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除米哚妥林之外:阿伐替尼在系统性肥大细胞增多症治疗中的作用

Beyond Midostaurin: Role of Avapritinib in Managing Systemic Mastocytosis.

作者信息

Pokima Ngowari, Khattar Georges, Keesari Praneeth R, Khan Salman, Asogwa Nnedindu, Niazi Muhammad, Zheng Ruifang, Dai Qun

机构信息

Internal Medicine, Staten Island University Hospital, Staten Island, USA.

Hematology and Medical Oncology, Northwell Health/Staten Island University Hospital, Staten Island, USA.

出版信息

Cureus. 2024 May 12;16(5):e60161. doi: 10.7759/cureus.60161. eCollection 2024 May.

DOI:10.7759/cureus.60161
PMID:38868249
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11166541/
Abstract

We present a case of an adult male who presented with pancytopenia accompanied by symptomatic anemia, necessitating chronic transfusions. He was diagnosed with systemic mastocytosis with an associated hematologic neoplasm. Following an inadequate response to midostaurin therapy, the patient was initiated on the newly approved avapritinib. The patient showed significant improvements in all three blood cell lines; however, he developed leg edema, blepharedema, and gum bleeding on this medication. This case underscores the intricacies of managing a patient with advanced systemic mastocytosis, the emerging role of highly selective KIT inhibition in its treatment, and the practical management of adverse medication effects.

摘要

我们报告一例成年男性病例,该患者表现为全血细胞减少并伴有症状性贫血,需要长期输血。他被诊断为系统性肥大细胞增多症并伴有相关血液肿瘤。在对米哚妥林治疗反应不佳后,该患者开始使用新批准的阿伐替尼。患者的所有三种血细胞系均有显著改善;然而,他在使用这种药物时出现了腿部水肿、眼睑水肿和牙龈出血。该病例强调了管理晚期系统性肥大细胞增多症患者的复杂性、高选择性KIT抑制在其治疗中的新兴作用以及药物不良反应的实际管理。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c5a3/11166541/5d3ff9f92b44/cureus-0016-00000060161-i03.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c5a3/11166541/5e070109bff0/cureus-0016-00000060161-i01.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c5a3/11166541/6857765cc2a3/cureus-0016-00000060161-i02.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c5a3/11166541/5d3ff9f92b44/cureus-0016-00000060161-i03.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c5a3/11166541/5e070109bff0/cureus-0016-00000060161-i01.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c5a3/11166541/6857765cc2a3/cureus-0016-00000060161-i02.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c5a3/11166541/5d3ff9f92b44/cureus-0016-00000060161-i03.jpg

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本文引用的文献

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Efficacy of avapritinib versus best available therapy in the treatment of advanced systemic mastocytosis.阿伐普利替尼对比最佳可用疗法治疗晚期系统性肥大细胞增多症的疗效。
Leukemia. 2022 Aug;36(8):2108-2120. doi: 10.1038/s41375-022-01615-z. Epub 2022 Jul 5.
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Indirect treatment comparisons of avapritinib versus midostaurin for patients with advanced systemic mastocytosis.阿伐替尼与米哚妥林用于晚期系统性肥大细胞增多症患者的间接治疗比较。
Future Oncol. 2022 Apr;18(13):1583-1594. doi: 10.2217/fon-2021-1509. Epub 2022 Feb 4.
3
Safety and efficacy of avapritinib in advanced systemic mastocytosis: the phase 1 EXPLORER trial.
在晚期系统性肥大细胞增多症中阿伐普利替尼的安全性和疗效:EXPLORER 期试验。
Nat Med. 2021 Dec;27(12):2183-2191. doi: 10.1038/s41591-021-01538-9. Epub 2021 Dec 6.
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Inhibitory effects of midostaurin and avapritinib on myeloid progenitors derived from patients with KIT D816V positive advanced systemic mastocytosis.米哚妥林和 avapritinib 对 KIT D816V 阳性晚期系统性肥大细胞增多症患者来源的髓系祖细胞的抑制作用。
Leukemia. 2019 May;33(5):1195-1205. doi: 10.1038/s41375-019-0450-8. Epub 2019 Mar 25.
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Midostaurin: A Multiple Tyrosine Kinases Inhibitor in Acute Myeloid Leukemia and Systemic Mastocytosis.米哚妥林:一种用于急性髓系白血病和系统性肥大细胞增多症的多酪氨酸激酶抑制剂。
Recent Results Cancer Res. 2018;212:199-214. doi: 10.1007/978-3-319-91439-8_10.
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A precision therapy against cancers driven by mutations.针对驱动突变的癌症的精准疗法。
Sci Transl Med. 2017 Nov 1;9(414). doi: 10.1126/scitranslmed.aao1690.
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Mastocytosis: 2016 updated WHO classification and novel emerging treatment concepts.肥大细胞增多症:2016年世界卫生组织更新分类及新出现的治疗理念。
Blood. 2017 Mar 16;129(11):1420-1427. doi: 10.1182/blood-2016-09-731893. Epub 2016 Dec 28.
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Midostaurin in Advanced Systemic Mastocytosis.米哚妥林治疗晚期系统性肥大细胞增多症
N Engl J Med. 2016 Jun 30;374(26):2605-7. doi: 10.1056/NEJMc1515403.
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