Arnaud François, Stremler-Le Bel Nathalie, Reynaud-Gaubert Martine, Mancini Julien, Gaubert Jean-Yves, Gorincour Guillaume
Service d'Imagerie Médicale, AP-HM Hôpital Nord, 13015 Marseille, France.
Centre de Ressource et de Compétences de la Mucoviscidose (CRCM) Pédiatrique, AP-HM Hôpital la Timone, 13005 Marseille, France.
J Clin Med. 2021 May 7;10(9):1999. doi: 10.3390/jcm10091999.
As Cystic Fibrosis (CF) treatments drastically improved in recent years, tools to assess their efficiency need to be properly evaluated, especially cross-sectional imaging techniques. High-resolution computed tomography (HRCT) scan response to combined lumacaftor- ivacaftor therapy (Orkambi) in patients with homozygous for F508del CFTR has not yet been assessed.
We conducted a retrospective observational study in two French reference centers in CF in Marseille hospitals, including teenagers (>12 years old) and adults (>18 years) who had received lumacaftor-ivacaftor and for whom we had at disposal at least two CT scans, one at before therapy and one at least six months after therapy start. CT scoring was performed by using the modified version of the Brody score.
34 patients have been included. The mean age was 26 years (12-56 years). There was a significant decrease in the total CT score (65.5 to 60.3, = 0.049) and mucous plugging subscore (12.3 to 8.7, = 0.009). Peribronchial wall thickening (PWT) was significantly improved only in the adult group (29.1 to 27.0, = 0.04). Improvements in total score, peribronchial thickening, and mucous pluggings were significantly correlated with improvement in FEV1 (forced expiratory volume in 1 s).
Treatment with lumacaftor-ivacaftor was associated with a significant improvement in the total CT score, which was mainly related to an improvement in mucous pluggings.
近年来，囊性纤维化（CF）的治疗方法有了显著改善，因此需要对评估其疗效的工具进行恰当评估，尤其是横断面成像技术。目前尚未评估F508del CFTR纯合子患者接受鲁马卡托-依伐卡托联合治疗（奥凯比）时高分辨率计算机断层扫描（HRCT）的反应。
我们在马赛医院的两个法国CF参考中心进行了一项回顾性观察研究，纳入了接受鲁马卡托-依伐卡托治疗的青少年（>12岁）和成年人（>18岁），且我们至少有他们治疗前和治疗开始后至少6个月的两次CT扫描结果。采用改良版的布罗迪评分进行CT评分。
共纳入34例患者。平均年龄为26岁（12 - 56岁）。CT总分（从65.5降至60.3，P = 0.049）和黏液嵌塞子评分（从12.3降至8.7，P = 0.009）显著降低。仅在成年组中，支气管壁增厚（PWT）有显著改善（从29.1降至27.0，P = 0.04）。总分、支气管周围增厚和黏液嵌塞的改善与第1秒用力呼气容积（FEV1）的改善显著相关。
鲁马卡托-依伐卡托治疗使CT总分显著改善，这主要与黏液嵌塞的改善有关。
