POEMS（多发性神经病、器官肿大、内分泌病、单克隆丙种球蛋白病和皮肤变化）综合征是浆细胞恶液质的一种罕见形式，通常采用大剂量化疗和自体干细胞移植 (ASCT) 进行治疗。 ASCT 取得了令人满意且持续的治疗结果。然而，大量患者最终会出现疾病进展，需要二线治疗。因此，确定 ASCT 后获得最佳长期生存的患者将具有进一步的益处。通过长期随访，充分揭示接受 ASCT 的患者的大系列结果。239 的长期结果对单中心接受 ASCT 的初诊 POEMS 综合征患者进行回顾性评估，血液学完全缓解率 (CRH) 和 VEGF (CRV) 完全缓解率分别为 57.3% 和 68.6%，其中 90.5% 的患者达到总体临床缓解。中位随访时间94个月后，5年OS（总生存）率为92.8%，5年TTNT（到下线治疗的时间）率为72.2%（中位TTNT：96个月） 。实现 CRH（5 年 TTNT 率 82.5% vs. 60.7%，P < 0.0001）或 CRV（5 年 TTNT 率 83.7% vs. 54.2%，P < 0.0001）的患者比非 CR 组患者具有更好的生存结果。双重血液学和 VEGF 完全缓解为生存带来进一步的益处（中位 TTNT：129 个月 vs. 68 个月，P < 0.0001）。记录了 7 例第二原发恶性肿瘤，均为实体瘤。一线 ASCT 为 POEMS 综合征患者带来了优异的长期生存率，其中实现血液学和 VEGF 双 CR 的患者生存率最高。版权所有 © 2023 年。由爱思唯尔公司出版。

POEMS (polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy and skin changes) syndrome is a rare form of plasma cell dyscrasia often treated with high-dose chemotherapy and autologous stem cell transplantation (ASCT). ASCT has resulted in satisfactory and sustained therapeutic outcomes. However, a substantial number of patients eventually experience disease progression, requiring second-line treatment. Therefore, it would be of further benefit to identify patients who will acquire the best long-term survival after ASCT.To fully reveal the outcomes of patients receiving ASCT in a large series with long-term follow-up.Long-term outcomes of 239 patients with newly diagnosed POEMS syndrome receiving ASCT at a single center were retrospectively evaluated.Rates of hematological complete response (CRH) and VEGF (CRV) complete response were 57.3% and 68.6%, respectively, with 90.5% of patients achieving an overall clinical response. After the median follow-up time of 94 months, the 5-year OS (overall survival) rate was 92.8%, and the 5-year TTNT (time to next-line treatment) rate was 72.2% (median TTNT: 96 months). Patients achieving CRH (5-year TTNT rate 82.5% vs. 60.7%, P < 0.0001) or CRV (5-year TTNT rate 83.7% vs. 54.2%, P < 0.0001) had better survival outcomes than non-CR group patients. Dual hematological and VEGF complete responses bring further benefit for survival (median TTNT: 129 months vs. 68 months, P < 0.0001). Seven cases of second primary malignancy were recorded, all of which were solid tumors.Front-line ASCT resulted in excellent long-term survival in patients with POEMS syndrome, and the best survival was observed in those achieving dual hematological and VEGF CRs.Copyright © 2023. Published by Elsevier Inc.