ixazomib and POEMS-Syndrome

To investigate the safety and efficacy of a new proteasome inhibitor Ixazomib followed by autologous hematopoietic stem cell transplantation (AHSCT) in the treatment of POEMS syndrome.. The clinical manifestations, diagnosis and treatment process and follow-up results of 4 patients with POEMS syndrome who were treated with Ixazomib-based regimen combined with AHSCT in Wuhan No.1 Hospital from February 2018 to July 2020 were analyzed retrospectively. All patients were male, aged from 37-54 years old, with varying degrees of peripheral neuropathy, organ enlargement (liver, spleen or lymph nodes), circulatory overload (peripheral edema and/or pleural effusion), osteosclerosis, endocrine diseases (thyroid, gonads, etc.), skin changes (pigmentation, hemangioma, white nails, etc.), M protein, papilledema and other clinical manifestations and characteristics at the time of initial treatment. Two patients were pathologically diagnosed as hyaline vascular Castleman disease by lymph node biopsy. Three patients underwent lumbar puncture examinations and all showed elevated cerebrospinal fluid protein. All patients received at least 2 cycles of sequential AHSCT after induction chemotherapy based on ixazomib. The follow-up time was 10-28 months, and the median follow-up time was 16 months.. All cases survived. The complications were controllable during the treatment. Moreover, the clinical symptoms related to the disease were improved to a certain extent after the treatment. The levels of vascular endothelial growth factor (VEGF) showed a gradual decline.. Ixazomib combined with AHSCT is safe and effective in the treatment of POEMS syndrome.. 以伊沙佐米为基础的方案序贯自体造血干细胞移植治疗POEMS综合征的临床观察.. 探讨新型蛋白酶体抑制剂伊沙佐米序贯自体造血干细胞移植（AHSCT）治疗POEMS综合征的安全性及疗效.. 回顾性分析2018年2月至2020年7月在武汉市第一医院以伊沙佐米为基础的方案联合AHSCT治疗的4例POEMS综合征患者的临床特征、诊治经过及随访结果，并结合相关文献进行讨论。所有患者均为男性，年龄37-54岁，初治时均不同程度地合并周围神经病变、肝脾或淋巴结肿大、循环超负荷（外周水肿、胸腹腔积液）、骨硬化症、内分泌病变（甲状腺、性腺等）、皮肤改变（色素沉着、血管瘤、白甲等）、M蛋白、视乳头水肿等临床表现及特征。2例患者合并Castleman病，3例患者行脑脊液检测且均提示脑脊液蛋白水平升高。所有患者均至少接受2周期以伊沙佐米为基础的方案诱导化疗后序贯AHSCT。随访时间10-28个月，中位随访时间16个月.. 无死亡病例。治疗过程中并发症可控，治疗后疾病相关的临床症状均得到一定程度的改善，血管内皮生长因子（VEGF）的水平均呈逐渐下降的趋势.. 伊沙佐米作为一种新型的、周围神经毒性更小的蛋白酶体抑制剂，联合AHSCT治疗POEMS综合征是安全、有效的.

Topics: Adult; Boron Compounds; Glycine; Hematopoietic Stem Cell Transplantation; Humans; Male; Middle Aged; POEMS Syndrome; Retrospective Studies; Transplantation, Autologous; Vascular Endothelial Growth Factor A

Objective Immunomodulatory drugs and proteasome inhibitors are therapeutic options for polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes (POEMS) syndrome. This study aimed to evaluate the efficacy and safety of the combination of ixazomib, lenalidomide, and dexamethasone (IRd) for POEMS syndrome. Methods Six consecutive patients with POEMS syndrome who were treated with the IRd regimen at Chiba University Hospital between April 2018 and August 2021 were included. Serum M-protein and serum vascular endothelial growth factor (sVEGF) levels, overall neuropathy limitation scales (ONLS), clinical symptoms, and adverse events were assessed. Results Of the six patients, five had received prior treatments. Patients received a median of 5 cycles (range, 3-28 cycles) of IRd. Following treatment, serum M-protein disappeared in two patients, sVEGF levels returned to normal in two patients, two patients showed a reduction in the ONLS of 1, and clinical symptoms improved in four patients. The median level of sVEGF decreased from 2,395 pg/mL (range, 802-6,120 pg/mL) to 1,428 pg/mL (range, 183-3,680 pg/mL) in three months. Adverse events, including rash, neutropenia, sensory peripheral neuropathy, and nausea, were observed in three patients, which necessitated dose reduction or discontinuation of treatment. Conclusion IRd can be a therapeutic option for POEMS syndrome, albeit with careful monitoring of adverse events.

Topics: Boron Compounds; Dexamethasone; Endocrine System Diseases; Glycine; Humans; Lenalidomide; Monoclonal Gammopathy of Undetermined Significance; POEMS Syndrome; Vascular Endothelial Growth Factor A