Fang Fang, Lan Xiao-Xi, Hu Rong-Hua, Hui Wu-Han, Zhao Hong, Guo Yi-Xian, Ji Bing-Xin, Liu Hong-Jun, Su Li, Sun Wan-Ling
Department of Hematology, Xuanwu Hospital, Capital Medical University, Beijing, China.
Center for Evidence-Based Medicine, Xuanwu Hospital, Capital Medical University, Beijing, China.
Ther Adv Neurol Disord. 2024 Jan 27;17:17562864231219151. doi: 10.1177/17562864231219151. eCollection 2024.
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.
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).
We conducted a retrospective study using real-world data from Capital Medical University, Xuanwu Hospital.
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.
The overall vascular endothelial growth factor (VEGF) response rate of this new treatment mode was 100%. The cumulative VEGF complete remission (CR) rate was 67.9%, and the cumulative complete hematological response (CR) 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% CR rate (median time from diagnosis to CR = 130 days) and 66.7% CR rate (median time from diagnosis to CR = 218 days). In addition, the VEGF response was less than the partial remission (PR) 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.
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.
由于多神经病、器官肿大、内分泌病、单克隆丙种球蛋白病和皮肤改变（POEMS）综合征较为罕见，尽管指南中有多种选择，但尚未确定最佳的一线治疗方案。首选治疗方法因医生的偏好和经验而异。
第一，分析一种新的POEMS综合征治疗模式的疗效，该模式采用四个周期的治疗作为诱导方案，随后对符合移植条件的患者进行序贯移植作为巩固方案，或对不符合移植条件的患者接受另外两个周期的治疗。第二，比较含蛋白酶体抑制剂（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）方案的疗效和安全性。
我们使用首都医科大学宣武医院的真实世界数据进行了一项回顾性研究。
共有34例新诊断的POEMS综合征患者符合Dispenzieri的诊断标准，纳入2013年7月至2021年3月期间完成至少四个周期治疗的患者。
这种新治疗模式的总体血管内皮生长因子（VEGF）反应率为100%。VEGF累积完全缓解（CR）率为67.9%，累积完全血液学缓解（CR）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方案的CR率为75%（从诊断到CR的中位时间=130天），CD±T方案的CR率为66.7%（从诊断到CR的中位时间=218天）。此外，VEGF反应低于四个周期诱导治疗后的部分缓解（PR），以及巩固治疗1年后总体神经功能受限量表评分下降少于3分，是独立的不良预后因素。
POEMS综合征患者对硼替佐米耐受性良好。与CD±T方案相比，BCD作为诱导方案可实现更好的VEGF反应和更早的血液学缓解。自体干细胞移植用作巩固治疗可进一步提高神经和血液学缓解率，并带来更好的OS和PFS。
