Midostaurin在先进的系统性肥大细胞增多症的临床疗效和安全性。

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Gotlib J, Kluin-Nelemans HC,乔治TI,类似C, Sotlar K, Hermine O, Awan英尺,亨E,毛罗·乔丹,斯特恩伯格DW, Villeneuve M,洪博培标签,斯坦内克EJ,哈特曼K,角质惠普,化合价的P, Reiter

Midostaurin在先进的系统性肥大细胞增多症的临床疗效和安全性。

郑传经地中海J。2016年6月30日,374 (26):2530 - 41。doi: 10.1056 / NEJMoa1513098。

PubMed ID

27355533 (在PubMed

]

文摘

背景:先进的系统性肥大细胞增多症包括罕见的血液肿瘤相关预后不良和缺乏有效的治疗方法。midostaurin multikinase抑制剂抑制工具包D816V,疾病发病机理的主要推动力。方法:我们进行了一次开放研究口服midostaurin 100毫克的剂量每天两次在116名患者,其中89年mastocytosis-related器官损伤都有资格列入人口的主要功效;16有侵略性的系统性肥大细胞增多症,57系统性肥大细胞增多症,血液肿瘤有关,和16能白血病。主要的结果是最好的总体响应。结果:总体响应率为60%(95%可信区间(CI), 49 - 70);45%的病人有一个主要的反应,它被定义为完整的至少一种类型的决议mastocytosis-related器官损伤。响应率相似不管亚型先进的系统性肥大细胞增多症,装备突变状态或暴露于先前的治疗。骨髓中最好的百分比变化能负担和血清类胰蛋白酶水平分别为-59%和-58%,分别。中位总生存期是28.7个月,中位无进展生存期是14.1个月。 Among the 16 patients with mast-cell leukemia, the median overall survival was 9.4 months (95% CI, 7.5 to not estimated). Dose reduction owing to toxic effects occurred in 56% of the patients; re-escalation to the starting dose was feasible in 32% of those patients. The most frequent adverse events were low-grade nausea, vomiting, and diarrhea. New or worsening grade 3 or 4 neutropenia, anemia, and thrombocytopenia occurred in 24%, 41%, and 29% of the patients, respectively, mostly in those with preexisting cytopenias. CONCLUSIONS: In this open-label study, midostaurin showed efficacy in patients with advanced systemic mastocytosis, including the highly fatal variant mast-cell leukemia. (Funded by Novartis Pharmaceuticals and others; ClinicalTrials.gov number, NCT00782067.).

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药物

Midostaurin