., Rockville, MD; Address for Correspondence: Dorothee Oberdhan, MS, Otsuka Pharmaceutical Development & Commercialization, Inc., 2440 Research Blvd., Rockville, MD 20850QualityMetric Incorporated, Johnston, RIQualityMetric Incorporated, Johnston, RIBlue Persimmon Group LLC, Washington, DCRationale & Objective: Using OVERTURE (NCT01430494) study data on patient-perceived health, health care utilization, and productivity in autosomal dominant polycystic kidney disease (ADPKD), this research was conducted to characterize the burden of illness in patients with ADPKD and assess whether patient-reported outcome (PRO) assessment scores predict clinical and health-economic outcomes. Study Design: Data were analyzed from a prospective, observational study. Setting & Participants: The study cohort comprised 3,409 individuals with ADPKD in 20 countries who were aged 12-78 years and were in chronic kidney disease (CKD) stages G1-G5 and Mayo risk subclasses 1A-1E. Predictors: Scores on PRO instruments, including disease-specific assessments [ADPKD-Impact Scale (ADPKD-IS), and ADPKD-Urinary Impact Scale (ADPKD-UIS)] and generic measures were assessed. Outcomes: Clinical variables [eg, height-adjusted total kidney volume (htTKV), estimated glomerular filtration rate (eGFR), and abdominal girth] and health-economic outcomes were assessed. Analytical Approach: Associations among variables were evaluated using Spearman correlations, logistic regression, and generalized linear mixed effects repeated measures models. Results: Baseline CKD stage and Mayo risk classification showed little correlation with baseline PRO scores; however, scores on disease-specific instruments and measures of physical functioning were worse at more severe CKD stages. PRO scores predicted hospitalizations and sick days at 6-18 months, with strongest associations noted for the ADPKD-IS. PRO scores were not associated with htTKV and eGFR, but worse PRO scores were associated with greater abdominal girth. Poor baseline ADPKD-IS scores were positively associated with occurrence of ADPKD-related symptoms up to 18 months, including kidney pain (OR, 5.30; 95% CI, 2.75-10.24), hematuria (OR, 4.58; 95% CI, 1.99-10.53), and urinary tract infection (OR, 4.41; 95% CI, 1.93-10.11; P < 0.001 for all). Limitations: A limitation of the study was the maximum 18 months of follow-up available to assess outcomes. Conclusions: PRO scores predicted clinical and health-economic outcomes, such as hospitalization and absence from work, underscoring the importance of quality of life assessment of individuals with ADPKD. Plain-Language Summary: Patient-reported outcomes (PROs) are increasingly recognized as important parameters for assessing the clinical and humanistic burden of autosomal dominant polycystic kidney disease (ADPKD). We analyzed data from the observational OVERTURE study to better characterize disease impact on quality of life and determine whether patient-perceived burden might predict outcomes. Scores on PRO assessment instruments predicted hospitalizations and sick days at 6-18 months, with associations strongest for the disease-specific ADPKD-Impact Scale. Compared to patients who rated their health-related quality of life as good, those with poor baseline scores were significantly more likely to report ADPKD-related signs and symptoms up to 18 months of follow-up. These findings support using disease-specific PRO assessment instruments to assess and predict the impact of ADPKD.http://www.sciencedirect.com/science/article/pii/S2590059523001735Autosomal dominant polycystic kidney disease (ADPKD)Autosomal Dominant Polycystic Kidney Disease-Impact Scale (ADPKD-IS)chronic kidney disease (CKD)cohort studypatient-reported outcomes (PRO)OVERTURE |
| spellingShingle | Dorothee Oberdhan Aaron Yarlas Jakob B. Bjorner Holly Krasa Patient-Reported Outcomes Measures, Polycystic Kidney Disease Burden, and Outcomes in Autosomal Dominant Polycystic Kidney Disease Kidney Medicine Autosomal dominant polycystic kidney disease (ADPKD) Autosomal Dominant Polycystic Kidney Disease-Impact Scale (ADPKD-IS) chronic kidney disease (CKD) cohort study patient-reported outcomes (PRO) OVERTURE |
| title | Patient-Reported Outcomes Measures, Polycystic Kidney Disease Burden, and Outcomes in Autosomal Dominant Polycystic Kidney Disease |
| title_full | Patient-Reported Outcomes Measures, Polycystic Kidney Disease Burden, and Outcomes in Autosomal Dominant Polycystic Kidney Disease |
| title_fullStr | Patient-Reported Outcomes Measures, Polycystic Kidney Disease Burden, and Outcomes in Autosomal Dominant Polycystic Kidney Disease |
| title_full_unstemmed | Patient-Reported Outcomes Measures, Polycystic Kidney Disease Burden, and Outcomes in Autosomal Dominant Polycystic Kidney Disease |
| title_short | Patient-Reported Outcomes Measures, Polycystic Kidney Disease Burden, and Outcomes in Autosomal Dominant Polycystic Kidney Disease |
| title_sort | patient reported outcomes measures polycystic kidney disease burden and outcomes in autosomal dominant polycystic kidney disease |
| topic | Autosomal dominant polycystic kidney disease (ADPKD) Autosomal Dominant Polycystic Kidney Disease-Impact Scale (ADPKD-IS) chronic kidney disease (CKD) cohort study patient-reported outcomes (PRO) OVERTURE |
| url | http://www.sciencedirect.com/science/article/pii/S2590059523001735 |
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