一年卢美他南-依伐卡托治疗对囊性纤维化患者葡萄糖耐量异常的影响。
Effect of one-year lumacaftor-ivacaftor treatment on glucose tolerance abnormalities in cystic fibrosis patients.
机构信息
Service d'endocrinologie, diabète et nutrition, Hôpitaux Universitaires de Strasbourg, place de l'hôpital, Strasbourg 67091, France.
Service de pneumologie et CRCM adulte, Hôpitaux Universitaires de Strasbourg, 1 place de l'hôpital, Strasbourg 67091, France.
出版信息
J Cyst Fibros. 2020 Sep;19(5):712-716. doi: 10.1016/j.jcf.2020.03.002. Epub 2020 Mar 19.
OBJECTIVES
To investigate the effects of 1-year lumacaftor-ivacaftor treatment on abnormalities in glucose tolerance (AGT) in Phe508del homozygous cystic fibrosis (CF) patients.
METHODS
Untreated CF patients with glucose intolerance or newly diagnosed diabetes were included in a prospective, observational study. After 1-year lumacaftor-ivacaftor treatment, AGT were evaluated by using oral glucose tolerance test.
RESULTS
Forty patients participated. 78% of patients had glucose intolerance and 22% diabetes at baseline. After one-year treatment, 50% of patients had normal glucose tolerance, 40% glucose intolerance, and 10% diabetes (p <0.001). The two-hour OGTT glycemia decreased from 171 (153-197) to 139 (117-162) mg/dL (p <0.001). 57.5% (n = 23) of patients improved their glucose tolerance with a significant decrease in both 1-hour (p<0.01) and 2-hour (p<0.001) OGTT glycemia.
CONCLUSION
Improvements in AGT were observed following 1-year lumacaftor-ivacaftor treatment. Larger studies are needed to comprehensively assess CF transmembrane conductance regulator (CFTR) modulators.
目的
研究 1 年卢美他尼-依伐卡托治疗对 Phe508del 纯合子囊性纤维化（CF）患者葡萄糖耐量异常（AGT）的影响。
方法
本前瞻性观察性研究纳入了葡萄糖耐量异常或新诊断为糖尿病的未经治疗的 CF 患者。在接受 1 年卢美他尼-依伐卡托治疗后，通过口服葡萄糖耐量试验评估 AGT。
结果
40 名患者参与了研究。基线时有 78%的患者存在葡萄糖耐量异常，22%的患者患有糖尿病。经过 1 年的治疗后，50%的患者血糖恢复正常，40%的患者血糖耐量异常，10%的患者患有糖尿病（p<0.001）。2 小时 OGTT 血糖从 171（153-197）降至 139（117-162）mg/dL（p<0.001）。57.5%（n=23）的患者改善了葡萄糖耐量，1 小时（p<0.01）和 2 小时（p<0.001）OGTT 血糖均显著降低。
结论
卢美他尼-依伐卡托治疗 1 年后观察到 AGT 改善。需要进一步开展更大规模的研究以全面评估囊性纤维化跨膜电导调节因子（CFTR）调节剂。
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