Efficacy and Long-Term Outcomes of Autologous Stem Cell Transplantation in POEMS Syndrome: A Nationwide Survey in Japan
Efficacy and Long-Term Outcomes of Autologous Stem Cell Transplantation in POEMS Syndrome: A Nationwide Survey in Japan
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DOI：
10.1016/j.bbmt.2018.01.026
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发表时间：
2018-06-01
影响因子：
4.3
通讯作者：
Nakaseko, Chiaki
中科院分区：
文献类型：
--
作者：
Kawajiri-Manako, Chika;Sakaida, Emiko;Nakaseko, Chiaki
POEMS综合征是一种罕见的浆细胞病液症，表现为多发性神经病、λ型M蛋白、血管内皮生长因子升高和全身表现。标准治疗尚未建立，但自体干细胞移植（ASCT）在这种综合征中表现出有效性。然而，ASCT的疗效和长期结局尚未得到系统研究。为了阐明日本接受ASCT治疗患者的疗效和长期结局，我们进行了一项多中心回顾性研究，评估了在日本造血细胞移植注册移植统一管理计划（TRUMP）数据库中注册的患者的临床病程。在2000年1月至2011年12月期间，95例患者（58例男性）登记到TRUMP数据库中，中位年龄为53岁（范围：28至72岁）。94例患者中有93例（99%）的预处理方案为美法仑，69例（74.2%）患者接受美法仑剂量≥ 200 mg/m2。中位CD 34细胞剂量为2.47 × 10（6）/kg（范围：0.31 - 20）。ASCT后，患者体能状态显著改善（东部肿瘤协作组体能状态0 - 1：20.0% vs 71.6%，P <0.0001）。中位随访46.6个月，10例患者死亡，5年总生存率为88.8%（n = 95）。3年无进展生存率为78.3%（n = 70;中位随访时间为54.4个月）。这些数据支持ASCT在POEMS综合征患者中延长生存期和改善生活质量的有希望的作用。然而，疾病复发仍然是长期生存者的主要问题。(C)2018年美国血液和骨髓移植协会。
POEMS syndrome is a rare plasma cell dyscrasia presenting with polyneuropathy, lambda-type M protein, vascular endothelial growth factor elevation, and systemic manifestations. The standard treatment has not been established, but autologous stem cell transplantation (ASCT) has exhibited effectiveness in this syndrome. However, the efficacy and long-term outcomes of ASCT have not been systematically studied. To clarify the efficacy and long-term outcomes of ASCT-treated patients in Japan, we performed a multicenter retrospective study assessing the clinical course of patients registered to the Japan Society for Hematopoietic Cell Transplantation Transplant Registry Unified Management Program (TRUMP) database. Between January 2000 and December 2011, 95 patients (58 men) were registered to the TRUMP database with a median age of 53 years (range, 28 to 72). The conditioning regimen was melphalan in 93 of 94 patients (99%), and 69 patients (74.2%) received a melphalan dose >= 200 mg/m(2). The median CD34 cell dose was 2.47 x 10(6)/kg (range, .31 to 20). After ASCT, patient performance status was dramatically improved (Eastern Cooperative Oncology Group performance status 0 to 1: 20.0% versus 71.6%, P < .0001). Over a median follow-up of 46.6 months 10 patients died, and 5-year overall survival was 88.8% (n = 95). Progression-free survival at 3 years was 78.3% (n = 70; median follow-up, 54.4 months). These data support the promising role of ASCT in patients with POEMS syndrome for both prolonging survival and improving quality of life. However, disease recurrence remains a major issue for longterm survivors. (C) 2018 American Society for Blood and Marrow Transplantation.