CFTR 调节剂对成年囊性纤维化患者 GH-IGF-1 轴损伤的影响。
Impact of CFTR-modulating drugs on GH-IGF-1 axis impairment in adult patients with cystic fibrosis.
机构信息
Section of Medical Pathophysiology, Endocrinology, Department Experimental Medicine, Sapienza University of Rome, 00161, Rome, Italy.
Cystic Fibrosis Unit, Bambino Gesù Children's Hospital, Rome, Italy.
出版信息
J Endocrinol Invest. 2019 Nov;42(11):1361-1363. doi: 10.1007/s40618-019-01051-4. Epub 2019 Apr 20.
INTRODUCTION
A new class of drugs in the treatment of cystic fibrosis (CF) includes two agents: lumacaftor, which corrects CFTR channel protein, and ivacaftor, which increases CFTR channel activity. In our previous study we recruited 50 stable adults with CF and 16 of them showed growth hormone deficit (GHD): 7 patients severe and 9 patients partial GHD.
MATERIAL AND METHODS
We decided to re-evaluate ten patients with the GHRH + arginine test of whom only five were treated with lumacaftor/ivacaftor.
RESULTS
All CF patients in therapy with lumacaftor/ivacaftor showed a marked improvement in GHD. Two patients moved from a severe GHD to a normal response to the GH/IGF-1 axis test, and three patients who had partial GHD moved to normal response.
CONCLUSION
The pituitary gland may be damaged by CF disease and could benefit of the action of correcting drugs.
简介
治疗囊性纤维化 (CF) 的一类新药包括两种药物：校正 CFTR 通道蛋白的 lumacaftor 和增加 CFTR 通道活性的 ivacaftor。在我们之前的研究中，我们招募了 50 名稳定期 CF 成人，其中 16 名存在生长激素缺乏症 (GHD)：7 名严重 GHD，9 名部分 GHD。
材料和方法
我们决定重新评估 10 名接受 GHRH+精氨酸试验的患者，其中只有 5 名接受 lumacaftor/ivacaftor 治疗。
结果
所有接受 lumacaftor/ivacaftor 治疗的 CF 患者的 GHD 均明显改善。2 名患者从严重 GHD 转变为 GH/IGF-1 轴测试的正常反应，3 名部分 GHD 患者转变为正常反应。
结论
CF 疾病可能损害垂体，并可能受益于校正药物的作用。
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