Kibleur Yves, Guffon Nathalie
Lucane Pharma, 172 rue de Charonne, 75011, Paris, France.
Hôpital Femme-Mère-Enfant, Centre de Référence des maladies héréditaires du métabolisme, 59 Boulevard Pinel, 69577, Bron Cedex, France.
Paediatr Drugs. 2016 Apr;18(2):139-44. doi: 10.1007/s40272-015-0159-8.
The aim was to describe the status of patients with urea cycle disorders (UCD) at the latest long-term clinical follow-up of treatment with a new taste-masked formulation of sodium phenylbutyrate (NaPB) granules (Pheburane). These patients are a subset of those treated under a cohort temporary utilisation study (ATU) previously reported and now followed for 2 years.
From a French cohort temporary utilization authorization (ATU) set up to monitor the use of Pheburane on a named-patient basis in UCD patients in advance of its marketing authorization, a subset of patients were followed up in the long term. Data on demographics, dosing characteristics of NaPB, concomitant medications, adverse events and clinical outcomes were collected at a follow-up visit after 1-2 years of treatment with the drug administered under marketing conditions. This paper reports on the subset of patients who were included in further long-term follow-up at the principal recruiting metabolic reference center involved in the original cohort.
No episode of metabolic decompensation was observed over a treatment period ranging from 8 to 30 months with Pheburane, and the range of ammonia and glutamine levels continued to improve and remained within the normal range, thus adding valuable longer-term feedback to the original ATU report. In all, no adverse events were reported with Pheburane treatment. These additional data demonstrate the maintenance of the safety and efficacy of Pheburane over time.
The recently developed taste-masked formulation of NaPB granules (Pheburane) improved the quality of life for UCD patients. The present post-marketing report on the use of the product confirms the original observations of improved compliance, efficacy and safety with this taste-masked formulation of NaPB.
本研究旨在描述尿素循环障碍（UCD）患者在接受新型掩味苯丁酸钠颗粒（Pheburane）治疗的最新长期临床随访中的状况。这些患者是先前报告的队列临时使用研究（ATU）中接受治疗患者的一个子集，目前已随访2年。
在法国，为在Pheburane上市许可前对UCD患者进行按患者命名的使用监测而设立了队列临时使用授权（ATU），其中一部分患者进行了长期随访。在按照上市条件给药治疗1 - 2年后的随访中，收集了有关人口统计学、苯丁酸钠的给药特征、伴随用药、不良事件和临床结局的数据。本文报告了在最初队列研究的主要招募代谢参考中心纳入进一步长期随访的患者子集情况。
使用Pheburane治疗8至30个月期间未观察到代谢失代偿事件，氨和谷氨酰胺水平范围持续改善并维持在正常范围内，从而为原始ATU报告增添了宝贵的长期反馈信息。总体而言，Pheburane治疗未报告不良事件。这些额外数据证明了Pheburane随时间推移安全性和有效性的维持。
最近开发的掩味苯丁酸钠颗粒（Pheburane）改善了UCD患者的生活质量。本产品上市后报告证实了最初关于这种掩味苯丁酸钠制剂改善依从性、有效性和安全性的观察结果。
