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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.
Bertholet-Thomas, Aurélia; Guittet, Catherine; Manso-Silván, Maria A; Joukoff, Sophie; Navas-Serrano, Victor; Baudouin, Véronique; Cailliez, Mathilde; Di Maio, Massimo; Gillion-Boyer, Olivia; Golubovic, Emilija; Harambat, Jérôme; Knebelmann, Bertrand; Nobili, François; Novo, Robert; Podracka, Ludmila; Roussey-Kesler, Gwenaëlle; Granier, Luc-André.
Afiliación
  • Bertholet-Thomas A; Centre de Référence des Maladies Rénales Rares-Néphrogones-Hôpital Femme Mère Enfant, Hospices Civils de Lyon-Filière ORKiD, Bron, France. aurelia.bertholet-thomas@chu-lyon.fr.
  • Guittet C; Advicenne, Nîmes, France.
  • Manso-Silván MA; Advicenne, Nîmes, France.
  • Joukoff S; Advicenne, Nîmes, France.
  • Navas-Serrano V; Advicenne, Nîmes, France.
  • Baudouin V; Service de Néphrologie Pédiatrique, Hôpital Robert Debré, Paris, France.
  • Cailliez M; Service de Pédiatrie Multidisciplinaire, Hôpital de la Timone, AP-HM, Marseille, France.
  • Di Maio M; Service de Réanimation Néonatale et Néonatologie, CHU de Nîmes, Nîmes, France.
  • Gillion-Boyer O; Service de Néphrologie Pédiatrique, Centre de Référence des Maladies Rénales Héréditaires de l'Enfant et de l'Adulte (MARHEA), Institut Imagine, Hôpital Necker-Enfants Malades, Université de Paris, Paris, France.
  • Golubovic E; Klinika za decije interne bolesti-Odeljenje za nefrologiju, Klinicki Centar Nis, Nis, Serbia.
  • Harambat J; Service de Pédiatrie, Centre de Référence Maladies Rénales Rares du Sud-ouest (SoRare), CHU de Bordeaux, Hôpital Pellegrin-Enfants, Bordeaux, France.
  • Knebelmann B; Service de Néphrologie adultes Hôpital Necker, Paris, France.
  • Nobili F; Service de Pédiatrie 2, Hôpital Jean Minjoz, CHU de Besançon, Besançon, France.
  • Novo R; Service de Néphrologie Pédiatrique, Hôpital Jeanne de Flandre, CHRU de Lille, Lille, France.
  • Podracka L; Department of Pediatrics, National Institute of Children's Health, Bratislava, Slovakia.
  • Roussey-Kesler G; Unité de Néphrologie et Hémodialyse Pédiatrique, Clinique Médicale Pédiatrique Hôpital Mère-Enfant, CHU de Nantes, Nantes, France.
  • Granier LA; Advicenne, Nîmes, France.
Pediatr Nephrol ; 36(7): 1765-1774, 2021 07.
Article en En | MEDLINE | ID: mdl-33635379
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.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Acidosis Tubular Renal / Bicarbonatos / Compuestos de Potasio / Citrato de Potasio Aspecto: Patient_preference Límite: Adult / Child / Humans Idioma: En Revista: Pediatr Nephrol Asunto de la revista: NEFROLOGIA / PEDIATRIA Año: 2021 Tipo del documento: Article País de afiliación: Francia Pais de publicación: Alemania

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Acidosis Tubular Renal / Bicarbonatos / Compuestos de Potasio / Citrato de Potasio Aspecto: Patient_preference Límite: Adult / Child / Humans Idioma: En Revista: Pediatr Nephrol Asunto de la revista: NEFROLOGIA / PEDIATRIA Año: 2021 Tipo del documento: Article País de afiliación: Francia Pais de publicación: Alemania