Safety, efficacy, and acceptability of ADV7103 during 24 months of treatment: an open-label study in pediatric and adult patients with distal renal tubular acidosis

Autor: Olivia Gillion-Boyer, Sophie Joukoff, François Nobili, Massimo Di Maio, Mathilde Cailliez, Aurélia Bertholet-Thomas, Catherine Guittet, Véronique Baudouin, Robert Novo, Maria A. Manso-Silván, Jérôme Harambat, Victor Navas-Serrano, Gwenaëlle Roussey-Kesler, Ludmila Podracka, Bertrand Knebelmann, Luc-André Granier, Emilija Golubovic
Přispěvatelé: Hôpital Femme Mère Enfant [CHU - HCL] (HFME), Hospices Civils de Lyon (HCL), Hôpital Robert Debré, Hôpital de la Timone [CHU - APHM] (TIMONE), Centre Hospitalier Universitaire de Nîmes (CHU Nîmes), Imagine - Institut des maladies génétiques (IHU) (Imagine - U1163), Institut National de la Santé et de la Recherche Médicale (INSERM)-Université de Paris (UP), Bordeaux population health (BPH), Université de Bordeaux (UB)-Institut de Santé Publique, d'Épidémiologie et de Développement (ISPED)-Institut National de la Santé et de la Recherche Médicale (INSERM), Hôpital Pellegrin, CHU Bordeaux [Bordeaux]-Groupe hospitalier Pellegrin
Jazyk: angličtina
Rok vydání: 2021
Předmět:
Nephrology
Adult
medicine.medical_specialty
Potassium Compounds
030232 urology & nephrology
[SDV.MHEP.UN]Life Sciences [q-bio]/Human health and pathology/Urology and Nephrology
Potassium bicarbonate
03 medical and health sciences
chemistry.chemical_compound
0302 clinical medicine
Patient satisfaction
Acceptability
Distal renal tubular acidosis
Internal medicine
Potassium Citrate
Medicine
Humans
030212 general & internal medicine
Adverse effect
Child
business.industry
dRTA
Acidosis
Renal Tubular
Adherence to treatment
medicine.disease
3. Good health
Discontinuation
Bicarbonates
Tolerability
chemistry
Pediatrics
Perinatology and Child Health
Cohort
Plasma bicarbonate
Potassium
Quality of Life
[SDV.SP.PHARMA]Life Sciences [q-bio]/Pharmaceutical sciences/Pharmacology
Original Article
Safety
business
Zdroj: Pediatric Nephrology
Pediatric Nephrology, Springer Verlag, In press, ⟨10.1007/s00467-020-04873-0⟩
Pediatric Nephrology (Berlin, Germany)
ISSN: 0931-041X
1432-198X
Popis: Background A new prolonged-release formulation of potassium citrate and potassium bicarbonate, ADV7103, has been shown to improve metabolic control, palatability, and gastrointestinal safety in patients with distal renal tubular acidosis (dRTA) when compared to standard of care (SoC) treatments. The present work evaluates safety and efficacy of ADV7103 during 24 months. Methods Thirty pediatric and adult patients were included in an open-label extension study after a phase II/III trial. Safety and tolerability were assessed. Plasma bicarbonate and potassium levels, as well as urine parameters, were evaluated over time. Acceptability, adherence, and quality of life were also assessed. The evolution of clinical consequences of dRTA in the cohort was explored. Results There were 104 adverse events (AEs) reported, but only 9 gastrointestinal events observed in five patients (17%) were considered to be related to ADV7103 treatment. There were no AEs leading to treatment discontinuation. Plasma bicarbonate and potassium levels were in the normal ranges at the different visits, respectively, in 69–86% and 83–93% of patients. Overall adherence rates were ≥ 75% throughout the whole study in 79% patients. An average improvement of quality of life of 89% was reported at 24 months of study. Conclusions Common AEs concerned metabolism and gastrointestinal disorders; the former being related to the disease. Less than half of the gastrointestinal AEs were related to ADV7103 treatment and they were mostly mild in severity. Metabolic parameters were maintained in the normal ranges in most patients. Patient satisfaction was high and adherence to treatment was good and remained stable. Trial registration number Registered as EudraCT 2013-003828-36 on the 3rd of September 2013. Graphical Abstract
Databáze: OpenAIRE
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