What is the best first-line treatment for POEMS syndrome: autologous transplantation, melphalan and dexamethasone, or lenalidomide and dexamethasone?

作者：Hao Zhao, Xufei Huang, Xuemin Gao, Hao Cai, Lu Zhang, Jun Feng, Xinxin Cao, Daobin Zhou, Li J · 发表于：Leukemia · 年份：2019 · DOI：10.1038/s41375-019-0391-2 · 被引用次数：62 · 研究领域：Multiple Myeloma Research and Treatments、Peripheral Neuropathies and Disorders、Platelet Disorders and Treatments

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 (CR H ) was 46.4%, vascular endothelial growth factor complete remission (CR V ) was 55.1%, and neurological remission (R N ) was 93.8%. CR H was better with ASCT (49.7%) than with MDex (37.7%, p = 0.001). CR V 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 CR H and CR V 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.