Adherence to cysteamine in nephropathic cystinosis: A unique electronic monitoring experience for a better understanding
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ORIGINAL ARTICLE
Adherence to cysteamine in nephropathic cystinosis: A unique electronic monitoring experience for a better understanding. A prospective cohort study: CrYSTobs Segolene Gaillard 1,2 & Laurent Roche 2,4 & Sandrine Lemoine 3 & 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 2,4 & Catherine Mercier 2,4 & Pierre Cochat 8 & Aurélia Bertholet-Thomas 8 Received: 20 May 2020 / Revised: 22 June 2020 / Accepted: 23 July 2020 # IPNA 2020
Abstract 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.
Keywords Nephropathic cystinosis . Cysteamine . Adherence . Medication event monitoring system
Introduction Nephropathic cystinosis (NC) is an orphan inherited autosomal recessive disease characterized as a generalized lysosomal storage disease due to a deficiency of the cystine Electronic supplementary material The online version of this article (https://doi.org/10.1007/s00467-020-04722-0) contains supplementary material, which is available to authorized users. * Segolene Gaillard [email protected] Extended author information available on the last page of the article
lysosomal transport protein, cystinosin [1]. It is encoded by the CTNS (Cystinosin) gene located on chromosome 17 (17p13.2) [2–4]. Its
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