What is the best first-line treatment for POEMS syndrome: autologous transplantation, melphalan and dexamethasone, or lenalidomide and dexamethasone?
What is the best first-line treatment for POEMS syndrome: autologous transplantation, melphalan and dexamethasone, or lenalidomide and dexamethasone?
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POEMS 综合征的最佳一线治疗方法是什么：自体移植、美法仑和地塞米松，还是来那度胺和地塞米松？
DOI：
10.1038/s41375-019-0391-2
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发表时间：
2019-04-01
期刊：
影响因子：
11.4
通讯作者：
Li, Jian
中科院分区：
文献类型：
--
作者：
Zhao, Hao;Huang, Xu-fei;Li, Jian
POEMS综合征是一种罕见的浆细胞病变。本研究比较了347例POEMS综合征患者接受三种一线治疗方案的疗效和生存率：自体干细胞移植（ASCT, N = 165）和美法兰+地塞米松（MDex, N = 79）或来那度胺+地塞米松（LDex, N = 103）。中位随访45个月后，总体血液学完全缓解（CRH）为46.4%，血管内皮生长因子完全缓解（CRV）为55.1%，神经学缓解（R-N）为93.8%。ASCT组CRH（49.7%）优于MDex组（37.7%,p = 0.001）。ASCT组CRV（66.2%）优于MDex组（38.5%,p = 0.001）或LDex组（47.7%,p = 0.008）。三种方案的R-N差异（91.5% vs. 100% vs. 93.8%, p = 0.234）无统计学意义。总3年无进展生存期（PFS）为80.5%，总3年总生存期（OS）为90.8%。ASCT组PFS为87.6%，LDex组为64.9% （p = 0.003）。三种方案的OS无差异（p = 0.079）。在中高危患者中，ASCT的CRH和CRV优于MDex， PFS优于LDex。因此，尽管这三种治疗都有合理的反应和生存，但高风险患者可能从ASCT治疗中获益更多。
POEMS syndrome is a rare plasma cell dyscrasia. This study compared the responses to and survival of 347 POEMS syndrome patients given three first-line treatment regimens: autologous stem cell transplantation (ASCT, N = 165) and melphalan + dexamethasone (MDex, N = 79), or lenalidomide + dexamethasone (LDex, N = 103). After a median 45-month follow-up, overall hematologic complete remission (CRH) was 46.4%, vascular endothelial growth factor complete remission (CRV) was 55.1%, and neurological remission (R-N) was 93.8%. CRH was better with ASCT (49.7%) than with MDex (37.7%, p = 0.001). CRV was better with ASCT (66.2%) than with MDex (38.5%, p = 0.001) or LDex (47.7%, p = 0.008). Differences in R-N achieved by three regimens (91.5% vs. 100% vs. 93.8%, p = 0.234) were not significant. Overall 3-year progression-free survival (PFS) was 80.5% and overall 3-year overall survival (OS) was 90.8%. PFS was 87.6% with ASCT and 64.9% with LDex (p = 0.003). OS in the three regimens did not differ (p = 0.079). In medium-high risk patients, ASCT had better CRH and CRV than MDex, and better PFS than LDex. Therefore, although all three treatments had reasonable responses and survivals, patients with higher risk may benefit more from ASCT treatment.