ESPN 54th Annual Meeting

ESPN 2022


 
Switching from immediate to extended release cysteamine in patients with nephropathic cystinosis in Spain: from clinical trials to clinical practice
GEMA ARICETA 1 Fernando Santos 2 Andrés Lopez Muñiz 3 Alvaro Hermida 4 Maria Luisa Matoses 5 Ana Ventura 6 Paloma Leticia Martín-Moreno 7 Esther González 8 Julia Vara 9

1- PEDIATRIC NEPHROLOGY. HOSPITAL UNIVERSITARI VALL D HEBRON. BARCELONA
2- Paediatric Nephrology. Hospital Universitario Central de Asturias. University of Oviedo
3- Nephrology department. Complejo Hospitalario Universitario a Coruña
4- Internal Medicine Service. Hospital Clínico Universitario de Santiago de Compostela
5- Paediatric nephrology department. Hospital Universitario La Fe (Valencia)
6- Nephrology department. Hospital Universitario La Fe (Valencia)
7- Department of Nephrology, Clinica Universidad de Navarra, Navarra Institute for Health Research (IdiSNA) (Pamplona)
8- Nephrology department. Hospital 12 de Octubre (Madrid)
9- Paediatric Nephrology department. Hospital 12 de Octubre (Madrid)
 
Introduction:

Objectives: The objective of RELUCIR study was to evaluate, under clinical practice conditions, the effectiveness and safety of switching from immediate-release (IR) to extended-release (ER) cysteamine in patients with nephropathic cystinosis (NC) in Spain.

Material and methods:

Observational, retrospective multicentre study in patients with NC of any age, that had received IR cysteamine for at least 12 months, had switched to ER cysteamine, and had been receiving ER cysteamine for at least 6 months prior to inclusion into the study.

Results:

A total of 9 patients, 4 children and 5 adults, 10.1-34.4 years old, were included. All 5 adult patients and one adolescent had received a kidney transplant (3 in one patient). Despite individual variations, no significant differences in grouped WBC (white blood cells) cystine levels were observed after the switch. In patients with preserved kidney function, eGFR remained stable after the switch. There was no significant difference in the cysteamine dose received before and after the switch. However, we observed that some patients were receiving <50% of recommended doses of cysteamine and showed elevated levels of WBC cystine. A significant improvement in height, weight and corresponding Z scores after the switch was observed, particularly in paediatric patients (p<0.05). There was a trend towards reduction in the number of hospitalizations and a significant reduction in the hospitalizations stays (days of hospitalization; p<0,0001) after the switch to ER-cysteamine, especially in those patients who were admitted due to medication or to the disease itself. A reduction in the appearance of halitosis, body odour and gastrointestinal effects, as well as PPI use were observed after the change to ER-cysteamine in some of the patients.

Conclusions:

Switching from IR to ER cysteamine in clinical practice might help to improve tolerability and growth in children with NC and to reduce hospitalization stays.