CFTR增强剂疗法可改善患有门控突变的CF患者受损的胰岛素分泌。
CFTR potentiator therapy ameliorates impaired insulin secretion in CF patients with a gating mutation.
作者信息
Tsabari Reuven, Elyashar Hila Iron, Cymberknowh Malena Cohen, Breuer Oded, Armoni Shoshana, Livnat Galit, Kerem Eitan, Zangen David Haim
机构信息
Division of Pediatric Pulmonology and CF center, Jerusalem 91240, Israel.
Israel CF center, Carmel Medical Center, Haifa, Israel.
出版信息
J Cyst Fibros. 2016 May;15(3):e25-7. doi: 10.1016/j.jcf.2015.10.012. Epub 2015 Nov 4.
OBJECTIVE
To investigate the effect of treatment with ivacaftor on insulin secretion in patients with cystic fibrosis (CF) (ΔF508\S549R) having CFRD/impaired insulin secretion.
METHODS
A standard OGTT was performed before and after 16weeks of treatment with ivacaftor in 2 sibling patients with CF carrying the S549R gating mutation. The area under the curve (AUC) for glucose and insulin was calculated using the trapezoidal estimation.
RESULTS
Before treatment, the OGTT of case 1 showed indeterminate glycemia; the OGTT of case 2 indicated CFRD. After ivacaftor treatment the OGTT demonstrated improved insulin secretion pattern mainly by increased first phase early insulin secretion, resulting in reduction of the AUC of glucose in both cases.
CONCLUSIONS
The treatment with ivacaftor in patients with CF carrying gating mutation can ameliorate impaired insulin secretion. Further studies and larger cohorts are needed to evaluate the impact of ivacaftor on insulin secretion in patients with CF carrying gating or other mutations.
目的
研究依伐卡托对患有囊性纤维化（CF）（ΔF508\S549R）且伴有囊性纤维化相关糖尿病（CFRD）/胰岛素分泌受损患者胰岛素分泌的影响。
方法
对2例携带S549R门控突变的CF同胞患者进行依伐卡托治疗16周前后的标准口服葡萄糖耐量试验（OGTT）。采用梯形估计法计算葡萄糖和胰岛素的曲线下面积（AUC）。
结果
治疗前，病例1的OGTT显示血糖情况不确定；病例2的OGTT提示患有CFRD。依伐卡托治疗后，OGTT显示胰岛素分泌模式改善，主要表现为早期胰岛素分泌的第一相增加，导致两例患者的葡萄糖AUC均降低。
结论
对携带门控突变的CF患者使用依伐卡托治疗可改善胰岛素分泌受损情况。需要进一步研究和更大规模的队列来评估依伐卡托对携带门控或其他突变的CF患者胰岛素分泌的影响。
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