伴超高水平血清游离轻链的轻链型淀粉样变患者临床特征和预后分析

Clinical presentation and prognosis in patients with light-chain amyloidosis with an ultra-high level of serum free light-chain

目的探讨伴超高水平血清游离轻链(FLC)的轻链型(AL型)淀粉样变患者的临床特征及预后。方法回顾性分析2009年1月至2020年1月北京协和医院确诊的595例AL型淀粉样变患者的临床资料,按FLC水平将其分为两组:超高FLC组[FLC差值(dFLC)>500 mg/L,124例]与非超高FLC组(dFLC≤500 mg/L,471例),比较两组患者的临床特征和预后。结果超高FLC组患者与非超高FLC组相比,心脏受累比例更高(82.3%对70.1%,P=0.007),2004梅奥分期Ⅲ期比例更高(41.8%对33.8%,P=0.029),肾脏受累比例更低(59.7%对71.8%,P=0.009)。超高FLC组患者与非超高FLC组相比,血液学缓解率降低(72.4%对82.3%,P=0.048),心脏缓解率降低(37.3%对54.7%,P=0.016),中位总生存期缩短(13.0个月对未达到,P<0.001),3个月内早期死亡率升高(28.2%对11.3%,P<0.001)。dFLC>500 mg/L是影响AL型淀粉样变患者预后的独立危险因素(HR=2.279,95%CI 1.685~3.083,P<0.001)。结论初诊时FLC超高AL型淀粉样变患者的心脏受累更多见,临床分期更晚,预后极差。

Objective To investigate the clinical features and outcomes of patients with light-chain(AL)amyloidosis with an ultra-high level of serum free light-chain(FLC).Methods Five hundred and ninety-five patients with AL amyloidosis were retrospectively reviewed between January 2009 and January 2020 at Peking Union Medical College Hospital.We analyzed the clinical features and prognosis of patients with ultra-high FLC levels[difference between involved and uninvolved light chains(dFLC)>500 mg/L;n=124]and those without ultra-high FLC levels(dFLC≤500 mg/L;n=471).Results Patients with ultra-high FLC presented with more frequent cardiac involvement(82.3%vs 70.1%,P=0.007),and a higher percentage of patients with 2004 MayoⅢstage(41.8%vs 33.8%,P=0.029),but less frequent renal involvement than patients without an ultra-high FLC(59.7%vs 71.8%,P=0.009).Patients with an ultra-high FLC achieved a lower proportion of hematologic(72.4%vs 82.3%,P=0.048)and cardiac response(37.3%vs 54.7%,P=0.016)and had shorter overall survival(13.0 months vs not reached,P<0.001)and a higher early death rate within 3 months(28.2%vs 11.3%,P<0.001)than those without an ultra-high FLC.Ultra-high FLC independently predicted worse prognosis in patients with AL amyloidosis(HR=2.279,95%CI 1.685-3.083,P<0.001).Conclusions Patients with an initially ultra-high FLC represented a subgroup with more common cardiac involvement,more advanced cardiac stages,and extremely poor prognosis.