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. 2021 Mar;36(3):581-589.
doi: 10.1007/s00467-020-04722-0. Epub 2020 Sep 9.

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  3   4 , Sandrine Lemoine  5 , Georges Deschênes  6 , Denis Morin  7 , Christine Vianey-Saban  8 , Cécile Acquaviva-Bourdain  8 , Bruno Ranchin  9 , Justine Bacchetta  9 , Behrouz Kassai  10   3 , Patrice Nony  10   3 , Eurielle Bodénan  10 , Valérie Laudy  10   3 , Cécile Rouges  11 , Setareh Zarrabian  12 , Fabien Subtil  3   4 , Catherine Mercier  3   4 , Pierre Cochat  9 , Aurélia Bertholet-Thomas  9
Affiliations

Adherence to cysteamine in nephropathic cystinosis: A unique electronic monitoring experience for a better understanding. A prospective cohort study: CrYSTobs

Segolene Gaillard et al. Pediatr Nephrol. 2021 Mar.

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. Graphical abstract.

Keywords: Adherence; Cysteamine; Medication event monitoring system; Nephropathic cystinosis.

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