Pediatric Unit, Provincial Centre for Rare Diseases, Department of Women's and Children's Health, "S. Chiara" Hospital, Azienda Provinciale per i Servizi Sanitari, Largo Medaglie d'oro 9, 38122, Trento, Italy.
Hospital Pharmacy Unit, Azienda Provinciale per i Servizi Sanitari, Largo Medaglie d'oro 9, Trento, Italy.
AAPS PharmSciTech. 2020 Mar 9;21(3):100. doi: 10.1208/s12249-020-01642-y.
Sodium benzoate, a common food preservative, is used in the treatment of patients with urea cycle disorders (UCDs) as it stimulates ammonia removal by a non-urea cycle-based pathway. Despite its use in the clinical routine, no commercially available oral formulations currently exist. Liquid formulation is normally well accepted in pediatric age and allows precise dosage according to the children's needs.
(1) To prepare an oral sodium benzoate solution in different tastes and determine its stability, palatability, and tolerability and (2) to describe the long-term follow-up of two pediatric patients with UCDs treated with our formulation.
We prepared five oral solutions of sodium benzoate (200 mg/ml) by adding different flavoring agents. We measured drug concentration in the samples by high-performance liquid chromatography (HPLC). We evaluated palatability and tolerability with adult volunteers. Long-term drug compliance and metabolic control were appraised in two pediatric patients.
All the oral solutions remained stable at room temperature along the 96-day test period, and they were well tolerated. The mint-flavored solution resulted the most palatable and preferred by adult volunteers. We report good drug compliance and good metabolic outcomes for both pediatric patients during the entire follow-up.
Our study highlighted the stability and tolerability of flavored sodium benzoate oral solutions. These solutions were well accepted during a long-term follow-up and guaranteed a good metabolic control. Since taste attributes are critical to ensure acceptable medication adherence in the pediatric age, flavored liquid formulations of sodium benzoate may be an efficient strategy to achieve therapeutic outcomes in UCD pediatric patients.
苯甲酸钠是一种常见的食品防腐剂，用于治疗尿素循环障碍（UCD）患者，因为它可以通过非尿素循环途径刺激氨的清除。尽管它在临床常规中使用，但目前尚无市售的口服制剂。液体制剂通常在儿科中被很好地接受，并允许根据儿童的需要精确给药。
（1）制备不同口味的口服苯甲酸钠溶液，并确定其稳定性、可接受性和耐受性；（2）描述我们的制剂治疗的两名 UCD 儿科患者的长期随访结果。
我们通过添加不同的调味剂制备了五种苯甲酸钠（200mg/ml）的口服溶液。我们通过高效液相色谱法（HPLC）测量样品中的药物浓度。我们用成人志愿者评估可接受性和耐受性。我们评估了两名儿科患者的长期药物依从性和代谢控制情况。
在 96 天的测试期内，所有口服溶液在室温下均保持稳定，且耐受性良好。薄荷味溶液最可口，最受成人志愿者欢迎。我们报告了两名儿科患者在整个随访期间的良好药物依从性和良好的代谢结果。
我们的研究强调了调味苯甲酸钠口服溶液的稳定性和耐受性。这些溶液在长期随访期间被很好地接受，并保证了良好的代谢控制。由于口味特征对于确保儿科患者接受可接受的药物治疗至关重要，因此苯甲酸钠的调味液体制剂可能是在 UCD 儿科患者中实现治疗效果的有效策略。
