Segolene Gaillard1,2, Laurent Roche3,4, Sandrine Lemoine5, Georges Deschênes6, Denis Morin7, Christine Vianey-Saban8, Cécile Acquaviva-Bourdain8, Bruno Ranchin9, Justine Bacchetta9, Behrouz Kassai10,3, Patrice Nony10,3, Eurielle Bodénan10, Valérie Laudy10,3, Cécile Rouges11, Setareh Zarrabian12, Fabien Subtil3,4, Catherine Mercier3,4, Pierre Cochat9, Aurélia Bertholet-Thomas9. 1. Hospices Civils de Lyon, EPICIME-CIC 1407 de Lyon, Inserm, Department of Clinical Epidemiology, CHU-Lyon, F-69677, Bron, France. segolene.gaillard@chu-lyon.fr. 2. Université de Lyon, F-69000, Lyon, Université Lyon 1; CNRS, UMR 5558, Laboratoire de Biométrie et Biologie Evolutive, F-69622, Villeurbanne, France. segolene.gaillard@chu-lyon.fr. 3. Université de Lyon, F-69000, Lyon, Université Lyon 1; CNRS, UMR 5558, Laboratoire de Biométrie et Biologie Evolutive, F-69622, Villeurbanne, France. 4. Hospices Civils de Lyon, Service de Biostatistique, F-69324, Lyon, France. 5. Service de Néphrologie, Dialyse, Hypertension artérielle, Hôpital Edouard Herriot, Hospices Civils de Lyon, Université de Lyon, Lyon, France. 6. APHP, Hôpital Robert Debré, Service de néphrologie pédiatrique, Paris, France. 7. CHU Montpellier, Service de néphrologie et endocrinologie pédiatrique, Montpellier, France. 8. Hospices Civils de Lyon, Service Biochimie et Biologie Moléculaire, UF Maladies Héréditaires du Métabolisme, F-69500, Bron, France. 9. Hospices Civils de Lyon, Service de Néphrologie Pédiatrique, et centre de référence maladies rénales et phosphocalciques rares- Néphrogones- Filière ORKiD -69500, Bron, France. 10. Hospices Civils de Lyon, EPICIME-CIC 1407 de Lyon, Inserm, Department of Clinical Epidemiology, CHU-Lyon, F-69677, Bron, France. 11. CHU Montpellier, Centre d'Investigation Clinique, Inserm CIC 1411, F-69500, Bron, Montpellier, France. 12. Centre d'Investigation Clinique - CIC 1426 Hôpital Robert Debre - Assistance Publique - Hopitaux de Paris (AP-HP), Paris, France.
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.
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.