Abstract
Introduction: Rare diseases are those with a prevalence of 1 in 2,000. These include urea cycle disorders (UCDs) and primary distal tubular metabolic acidosis (DTA). Their treatment relies on the use of oral sodium benzoate and sodium bicarbonate, respectively. The aim of this study was to formulate capsules of sodium benzoate and sodium bicarbonate of 250 mg and to demonstrate their role in the treatment of these rare diseases. Materials and Methods: A retrospective descriptive study was carried out over a 3-year period evaluating requests for compounded medicines. Formulation and quality control of sodium benzoate and sodium bicarbonate capsules dosed at 250 mg were performed according to Good Hospital Preparation Practices. Treatment efficacy and adverse effects were evaluated. Results and Discussion: A total of 10106 drugs were compounded during the study period, including 325 preparations based on drugs for metabolic disorders, i.e. 3.22% of all preparations. Sodium benzoate accounted for 12.31% and sodium bicarbonate for 10.15% of these preparations for metabolic disorders. The capsules were formulated with a consistent appearance, weight uniformity and drug content with a stability of 3 months. Treatment efficacy was followed in a population of 20 patients, 11 of whom completed the questionnaire. A male predominance was observed in patients with UCD and primary DTA, with the majority of patients with UCD and primary DTA having onset of symptoms before 1 year. Treatment efficacy was observed in 9 patients with no adverse events reported. Conclusions: The present study confirms the stability and efficacy of compounded medicines based on sodium benzoate and sodium bicarbonate in the treatment of UCD and DTA, making them an excellent alternative for their treatment.
Published Version
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