1. Adherence to cysteamine in nephropathic cystinosis: A unique electronic monitoring experience for a better understanding. A prospective cohort study: CrYSTobs.
- Author
-
Gaillard, Segolene, Roche, Laurent, Lemoine, Sandrine, Deschênes, Georges, Morin, Denis, Vianey-Saban, Christine, Acquaviva-Bourdain, Cécile, Ranchin, Bruno, Bacchetta, Justine, Kassai, Behrouz, Nony, Patrice, Bodénan, Eurielle, Laudy, Valérie, Rouges, Cécile, Zarrabian, Setareh, Subtil, Fabien, Mercier, Catherine, Cochat, Pierre, and Bertholet-Thomas, Aurélia
- Subjects
- *
AMINES , *DRUG monitoring , *DRUGS , *HEALTH care teams , *HEALTH education , *INTEGRATED health care delivery , *KIDNEY diseases , *LONGITUDINAL method , *MEDICAL care , *INBORN errors of metabolism , *PATIENT compliance , *DISEASE progression , *INDIVIDUALIZED medicine , *DESCRIPTIVE statistics - 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 [ABSTRACT FROM AUTHOR]
- Published
- 2021
- Full Text
- View/download PDF