Eligibility for hematopoietic stem-cell transplantation for primary systemic amyloidosis is a favorable prognostic factor for survival
Eligibility for hematopoietic stem-cell transplantation for primary systemic amyloidosis is a favorable prognostic factor for survival
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DOI：
10.1200/jco.2001.19.14.3350
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发表时间：
2001-07-15
影响因子：
45.3
通讯作者：
Gertz, MA
中科院分区：
文献类型：
--
作者：
Dispenzieri, A;Lacy, MQ;Gertz, MA
目的：基于造血干细胞移植（HSCT）治疗多发性骨髓瘤的成功，HSCT正被用于治疗原发性系统性淀粉样变性（AL）患者。本文讨论了在何种程度上接受HSCT的资格是一个有利的预后特征，并探讨了预后因素的子集内的合格患者。患者和方法：查询马约诊所淀粉样蛋白数据库的所有患者与AL看到在马约诊所从1983年到1997年谁将有资格进行外周血干细胞移植。入选标准包括活检证实的淀粉样蛋白、症状性疾病、无多发性骨髓瘤临床诊断、年龄小于或等于70岁、心脏室间隔厚度小于或等于15 mm、心脏射血分数大于55%、血清肌酐小于或等于2 mg/dL、直接胆红素小于或等于2.0 mg/dL。中位年龄为56岁（范围：25 - 70岁），其中79例（34%）年龄大于60岁。100例患者有早期心脏受累; 41例，肝脏受累; 167例，肾脏受累; 39例，神经受累。229名患者的中位随访时间为52个月，151人死亡。中位生存期为42个月，5年和10年生存率分别为36%和15%。生存的重要预测因素是24小时尿中AN组分的大小、受累器官的数量、碱性磷酸酶、性能恐慌和体重减轻。符合HSCT条件的患者是化疗效果相对较好的高风险人群（中位生存期，42个月），显著优于所有AL患者的预期中位生存期18个月。需要随机试验来评估HSCT的真实效果。(C)2001年，美国临床肿瘤学会。
Purpose: Based on the success of hematopoietic stem-cell transplantation (HSCT) for multiple myeloma, HSCT is being used to treat patients with primary systemic amyloidosis (AL). This article addresses the extent to which eligibility to undergo HSCT is a favorable prognostic feature and explores prognostic factors within the subset of eligible patients.patients and Methods: The Mayo Clinic amyloid database was queried for all patients with AL seen at the Mayo Clinic from 1983 through 1997 who would have been eligible for peripheral-blood stem-cell transplantation. Inclusion criteria included biopsy-proven amyloid, symptomatic disease, absence of a clinical diagnosis of multiple myeloma, age less than or equal to 70 years, cardiac interventricular septal thickness less than or equal to 15 mm, cardiac ejection fraction more than 55%, serum creatinine less than or equal to 2 mg/dL, and direct bilirubin less than or equal to 2.0 mg/dL.Results: Median age was 56 years (range, 25 to 70) with 79 (34%) older than 60 years. One hundred patients had early cardiac involvement; 41, hepatic involvement; 167, renal involvement; and 39, nerve involvement. The 229 patients have had a median follow-up of 52 months, and 151 have died. The median survival was 42 months with 5- and 10-year survival rates of 36% and 15%, respectively. Important predictors of survival were size of an-component in 24-hour urine, number of involved organs, alkaline phosphatase, performance scare, and weight loss.Conclusion: The same patients who are eligible for HSCT are a good-risk population who do relatively well with chemotherapy (median survival, 42 months), substantially better than the expected median survival of 18 months for all patients with AL. A randomized trial is needed to assess the true effect of HSCT. (C) 2001 by American Society of Clinical Oncology.