Prokaeva Tatiana, Spencer Brian, Kaut Maurya, Ozonoff Al, Doros Gheorghe, Connors Lawreen H, Skinner Martha, Seldin David C
Boston University School of Medicine, Boston, Massachusetts 02118-2526, USA.
Arthritis Rheum. 2007 Nov;56(11):3858-68. doi: 10.1002/art.22959.
To characterize symptoms and signs of AL amyloidosis that may bring patients to the attention of rheumatologists, evaluate Ig V(L) gene usage in this subgroup of patients, and assess the impact of soft tissue and bone involvement and V(L) gene usage on survival.
Clinical features of soft tissue and bone involvement were assessed in 191 patients with AL amyloidosis. V(L) gene sequencing was carried out to determine light-chain family, rate of somatic mutation, and evidence of antigen selection. The association of soft tissue and bone involvement with V(L) gene usage was assessed by logistic regression analysis, and survival time was analyzed using log rank tests and Cox regression models.
Soft tissue and bone involvement occurred in 42.9% of the patients, and 9.4% had dominant soft tissue and bone involvement. The most common manifestations were submandibular gland enlargement, macroglossia, and carpal tunnel syndrome. Dominant soft tissue and bone involvement was significantly associated with V(L)kappaI gene usage. Mutation rate and evidence of antigen selection in the V(L) genes were not found to be confounding factors, providing evidence against a contribution of autoimmunity in this type of AL amyloidosis. Survival time was initially longer in patients with dominant soft tissue and bone involvement than in patients with other dominant organ involvement; however, this difference diminished over time.
Amyloid infiltration into soft tissue, joints, periarticular structures, and bones can bring patients with AL amyloidosis to the attention of rheumatologists. Recognition of the presenting symptoms is essential for accurate diagnosis and appropriate treatment, since the long-term outlook for untreated patients with dominant soft tissue and bone involvement is not better than that for patients with other dominant features of AL amyloidosis.
明确可能引起风湿病学家关注的AL淀粉样变性的症状和体征,评估该亚组患者中Ig V(L)基因的使用情况,并评估软组织和骨骼受累情况以及V(L)基因使用对生存的影响。
对191例AL淀粉样变性患者的软组织和骨骼受累的临床特征进行评估。进行V(L)基因测序以确定轻链家族、体细胞突变率和抗原选择证据。通过逻辑回归分析评估软组织和骨骼受累与V(L)基因使用的关联,并使用对数秩检验和Cox回归模型分析生存时间。
42.9%的患者出现软组织和骨骼受累,9.4%的患者以软组织和骨骼受累为主。最常见的表现为颌下腺肿大、巨舌和腕管综合征。以软组织和骨骼受累为主与V(L)κI基因的使用显著相关。未发现V(L)基因的突变率和抗原选择证据是混杂因素,这为自身免疫在这种类型的AL淀粉样变性中的作用提供了反证。最初,以软组织和骨骼受累为主的患者的生存时间比其他主要器官受累的患者更长;然而,随着时间的推移,这种差异逐渐减小。
淀粉样蛋白浸润到软组织、关节、关节周围结构和骨骼可引起AL淀粉样变性患者被风湿病学家关注。识别出现的症状对于准确诊断和适当治疗至关重要,因为未经治疗的以软组织和骨骼受累为主的患者的长期预后并不优于具有AL淀粉样变性其他主要特征的患者。