Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham (UAB), Birmingham, Alabama, USA.
Division of Pulmonary and Sleep Medicine, Department of Pediatrics, University of Alabama at Birmingham, Birmingham, Alabama, USA.
Clin Transl Sci. 2023 Apr;16(4):662-672. doi: 10.1111/cts.13479. Epub 2023 Feb 9.
Although major advancements have been made in the therapeutics for people with cystic fibrosis (PwCF), many still require the use of multiple medications to manage acute exacerbations of disease and maintain health. Iterative trial and error processes of pharmacotherapeutic management can be optimized by assessing and incorporating pharmacogenetics. For 82 PwCF, we reviewed 2 years of medication use and response history and interrogated metabolizer status for common pharmacogenes, revealing 3336 medication exposure events (MEEs) to 286 unique medications. As expected, the more frequent MEEs were those commonly used to treat cystic fibrosis (CF), such as antibiotics and respiratory medications. Antibiotics also comprised 56.7% of the undesirable drug responses. The impact of gene variants on drug responses was assessed using Pharmacogenomics Knowledgebase (PharmGKB) and Clinical Pharmacogenetics Implementation Consortium (CPIC) guidelines. Thirty-three (11.5%) medications have strong evidence of genetic influence on response as evidenced by gene-based dosing guidelines. 110 (38.5%) unique medications had at least one association with a very important pharmacogene, whereas 143 (50%) were associated with at least one clinical or variant annotation. Over 97% of participants had at least one actionable genotype. Eleven (13.4%) patients with an actionable genotype, taking the impacted medication, had an undesirable drug response described in the CPIC guidelines that could potentially have been mitigated with a priori knowledge of the genotype. PwCF take many medications for disease management, with frequent dose changes to elicit a desired clinical effect. As CF care evolves, implementing pharmacogenetics testing can improve efficiency and safety of prescribing practices using precision selection and dosing at medication initiation.
尽管囊性纤维化 (PwCF) 患者的治疗方法已经取得了重大进展,但许多人仍需要使用多种药物来治疗疾病的急性加重并维持健康。通过评估和纳入药物遗传学,可以优化药物治疗管理的迭代试验和错误过程。我们回顾了 82 名 PwCF 的药物使用和反应史,并研究了常见药物遗传学的代谢物状态,共发现 3336 次药物暴露事件 (MEE) 和 286 种独特药物。正如预期的那样,更频繁的 MEE 是那些常用于治疗囊性纤维化 (CF) 的药物,如抗生素和呼吸系统药物。抗生素也构成了 56.7%的不良药物反应。使用 Pharmacogenomics Knowledgebase (PharmGKB) 和 Clinical Pharmacogenetics Implementation Consortium (CPIC) 指南评估基因变异对药物反应的影响。33 种 (11.5%)药物具有很强的遗传影响药物反应的证据,这是由基于基因的剂量指南证明的。110 种 (38.5%)独特药物至少有一种与非常重要的药物基因相关联,而 143 种 (50%)药物与至少一种临床或变体注释相关联。超过 97%的参与者至少有一种可操作的基因型。11 名携带可操作基因型的患者 (13.4%)正在服用受影响的药物,出现 CPIC 指南中描述的不良药物反应,这些反应本可以通过预先了解基因型来减轻。PwCF 需要服用许多药物来进行疾病管理,经常调整剂量以产生所需的临床效果。随着 CF 护理的发展,实施药物遗传学测试可以通过在药物开始时进行精确选择和剂量调整来提高处方实践的效率和安全性。