参考资料:
1.张志军,张志军,等。治疗晚期系统性肥大细胞增多症的新方法[J]。临床药理学:进展与应用,2019,11: 77。
2.林克汉,帕尔达纳尼A,巴特菲尔德J H,等。干扰素、羟基脲、甲磺酸伊马替尼或2-氯脱氧腺苷治疗期间108例系统性肥大细胞增多症成人患者的细胞减灭治疗:结果分析和反应预测[J]。美国血液学杂志,2009,84(12): 790-794。
3.王志平,王志军,等。克拉屈滨治疗成人肥大细胞增多症的长期疗效和安全性[J]。血,2015,126(8): 1009-1016。
4.戈德利,克鲁因-尼莱曼斯H C,乔治T . I,等。米多司桃林治疗晚期系统性肥大细胞增多症的疗效和安全性[J]。新英格兰医学杂志,2016,374(26): 2530-2541。
5.[2]dean gelo D J,George T I,Linder A,等。米多司桃林治疗晚期系统性肥大细胞增多症患者的疗效和安全性:一项二期试验的10年中位随访[J]。白血病,2018,32(2): 470-478。
: border-box;">6.Shomali W, Gotlib J. Response Criteria in Advanced Systemic Mastocytosis: Evolution in the Era of KIT Inhibitors[J]. International Journal of Molecular Sciences, 2021, 22(6): 2983.7.Evans E K, Gardino A K, Kim J L, et al. A precision therapy against cancers driven by KIT/PDGFRA mutations[J]. Science Translational Medicine, 2017, 9(414).
8.Drummond M W, DeAngelo D J, Deininger M W, et al. Preliminary safety and clinical activity in a phase 1 study of Blu-285, a potent, highly-selective inhibitor of KIT D816V in advanced systemic mastocytosis (SM)[J]. Blood, 2016, 128(22): 477.
9.Gotlib J R, Radia D, DeAngelo D J, et al. Avapritinib, a potent and selective inhibitor of KIT D816V, improves symptoms of advanced systemic mastocytosis (AdvSM): analyses of patient reported outcomes (PROs) from the phase 1 (EXPLORER) study using the (AdvSM) symptom assessment form (AdvSM-SAF), a new PRO questionnaire for (AdvSM)[J]. Blood, 2018, 132: 351.
10.Radia D, Deininger M W, Gotlib J, et al. S830 AVAPRITINIB, A POTENT AND SELECTIVE INHIBITOR OF KIT D816V, INDUCES COMPLETE AND DURABLE RESPONSES IN PATIENTS (PTS) WITH ADVANCED SYSTEMIC MASTOCYTOSIS (ADVSM)[J]. HemaSphere, 2019, 3(S1): 368.
11.Gotlib J, Radia D H, George T I, et al. Pure pathologic response is associated with improved overall survival in patients with advanced systemic mastocytosis receiving avapritinib in the phase I EXPLORER study[J]. Blood, 2020, 136(Suppl 1): 37-38.
12.Deangelo D J, Reiter A, Radia D, et al. Abstract CT023 PATHFINDER: Interim analysis of avapritinib (ava) in patients (pts) with advanced systemic mastocytosis (AdvSM)[J]. Cancer Research, 2021, 81(13 Supplement): CT023.
13.Escribano L, lvarez-Twose I, Sánchez-Muoz L, et al. Prognosis in adult indolent systemic mastocytosis: a long-term study of the Spanish Network on Mastocytosis in a series of 145 patients[J]. Journal of Allergy and Clinical Immunology, 2009, 124(3): 514-521.
14.Tefferi A, Shah S, Reichard K K, et al. Smoldering mastocytosis: Survival comparisons with indolent and aggressive mastocytosis[J]. American Journal of Hematology, 2019, 94(1): E1-E2.
15.Akin C, Elberink H O, Gotlib J, et al. Results from PIONEER: a randomized, double-blind, placebo-controlled, phase 2 study of avapritinib in patients with indolent systemic mastocytosis[J]. Blood, 2020, 136.
本文作者丨谭硕