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1
Imatinib has limited therapeutic activity for hypereosinophilic syndrome patients with unknown or negative PDGFRalpha mutation status.伊马替尼对血小板衍生生长因子受体α（PDGFRα）突变状态未知或呈阴性的高嗜酸性粒细胞综合征患者的治疗活性有限。
Leuk Res. 2009 Jun;33(6):837-9. doi: 10.1016/j.leukres.2008.10.004. Epub 2008 Nov 14.
2
The efficacy of imatinib mesylate in patients with FIP1L1-PDGFRalpha-positive hypereosinophilic syndrome. Results of a multicenter prospective study.甲磺酸伊马替尼治疗FIP1L1-PDGFRα阳性高嗜酸性粒细胞综合征患者的疗效。一项多中心前瞻性研究的结果
Haematologica. 2007 Sep;92(9):1173-9. doi: 10.3324/haematol.11420. Epub 2007 Aug 1.
3
FIP1L1-PDGFRalpha alone or with other genetic abnormalities reveals disease progression in chronic eosinophilic leukemia but good response to imatinib.单独的FIP1L1-PDGFRα或伴有其他基因异常均提示慢性嗜酸性粒细胞白血病的疾病进展，但对伊马替尼反应良好。
Chin Med J (Engl). 2008 May 20;121(10):867-73.
4
Antitumor activity of S116836, a novel tyrosine kinase inhibitor, against imatinib-resistant FIP1L1-PDGFRα-expressing cells.新型酪氨酸激酶抑制剂S116836对表达伊马替尼耐药的FIP1L1-PDGFRα细胞的抗肿瘤活性。
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Success of short-term, higher-dose imatinib mesylate to induce clinical response in FIP1L1-PDGFRalpha-negative hypereosinophilic syndrome.短期、高剂量甲磺酸伊马替尼诱导FIP1L1-PDGFRα阴性高嗜酸性粒细胞综合征临床反应的成功案例。
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Imatinib mesylate may induce long-term clinical response in FIP1L1-PDGFRα-negative hypereosinophilic syndrome.甲磺酸伊马替尼可能诱导 FIP1L1-PDGFRα 阴性嗜酸性粒细胞增多综合征的长期临床缓解。
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7
The FIP1L1-PDGFRalpha fusion tyrosine kinase in hypereosinophilic syndrome and chronic eosinophilic leukemia: implications for diagnosis, classification, and management.嗜酸性粒细胞增多综合征和慢性嗜酸性粒细胞白血病中的FIP1L1-PDGFRα融合酪氨酸激酶：对诊断、分类和管理的意义。
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8
Safety and efficacy of imatinib in chronic eosinophilic leukaemia and hypereosinophilic syndrome: a phase-II study.伊马替尼治疗慢性嗜酸性粒细胞白血病和高嗜酸性粒细胞综合征的安全性和有效性：一项II期研究。
Br J Haematol. 2008 Dec;143(5):707-15. doi: 10.1111/j.1365-2141.2008.07294.x. Epub 2008 Oct 17.
9
Systemic mastocytosis (SM) associated with chronic eosinophilic leukemia (SM-CEL): detection of FIP1L1/PDGFRalpha, classification by WHO criteria, and response to therapy with imatinib.与慢性嗜酸性粒细胞白血病相关的系统性肥大细胞增多症（SM-CEL）：FIP1L1/PDGFRα的检测、按世界卫生组织标准分类以及对伊马替尼治疗的反应
Leuk Res. 2006 Sep;30(9):1201-5. doi: 10.1016/j.leukres.2005.11.014. Epub 2006 Jan 6.
10
Cessation of imatinib mesylate may lead to sustained hematologic and molecular remission in FIP1L1-PDGFRA-mutated hypereosinophilic syndrome.甲磺酸伊马替尼的停药可能导致FIP1L1-PDGFRA突变的嗜酸性粒细胞增多综合征出现持续的血液学和分子学缓解。
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引用本文的文献

