Gertz M A, Kyle R A
Division of Hematology and Internal Medicine, Mayo Clinic, Rochester, MN 55905.
Semin Arthritis Rheum. 1994 Oct;24(2):124-38. doi: 10.1016/s0049-0172(05)80006-x.
The objective of this study was to review (1) the factors that have been linked to prediction of clinical outcome and survival in amyloidosis and (2) the available studies on the therapy for localized and systemic forms of amyloidosis. We made a retrospective review of the relevant literature on treatment and prognosis in localized and systemic amyloidosis dating back to 1975. The most important prognostic factors in amyloidosis are the presence of congestive heart failure, beta 2-microglobulin, and whether peripheral neuropathy dominates the presentation. The presence of a monoclonal light chain in serum or urine, multiple myeloma, and hepatic involvement are also important adverse factors. Colchicine is beneficial in treating familial Mediterranean fever and may play a role in managing secondary amyloidosis in inflammatory bowel disease. Chlorambucil is particularly useful in juvenile rheumatoid arthritis with amyloidosis. Dimethyl sulfoxide provides benefit in bladder and lichen amyloidosis. A trial of alkylating agent-based chemotherapy is reasonable in symptomatic primary systemic amyloidosis. Advances have been made in the treatment of amyloidosis and include chemotherapy, dialysis, transplantation, and improved supportive care. Definite disease regressions with long-term survival (> 10 years) are seen. Unfortunately, alternatives still need to be developed: Of 859 patients with primary systemic amyloidosis seen at the Mayo Clinic from 1982 to 1992, the median survival was 2.1 years.
（1）与淀粉样变性临床结局和生存预测相关的因素；（2）关于局限性和全身性淀粉样变性治疗的现有研究。我们对1975年以来有关局限性和全身性淀粉样变性治疗及预后的相关文献进行了回顾。淀粉样变性最重要的预后因素是充血性心力衰竭、β2微球蛋白的存在，以及周围神经病变是否在临床表现中占主导地位。血清或尿液中存在单克隆轻链、多发性骨髓瘤和肝脏受累也是重要的不利因素。秋水仙碱对治疗家族性地中海热有益，可能在炎症性肠病继发性淀粉样变性的管理中发挥作用。苯丁酸氮芥对伴有淀粉样变性的青少年类风湿关节炎特别有用。二甲亚砜对膀胱和苔藓样淀粉样变性有益。对于有症状的原发性全身性淀粉样变性，基于烷化剂的化疗试验是合理的。淀粉样变性的治疗已取得进展，包括化疗、透析、移植和改善支持治疗。已观察到明确的疾病缓解和长期生存（>10年）。不幸的是，仍需开发其他治疗方法：1982年至1992年在梅奥诊所就诊的859例原发性全身性淀粉样变性患者中，中位生存期为2.1年。
