Recent Developments in the Treatment of Pediatric Distal Renal Tubular Acidosis.
Journal
Paediatric drugs
ISSN: 1179-2019
Titre abrégé: Paediatr Drugs
Pays: Switzerland
ID NLM: 100883685
Informations de publication
Date de publication:
26 Sep 2024
26 Sep 2024
Historique:
accepted:
19
08
2024
medline:
26
9
2024
pubmed:
26
9
2024
entrez:
26
9
2024
Statut:
aheadofprint
Résumé
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.
Identifiants
pubmed: 39325135
doi: 10.1007/s40272-024-00651-9
pii: 10.1007/s40272-024-00651-9
doi:
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Informations de copyright
© 2024. The Author(s), under exclusive licence to Springer Nature Switzerland AG.
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