Altes Talissa A, Johnson Mac, Fidler Meredith, Botfield Martyn, Tustison Nicholas J, Leiva-Salinas Carlos, de Lange Eduard E, Froh Deborah, Mugler John P
Department of Radiology, One Hospital Drive, University of Missouri, Columbia, MO 65212, USA.
Vertex Pharmaceuticals Incorporated, 50 Northern Ave Boston, MA 02210, USA.
J Cyst Fibros. 2017 Mar;16(2):267-274. doi: 10.1016/j.jcf.2016.12.004. Epub 2017 Jan 26.
This pilot study evaluated the effect of short- and long-term ivacaftor treatment on hyperpolarized He-magnetic resonance imaging (MRI)-defined ventilation defects in patients with cystic fibrosis aged ≥12years with a G551D-CFTR mutation.
Part A (single-blind) comprised 4weeks of ivacaftor treatment; Part B (open-label) comprised 48weeks of treatment. The primary outcome was change from baseline in total ventilation defect (TVD; total defect volume:total lung volume ratio).
Mean change in TVD ranged from -8.2% (p=0.0547) to -12.8% (p=0.0078) in Part A (n=8) and -6.3% (p=0.1953) to -9.0% (p=0.0547) in Part B (n=8) as assessed by human reader and computer algorithm, respectively.
TVD responded to ivacaftor therapy. He-MRI provides an individual quantification of disease burden that may be able to detect aspects of the disease missed by population-based spirometry metrics. Assessments by human reader and computer algorithm exhibit similar trends, but the latter appears more sensitive. www.clinicaltrials.gov identifier: NCT01161537.
本初步研究评估了短期和长期使用依伐卡托治疗对年龄≥12岁、携带G551D-CFTR突变的囊性纤维化患者经超极化氦磁共振成像（MRI）定义的通气缺陷的影响。
A部分（单盲）包括4周的依伐卡托治疗；B部分（开放标签）包括48周的治疗。主要结局是总通气缺陷（TVD；总缺陷体积与总肺体积之比）相对于基线的变化。
根据人工阅片者和计算机算法评估，A部分（n = 8）中TVD的平均变化范围分别为-8.2%（p = 0.0547）至-12.8%（p = 0.0078），B部分（n = 8）中为-6.3%（p = 0.1953）至-9.0%（p = 0.0547）。
TVD对依伐卡托治疗有反应。氦MRI可对疾病负担进行个体量化，这可能能够检测出基于人群的肺量计指标所遗漏的疾病方面。人工阅片者和计算机算法的评估显示出相似趋势，但后者似乎更敏感。www.clinicaltrials.gov标识符：NCT01161537。
