Perrone Ronald D, Oberdhan Dorothee, Ouyang John, Bichet Daniel G, Budde Klemens, Chapman Arlene B, Gitomer Berenice Y, Horie Shigeo, Ong Albert C M, Torres Vicente E, Turner A Neil, Krasa Holly
Division of Nephrology, Tufts Medical Center, Tufts University School of Medicine, Boston, Massachusetts, USA.
Otsuka Pharmaceutical Development & Commercialization, Inc., Rockville, Maryland, USA.
Kidney Int Rep. 2023 Feb 13;8(5):989-1001. doi: 10.1016/j.ekir.2023.02.1073. eCollection 2023 May.
The course of autosomal dominant polycystic kidney disease (ADPKD) varies greatly among affected individuals, necessitating natural history studies to characterize the determinants and effects of disease progression. Therefore, we conducted an observational, longitudinal study (OVERTURE; NCT01430494) of patients with ADPKD.
This prospective study enrolled a large international population ( = 3409) encompassing a broad spectrum of ages (12-78 years), chronic kidney disease (CKD) stages (G1-G5), and Mayo imaging classifications (1A-1E). Outcomes included kidney function, complications, quality of life, health care resource utilization, and work productivity.
Most subjects (84.4%) completed ≥12 months of follow-up. Consistent with earlier findings, each additional l/m of height-adjusted total kidney volume (htTKV) on magnetic resonance imaging (MRI) was associated with worse outcomes, including lower estimated glomerular filtration rate (eGFR) (regression coefficient 17.02, 95% confidence interval [CI] 15.94-18.11) and greater likelihood of hypertension (odds ratio [OR] 1.25, 95% CI 1.17-1.34), kidney pain (OR 1.22, 95% CI 1.11-1.33), and hematuria (OR 1.35, 95% CI 1.21-1.51). Greater baseline htTKV was also associated with worse patient-reported health-related quality of life (e.g., ADPKD Impact Scale physical score, regression coefficient 1.02, 95% CI 0.65-1.39), decreased work productivity (e.g., work days missed, regression coefficient 0.55, 95% CI 0.18-0.92), and increased health care resource utilization (e.g., hospitalizations, OR 1.48, 95% CI 1.33-1.64) during follow-up.
Although limited by a maximum 3-year duration of follow-up, this observational study characterized the burden of ADPKD in a broad population and indicated the predictive value of kidney volume for outcomes other than kidney function.
常染色体显性遗传性多囊肾病（ADPKD）患者的病程在个体间差异很大，因此需要进行自然史研究来确定疾病进展的决定因素和影响。为此，我们对ADPKD患者开展了一项观察性纵向研究（OVERTURE；NCT01430494）。
这项前瞻性研究纳入了一个庞大的国际人群（n = 3409），涵盖了广泛的年龄范围（12 - 78岁）、慢性肾脏病（CKD）分期（G1 - G5）以及梅奥影像分类（1A - 1E）。研究结局包括肾功能、并发症、生活质量、医疗资源利用和工作生产力。
大多数受试者（84.4%）完成了≥12个月的随访。与早期研究结果一致，磁共振成像（MRI）上每增加1升/米身高校正后的总肾体积（htTKV），都与更差的结局相关，包括更低的估计肾小球滤过率（eGFR）（回归系数17.02，95%置信区间[CI] 15.94 - 18.11）以及更高的高血压发生可能性（比值比[OR] 1.25，95% CI 1.17 - 1.34）、肾区疼痛（OR 1.22，95% CI 1.11 - 1.33）和血尿（OR 1.35，95% CI 1.21 - 1.51）。更高的基线htTKV还与患者报告的健康相关生活质量较差相关（例如，ADPKD影响量表身体评分，回归系数1.02，95% CI 0.65 - 1.39）、工作生产力下降（例如，缺勤天数，回归系数0.55，95% CI 0.18 - 0.92）以及随访期间医疗资源利用增加（例如，住院，OR 1.48，95% CI 1.33 - 1.64）。
尽管本观察性研究受限于最长3年的随访时间，但它描述了广泛人群中ADPKD的负担，并表明肾体积对除肾功能外的其他结局具有预测价值。
