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Elexacaftor/Tezacaftor/Ivacaftor therapy in cystic fibrosis children previously CFSPID: Is it over-medicalization?

作者信息

Terlizzi V

机构信息

Department of Pediatric Medicine, Meyer Children's Hospital IRCCS, Cystic Fibrosis Regional Reference Center, Florence, Italy.

出版信息

J Cyst Fibros. 2024 Mar;23(2):366-367. doi: 10.1016/j.jcf.2023.10.008. Epub 2023 Oct 13.

DOI:10.1016/j.jcf.2023.10.008
PMID:37838487
Abstract
摘要

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1
Elexacaftor/Tezacaftor/Ivacaftor therapy in cystic fibrosis children previously CFSPID: Is it over-medicalization?针对既往患有CFSPID的囊性纤维化儿童的依列卡福/替扎卡福/依伐卡托疗法：这是否属于过度医疗？
J Cyst Fibros. 2024 Mar;23(2):366-367. doi: 10.1016/j.jcf.2023.10.008. Epub 2023 Oct 13.
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Eur Respir J. 2024 Sep 26;64(3). doi: 10.1183/13993003.00929-2024. Print 2024 Sep.
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Elexacaftor/Tezacaftor/Ivacaftor Effectiveness in N1303K Variant in Adult People With Cystic Fibrosis.依列卡福妥/替扎卡福妥/依伐卡托对成年囊性纤维化患者N1303K变异体的有效性
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Reply: Reversal of structural abnormalities with elexacaftor/tezacaftor/ivacaftor in adults with cystic fibrosis: the earlier you start, the better the outcome will be!回复：在成年囊性纤维化患者中，使用依列卡福/替扎卡福/依伐卡福可逆转结构异常：越早开始治疗，效果越好！
Eur Respir J. 2024 Sep 26;64(3). doi: 10.1183/13993003.01247-2024. Print 2024 Sep.
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Clinical effectiveness of elexacaftor/tezacaftor/ivacaftor in a man with A559T and 3120+1G>CFTR variants.依列卡福妥/替扎卡福妥/依伐卡托对一名携带A559T和3120+1G>CFTR变异的男性的临床疗效。
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Reply to Piehler : Depression Symptoms in Patients with Cystic Fibrosis Fluctuate at Baseline and Improve with Elexacaftor/Tezacaftor/Ivacaftor Therapy.对皮勒的回复：囊性纤维化患者的抑郁症状在基线时波动，并随依列卡福/替扎卡福/依伐卡福治疗而改善。
Am J Respir Crit Care Med. 2024 Aug 1;210(3):367. doi: 10.1164/rccm.202404-0852LE.
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Patients eligible for modulator drugs: Data from cystic fibrosis registry of Turkey.适合调节剂药物的患者：来自土耳其囊性纤维化登记处的数据。
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Depression Symptoms in Patients with Cystic Fibrosis Fluctuate at Baseline and Improve with Elexacaftor/Tezacaftor/Ivacaftor Therapy.囊性纤维化患者的抑郁症状在基线时波动，并随着依列卡福/替扎卡福/依伐卡福治疗而改善。
Am J Respir Crit Care Med. 2024 Aug 1;210(3):365-367. doi: 10.1164/rccm.202404-0787LE.
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Efficacy of elexacaftor-tezacaftor-ivacaftor in portuguese adolescents and adults with cystic fibrosis carrying non-F508del variants.依列卡福妥-替扎卡福妥-依伐卡福妥对携带非F508del突变的葡萄牙囊性纤维化青少年和成人的疗效。
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Case report of two adults with F508del/3849+10 kb C > T genotype regaining exocrine pancreatic function following treatment with elexacaftor/tezacaftor/ivacaftor.两名携带F508del/3849+10 kb C>T基因型的成年人在接受依列卡福/替扎卡福/依伐卡福治疗后恢复外分泌胰腺功能的病例报告。
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Clinical Significance of the 5T;12TG Genotype in Pediatric CFSPID: A Retrospective Study.5T;12TG基因型在儿童CFSPID中的临床意义：一项回顾性研究
Children (Basel). 2025 Jun 14;12(6):778. doi: 10.3390/children12060778.