■由于多发性神经病的罕见性,器官肿大,内分泌病,单克隆丙种球蛋白病,和皮肤变化(POEMS)综合征,最佳的一线治疗尚未建立,尽管指南中有几种选择。优选的治疗根据医生的偏好和轶事而变化。
■首先,分析以四个周期治疗为诱导方案的POEMS综合征治疗新模式的疗效,随后序贯移植作为符合移植条件的患者的巩固方案,或接受另一个两个周期的治疗移植不合格的患者。第二,比较蛋白酶体抑制剂(硼替佐米-环磷酰胺-地塞米松,BCD)或不含蛋白酶体抑制剂(环磷酰胺-地塞米松±沙利度胺,CD±T)。
■我们使用首都医科大学的真实数据进行了回顾性研究,宣武医院。
■共有34名新诊断的POEMS综合征患者符合Dispenzieri的诊断标准,以及从2013年7月至2021年3月完成至少4个周期治疗的患者纳入.
■这种新治疗模式的总体血管内皮生长因子(VEGF)反应率为100%。累计VEGF完全缓解(CRV)率为67.9%,累积完全血液学应答(CRH)率为55.6%。在中位49个月的随访期间,5年总生存率(OS)为90.7%,3年无进展生存率(PFS)为78.4%,5年PFS率为73.8%。BCD方案实现了75%的CRV率(从诊断到CRV的中位时间=130天)和66.7%的CRH率(从诊断到CRH的中位时间=218天)。此外,4个周期诱导治疗后,VEGF反应小于部分缓解(PRV),which,合并治疗后1年,总体神经限制量表减少少于3分,是一个独立的不良预后因素。
■POEMS综合征患者对硼替佐米的耐受性良好。与CD±T方案相比,BCD作为诱导方案实现了更好的VEGF反应和更早的血液学缓解。自体干细胞移植作为巩固治疗,进一步提高了神经和血液学的缓解率,导致更好的OS和PFS。
UNASSIGNED: Due to the rarity of polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes (POEMS) syndrome, the best first-line treatment has not been established, although there are several options in guidelines. The preferred treatments vary according to the preference of the physician and anecdote.
UNASSIGNED: First, to analyze the efficacy of a new treatment mode in POEMS syndrome that uses the four-cycle treatment as the induction regimen, followed by sequential transplantation as the consolidation regimen for transplantation-eligible patients, or received another two-cycle treatment for transplantation-ineligible patients. Second, to compare the efficacy and safety of regimens with a proteasome inhibitor (bortezomib-cyclophosphamide-dexamethasone, BCD) or without a proteasome inhibitor (cyclophosphamide-dexamethasone ± thalidomide, CD ± T).
UNASSIGNED: We conducted a retrospective study using real-world data from Capital Medical University, Xuanwu Hospital.
UNASSIGNED: A total of 34 newly diagnosed POEMS syndrome patients met Dispenzieri\'s diagnostic criteria, and those who completed at least four cycles of treatment from July 2013 to March 2021 were included.
UNASSIGNED: The overall vascular endothelial growth factor (VEGF) response rate of this new treatment mode was 100%. The cumulative VEGF complete remission (CRV) rate was 67.9%, and the cumulative complete hematological response (CRH) rate was 55.6%. During the median 49-month follow-up, the 5-year-overall survival (OS) rate was 90.7%, the 3-year-progression-free survival (PFS) rate was 78.4%, and the 5-year-PFS rate was 73.8%. The BCD regimen achieved a 75% CRV rate (median time from diagnosis to CRV = 130 days) and 66.7% CRH rate (median time from diagnosis to CRH = 218 days). In addition, the VEGF response was less than the partial remission (PRV) after four-cycle induction treatment, which, together with a decrease on the Overall Neurological Limitation Scale of less than three points 1 year after consolidation treatment, was an independent poor prognostic factor.
UNASSIGNED: Bortezomib was well-tolerated by patients with POEMS syndrome. Compared with CD ± T regimen, BCD as the induction regimen achieved better VEGF response and earlier hematological remission. Autologous stem cell transplantation used as consolidation therapy further improved the neurological and hematological remission rates, resulting in better OS and PFS.