囊性纤维化儿童和成人在开始接受高效 CFTR 调节剂治疗后维生素和微量元素的变化 - 现实生活的启示。

IF 2.4 Q1 PEDIATRICS
Dorit Fabricius, Tina Knieling, Noelle Zurmuehl, Leandra Makedon, Joachim Freihorst, Hanna Schmidt, Sebastian Bode
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引用次数: 0

摘要

背景:使用依来沙/替扎/伊伐卡夫托(ETI)进行高效的CFTR调节剂治疗可改善囊性纤维化(CF)患者的肺功能、汗液氯化物、体重指数(BMI)和生活质量。有报道称,CFTR调节剂可改善脂溶性维生素和微量营养素的吸收,但缺乏有关ETI疗法的数据:这项单中心回顾性研究评估了符合 ETI 治疗条件的儿童和成人的一秒用力呼气容积(FEV-1)、汗液氯化物、体重指数、转氨酶(AST、ALT)、胆红素、维生素 A、D、E、锌和硒。在开始 ETI 之前和之后一年内对各项参数进行评估:结果:58 名患者(中位年龄 m = 28 岁,SD ± 11.6 岁,51.7% 为女性)14 结论:这项研究为 ETI 后某些脂溶性维生素水平的改善提供了更多证据。微量营养素没有发生变化。建议进行个体化随访和补充。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Changes in vitamins and trace elements after initiation of highly effective CFTR modulator therapy in children and adults with cystic fibrosis - a real-life insight.

Background: Highly-effective CFTR-modulator therapy with elexa-/teza-/ivacaftor (ETI) has led to improvements in pulmonary outcomes, sweat chloride, body mass index (BMI) and quality of life in people with cystic fibrosis (CF). Improved uptake of fat-soluble vitamins and micronutrients has been reported for CFTR-modulators but data regarding ETI therapy is lacking.

Methods: This single-center retrospective study evaluated forced expiratory volume in one second (FEV-1), sweat chloride, BMI, transaminases (AST, ALT), bilirubin, vitamins A, D, E, zinc and selenium in children and adults eligible for ETI. Parameters were assessed before and up to one year after initiation of ETI.

Results: 58 patients (median age m = 28 years, SD ± 11.6 years, 51.7% female14 < 18 years old) were included. FEV-1 and sweat chloride improved significantly after ETI. There were no changes in BMI or AST. ALT was increased significantly after 4 weeks of ETI but returned to normal levels in further course. Bilirubin levels remained elevated after ETI. Vitamin A was significantly higher 12 months after ETI. No changes were found for vitamins D, E, zinc and selenium.

Conclusions: This study adds to the evidence that improvements of some fat-soluble vitamin levels can be found after ETI. No changes regarding micronutrients were noted. Individualized follow-up and supplementation are recommended.

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