Abstract：
Young autosomal dominant polycystic kidney disease (ADPKD) patients are becoming the new target population for the development of new treatment options. Determination of a reliable equation for estimated glomerular filtration rate (eGFR) from early stages is needed with the promising potential interventional therapies.
Prospective and longitudinal study on a cohort of 68 genotyped ADPKD patients (age range 0-23 years) with long-term follow-up. Commonly used equations for eGFR were compared for their relative performance.
The revised Schwartz formula (CKiD) showed a highly significant decline in eGFR with aging (- 3.31 mL/min/1.73 m2/year, P < 0.0001). The recently updated equation by the Schwartz group (CKiDU25) showed a smaller (- 0.90 mL/min/1.73 m2/year) but significant (P = 0.001) decline in eGFR with aging and also showed a significant sex difference (P < 0.0001), not observed by the other equations. In contrast, the full age spectrum (FAS) equations (FAS-SCr, FAS-CysC, and the combined) showed no age and sex dependency. The prevalence of hyperfiltration is highly dependent on the formula used, and the highest prevalence was observed with the CKiD Equation (35%).
The most widely used methods to calculate eGFR in ADPKD children (CKiD and CKiDU25 equations) were associated with unexpected age or sex differences. The FAS equations were age- and sex-independent in our cohort. Hence, the switch from the CKiD to CKD-EPI equation at the transition from pediatric to adult care causes implausible jumps in eGFR, which could be misinterpreted. Having reliable methods to calculate eGFR is indispensable for clinical follow-up and clinical trials. A higher resolution version of the Graphical abstract is available as Supplementary information.
Prospective and longitudinal study on a cohort of 68 genotyped ADPKD patients (age range 0-23 years) with long-term follow-up. Commonly used equations for eGFR were compared for their relative performance.
The revised Schwartz formula (CKiD) showed a highly significant decline in eGFR with aging (- 3.31 mL/min/1.73 m2/year, P < 0.0001). The recently updated equation by the Schwartz group (CKiDU25) showed a smaller (- 0.90 mL/min/1.73 m2/year) but significant (P = 0.001) decline in eGFR with aging and also showed a significant sex difference (P < 0.0001), not observed by the other equations. In contrast, the full age spectrum (FAS) equations (FAS-SCr, FAS-CysC, and the combined) showed no age and sex dependency. The prevalence of hyperfiltration is highly dependent on the formula used, and the highest prevalence was observed with the CKiD Equation (35%).
The most widely used methods to calculate eGFR in ADPKD children (CKiD and CKiDU25 equations) were associated with unexpected age or sex differences. The FAS equations were age- and sex-independent in our cohort. Hence, the switch from the CKiD to CKD-EPI equation at the transition from pediatric to adult care causes implausible jumps in eGFR, which could be misinterpreted. Having reliable methods to calculate eGFR is indispensable for clinical follow-up and clinical trials. A higher resolution version of the Graphical abstract is available as Supplementary information.
摘要：
背景：年轻常染色体显性遗传多囊肾病（ADPKD）患者正在成为开发新治疗方案的新目标人群。对于有前途的潜在介入疗法，需要从早期阶段确定估算的肾小球滤过率（eGFR）的可靠方程。
方法：对68名基因分型ADPKD患者（年龄范围0-23岁）进行长期随访的前瞻性和纵向研究。比较了eGFR的常用方程的相对性能。
结果：修订的Schwartz公式（CGiD）显示，随着老化，eGFR显着下降（-3.31mL/min/1.73m2/年，P<0.0001)。Schwartz组（CGIDU25）最近更新的方程显示，随着年龄的增长，eGFR下降较小（-0.90mL/min/1.73m2/年），但显著（P=0.001），也显示出显著的性别差异（P<0.0001）。其他方程没有观察到。相比之下,全年龄谱(FAS)方程(FAS-SCr，FAS-CysC,并且合并）没有显示年龄和性别依赖性。超滤的患病率高度依赖于所使用的配方,并观察到最高的患病率与CGiD方程（35％）。
结论：计算ADPKD儿童eGFR的最广泛使用的方法（CGiD和CGiDU25方程）与意外的年龄或性别差异有关。在我们的队列中，FAS方程与年龄和性别无关。因此，从儿童护理过渡到成人护理时，从CKD-EPI方程的转换会导致eGFR的不合理跳跃，这可能会被误解。具有可靠的方法来计算eGFR对于临床随访和临床试验是必不可少的。更高分辨率版本的图形摘要可作为补充信息。
方法：对68名基因分型ADPKD患者（年龄范围0-23岁）进行长期随访的前瞻性和纵向研究。比较了eGFR的常用方程的相对性能。
结果：修订的Schwartz公式（CGiD）显示，随着老化，eGFR显着下降（-3.31mL/min/1.73m2/年，P<0.0001)。Schwartz组（CGIDU25）最近更新的方程显示，随着年龄的增长，eGFR下降较小（-0.90mL/min/1.73m2/年），但显著（P=0.001），也显示出显著的性别差异（P<0.0001）。其他方程没有观察到。相比之下,全年龄谱(FAS)方程(FAS-SCr，FAS-CysC,并且合并）没有显示年龄和性别依赖性。超滤的患病率高度依赖于所使用的配方,并观察到最高的患病率与CGiD方程（35％）。
结论：计算ADPKD儿童eGFR的最广泛使用的方法（CGiD和CGiDU25方程）与意外的年龄或性别差异有关。在我们的队列中，FAS方程与年龄和性别无关。因此，从儿童护理过渡到成人护理时，从CKD-EPI方程的转换会导致eGFR的不合理跳跃，这可能会被误解。具有可靠的方法来计算eGFR对于临床随访和临床试验是必不可少的。更高分辨率版本的图形摘要可作为补充信息。
