Institute for Experimental Paediatric Endocrinology, Charité Berlin, Berlin, Germany.
Department of Clinical Pharmacy and Biochemistry, Institute of Pharmacy, Freie Universität Berlin, Berlin, Germany.
Eur J Endocrinol. 2017 Aug;177(2):239-242. doi: 10.1530/EJE-17-0248. Epub 2017 May 16.
Due to the lack of paediatric-licensed formulations, children are often treated with individualized pharmacy-compounded adult medication. An international web-based survey about the types of medication in children with adrenal insufficiency (AI) revealed that the majority of paediatric physicians are using pharmacy-compounded medication to treat children with AI. Observations of loss of therapy control in children with congenital adrenal hyperplasia with compounded hydrocortisone capsules and regained control after prescribing a new hydrocortisone batch led to this 'real world' evaluation of pharmacy-compounded paediatric hydrocortisone capsules.
Capsule samples were collected randomly from volunteering parents of treated children suffering from congenital adrenal hyperplasia from all over Germany. Analysis of net mass and hydrocortisone content by high-performance liquid chromatography with ultraviolet (HPLC-UV) detection method was performed based on the European Pharmacopeia.
In a total of 61 batches that were sent, 5 batches could not be analysed because of missing dose information, insufficient number of capsules or were not possible to be evaluated. Fifty-six batches containing 1125 capsules were evaluated. 21.4% of the batches revealed insufficiency in uniformity of net mass or drug content and additional 3.6% failed because they did not contain the labelled drug.
Compounded medication is a possible cause of variation of steroid doses in children with adrenal insufficiency or congenital adrenal hyperplasia, putting these vulnerable patients at risk of poor disease control and adrenal crisis. These data may apply to other individualized compounded oral medication as well, emphasizing the need for development of licensed paediatric formulations approved by regulatory authorities.
由于缺乏儿科许可制剂，儿童通常使用个体化药剂配制的成人药物进行治疗。一项关于肾上腺功能减退症（AI）患儿用药类型的国际网络调查显示，大多数儿科医生正在使用药剂配制药物来治疗 AI 患儿。在使用复合氢化可的松胶囊治疗先天性肾上腺增生症的儿童中，观察到治疗失控的情况，在开出处方的新一批氢化可的松后，这种情况得到了控制，这导致了对药剂配制儿科氢化可的松胶囊的“真实世界”评估。
从德国各地接受治疗的先天性肾上腺增生症患儿的治疗父母中随机收集胶囊样本。根据欧洲药典，采用高效液相色谱法-紫外检测法（HPLC-UV）对净质量和氢化可的松含量进行分析。
共寄出 61 批，因剂量信息缺失、胶囊数量不足或无法评估而无法分析 5 批。评估了含有 1125 粒胶囊的 56 批。21.4%的批次在净质量或药物含量均匀度方面存在不足，另外 3.6%的批次因不含有标签药物而失败。
配制药物可能是肾上腺功能减退症或先天性肾上腺增生症患儿类固醇剂量变化的原因，使这些脆弱的患者面临疾病控制不良和肾上腺危象的风险。这些数据可能也适用于其他个体化的配制口服药物，强调需要开发经监管机构批准的儿科许可制剂。
