Eliglustat, an investigational oral therapy for Gaucher disease type 1: Phase 2 trial results after 4 years of treatment
Eliglustat, an investigational oral therapy for Gaucher disease type 1: Phase 2 trial results after 4 years of treatment
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DOI：
10.1016/j.bcmd.2014.04.002
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发表时间：
2014-12-01
影响因子：
2.3
通讯作者：
Peterschmitt, Judith M.
中科院分区：
文献类型：
--
作者：
Lukina, Elena;Watman, Nora;Peterschmitt, Judith M.
Eliglustat是一种用于1型戈谢病（GD 1）的研究性口服底物减少疗法。19例初治患者现已完成4年的开放标签研究（NCT 00358150）。平均血红蛋白水平和血小板计数分别增加2.3 +/- 1.5 g/dL（基线：11.3 +/- 1.5 g/dL）和95%（基线：68，700 +/-21，200/mm（3））。平均脾脏和肝脏体积（正常值的倍数，MN）分别减少63%（基线：17.3 +/- 9.5 MN）和28%（基线：1.7 +/- 0.4 MN）。中位壳三糖苷酶和CCL-18均降低82%;血浆葡萄糖神经酰胺和GM 3正常化。腰椎平均骨密度T评分增加0.8（60%）（基线：-1.6 +/- 1.1）。17/18例患者的股骨暗骨髓（戈谢细胞浸润骨髓的反映）减少或稳定。无骨危象。大多数不良事件是轻度的，与治疗无关。这些结果将1年和2年时报告的eliglustat的安全性和疗效延长至4年。(C)2014 Genzyme，赛诺菲旗下公司。由爱思唯尔公司出版
Eliglustat is an investigational, oral substrate reduction therapy for Gaucher disease type 1 (GD1). Nineteen treatment-naive patients have now completed 4 years of an open-label study (NCT00358150). Mean hemoglobin level and platelet count increased by 2.3 +/- 1.5 g/dL (baseline: 11.3 +/- 1.5 g/dL) and 95% (baseline: 68,700 +/- 21,200/mm(3)), respectively. Mean spleen and liver volumes (multiples of normal, MN) decreased by 63% (baseline: 17.3 +/- 9.5 MN) and 28% (baseline: 1.7 +/- 0.4 MN), respectively. Median chitotriosidase and CCL-18 each decreased by 82%; plasma glucosylceramide and GM3 normalized. Mean bone mineral density T-score for the lumbar spine increased by 0.8 (60%) (baseline: -1.6 +/- 1.1). Femur dark marrow, a reflection of Gaucher cell infiltration into bone marrow, was reduced or stable in 17/18 patients. There were no bone crises. Most adverse events were mild and unrelated to treatment. These results extend the safety and efficacy of eliglustat reported at 1 and 2 years to 4 years. (C) 2014 Genzyme, a Sanofi company. Published by Elsevier B.V.