恩格列净治疗成年糖原贮积病Ib型同胞患者的总体获益：一年经验
The overall benefits of empagliflozin treatment in adult siblings with glycogen storage disease type Ib: one year experience.
作者信息
Bidiuk Joanna, Gaciong Zbigniew A, Sobieraj Piotr
机构信息
Department of Internal Medicine, Hypertension and Vascular Diseases, Medical University of Warsaw, Warsaw, Poland.
出版信息
Arch Med Sci. 2022 Jun 23;18(4):1095-1099. doi: 10.5114/aoms/150029. eCollection 2022.
INTRODUCTION
Recently published case reports suggest the benefit of empagliflozin use in subjects with glycogen storage disease Ib (GSD Ib).
METHODS
We present the clinical and laboratory data of 2 adult brothers with GSD Ib treated with empagliflozin for 12 months.
RESULTS
There was no severe infection during administration of empagliflozin. The improvement of clinical symptoms of inflammatory bowel disease and arthritis along with reduction in serum CRP levels and urinary albumin excretion was noted. Neutrophil count increased, allowing for reduction or temporary withdrawal of G-CSF treatment.
CONCLUSIONS
Empagliflozin may be a new safe treatment in GSD Ib patients with an advanced stage of the disease.
引言
最近发表的病例报告表明，恩格列净用于治疗糖原贮积病Ib型（GSD Ib）患者有益。
方法
我们展示了2名成年兄弟GSD Ib患者接受恩格列净治疗12个月的临床和实验室数据。
结果
恩格列净给药期间未发生严重感染。观察到炎症性肠病和关节炎的临床症状有所改善，同时血清CRP水平和尿白蛋白排泄减少。中性粒细胞计数增加，使得G-CSF治疗得以减少或暂时停用。
结论
恩格列净可能是处于疾病晚期的GSD Ib患者的一种新的安全治疗方法。
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