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Long-Term Efficacy and Safety of Benralizumab Treatment for PDGFRA-Negative Hypereosinophilic Syndrome.贝那利珠单抗治疗血小板衍生生长因子受体A阴性高嗜酸性粒细胞综合征的长期疗效和安全性
J Allergy Clin Immunol Pract. 2025 Jun;13(6):1421-1429.e2. doi: 10.1016/j.jaip.2025.03.016. Epub 2025 Mar 20.
2
Tumour-agnostic kinase inhibitors.肿瘤非特异性激酶抑制剂。
Nat Rev Drug Discov. 2025 Mar 6. doi: 10.1038/s41573-025-01147-y.
3
Imatinib is effective in some PDGFRA/B-negative hypereosinophilic syndromes: A step closer to unveiling underlying mechanisms.伊马替尼对某些血小板衍生生长因子受体A/B阴性的高嗜酸性粒细胞综合征有效：向揭示潜在机制迈进了一步。
Br J Haematol. 2024 Dec;205(6):2136-2138. doi: 10.1111/bjh.19853. Epub 2024 Oct 28.
4
Phase II trial of imatinib mesylate in patients with PDGFRA/B-negative hypereosinophilic syndrome.甲磺酸伊马替尼治疗血小板衍生生长因子受体A/B阴性高嗜酸性粒细胞综合征患者的II期试验。
Br J Haematol. 2024 Dec;205(6):2305-2314. doi: 10.1111/bjh.19828. Epub 2024 Oct 10.
5
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Clin Rheumatol. 2014 Nov;33(11):1685-8. doi: 10.1007/s10067-014-2566-6. Epub 2014 Mar 8.

本文引用的文献

1
The efficacy of imatinib mesylate in patients with FIP1L1-PDGFRalpha-positive hypereosinophilic syndrome. Results of a multicenter prospective study.甲磺酸伊马替尼治疗FIP1L1-PDGFRα阳性高嗜酸性粒细胞综合征患者的疗效。一项多中心前瞻性研究的结果
Haematologica. 2007 Sep;92(9):1173-9. doi: 10.3324/haematol.11420. Epub 2007 Aug 1.
2
Characterization of three new imatinib-responsive fusion genes in chronic myeloproliferative disorders generated by disruption of the platelet-derived growth factor receptor beta gene.血小板衍生生长因子受体β基因破坏产生的慢性骨髓增殖性疾病中三种新的伊马替尼反应性融合基因的特征分析。
Haematologica. 2007 Feb;92(2):163-9. doi: 10.3324/haematol.10980.
3
The results of imatinib therapy for patients with primary eosinophilic disorders.伊马替尼治疗原发性嗜酸性粒细胞疾病患者的结果。
Eur J Haematol. 2006 Jun;76(6):535-6. doi: 10.1111/j.1600-0609.2006.00652.x. Epub 2006 Apr 11.
4
Identification of a novel imatinib responsive KIF5B-PDGFRA fusion gene following screening for PDGFRA overexpression in patients with hypereosinophilia.在对嗜酸性粒细胞增多症患者进行血小板衍生生长因子受体α（PDGFRA）过表达筛查后，鉴定出一种新型的对伊马替尼敏感的驱动蛋白家族成员5B（KIF5B）-血小板衍生生长因子受体α（PDGFRA）融合基因。
Leukemia. 2006 May;20(5):827-32. doi: 10.1038/sj.leu.2404154.
5
FIP1L1-PDGFRA in eosinophilic disorders: prevalence in routine clinical practice, long-term experience with imatinib therapy, and a critical review of the literature.嗜酸性疾病中的FIP1L1-PDGFRA：常规临床实践中的患病率、伊马替尼治疗的长期经验及文献综述
Leuk Res. 2006 Aug;30(8):965-70. doi: 10.1016/j.leukres.2005.11.011. Epub 2006 Jan 6.
6
Imatinib mesylate as a novel treatment option for hypereosinophilic syndrome: two case reports and a comprehensive review of the literature.甲磺酸伊马替尼作为嗜酸性粒细胞增多综合征的一种新型治疗选择：两例病例报告及文献综述
Ann Hematol. 2006 Jan;85(1):1-16. doi: 10.1007/s00277-005-1084-7. Epub 2005 Sep 1.
7
The hypereosinophilic syndrome: fluorescence in situ hybridization detects the del(4)(q12)-FIP1L1/PDGFRA but not genomic rearrangements of other tyrosine kinases.高嗜酸性粒细胞综合征：荧光原位杂交检测到del(4)(q12)-FIP1L1/PDGFRA，但未检测到其他酪氨酸激酶的基因组重排。
Haematologica. 2005 May;90(5):596-601.
8
Molecular characterization of the idiopathic hypereosinophilic syndrome (HES) in 35 French patients with normal conventional cytogenetics.35例常规细胞遗传学正常的法国特发性嗜酸性粒细胞增多综合征（HES）患者的分子特征分析
Leukemia. 2005 May;19(5):792-8. doi: 10.1038/sj.leu.2403722.
9
FIP1L1-PDGFRA fusion: prevalence and clinicopathologic correlates in 89 consecutive patients with moderate to severe eosinophilia.FIP1L1-PDGFRA融合基因：89例连续性中重度嗜酸性粒细胞增多患者中的发生率及临床病理相关性
Blood. 2004 Nov 15;104(10):3038-45. doi: 10.1182/blood-2004-03-0787. Epub 2004 Jul 29.
10
Molecular remission and reversal of myelofibrosis in response to imatinib mesylate treatment in patients with the myeloproliferative variant of hypereosinophilic syndrome.伊马替尼甲磺酸盐治疗嗜酸性粒细胞增多综合征骨髓增殖性变异型患者后分子缓解及骨髓纤维化逆转
Blood. 2004 Jan 15;103(2):473-8. doi: 10.1182/blood-2003-08-2798. Epub 2003 Sep 22.

