伊马替尼布用于全身性巨细胞疾病
A Pardanani1, M Elliott, T Reeder
1Division of Hematology, Mayo Clinic, 200 First Street SW, Rochester, MN, USA.
Lancet (London, England)
|August 23, 2003
概括
伊马替尼有效地通过向c-kit来治疗全身性乳腺细胞疾病. 50%的患者表现出显著的反应,有些人实现了完全缓解,突出显示伊马替尼布.
科学领域:
- 血液学 血液学 血液学
- 在瘤学瘤学.
- 药理学 药理学是指药理学的学科.
背景情况:
- 系统性乳腺细胞疾病 (SMCD) 是一种罕见的疾病,通常是由c-kit原型瘤基因的突变驱动的.
- 伊马替尼是已知的氨酸激酶抑制剂,向c-kit,并在某些恶性瘤中显示出有效性.
研究的目的:
- 为了评估伊马替尼的疗效和安全性,成年患者有症状的全身性巨细胞疾病.
- 评估不同剂量的伊马替尼布治疗患者的应答率和临床结果.
主要方法:
- 一项前性研究涉及12名患有症状SMCD的成年患者.
- 患者接受了每日100毫克或400毫克剂量的意马替尼.
- 通过临床,组织学和血液学参数来评估反应,包括巨细胞细胞减少和eosinophilia.
主要成果:
- 50% (10名可评估患者中5名) 显示对伊马替尼布有可测量的反应.
- 四名患者实现了显著的巨细胞细胞减少,两名患者实现了完全的临床和组织学缓解.
- 五分之三的eosinophilia患者实现了完全的临床和血液学缓解. 没有反应的人拥有c-kit D816V突变.
结论:
- 伊马替尼在全身性巨细胞疾病中表现出显著的临床活性.
- 该药物似乎对野生类型的c-kit驱动疾病有效,对携带c-kit D816V突变的患者有效性有限.
- 伊马替尼可能会抑制野生型c-kit的增长促进作用或向SMCD中的其他瘤性激酶.
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