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Breast development in a 7 year old girl with CF treated with ivacaftor: An indication for personalized dosing?
Journal of Cystic Fibrosis ( IF 5.2 ) Pub Date : 2021-06-24 , DOI: 10.1016/j.jcf.2021.06.001
Joshena Jeyaratnam 1 , Renske van der Meer 2 , Gitte Berkers 1 , Harry Gm Heijerman 3 , Jeffrey M Beekman 1 , Cornelis K van der Ent 1
Affiliation  

Substantial progress has been made in the treatment of Cystic fibrosis due to introduction of CFTR modulators. However, little is known about the long term side effects of treatment with these drugs. We here present a 7 year old girl with CF who presented with breast development as a rare dose dependent side effect of treatment with ivacaftor and we report data on the correlation between drug plasma concentration and clinical effect, bodyweight, and BSA in 16 patients.

Higher plasma concentrations did not correlate with clinical effect, as change in FEV1 and sweat chloride concentration. Patients with low bodyweight or BSA tended to have higher plasma concentrations. This might indicate that the current recommended dose of ivacaftor is at the top of the dose-response curve and that some patients can be treated with lower doses of ivacaftor with similar clinical effect.



中文翻译:

用依伐卡托治疗 CF 的 7 岁女孩的乳房发育:个性化给药的指征?

由于引入 CFTR 调节剂,治疗囊性纤维化取得了实质性进展。然而,人们对这些药物治疗的长期副作用知之甚少。我们在这里介绍了一名 7 岁的 CF 女孩,她的乳房发育是 ivacaftor 治疗的一种罕见的剂量依赖性副作用,我们报告了 16 名患者的药物血浆浓度与临床效果、体重和 BSA 之间相关性的数据。

较高的血浆浓度与临床效果无关,如 FEV1 和汗液氯化物浓度的变化。低体重或 BSA 的患者往往具有较高的血浆浓度。这可能表明目前推荐的ivacaftor剂量处于剂量反应曲线的顶部,并且一些患者可以用较低剂量的ivacaftor进行治疗,具有相似的临床效果。

更新日期:2021-06-24
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