Heterogeneity in Reported Side Effects Following Initiation of Elexacaftor-Tezacaftor-Ivacaftor: Experiences at a Quaternary CF Care Center.

IF 2.7 3区 医学 Q1 PEDIATRICS
Lena Papadakis, Tayler Stander, Jacqueline Mombourquette, Christopher J Richards, Lael M Yonker, Brenden Lawton, Margot Hardcastle, Julia Zweifach, Leonard Sicilian, Lindsay Bringhurst, Isabel P Neuringer
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Abstract

Background: The benefits of Elexecaftor-Tezacaftor-Ivacaftor (ETI) therapy on the health and wellbeing of people with CF (pwCF) are well documented. Since approval, however, a growing number of potential side effects have emerged in reports from clinical practice. With current understanding of ETI tolerability limited to data from clinical trials, the prevalence of side effects and their impact on care decision making remains poorly categorized.

Methods: A 10-question survey was developed and distributed to patients 18 years or older who were treated at the Massachusetts General Hospital CF centers. Reports of side effects were measured across 12 distinct categories, and dose adjustments and discontinuation due to side effects were collected. If a patient reported no side effects, they did not have to complete the entire survey.

Results: Among 92 respondents initiated on ETI, 51 respondents (55.4%) reported potential side effects and 41 (44.5%) respondents reported no adverse events. The most commonly reported side effects were mental health, changes in appearance, and gastrointestinal complaints, which were reported by 22.8%, 30.4%, and 21.7% of patients, respectively. Eighteen (19.6%) respondents modified their dosing in response to side effects, and six discontinued treatment permanently (6.52%) due to persistent side effects.

Conclusions: Responses demonstrated marked heterogeneity, with most respondents reporting at least one side effect following initiation. Dose modification was commonly utilized to mitigate adverse effects, however few respondents had to discontinue treatment. These findings demonstrate the importance of monitoring for potential drug-related side effects of ETI in clinical settings.

开始使用 Elexacaftor-Tezacaftor-Ivacaftor 后报告副作用的异质性:一家四级 CF 护理中心的经验。
背景:Elexecaftor-Tezacaftor-Ivacaftor(ETI)疗法对 CF 患者(pwCF)的健康和福祉的益处有据可查。然而,自获得批准以来,临床实践报告中出现了越来越多的潜在副作用。由于目前对 ETI 耐受性的了解仅限于临床试验数据,因此副作用的发生率及其对护理决策的影响仍未得到很好的分类:方法:我们编制了一份包含 10 个问题的调查问卷,并分发给在麻省总医院 CF 中心接受治疗的 18 岁及以上患者。对 12 个不同类别的副作用报告进行了测量,并收集了因副作用导致的剂量调整和停药情况。如果患者报告没有副作用,则无需完成整个调查:在 92 名开始使用 ETI 的受访者中,51 名受访者(55.4%)报告了潜在的副作用,41 名受访者(44.5%)报告未出现不良事件。最常报告的副作用是精神健康、外观变化和胃肠道不适,分别有 22.8%、30.4% 和 21.7% 的患者报告了这些副作用。18名受访者(19.6%)因副作用而改变了用药剂量,6名受访者(6.52%)因持续副作用而永久停止治疗:结论:受访者的回答具有明显的异质性,大多数受访者在开始治疗后都报告了至少一种副作用。为减轻不良反应,通常会调整剂量,但很少有受访者不得不中断治疗。这些发现表明,在临床环境中监测 ETI 潜在的药物相关副作用非常重要。
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来源期刊
Pediatric Pulmonology
Pediatric Pulmonology 医学-呼吸系统
CiteScore
6.00
自引率
12.90%
发文量
468
审稿时长
3-8 weeks
期刊介绍: Pediatric Pulmonology (PPUL) is the foremost global journal studying the respiratory system in disease and in health as it develops from intrauterine life though adolescence to adulthood. Combining explicit and informative analysis of clinical as well as basic scientific research, PPUL provides a look at the many facets of respiratory system disorders in infants and children, ranging from pathological anatomy, developmental issues, and pathophysiology to infectious disease, asthma, cystic fibrosis, and airborne toxins. Focused attention is given to the reporting of diagnostic and therapeutic methods for neonates, preschool children, and adolescents, the enduring effects of childhood respiratory diseases, and newly described infectious diseases. PPUL concentrates on subject matters of crucial interest to specialists preparing for the Pediatric Subspecialty Examinations in the United States and other countries. With its attentive coverage and extensive clinical data, this journal is a principle source for pediatricians in practice and in training and a must have for all pediatric pulmonologists.
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