Theodorakakou Foteini, Fotiou Despina, Apostolakou Filia, Papassotiriou Ioannis, Spiliopoulou Vasiliki, Ntanasis-Stathopoulos Ioannis, Malandrakis Panagiotis, Migkou Magdalini, Kanellias Nikolaos, Eleutherakis-Papaiakovou Evangelos, Psimenou Erasmia, Papanikolaou Asimina, Gakiopoulou Charikleia, Marinaki Smaragdi, Giannouli Stavroula, Gavriatopoulou Maria, Terpos Evangelos, Dimopoulos Meletios-Athanasios, Kastritis Efstathios
Department of Clinical Therapeutics, National and Kapodistrian University of Athens, Athens, Greece.
Department of Clinical Biochemistry, "Aghia Sophia" Children's Hospital, Athens, Greece.
Am J Hematol. 2025 Aug;100(8):1305-1313. doi: 10.1002/ajh.27716. Epub 2025 May 19.
Cystatin C (CysC) has emerged as a novel and potentially more reliable biomarker for the estimation of glomerular filtration in the general population in patients with various conditions. In AL amyloidosis, the current renal staging system and renal response criteria are based on proteinuria and creatinine-based eGFR. We explored the prognostic role of CysC and of estimation of eGFR based on CysC-based equations in a cohort of 195 patients with newly diagnosed AL amyloidosis with renal involvement. Baseline CysC level was strongly and independently associated with progression to dialysis, and CysC levels ≥ 1.9 mg/L can be used in combination with the current renal staging system to identify patients with different risk of progression to dialysis among renal stages 2 and 3. eGFR based on CysC performed at least similarly to eGFR based on creatinine alone (by CKD-EPI race free formula) and the cutoff of 30 mL/min/1.73 m could better predict progression to dialysis at 2 years. At 6 months landmark, an increase in CysC by ≥ 1 mg/L was associated with higher risk of progression to dialysis (HR: 19.8, 95% CI 6.5-60.5, p < 0.001); a reduction of CysC based eGFR ≥ 30% was also associated with poor renal outcome, with a prognostic performance similar to current renal progression criteria. In conclusion, CysC provides prognostic information regarding the renal outcomes in patients with AL amyloidosis independently of the established biomarkers, but requires further validation.
胱抑素C（CysC）已成为一种新型且可能更可靠的生物标志物，用于评估普通人群及各种疾病患者的肾小球滤过功能。在AL淀粉样变性中，目前的肾脏分期系统和肾脏反应标准是基于蛋白尿和基于肌酐的估算肾小球滤过率（eGFR）。我们在195例新诊断的有肾脏受累的AL淀粉样变性患者队列中，探讨了CysC的预后作用以及基于CysC方程估算eGFR的情况。基线CysC水平与进展至透析密切且独立相关，CysC水平≥1.9mg/L可与当前肾脏分期系统联合使用，以识别2期和3期肾脏疾病中进展至透析风险不同的患者。基于CysC的eGFR表现至少与仅基于肌酐的eGFR（采用CKD-EPI无种族公式）相似，30mL/min/1.73m²的截断值能更好地预测2年时进展至透析的情况。在6个月的时间节点，CysC升高≥1mg/L与进展至透析的较高风险相关（风险比：19.8，95%可信区间6.5 - 60.5，p<0.001）；基于CysC的eGFR降低≥30%也与不良肾脏结局相关，其预后性能与当前肾脏进展标准相似。总之，CysC独立于已有的生物标志物，提供了有关AL淀粉样变性患者肾脏结局的预后信息，但需要进一步验证。
