Patients with myeloid malignancies bearing PDGFRB fusion genes achieve durable long-term remissions with imatinib
Patients with myeloid malignancies bearing PDGFRB fusion genes achieve durable long-term remissions with imatinib
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DOI：
10.1182/blood-2014-02-555607
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发表时间：
2014-06-05
期刊：
影响因子：
20.3
通讯作者：
Seymour, John F.
中科院分区：
文献类型：
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作者：
Cheah, Chan Y.;Burbury, Kate;Seymour, John F.
髓系肿瘤和伴有PDGFRb重排的嗜酸性粒细胞增多症是罕见的费城阴性骨髓增殖性肿瘤。患者通常为男性，形态特征为费城阴性的慢性骨髓增殖性综合征或伴有嗜酸性粒细胞增多的慢性粒单核细胞白血病。涉及PDGFRb的相互易位导致融合基因与结构性激活的受体酪氨酸激酶，对伊马替尼的抑制敏感。我们提出了一项对26名接受伊马替尼治疗的此类患者的最新和扩展的分析。中位随访10.2年(1.8年~17年)，10年总生存率90%(95%可信区间，%~97%)，中位伊马替尼疗程6.6年(0.1年~12年)，6年无进展生存率88%(95%可信区间，65%~96%)。在这些患者中，96%有反应；获得完全细胞遗传学(N 5 13)或分子(n=8)缓解的患者没有一例失去反应或进展为白血病危象。伊马替尼耐受性良好，在PDGFRb重排患者中取得了良好的长期疗效。
Myeloid neoplasms and eosinophilia with rearrangements of PDGFRB are uncommon Philadelphia-negative myeloproliferative neoplasms. Patients are typically male, with morphologic features of a Philadelphia-negative chronic myeloproliferative syndrome or chronic myelomonocytic leukemia with eosinophilia. Reciprocal translocations involving PDGFRB result in fusion genes with constitutively activated receptor tyrosine kinase sensitive to inhibition with imatinib. We present an updated and expanded analysis of a cohort of 26 such patients treated with imatinib. After a median follow-up of 10.2 years (range, 1.8-17 years), the 10-year overall survival rate was 90% (95% confidence interval, 64%-97%); after median imatinib duration of 6.6 years (range, 0.1-12 years), the 6-year progression-free survival rate was 88% (95% confidence interval, 65%-96%). Of the patients, 96% responded; no patients who achieved a complete cytogenetic (n 5 13) or molecular (n = 8) remission lost their response or progressed to blast crisis. Imatinib is well-tolerated and achieves excellent long-term responses in patients with PDGFRB rearrangements.