PDF(Pubmed)
Abstract：
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.
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.
摘要：
背景：使用elexa-/teza-/ivacaftor（ETI）进行高效CFTR调节剂治疗可改善肺部预后，汗液氯化物，囊性纤维化（CF）患者的体重指数（BMI）和生活质量。据报道，CFTR调节剂对脂溶性维生素和微量营养素的吸收有所改善，但缺乏有关ETI治疗的数据。
方法：这项单中心回顾性研究评估了一秒用力呼气量（FEV-1），汗液氯化物，BMI,转氨酶（AST，ALT),胆红素,维生素A,D,E,符合ETI条件的儿童和成人的锌和硒。在ETI开始之前和之后一年内评估参数。
结果：58例患者（中位年龄m=28岁，SD±11.6年，包括51.7%的女性14<18岁)。ETI后FEV-1和汗液氯化物明显改善。BMI或AST没有变化。ETI4周后ALT显著升高,但在进一步的过程中恢复至正常水平。ETI后胆红素水平仍然升高。维生素A在ETI后12个月显著升高。没有发现维生素D的变化，E,锌和硒。
结论：这项研究增加了证据，证明ETI后可以发现一些脂溶性维生素水平的改善。没有发现微量营养素方面的变化。建议进行个性化随访和补充。
方法：这项单中心回顾性研究评估了一秒用力呼气量（FEV-1），汗液氯化物，BMI,转氨酶（AST，ALT),胆红素,维生素A,D,E,符合ETI条件的儿童和成人的锌和硒。在ETI开始之前和之后一年内评估参数。
结果：58例患者（中位年龄m=28岁，SD±11.6年，包括51.7%的女性14<18岁)。ETI后FEV-1和汗液氯化物明显改善。BMI或AST没有变化。ETI4周后ALT显著升高,但在进一步的过程中恢复至正常水平。ETI后胆红素水平仍然升高。维生素A在ETI后12个月显著升高。没有发现维生素D的变化，E,锌和硒。
结论：这项研究增加了证据，证明ETI后可以发现一些脂溶性维生素水平的改善。没有发现微量营养素方面的变化。建议进行个性化随访和补充。