伊马替尼对血小板衍生生长因子受体α（PDGFRα）突变状态未知或呈阴性的高嗜酸性粒细胞综合征患者的治疗活性有限。

Imatinib has limited therapeutic activity for hypereosinophilic syndrome patients with unknown or negative PDGFRalpha mutation status.

作者信息

Jain Nitin, Cortes Jorge, Quintás-Cardama Alfonso, Manshouri Taghi, Luthra Raja, Garcia-Manero Guillermo, Kantarjian Hagop, Verstovsek Srdan

机构信息

Department of Leukemia, University of Texas, M.D. Anderson Cancer Center, Houston, TX 77030, USA.

出版信息

Leuk Res. 2009 Jun;33(6):837-9. doi: 10.1016/j.leukres.2008.10.004. Epub 2008 Nov 14.

DOI:10.1016/j.leukres.2008.10.004
PMID:19013640
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC4422052/
Abstract

Hypereosinophilic syndrome (HES) is characterized by sustained non-clonal blood and tissue eosinophilia, leading to end-organ damage. With a molecular/cytogenetic clonality marker, the disease is classified as chronic eosinophilic leukemia (CEL). Efficacy of imatinib mesylate is well established in CEL with FIP1L1-platelet-derived growth factor-alpha (PDGFRalpha) rearrangement. We treated with imatinib 18 HES patients (11 PDGFRalpha-negative and 7 PDGFRalpha-status unknown). One patient with unknown PDGFRalpha status achieved complete hematologic response, and two (one PDGFRalpha negative and one status unknown) achieved partial hematologic response. Our results confirm low response rate to imatinib in HES patients with unknown or negative PDGFRalpha status, and underscore the need for new therapeutic options for this disorder.

摘要

高嗜酸性粒细胞综合征（HES）的特征是血液和组织中嗜酸性粒细胞持续非克隆性增多，导致终末器官损害。使用分子/细胞遗传学克隆性标志物，该疾病被分类为慢性嗜酸性粒细胞白血病（CEL）。甲磺酸伊马替尼在伴有FIP1L1-血小板衍生生长因子α（PDGFRα）重排的CEL中的疗效已得到充分证实。我们用伊马替尼治疗了18例HES患者（11例PDGFRα阴性和7例PDGFRα状态未知）。1例PDGFRα状态未知的患者获得完全血液学缓解，2例（1例PDGFRα阴性和1例状态未知）获得部分血液学缓解。我们的结果证实，PDGFRα状态未知或阴性的HES患者对伊马替尼的缓解率较低，并强调了针对这种疾病需要新的治疗选择。