Sandritter Tracy L, Dinh Jean C, Wagner Jennifer A, Lowry Jennifer A
Division of Clinical Pharmacology/Medical Toxicology and Therapeutic Innovation, Children's Mercy Hospital, 2401 Gillham Road, Kansas City, MO 64108, USA.
Children (Basel). 2019 Feb 25;6(2):35. doi: 10.3390/children6020035.
The GOLDILOKs (Genomic and Ontogeny-Linked Dose Individualization and cLinical Optimization for KidS) Clinic aims to provide families and physicians with data to make more informed decisions with regard to pharmacological therapy by using innovative therapy and genomic technologies. The objectives are two-fold: (1) To describe the utility of the GOLDILOKs Clinic to referring prescribers by evaluating the type of referrals made to the GOLDILOKs Clinic and (2) to assess the most often utilized technologies (e.g., genotyping) completed to formulate therapy recommendations. Patient data from July 2010 to June 2016 was retrospectively reviewed following Institutional Review Board (IRB) approval. The GOLDILOKs Clinic evaluated 306 patients and had increases in annual referrals from 14 in 2010⁻2011 to 84 in 2016⁻2017. The children that were referred were predominately Caucasian (82%) and male (59%) with an average age of 12.4 ± 5.9 years. Subspecialty versus primary care referrals accounted for 82% and 18% of referrals, respectively. Adverse drug reactions (n = 166) and poor medication response (n = 179) were the major reasons for referral. However, it must be noted that patients could have multiple reasons for referral. Pharmacogenetic results were extensively used to provide guidance for future therapy in patients with medication-related problems. Genotyping of drug metabolizing enzymes and drug target receptors was performed in 221 patients (72.2%). Recommendations were fully accepted by 63% and partially accepted by 22% of internal provider referrals.
GOLDILOKs(儿童基因组与个体发育关联剂量个体化及临床优化)诊所旨在通过运用创新疗法和基因组技术,为家庭和医生提供数据,以便在药物治疗方面做出更明智的决策。其目标有两个:(1)通过评估转介至GOLDILOKs诊所的转诊类型,描述该诊所对转诊开方者的效用;(2)评估为制定治疗建议而完成的最常用技术(如基因分型)。在获得机构审查委员会(IRB)批准后,对2010年7月至2016年6月的患者数据进行了回顾性审查。GOLDILOKs诊所评估了306名患者,年度转诊量从2010 - 2011年的14例增加到2016 - 2017年的84例。转诊的儿童以白种人为主(占82%),男性居多(占59%),平均年龄为12.4±5.9岁。专科转诊与初级保健转诊分别占转诊的82%和18%。药物不良反应(n = 166)和药物反应不佳(n = 179)是转诊的主要原因。然而,必须指出的是,患者可能有多种转诊原因。药物遗传学结果被广泛用于为有药物相关问题的患者提供未来治疗指导。221名患者(72.2%)进行了药物代谢酶和药物靶受体的基因分型。内部提供者转诊中有63%的建议被完全接受,22%被部分接受。