FDA药物批准简介:阿扎胞苷(5-azacytidine Vidaza)注射悬浮液。
文章的细节
-
引用
复制到剪贴板 -
Kaminskas E,法雷尔,王YC, Sridhara R, Pazdur R
FDA药物批准简介:阿扎胞苷(5-azacytidine Vidaza)注射悬浮液。
肿瘤学家。2005年3月,10 (3):176 - 82。
- PubMed ID
-
15793220 (在PubMed]
- 文摘
-
2004年5月19日,阿扎胞苷(5-azacytidine;Vidaza(商标);Pharmion公司,博尔德公司,http://www.pharmion.com)注射悬挂获得定期批准由美国食品和药物管理局(FDA)治疗骨髓增生异常综合征(MDS)的所有亚型。这份报告总结了这个批准的基础。效果演示了在一个随机,对照试验比较阿扎胞苷和最好的支持性护理管理南卡罗来纳州单组研究(观察组)和两个,一个是阿扎胞苷的管理南卡罗来纳州,另一个是它的管理增长值阿扎胞苷的剂量,75 mg / m2 /日7天每28天,在所有三个研究中是相同的。在随机试验中,研究参与者被匹配对年龄,性别,种族,性能状态,MDS亚型,并使用输血前的3个月期间研究的条目。病人在观察手臂被协议允许跨越阿扎胞苷治疗如果他们的疾病进展根据预定的标准。在研究过程中,超过一半的患者在观察手臂交叉在阿扎胞苷治疗手臂。主要疗效终点是整体回应率。响应由完全或部分血细胞计数和正常化的骨髓形态学。 The response rate in the azacitidine arm was about 16%; there were no responses in the observation arm. The response rates in the two single-arm studies were similar (13% and 19%). The responses were sustained, with median durations of 11 months and 17 months respectively. Responding patients who were transfusion dependent at study entry lost the need for transfusions. In addition, about 19% of patients had less than partial responses (termed improvement), and two-thirds of them became transfusion independent. Common adverse events associated with azacitidine treatment were gastrointestinal (nausea, vomiting, diarrhea, constipation, and anorexia), hematologic (neutropenia, thrombocytopenia), fevers, rigors, ecchymoses, petechiae, injection site events, arthralgia, headache, and dizziness. Liver function abnormalities occurred in 16% of patients with intercurrent hepatobiliary disorders and in two patients with previously diagnosed liver cirrhosis. Renal failure occurred in patients during sepsis and hypotension. There were no deaths attributed to azacitidine. Azacitidine, the first drug approved by the U.S. FDA for MDS, has a favorable safety profile and provides a clinical benefit of eliminating transfusion dependence and complete or partial normalization of blood counts and bone marrow blast percentages in responding patients.