POEMS syndrome is a life-threatening plasma cell neoplasm with the main clinical manifestations of polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy and skin changes. The syndrome is rare and has a poor prognosis. Despite the increasing understanding of POEMS syndrome, the exact pathogenesis is not fully understood, and there is no standard treatment. Systemic therapy, including short-course induction therapy before autologous stem cell transplantation (ASCT) and ASCT, has been proposed as a potentially effective treatment. These therapies are designed to control disease progression, improve symptoms, and improve patient survival.

This study aims to evaluate the efficacy and safety of therapy of short-term induction regimens, including the combination of melphalan and dexamethasone (MD) regimen, immunomodulators (thalidomide or lenalidomide) combined with Dexamethasone (TD or RD regimens), and ASCT for POEMS syndrome patients, providing evidence-based medicine for clinical treatment.

Computer retrieval was conducted in PubMed, Embase, and the Cochrane Library, with the search period ranging from the establishment of the databases to May 2024. At the same time, other search methods were combined to collect randomized controlled trials (RCT), non-RCT, case-control studies, cohort studies, etc. on the systemic therapies for POEMS syndrome. Meta-analysis of single-arm rates, sensitivity analysis, and publication bias analysis were performed using Stata 15.1 software.

Fourteen studies including 794 patients with POEMS syndrome were included in this study. The meta-analysis showed that the response rates for the MD regimen, immune modulators, and ASCT in treating POEMS syndrome were 0.800 (95%CI=0.715-0.874) for hematological responses, 0.872 (95%CI=0.751-0.962) for VEGF responses, 0.796 (95%CI=0.603-0.941) for neurological responses, 0.108 (95%CI=0.000-0.309) for grade 3 or higher adverse reactions, and 0.940 (95%CI=0.908-0.966) for survival to last follow-up. Subgroup analysis showed that the response rates for the three treatments were 0.806, 0.726, and 83.0% for hematological responses; 95.8%, 88.7%, and 80.0% for vascular endothelial growth factor (VEGF) responses; 1.000, 0.642, and 0.926 for neurological responses; and 1.000, 0.978, and 0.908 for survival to last follow-up. Sensitivity analysis showed that the results were robust and were not significantly affected by the exclusion of individual studies. The Egger's test was used to assess publication bias, and the results showed no publication bias for hematological response rates (P=0.777), VEGF response rates (P=0.981), neurological response rates (P=0.060), grade 3 or higher adverse reaction rates (P=0.117), survival to last follow-up (P=0.088), and recurrence rates at last follow-up (P=0.885) .

In systemic therapy for POEMS syndrome, the hematological response rate is good for all three types of treatment, with particularly high neurological response rates for the MD and ASCT regimens. However, the RD regimen has a lower incidence of grade 3 or higher adverse reactions, which is beneficial for long-term treatment. Therefore, the RD regimen can be considered as the first-line treatment option for POEMS syndrome patients, and for young patients who are tolerant, it is recommended to undergo RD regimen-induced treatment followed by high-dose melphalan treatment and ASCT. The RD regimen can be considered as the first-line treatment option for POEMS syndrome patients, and for young patients who are tolerant, it is recommended to undergo RD regimen-induced treatment followed by high-dose melphalan treatment and ASCT treatment.

POEMS综合征是一种危及生命的浆细胞肿瘤，以周围神经病变、脏器肿大、内分泌障碍、M蛋白病变为主要临床表现。该综合征发病率低，且预后不良。尽管对POEMS综合征的认识逐渐增加，但其确切发病机制尚不完全清晰，也无标准治疗方案。全身性系统性治疗包括自体干细胞移植（ASCT）前的短程诱导治疗和ASCT治疗，已被作为潜在的有效治疗手段。这些治疗方法旨在控制病情进展，改善症状，并提高患者生存率。

评价诱导治疗方案马法兰联合地塞米松（MD）方案、免疫调节剂（沙利度胺或来那度胺）联合地塞米松（TD或RD方案），以及ASCT用于POEMS综合征的疗效和安全性。

计算机检索PubMed、Embase、Cochrane Library，检索时限自建库至2024年5月，同时配合其他检索方式，收集POEMS综合征系统性疗法的随机对照试验（RCT）、非RCT、病例对照研究、队列研究等文献，采用Stata 15.1软件进行单组率Meta分析、敏感性分析和发表偏倚分析。

共纳入14项研究，共计794例POEMS综合征患者。Meta分析结果显示：MD方案、免疫调节剂及ASCT用于治疗POEMS综合征的血液学反应率为0.800（95%CI=0.715~0.874），血管内皮生长因子（VEGF）反应率为0.872（95%CI=0.751~0.962），神经系统反应率0.796（95%CI=0.603~0.941），≥3级不良反应发生率为0.108（95%CI=0.000~0.309），末次随访时的存活率为0.940（95%CI=0.908~0.966），末次随访时的复发率为7.2%（95%CI=0.013~0.160），5年总生存率为0.917（95%CI=0.893~0.941）。亚组分析显示：血液学反应率方面，以上三种疗法分别为0.806、0.726、0.830；VEGF反应率方面，三者分别为0.958、0.887、0.800；神经系统反应率方面，三者分别为1.000、0.642、0.926；末次随访时的存活率方面，三者分别为1.000、0.978、0.908。敏感性分析显示，在逐一剔除文献后，所得结果未受到明显影响，提示本研究结果稳健。采用Egger's检验评估发表偏倚风险，经过Egger's检验，血液学反应率（P=0.777）、VEGF反应率（P=0.981）、神经系统反应率（P=0.060）、≥3级不良反应发生率（P=0.117）、末次随访时的存活率（P=0.088）及末次随访时的复发率（P=0.885）均不存在发表偏倚。

在POEMS综合征全身性系统性治疗中，血液学反应率方面，MD方案、免疫调节剂及ASCT的治疗反应率均佳，尤其是MD方案和ASCT神经系统反应率更高，但RD方案3级以上不良反应较低，有利于长期治疗。因此，RD方案可作为POEMS综合征患者的一线治疗选择，对于年轻可耐受患者，推荐RD方案诱导治疗后接受大剂量马法兰治疗并进行ASCT。