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CFTR modulator therapies – Effect on life expectancy in people with cystic fibrosis
Paediatric Respiratory Reviews ( IF 4.7 ) Pub Date : 2020-05-26 , DOI: 10.1016/j.prrv.2020.05.002
I M Balfour-Lynn 1 , J A King 1
Affiliation  

CFTR modulators have dramatically changed the clinical course of CF in those fortunate enough to receive them. Inevitably, randomised controlled trials during the development of these drugs are too short to use mortality as an outcome. Evidence for their effect on life expectancy are best gained from real world registry studies specifically looking at mortality, but these are only available for ivacaftor to date. Therefore, indirect evidence must be obtained by looking at outcomes known to affect mortality and seeing the effect of these drugs on those outcomes.



中文翻译:

CFTR 调节剂疗法——对囊性纤维化患者预期寿命的影响

CFTR 调节剂极大地改变了那些有幸接受它们的 CF 的临床过程。不可避免的是,这些药物开发过程中的随机对照试验太短,无法将死亡率作为结果。它们对预期寿命影响的证据最好从专门关注死亡率的真实世界登记研究中获得,但迄今为止这些仅适用于ivacaftor。因此,必须通过查看已知会影响死亡率的结果并查看这些药物对这些结果的影响来获得间接证据。

更新日期:2020-05-26
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