Abstract：
BACKGROUND: Cystine-depleting therapy in nephropathic cystinosis is currently monitored via the white blood cell cystine assay, although its application and usefulness are limited by practical and technical issues. Therefore, alternative biomarkers that are widely available, more economical and less technically demanding, while reliably reflecting long-term adherence to cysteamine treatment, are desirable. Recently, we proposed chitotriosidase enzyme activity as a potential novel biomarker for the therapeutic monitoring of cysteamine treatment in cystinosis. In this study, we aimed to validate our previous findings and to confirm the value of chitotriosidase in the management of cystinosis therapy.
METHODS: A retrospective study was conducted on 12 patients treated at the National Institutes of Health Clinical Center and followed up for at least 2 years. Plasma chitotriosidase enzyme activity was correlated with corresponding clinical and biochemical data.
RESULTS: Plasma chitotriosidase enzyme activity significantly correlated with WBC cystine levels, cysteamine total daily dosage and a Composite compliance score. Moreover, plasma chitotriosidase was a significant independent predictor for WBC cystine levels, and cut-off values were established in both non-kidney transplanted and kidney transplanted cystinosis patients to distinguish patients with a good versus poor compliance with cysteamine treatment. Our observations are consistent with those of our previous study and validate our findings.
CONCLUSIONS: Chitotriosidase enzyme activity is a valid potential alternative biomarker for monitoring cysteamine treatment in nephropathic cystinosis patients.
CONCLUSIONS: Chitotriosidase enzyme activity is a valid potential alternative biomarker for monitoring cysteamine treatment in nephropathic cystinosis patients.
METHODS: A retrospective study was conducted on 12 patients treated at the National Institutes of Health Clinical Center and followed up for at least 2 years. Plasma chitotriosidase enzyme activity was correlated with corresponding clinical and biochemical data.
RESULTS: Plasma chitotriosidase enzyme activity significantly correlated with WBC cystine levels, cysteamine total daily dosage and a Composite compliance score. Moreover, plasma chitotriosidase was a significant independent predictor for WBC cystine levels, and cut-off values were established in both non-kidney transplanted and kidney transplanted cystinosis patients to distinguish patients with a good versus poor compliance with cysteamine treatment. Our observations are consistent with those of our previous study and validate our findings.
CONCLUSIONS: Chitotriosidase enzyme activity is a valid potential alternative biomarker for monitoring cysteamine treatment in nephropathic cystinosis patients.
CONCLUSIONS: Chitotriosidase enzyme activity is a valid potential alternative biomarker for monitoring cysteamine treatment in nephropathic cystinosis patients.
摘要：
背景：目前通过白细胞胱氨酸测定法监测肾病性膀胱炎的半胱氨酸消耗疗法，尽管它的应用和有用性受实际和技术问题的限制。因此,广泛可用的替代生物标志物，更经济，技术要求更低，同时可靠地反映了对半胱胺治疗的长期坚持，是可取的。最近,我们提出了壳三糖苷酶酶活性作为一个潜在的新的生物标志物，用于治疗性监测半胱胺治疗的胱氨酸病。在这项研究中,我们的目的是验证我们之前的研究结果,并确认壳三糖苷酶在胱氨酸病治疗中的价值.
方法：对在美国国立卫生研究院临床中心接受治疗的12名患者进行了回顾性研究，并随访了至少2年。血浆壳三糖苷酶酶活性与相应的临床和生化数据相关。
结果：血浆壳三糖苷酶活性与白细胞胱氨酸水平显著相关，半胱胺总每日剂量和综合依从性评分。此外，血浆壳三糖苷酶是白细胞胱氨酸水平的显著独立预测因子，并在非肾移植和肾移植的胱氨酸病患者中建立了临界值，以区分对半胱胺治疗依从性好和差的患者。我们的观察结果与我们之前的研究结果一致，并验证了我们的发现。
结论：壳三糖苷酶活性是监测肾病性膀胱炎患者半胱胺治疗的有效的潜在替代生物标志物。
结论：壳三糖苷酶活性是监测肾病性膀胱炎患者半胱胺治疗的有效的潜在替代生物标志物。
方法：对在美国国立卫生研究院临床中心接受治疗的12名患者进行了回顾性研究，并随访了至少2年。血浆壳三糖苷酶酶活性与相应的临床和生化数据相关。
结果：血浆壳三糖苷酶活性与白细胞胱氨酸水平显著相关，半胱胺总每日剂量和综合依从性评分。此外，血浆壳三糖苷酶是白细胞胱氨酸水平的显著独立预测因子，并在非肾移植和肾移植的胱氨酸病患者中建立了临界值，以区分对半胱胺治疗依从性好和差的患者。我们的观察结果与我们之前的研究结果一致，并验证了我们的发现。
结论：壳三糖苷酶活性是监测肾病性膀胱炎患者半胱胺治疗的有效的潜在替代生物标志物。
结论：壳三糖苷酶活性是监测肾病性膀胱炎患者半胱胺治疗的有效的潜在替代生物标志物。
