Papadakis Lena, Stander Tayler, Mombourquette Jacqueline, Richards Christopher J, Yonker Lael M, Lawton Brenden, Hardcastle Margot, Zweifach Julia, Sicilian Leonard, Bringhurst Lindsay, Neuringer Isabel P
Pulmonary Division, Massachusetts General Hospital for Children, MGH Cystic Fibrosis Center, Boston, Massachusetts, USA.
Pulmonary Division, Massachusetts General Hospital, MGH Cystic Fibrosis center, Boston, Massachusetts, USA.
Pediatr Pulmonol. 2025 Jan;60(1):e27382. doi: 10.1002/ppul.27382. Epub 2024 Nov 6.
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.
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.
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.
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.
依列卡福妥-替扎卡福妥-依伐卡福妥（ETI）疗法对囊性纤维化患者（pwCF）健康和福祉的益处已有充分记录。然而，自获批以来，临床实践报告中出现了越来越多潜在的副作用。鉴于目前对ETI耐受性的了解仅限于临床试验数据，副作用的发生率及其对治疗决策的影响仍分类不清。
设计了一项包含10个问题的调查问卷，并分发给在麻省总医院囊性纤维化中心接受治疗的18岁及以上患者。对12个不同类别的副作用报告进行了统计，并收集了因副作用而进行的剂量调整和停药情况。如果患者报告无副作用，则无需完成整个调查问卷。
在92名开始接受ETI治疗的受访者中，51名受访者（55.4%）报告了潜在副作用，41名受访者（44.5%）报告无不良事件。最常报告的副作用是心理健康问题、外貌变化和胃肠道不适，分别有22.8%、30.4%和21.7%的患者报告。18名受访者（19.6%）因副作用调整了剂量，6名受访者（6.52%）因持续副作用永久停药。
调查结果显示出明显的异质性，大多数受访者在开始治疗后报告了至少一种副作用。剂量调整常用于减轻不良反应，但很少有受访者不得不停药。这些发现表明在临床环境中监测ETI潜在药物相关副作用的重要性。
