期刊文献+

极高危原发性轻链型淀粉样变患者的临床特征和预后分析

Analysis of clinical characteristics and outcome immunoglobulin light- chain amyloidosis
原文传递
导出
摘要 目的探讨极高危原发性轻链型淀粉样变(pAL)患者的临床特征及其预后。方法回顾性分析2009年1月至2016年2月在北京协和医院确诊的205例pAL患者的临床资料,将梅奥2004分期111b期或梅奥2012分期4期的患者定义为极高危患者。结果34例(16.6%)为极高危pAL患者,中位年龄57(20~84)岁,男性22例(64.7%)。所有患者均有心脏受累,15例(44.1%)患者的受累脏器≥3个。27例(81.8%)患者的心功能分级为3-4级,中位血清肌钙蛋白I为0.25(0.08-1.23)μg/L,中位血清N末端B型利钠肽前体为11733(1892~103277)ng/L,中位血清游离轻链差值为403.0(18.1~1911.6)mg/L,8例(24.2%)患者的骨髓浆细胞比例I〉0.100。16例(47.1%)患者采用硼替佐米为主的化疗方案,总体血液学缓解率为58.3%。中位随诊27(1-40)个月,14例(41.2%)患者在诊断后3个月内死亡,中位生存时间仅为4个月。3、6、12和24个月的预期生存率分别为51.3%、44.0%、35.2%和29.6%。一线化疗后获得血液学缓解、未获得血液学缓解以及姑息治疗的患者1年预计存活率分别为90.9%、11.1%及0(P〈0.001)。结论极高危pAL患者的预后极差,早期病死率高,获得血液学缓解的pAL患者有着明显更好的预后。 Objective To evaluate the clinical characteristics and outcomes of very high risk patients with primary immunoglobulin light-chain amyloidosis (pAL) at a single center in China. Method Clinical data, treatment and outcome of 205 pAL patients in Peking Union Medical College Hospital from January 2009 to February 2016 were retrospectively analyzed. A 'very high risk' group includes patients with Mayo 2004 stage II1 b and Mayo 2012 stage 4. Results Of 205 patients, 34 (16.6%) were defined as very high risk pAL patients. The median age at diagnosis was 57 (20 - 84) years, and 22 patients (64.7%) were male. All 34 patients were diagnosed with cardiac involvement, multi-organ involvement was observed in 15 patients (44.1% ), and 27 ( 81.8% ) had New York Heart Association Class 111 or 1W. Median values of serum cTnI, NT-proBNP, and free light chains difference were 0.25 ktg/L, 11 733 ng/L, and 403 mg/L, respectively. Eight (24.2%) had more than 10% plasma cell on the bone marrow aspirate. Sixteen (47.1%) patients received bortezomib based chemotherapy and overall hematologic response rate was 58.3%. Median overall survival (OS) was 4 months. The estimated OS at 3, 6, 12, and 24 months was 51.3%, 44.0%, 35.2%, and 29.6%, respectively. Fourteen (41.2%) patients died within 3 months after the diagnosis. The estimated 1-year survival rate for the patients who got hematologic response, without hematologic response, and palliative treatment was 90.9%, 11.1%, and 0, respectively (P〈0.001).Conclusion Patients with very high risk pAL had very poor prognosis and the early death rate remained high. Those patients who obtained hematologic remission would have significantly better outcomes.
作者 冯俊 黄栩芾 张聪丽 沈恺妮 张春兰 孙健 田庄 曹欣欣 张路 周道斌 李剑 Feng Jun Huang Xufei Zhang Congli Shen Kaini Zhang Chunlan Sun Jian Tian Zhuang Cao Xinxin Zhang Lu Zhou Daobin Li Jian(Department of Hematology, Peking Union of patients with very high risk primary Medical College Hospital, Chinese Academy of Medical Sciences, Beijing 100730, China)
出处 《中华血液学杂志》 CAS CSCD 北大核心 2017年第2期107-111,共5页 Chinese Journal of Hematology
关键词 原发性轻链型淀粉样变 极高危 硼替佐米 Primary light chain amyloidosis Very high risk Bortezomib
  • 相关文献

参考文献1

二级参考文献15

  • 1Merlini G, Bellotti V. Molecular mechanisms of amyloidosis [J]. N Engl J Med, 2003, 349(6):583-596.
  • 2Sipe JD, Benson MD, BuxbaumJN, et al. Amyloid fibril proteinnomenclature: 2010 recommendations from the nomenclature committee of the International Society of Amyloidosis [J]. Amyloid, 2010, 17(3-4):101-104.
  • 3Merlini G, Seldin DC, Gertz MA. Amyloidosis: pathogenesis and new therapeutic options [J]. J Clin Oncol, 2011, 29 (14): 1924-1933.
  • 4Vrana JA, Gamez JD, Madden B J, et al. Classification of amyloi- dosis by laser microdissection and mass spectrometry-based proteomic analysis in clinical biopsy specimens [J]. Blood, 2009, 114(24):4957-4959.
  • 5Klein CJ, Vrana JA, Theis JD, et al. Mass spectrometric-based proteomic analysis of amyloid neuropathy type in nerve tissue [J]. Arch Neurol, 2011, 68(2): 195-199.
  • 6Arike L, Peil L. Spectral counting label-free proteomics [J]. Methods Mol Biol, 2014, 1156:213-222.
  • 7Lavatelli F, Vrana JA. Proteomic typing of amyloid deposits in systemic amyloidoses [J]. Amyloid, 2011, 18 (4) : 177-182.
  • 8Brambilla F, Lavatelli F, Merlini G, et al. Clinical proteomics for diagnosis and typing of systemic amyloidoses [J]. Proteomics Clin Appl, 2013, 7 ( 1-2 ) : 136-143.
  • 9Mollee P, Renaut P, Gottlieb D, et al. How to diagnose amyloido- sis[J]. Intern Med J, 2014, 44( 1 ):7-17.
  • 10Rodriguez F J, Gamez JD, Vrana JA, et al. Immunoglobulin derived depositions in the nervous system: novel mass spectrometry application for protein characterization in formalin- fixed tissues[J]. Lab Invest, 2008, 88( 10):1024-1037.

共引文献13

相关作者

内容加载中请稍等...

相关机构

内容加载中请稍等...

相关主题

内容加载中请稍等...

浏览历史

内容加载中请稍等...
;
使用帮助 返回顶部