Superior survival in primary systemic amyloidosis patients undergoing peripheral blood stem cell transplantation: a case-control study
Superior survival in primary systemic amyloidosis patients undergoing peripheral blood stem cell transplantation: a case-control study
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DOI：
10.1182/blood-2003-12-4192
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发表时间：
2004-05-15
期刊：
影响因子：
20.3
通讯作者：
Gertz, MA
中科院分区：
文献类型：
--
作者：
Dispenzieri, A;Kyle, RA;Gertz, MA
原发性系统性淀粉样变性（AL）是一种浆细胞病变，导致多系统功能衰竭和死亡。与标准化疗相比，大剂量化疗与外周血干细胞移植（PBSCT）具有更高的反应率和更高的总生存率。然而，选择偏差混淆了对这些结果的解释。我们进行了一项病例匹配对照研究，比较63例接受移植的AL患者和63例未接受移植的AL患者的总生存率。匹配标准包括年龄、性别、发病时间、左心室射血分数、血清肌酐、间隔厚度、神经受累程度、24小时尿蛋白和血清碱性磷酸酶。根据设计，在性别（57%为男性）、年龄（中位数为53岁）、左心室射血分数（65%）、周围神经受累患者数（17%）、心室间隔壁厚度（12 mm）、血清肌酐（1.1 mg/dL [97.24 mumol/L]）和骨髓浆细胞增多症（8%）方面，两组间无差异。66例患者死亡（16例病例和50例对照）。对于PBSCT和对照组，1年、2年和4年的总生存率分别为89%和71%；81%和55%；71%和41%。在一项随机临床试验之外，这些结果提供了最有力的数据，支持PBSCT在选定的AL患者中的作用。(C) 2004年由美国血液学学会发表。
Primary systemic amyloidosis (AL) is a plasma cell dyscrasia resulting in multisystem failure and death. High-dose chemotherapy with peripheral blood stem cell transplantation (PBSCT) has been associated with higher response rates and seemingly higher overall survival than standard chemotherapy. Selection bias, however, confounds interpretation of these results. We performed a case-match-control study comparing overall survival of 63 AL patients undergoing transplantation with 63 patients not under-going transplantation. Matching criteria included age, sex, time to presentation, left ventricular ejection fraction, serum creatinine, septal thickness, nerve involvement, 24-hour urine protein, and serum alkaline phosphatase. According to design, there was no difference between the groups with respect to sex (57% males), age (median, 53 years), left ventricular ejection fraction (65%), number of patients with peripheral nerve involvement (17%), cardiac interventricular septal wall thickness (12 mm), serum creatinine (1.1 mg/dL [97.24 mumol/L]), and bone marrow plasmacytosis (8%). Sixty-six patients have died (16 cases and 50 controls). For PBSCT and control groups, respectively, the 1-, 2-, and 4-year overall survival rates are 89% and 71%; 81% and 55%; and 71 % and 41 %. Outside a randomized clinical trial, these results present the strongest data supporting the role of PBSCT in selected patients with AL. (C) 2004 by The American Society of Hematology.