Imatinib in myeloid/lymphoid neoplasms with eosinophilia and rearrangement of PDGFRB in chronic or blast phase
Imatinib in myeloid/lymphoid neoplasms with eosinophilia and rearrangement of PDGFRB in chronic or blast phase
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DOI：
10.1007/s00277-017-3067-x
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发表时间：
2017-09-01
影响因子：
3.5
通讯作者：
Metzgeroth, Georgia
中科院分区：
文献类型：
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作者：
Jawhar, Mohamad;Naumann, Nicole;Metzgeroth, Georgia
我们评估了 22 例伴有嗜酸性粒细胞增多和 PDGFRB 重排的骨髓/淋巴肿瘤患者的临床特征和伊马替尼治疗结果。中位年龄为 49 岁（范围 20-80 岁），其中 91% 为男性。鉴定出 15 种不同的 PDGFRB 融合基因。 4/19 (21%) 病例不存在嗜酸性粒细胞增多，仅 11/19 (58%) 病例嗜酸性粒细胞>= 1.5x10(9)/L。服用伊马替尼后，17/17 (100%) 的慢性期患者在中位 2 个月（范围 0-13）aEuroi 后达到完全血液学缓解。中位 10 个月（范围 3-34）和 19 个月（范围 7-110）后，分别有 12/13（92%）和 12/14（86%）的患者达到完全细胞遗传学缓解和/或完全分子缓解（通过 RT-PCR）。对于急变期患者（骨髓，n = 2；淋巴，n = 3），治疗包括伊马替尼（n = 5）、强化化疗（n = 3）和/或同种异体干细胞移植（n = 3）的组合。所有 3 名移植患者（复杂核型，n = 2）均出现早期复发。最初，患者接受伊马替尼 400 mg/天（n = 15）或 100 mg/天（n = 7）治疗，随访期间 9 名患者的剂量从 400 mg/天减少至 100 mg/天。中位治疗71个月（范围1-135）后，5年生存率为83%； 4/22 (18%) 患者因疾病进展 (n = 3) 或缓解期合并症 (n = 1) 死亡（慢性期；n = 2；急变期，n = 2）。值得注意的是，3/4 的患者具有复杂的核型。总之，PDGFRB重排的骨髓/淋巴肿瘤最重要的特征包括（a）男性为主，（b）经常缺乏嗜酸性粒细胞增多，（c）处于慢性或急变期，（d）对低剂量伊马替尼的快速反应和长期缓解，以及（e）复杂核型可能产生的不良预后影响。
We evaluated clinical characteristics and outcome on imatinib of 22 patients with myeloid/lymphoid neoplasms with eosinophilia and rearrangement of PDGFRB. Median age was 49 years (range 20-80), 91% were male. Fifteen different PDGFRB fusion genes were identified. Eosinophilia was absent in 4/19 (21%) cases and only 11/19 (58%) cases had eosinophils >= 1.5x10(9)/L. On imatinib, 17/17 (100%) patients in chronic phase achieved complete hematologic remission after median 2 months (range 0-13)aEuroi. Complete cytogenetic remission and/or complete molecular remission by RT-PCR were achieved in 12/13 (92%) and 12/14 patients (86%) after median 10 (range 3-34) and 19 months (range 7-110), respectively. In patients with blast phase (myeloid, n = 2; lymphoid, n = 3), treatment included combinations of imatinib (n = 5), intensive chemotherapy (n = 3), and/or allogeneic stem cell transplantation (n = 3). All 3 transplanted patients (complex karyotype, n = 2) experienced early relapse. Initially, patients were treated with imatinib 400 mg/day (n = 15) or 100 mg/day (n = 7), the dose was reduced from 400 mg/day to 100 mg/day during follow-up in 9 patients. After a median treatment of 71 months (range 1-135), the 5-year survival rate was 83%; 4/22 (18%) patients died (chronic phase; n = 2; blast phase, n = 2) due to progression (n = 3) or comorbidity while in remission (n = 1). Of note, 3/4 patients had a complex karyotype. In summary, the most important characteristics of myeloid/lymphoid neoplasms with rearrangement of PDGFRB include (a) male predominance, (b) frequent lack of hypereosinophilia, (c) presentation in chronic or blast phase, (d) rapid responses and long-term remission on low-dose imatinib, and (e) possible adverse prognostic impact of a complex karyotype.