[Analysis of clinical characteristics and outcome of patients with very high risk primary immunoglobulin light-chain amyloidosis]

J Feng, X F Huang, C L Zhang, K N Shen, C L Zhang, J Sun, Z Tian, X X Cao, L Zhang, D B Zhou, J Li, J Feng, X F Huang, C L Zhang, K N Shen, C L Zhang, J Sun, Z Tian, X X Cao, L Zhang, D B Zhou, J Li

Abstract

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 Ⅲb 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 Ⅲ or Ⅳ. Median values of serum cTnI, NT-proBNP, and free light chains difference were 0.25 μg/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.

Keywords: Bortezomib; Primary light chain amyloidosis; Very high risk.

Figures

图1. 极高危及非极高危原发轻链型淀粉样变患者的生存曲线
图1. 极高危及非极高危原发轻链型淀粉样变患者的生存曲线
图2. 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极高危原发轻链型淀粉样变患者生存曲线
图2. 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极高危原发轻链型淀粉样变患者生存曲线

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