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Real-Life Safety and Effectiveness of Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis.

Lumacaftor-Ivacaftor 在囊性纤维化患者中的真实安全性和有效性。

  • 影响因子:5.24
  • DOI:10.1164/rccm.201906-1227OC
  • 作者列表:"Burgel PR","Munck A","Durieu I","Chiron R","Mely L","Prevotat A","Murris-Espin M","Porzio M","Abely M","Reix P","Marguet C","Macey J","Sermet-Gaudelus I","Corvol H","Bui S","Lemonnier L","Dehillotte C","Da Silva J","Paillasseur JL","Hubert D","French Cystic Fibrosis Reference Network Study Group.
  • 发表时间:2020-01-15
Abstract

:Rationale: Lumacaftor-ivacaftor is a CFTR (cystic fibrosis transmembrane conductance regulator) modulator combination recently approved for patients with cystic fibrosis (CF) homozygous for the Phe508del mutation.Objectives: To evaluate the safety and effectiveness of lumacaftor-ivacaftor in adolescents (≥12 yr) and adults (≥18 yr) in a real-life postapproval setting.Methods: The study was conducted in the 47 CF reference centers in France. All patients who initiated lumacaftor-ivacaftor from January 1 to December 31, 2016, were eligible. Patients were evaluated for lumacaftor-ivacaftor safety and effectiveness over the first year of treatment following the French CF Learning Society's recommendations.Measurements and Main Results: Among the 845 patients (292 adolescents and 553 adults) who initiated lumacaftor-ivacaftor, 18.2% (154 patients) discontinued treatment, often owing to respiratory (48.1%, 74 patients) or nonrespiratory (27.9%, 43 patients) adverse events. In multivariable logistic regression, factors associated with increased rates of discontinuation included adult age group, percent predicted FEV1 (ppFEV1) less than 40%, and numbers of intravenous antibiotic courses during the year before lumacaftor-ivacaftor initiation. Patients with continuous exposure to lumacaftor-ivacaftor showed an absolute increase in ppFEV1 (+3.67%), an increase in body mass index (+0.73 kg/m2), and a decrease in intravenous antibiotic courses by 35%. Patients who discontinued treatment had significant decrease in ppFEV1, without improvement in body mass index or decrease in intravenous antibiotic courses.Conclusions: Lumacaftor-ivacaftor was associated with improvement in lung disease and nutritional status in patients who tolerated treatment. Adults who discontinued lumacaftor-ivacaftor, often owing to adverse events, were found at high risk of clinical deterioration.

摘要

: 原理: Lumacaftor-ivacaftor 是一种 CFTR (囊性纤维化跨膜传导调节因子) 调节剂组合,最近被批准用于囊性纤维化 (CF) 纯合子 Phe508del 突变患者。目的: 在真实的批准后环境中,评价 lumacaftor-ivacaftor 在青少年 (≥ 12 岁) 和成人 (≥ 18 岁) 中的安全性和有效性。方法:该研究在法国的 47 个 CF 参考中心进行。从 1月1日至 2016年12月31日启动 lumacaftor-ivacaftor 的所有患者均符合条件。按照法国 CF 学习协会的建议,对患者进行 lumacaftor-ivacaftor 治疗第一年的安全性和有效性评价。测量和主要结果: 在启动 lumacaftor-ivacaftor 的 845 例患者 (292 例青少年和 553 例成人) 中,18.2% 例 (154 例患者) 因呼吸系统中断治疗 (48.1% 例,74 例患者)或非呼吸 (27.9%,43 例患者) 不良事件。在多变量 logistic 回归中,与停药率增加相关的因素包括成人年龄组,预计 FEV1 (ppFEV1) 百分比小于 40%, 和 lumacaftor-ivacaftor 开始前一年静脉注射抗生素疗程的次数。持续暴露于 lumacaftor-ivacaftor 的患者显示 ppFEV1 绝对增加 (+ 3.67%),体重指数增加 (+ 0.73千克/m2), 静脉注射抗生素疗程减少 35%。停止治疗的患者 ppFEV1 显著降低,体重指数无改善或静脉注射抗生素疗程减少。结论: Lumacaftor-ivacaftor 与耐受治疗的患者肺部疾病和营养状况的改善相关。停用 lumacaftor-ivacaftor 的成人,通常由于不良事件,被发现具有临床恶化的高风险。

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作者列表:["Vadillo C","Nieto MA","Romero-Bueno F","Leon L","Sanchez-Pernaute O","Rodriguez-Nieto MJ","Freites D","Jover JA","Álvarez-Sala JL","Abasolo L"]

METHODS:OBJECTIVES:To asses the clinical course in RA-related interstitial lung disease (RA-ILD) patients with and without rituximab (RTX). The influence of other variables was also evaluated. METHODS:A longitudinal multicentre study was conducted in RA diagnosed with ILD from 2007 until 2018 in Madrid. Patients were included in a registry [pNEumology RhEumatology Autoinmune diseases (NEREA)] from the time of ILD diagnosis. The main endpoint was functional respiratory impairment (FI), when there was a decline ≥5% in the predicted forced vital capacity compared with the previous one. Pulmonary function was measured at baseline and in follow-up visits every 6-12 months. The independent variable was therapy with RTX. Covariables included sociodemographic, clinical, radiological and other therapies. Survival techniques were used to estimate the incidence rate (IR) and 95% CI of functional impairment, expressed per 100 patient-semesters. Cox multivariate regression models were run to examine the influence of RTX and other covariates on FI. Results were expressed as the hazard ratio (HR) and CI. RESULTS:A total of 68 patients were included. FI occurred in 42 patients [IR 23.5 (95% CI 19, 29.1)] and 50% of them had FI within 1.75 years of an ILD diagnosis. A multivariate analysis showed that RTX exposure resulted in a lower risk of FI compared with non-exposure [HR 0.51 (95% CI 0.31, 0.85)]. Interstitial pneumonia, glucocorticoids, disease activity and duration also influenced FI. CONCLUSION:RA-ILD patients deteriorate over time, with the median time free of impairment being <2 years. Patients exposed to RTX had a higher probability of remaining free of FI compared with other therapies. Other factors have also been identified. Key words: rheumatoid arthritis, interstitial lung disease, observational study, rituximab and prognosis

翻译标题与摘要 下载文献
影响因子:4.40
发表时间:2020-01-01
DOI:10.1007/s00262-019-02431-8
作者列表:["Shibaki, Ryota","Murakami, Shuji","Matsumoto, Yuji","Yoshida, Tatsuya","Goto, Yasushi","Kanda, Shintaro","Horinouchi, Hidehito","Fujiwara, Yutaka","Yamamoto, Nobuyuki","Kusumoto, Masahiko","Yamamoto, Noboru","Ohe, Yuichiro"]

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翻译标题与摘要 下载文献
影响因子:4.04
发表时间:2020-01-25
来源期刊:New biotechnology
DOI:10.1016/j.nbt.2019.08.006
作者列表:["Sousa SA","Soares-Castro P","Seixas AMM","Feliciano JR","Balugas B","Barreto C","Pereira L","Santos PM","Leitão JH"]

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