Efficacy and safety of BD regimen sequential autologous hematopoietic stem cell transplantation for POEMS syndrome
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摘要: 目的 探讨蛋白酶体抑制剂硼替佐米联合地塞米松方案(BD方案)序贯自体造血干细胞移植(autologous hematopoietic stem cell transplantation，auto-HSCT)治疗POEMS综合征的安全性及疗效的临床观察。方法 回顾性分析2019年2月—2023年11月在甘肃省人民医院确诊的6例POEMS综合征患者的临床特征、诊治方案及随访结果，并结合相关文献进行讨论。结果 6例患者中男1例，女5例，中位年龄为49(37~58)岁。6例患者均接受BD方案化疗至少2个周期，均采用环磷酰胺联合粒细胞集落刺激因子动员，获得中位单个核细胞数3.5×108/kg和中位CD34+细胞数3.6×106/kg。后经大剂量马法兰预处理后行auto-HSCT。所有患者化疗及移植期间耐受性好，粒细胞植入中位时间为+10.5 d，血小板植入中位时间为+12.5 d，无移植相关死亡。中位随访时间34(16~57)个月，总生存率为83%(5/6)。结论 6例POEMS综合征患者经BD方案序贯auto-HSCT后血管内皮生长因子完全达到正常，BD方案序贯auto-HSCT治疗POEMS综合征是一种安全有效的选择。Abstract: Objective To explore the safety and efficacy of proteasome inhibitor bortezomib combined with dexamethasone(BD) regimen followed by autologous hematopoietic stem cell transplantation(auto-HSCT) in the treatment of POEMS syndrome.Methods The clinical characteristics, diagnosis and treatment regimens and follow-up results of 6 patients with POEMS syndrome diagnosed in Gansu Provincial Hospital from February 2019 to November 2023 were retrospectively analyzed, and the relevant literature was discussed.Results Among the 6 patients, 5 were female and 1 was male, with a median age of 49 (37~58) years old. All 6 patients received BD chemotherapy for at least 2 cycles, and all 6 patients were mobilized with cyclophosphamide combined with granulocyte colony stimulating factor, and the median number of mononuclear cells was 3.5×108/kg and the median number of CD34+ cells was 3.6×106/kg. After high-dose melphalan preconditioning, auto-HSCT was performed. All patients had good tolerance during chemotherapy and transplantation, with a median time of granulocyte implantation +10.5 d, a median time of platelet implantation +12.5 d, and no transplantation-related death. The median follow-up time was 34(16~57)months, and the overall survival rate was 83%(5/6).Conclusion After 6 patients with POEMS syndrome underwent the BD regimen followed by auto-HSCT, the normalization of the serum vascular endothelial growth factor(VEGF) level, the complete response of VEGF. The BD regimen followed by auto-HSCT is a safe and effective option for the treatment of POEMS syndrome.
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表 1 6例患者的临床特征
病例 性别 年龄/岁 ONLS得分/分 免疫固定电泳 淋巴结活检 硬化性骨病变 VEGF/(pg/mL) 1 女 58 7 IgA-λ / 有 777.88 2 女 53 4 IgA-λ / 有 787.17 3 女 45 3 IgA-λ / 无 698.55 4 女 37 2 IgA-λ Castleman病 无 800.00 5 男 39 1 IgA-λ Castleman病 无 763.16 6 女 57 2 IgG-κ / 有 260.07 注：/表示未做。 
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表 2 疗效评估
病例 免疫固定电泳 VEGF/(pg/mL) 中期评估 auto-HSCT后评估 确诊时 中期 HSCT后 中期 HSCT后 免疫固定电泳 VEGF 免疫固定电泳 VEGF ONLS得分/分 1 + ± - 172.85 90.07 NCRH PRV CRH CRV 2 2 + - - 180.02 100.96 CRH PRV CRH CRV 1 3 + + ± 300.00 150.90 NCRH PRV NCRH CRV 0 4 + - - 145.21 91.79 CRH CRV CRH CRV 0 5 + + ± 167.23 96.76 NCRH PRV NCRH CRV 0 6 + - - 197.84 109.73 CRH NRV CRH CRV 0 注：VEGF正常参考范围为0~160 pg/mL。
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