Pediatric Nephrology ( IF 3 ) Pub Date : 2020-09-09 , DOI: 10.1007/s00467-020-04722-0 Segolene Gaillard 1, 2 , Laurent Roche 2, 3 , Sandrine Lemoine 4 , Georges Deschênes 5 , Denis Morin 6 , Christine Vianey-Saban 7 , Cécile Acquaviva-Bourdain 7 , Bruno Ranchin 8 , Justine Bacchetta 8 , Behrouz Kassai 1, 2 , Patrice Nony 1, 2 , Eurielle Bodénan 1 , Valérie Laudy 1, 2 , Cécile Rouges 9 , Setareh Zarrabian 10 , Fabien Subtil 2, 3 , Catherine Mercier 2, 3 , Pierre Cochat 8 , Aurélia Bertholet-Thomas 8
Introduction
In nephropathic cystinosis (NC), adherence to cysteamine remains challenging; poor adherence is worsening the disease progression with a decline of kidney function and increase of extrarenal morbidities. Our objective was to describe adherence to cysteamine in NC patients, using electronic monitoring systems.
Methods
Patients with confirmed NC, aged > 4 years and receiving oral cysteamine (short acting or delayed release formulation as standard of care) from 3 French reference centers, were included. Adherence to treatment was primarily assessed as the percentage of days with a good adherence score, adherence score rating from 0 (poor) to 2 (good). A descriptive analysis was performed after 1-year follow-up.
Results
Seventeen patients (10 girls, median age: 13.9 (5.4–33.0) years) were included. Median age at diagnosis was 17.0 (3.0–76.9) months and age at start of cysteamine was 21.0 (15.5–116.3) months. Median daily dose of cysteamine was 1.05 (0.55–1.63) g/m2/day. Over the year, the median percentage of days with a good adherence score was 80 (1–99)% decreasing to 68 (1–99)% in patients > 11 years old. The median of average number of hours covered by treatment in a day was 22.5 (6.1–23.9) versus 14.9 (9.2–20.5) hours for delayed release versus short acting cysteamine.
Conclusion
Our data are the first describing a rather good adherence to cysteamine, decreasing in adolescents and adults. We described a potential interest of the delayed release formulation. Our data highlight the need for a multidisciplinary approach including therapeutic education and individualized approaches in NC patients transitioning to adulthood.
Graphical abstract
中文翻译:
肾病性胱氨酸病中半胱胺的依从性:一种独特的电子监测体验,可以更好地理解。一项前瞻性队列研究:CrYSTobs。
介绍
在肾病性胱氨酸病 (NC) 中,对半胱胺的依从性仍然具有挑战性;依从性差正在恶化疾病进展,肾功能下降和肾外发病率增加。我们的目标是使用电子监测系统描述 NC 患者对半胱胺的依从性。
方法
包括确诊 NC、年龄 > 4 岁并接受来自 3 个法国参考中心的口服半胱胺(短效或延迟释放制剂作为标准护理)的患者。治疗依从性主要评估为具有良好依从性评分的天数百分比,依从性评分从 0(差)到 2(好)。随访 1 年后进行描述性分析。
结果
包括 17 名患者(10 名女孩,中位年龄:13.9 (5.4-33.0) 岁)。诊断时的中位年龄为 17.0 (3.0-76.9) 个月,半胱胺开始时的年龄为 21.0 (15.5-116.3) 个月。半胱胺的中位日剂量为 1.05 (0.55–1.63) g/m 2 /天。在一年中,具有良好依从性评分的天数中位数百分比从 80 (1–99)% 下降到 > 11 岁患者的 68 (1–99)%。对于延迟释放与短效半胱胺,一天中治疗涵盖的平均小时数的中位数为 22.5 (6.1-23.9) 小时和 14.9 (9.2-20.5) 小时。
结论
我们的数据首次描述了对半胱胺的良好依从性,在青少年和成人中有所下降。我们描述了延迟释放制剂的潜在利益。我们的数据强调需要多学科方法,包括过渡到成年的 NC 患者的治疗教育和个性化方法。
图形概要