Recent Developments in the Treatment of Pediatric Distal Renal Tubular Acidosis.

Autor: Boyer O; Néphrologie Pédiatrique, Centre de Référence des Maladies Rénales Héréditaires de l'Enfant et l'Adulte (MARHEA), Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (APHP), Institut Imagine, Laboratory of Hereditary Kidney Diseases, INSERM U1163, Université Paris Cité, 149 Rue de Sèvres, 75015, Paris, France. olivia.boyer@aphp.fr., Ould Rabah M; Explorations Fonctionnelles, Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (APHP), Paris, France., Preka E; Néphrologie Pédiatrique, Centre de Référence des Maladies Rénales Héréditaires de l'Enfant et l'Adulte (MARHEA), Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (APHP), Institut Imagine, Laboratory of Hereditary Kidney Diseases, INSERM U1163, Université Paris Cité, 149 Rue de Sèvres, 75015, Paris, France.; INSERM U970, PARCC, Paris Translational Research Centre for Organ, Transplantation, Paris, France.
Jazyk: angličtina
Zdroj: Paediatric drugs [Paediatr Drugs] 2024 Nov; Vol. 26 (6), pp. 649-657. Date of Electronic Publication: 2024 Sep 26.
DOI: 10.1007/s40272-024-00651-9
Abstrakt: Distal renal tubular acidosis (dRTA) is characterized by a primary defect in proton secretion by α-intercalated cells of the collecting duct, leading to impaired urine acidification and resulting in metabolic acidosis, hypokalemia, and hypercalciuria. Inherited forms of dRTA are currently associated with variants in five genes (SLC4A1, ATP6V1B1, ATP6V0A4, FOXI1, and WDR72), each being associated with specific extra-renal manifestations. Acquired forms can result from autoimmune diseases or drug side effects. Classical complications include nephrolithiasis, nephrocalcinosis, reduced glomerular filtration rate (GFR), bone demineralization, and growth failure. Treatment focuses on correcting the acid-base imbalance through alkali supplementation (potassium, sodium, or magnesium bicarbonate or citrate) to reduce renal disease progression and promote normal growth and mineralization. Traditional treatments (alkali and potassium supplementation) often suffer from poor adherence due to frequent day and night administrations, gastrointestinal discomfort, and unpleasant taste. A novel investigational drug, ADV7103, which contains potassium citrate and potassium bicarbonate in an extended-release formulation, has recently been approved by the European Medicine Agency (EMA) for dRTA. Recent studies support its use as a first-line treatment, given its efficacy and safety profile.
(© 2024. The Author(s), under exclusive licence to Springer Nature Switzerland AG.)
Databáze: MEDLINE
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