一项关于阿贝亭治疗戈谢病的多中心、开放标签、III期研究。
A multicenter, open-label, phase III study of Abcertin in Gaucher disease.
作者信息
Lee Beom Hee, Abdalla Ahmed Fathy, Choi Jin-Ho, Beshlawy Amal El, Kim Gu-Hwan, Heo Sun Hee, Megahed Ahmed Megahed Hassan, Elsayed Mona Abdel Latif, Barakat Tarik El-Sayed Mohammad, Eid Khaled Mohamed Abd El-Azim, El-Tagui Mona Hassan, Mahmoud Mona Mohamed Hamdy, Fateen Ekram, Park June-Young, Yoo Han-Wook
机构信息
Department of Pediatrics, Asan Medical Center Children's Hospital, University of Ulsan College of Medicine, Seoul, Korea Paediatric GIT Unit, Mansoura University Children's Hospital Abou El Reesh Children's Hospital, Cairo University, Egypt Medical Genetics Center, Asan Medical Center Asan Institute for Life Sciences, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea Biochemical Genetics Department, National Research Centre, Cairo, Egypt ISU ABXIS Co., Ltd, Seongnam, Korea.
出版信息
Medicine (Baltimore). 2017 Nov;96(45):e8492. doi: 10.1097/MD.0000000000008492.
BACKGROUND
Gaucher disease (GD) is caused by a deficiency in the lysosomal enzyme glucocerebrosidase. Enzyme replacement therapy (ERT) is recommended for clinical improvement.
METHODS
The efficacy and safety of a new imiglucerase, Abcertin, were assessed in 7 Egyptian patients with treatment-naïve type 1 GD. Each patient was administered a biweekly 60 U/kg dose of Abcertin for 6 months. The primary endpoint was the change in hemoglobin concentration. The secondary endpoints were changes from baseline in platelet counts, spleen and liver volumes, biomarker levels, skeletal parameters, and bone mineral density.
RESULTS
The hemoglobin concentration increased by a mean of 1.96 ± 0.91 g/dL (range 1.11-2.80 g/dL) or 20.6% (P = .001). Statistically significant increases in the platelet count and decreases in the spleen volume and biomarker levels were also observed. There were no severe drug-related adverse events. One patient developed anti-imiglucerase antibodies without neutralizing activity.
CONCLUSION
Our study results demonstrate the efficacy and safety of Abcertin in patients with type 1 GD. This suggests that Abcertin can be an alternative ERT option for type 1 GD.
背景
戈谢病（GD）由溶酶体酶葡萄糖脑苷脂酶缺乏引起。推荐采用酶替代疗法（ERT）来实现临床改善。
方法
对7例未经治疗的1型GD埃及患者评估了一种新型伊米苷酶Abertin的疗效和安全性。每位患者每两周接受一次60 U/kg剂量的Abertin治疗，持续6个月。主要终点是血红蛋白浓度的变化。次要终点是血小板计数、脾脏和肝脏体积、生物标志物水平、骨骼参数和骨密度相对于基线的变化。
结果
血红蛋白浓度平均增加1.96±0.91 g/dL（范围为1.11 - 2.80 g/dL），即增加20.6%（P = 0.001）。还观察到血小板计数有统计学意义的增加，脾脏体积和生物标志物水平有下降。未出现严重的药物相关不良事件。1例患者产生了无中和活性的抗伊米苷酶抗体。
结论
我们的研究结果证明了Abertin对1型GD患者的疗效和安全性。这表明Abertin可以成为1型GD的一种替代ERT选择。
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