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Females with cystic fibrosis have a larger decrease in sweat chloride in response to lumacaftor/ivacaftor compared to males
Journal of Cystic Fibrosis ( IF 5.2 ) Pub Date : 2021-01-01 , DOI: 10.1016/j.jcf.2020.05.004 B L Aalbers 1 , R W Hofland 1 , I Bronsveld 1 , K M de Winter-de Groot 2 , H G M Arets 2 , A C de Kiviet 2 , M M M van Oirschot-van de Ven 2 , M A Kruijswijk 1 , S Schotman 1 , S Michel 2 , C K van der Ent 2 , H G M Heijerman 1
Journal of Cystic Fibrosis ( IF 5.2 ) Pub Date : 2021-01-01 , DOI: 10.1016/j.jcf.2020.05.004 B L Aalbers 1 , R W Hofland 1 , I Bronsveld 1 , K M de Winter-de Groot 2 , H G M Arets 2 , A C de Kiviet 2 , M M M van Oirschot-van de Ven 2 , M A Kruijswijk 1 , S Schotman 1 , S Michel 2 , C K van der Ent 2 , H G M Heijerman 1
Affiliation
AIM
To explore which patient-related factors influence sweat test response to CFTR modulators, as well as examining the correlation between the sweat chloride response and ppFEV1 or BMI response, using systematically collected real-life clinical data. METHODS
160 CF patients were identified who had used lumacaftor/ivacaftor for at least six months. Of these patients, age, sweat chloride levels, ppFEV1 weight and BMI at the start of treatment and after 6 months were collected retrospectively. Pearson and Spearman tests were performed to assess correlations. RESULTS
Females compared to males in this group showed a larger response in sweat chloride (mean difference 10.6 mmol/l, 95% CI: 5.7-15.4) and BMI (mean difference 0.27 kg/m2, 95% CI: 0.01-0.54). A modest but significant correlation was found between patient weight and sweat chloride response (Pearson R = 0.244, p = 0.001), which diminished upon correction for the other factors. The correlation between sex and sweat chloride response remained; R = 0.253, p = 0.001. Sweat chloride response did not correlate with ppFEV1 change or BMI change at 6 months after start of therapy. CONCLUSION
Sweat chloride response is larger in females compared to males, which also explains the negative correlation of weight with the response in sweat chloride concentration after start of lumacaftor/ivacaftor. Sweat chloride response does not correlate with the responses in ppFEV1 and BMI. This information may help the interpretation of sweat test results acquired for the follow up and evaluation of CFTR modulating treatments, and warrants further investigation into the underlying mechanisms of sex differences in response to CFTR modulators.
中文翻译：
与男性相比，患有囊性纤维化的女性在接受 lumacaftor/ivacaftor 后汗液氯化物的减少幅度更大
目的 使用系统收集的真实临床数据，探索哪些患者相关因素影响汗液测试对 CFTR 调节剂的反应，以及检查汗液氯化物反应与 ppFEV1 或 BMI 反应之间的相关性。方法 确定了 160 名使用 lumacaftor/ivacaftor 至少六个月的 CF 患者。在这些患者中，回顾性收集治疗开始时和 6 个月后的年龄、汗液氯化物水平、ppFEV1 体重和 BMI。进行 Pearson 和 Spearman 检验以评估相关性。结果 在该组中，与男性相比，女性在汗液氯化物（平均差异 10.6 mmol/l，95% CI：5.7-15.4）和 BMI（平均差异 0.27 kg/m2，95% CI：0.01-0.54）方面表现出更大的反应。在患者体重和汗液氯化物反应之间发现了适度但显着的相关性（Pearson R = 0.244，p = 0.001），在校正其他因素后该相关性减弱。性别和汗液氯化物反应之间的相关性仍然存在；R = 0.253，p = 0.001。汗液氯化物反应与治疗开始后 6 个月时的 ppFEV1 变化或 BMI 变化无关。结论与男性相比，女性的汗液氯化物反应更大，这也解释了体重与 lumacaftor/ivacaftor 开始后汗液氯化物浓度反应的负相关。汗液氯化物反应与 ppFEV1 和 BMI 的反应无关。此信息可能有助于解释为跟踪和评估 CFTR 调节治疗而获得的汗液测试结果，
更新日期：2021-01-01
中文翻译：
与男性相比，患有囊性纤维化的女性在接受 lumacaftor/ivacaftor 后汗液氯化物的减少幅度更大
目的 使用系统收集的真实临床数据，探索哪些患者相关因素影响汗液测试对 CFTR 调节剂的反应，以及检查汗液氯化物反应与 ppFEV1 或 BMI 反应之间的相关性。方法 确定了 160 名使用 lumacaftor/ivacaftor 至少六个月的 CF 患者。在这些患者中，回顾性收集治疗开始时和 6 个月后的年龄、汗液氯化物水平、ppFEV1 体重和 BMI。进行 Pearson 和 Spearman 检验以评估相关性。结果 在该组中，与男性相比，女性在汗液氯化物（平均差异 10.6 mmol/l，95% CI：5.7-15.4）和 BMI（平均差异 0.27 kg/m2，95% CI：0.01-0.54）方面表现出更大的反应。在患者体重和汗液氯化物反应之间发现了适度但显着的相关性（Pearson R = 0.244，p = 0.001），在校正其他因素后该相关性减弱。性别和汗液氯化物反应之间的相关性仍然存在；R = 0.253，p = 0.001。汗液氯化物反应与治疗开始后 6 个月时的 ppFEV1 变化或 BMI 变化无关。结论与男性相比，女性的汗液氯化物反应更大，这也解释了体重与 lumacaftor/ivacaftor 开始后汗液氯化物浓度反应的负相关。汗液氯化物反应与 ppFEV1 和 BMI 的反应无关。此信息可能有助于解释为跟踪和评估 CFTR 调节治疗而获得的汗液测试结果，
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