甲磺酸伊马替尼治疗系统性肥大细胞增多症:第二阶段试验。

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Droogendijk黄建忠、Kluin-Nelemans HJ van Doormaal JJ,荷兰队美联社,van de Loosdrecht AA,范Daele PL

甲磺酸伊马替尼治疗系统性肥大细胞增多症:第二阶段试验。

癌症。2006年7月15日,107 (2):345 - 51。

PubMed ID

16779792 (在PubMed

]

文摘

背景:肥大细胞增多症的特点是肥大细胞的异常增殖1或多个器官。在大多数患者中,基因的变异存在于c - kit,导致管制的c - kit受体。甲磺酸伊马替尼是一个c - kit受体酪氨酸激酶活性的有效抑制剂。因此,作者评价甲磺酸伊马替尼治疗的疗效和安全性进行系统性肥大细胞增多症患者。方法:系统性肥大细胞增多症患者接受了甲磺酸伊马替尼的剂量口服400毫克每日一次,3 - 6个月。低剂量的强的松中加入前2周。端点是减少血清类胰蛋白酶,尿N-methylhistamine排泄,皮肤损伤,肥大细胞的数量在骨髓的章节中,肝肿大和/或脾肿大,症状。结果:14个病人包括在这项研究中,11个患者D816V突变。一个病人表达了FIP1L1-PDGFR-alpha重排基因。在2例,没有发现突变。 In 10 patients, serum tryptase levels decreased >20%. In all patients, urinary N-methylhistamine excretion was reduced. In 8 of 13 evaluable patients, the number of mast cells in the bone marrow decreased. Skin symptoms diminished in 5 of 9 patients. Hepatosplenomegaly improved in 3 of 6 patients. Symptoms decreased in 8 of 13 patients. In all patients who had the D816V mutation, reductions in > or =2 endpoints were achieved. In the patient who expressed the FIP1L1-PDGFR-alpha rearrangement gene, a complete response was attained. In general, imatinib mesylate was tolerated well. CONCLUSIONS: Imatinib mesylate was effective in patients with systemic mastocytosis, including those who had the D816V mutation.

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药物

伊马替尼