Kovacsovics T
Département de médecine, CHUV, Lausanne.
Rev Med Suisse Romande. 2000 Oct;120(10):771-5.
AL amyloidosis is a plasma cell disorder characterized by the tissue accumulation of immunoglobulin light chains. The prognosis of AL amyloidosis is poor, with a median survival inferior to two years. Amyloid deposits, responsible for the clinical manifestations of this disease, can regress at least partially after the suppression of the production of amyloid precursors. Therefore, current treatment strategies in AL amyloidosis aim at reducing the plasma cell clones, using chemotherapy regimens applied in multiple myeloma. A combination of melphalan and prednisone is the standard therapy of AL amyloidosis, but its results remain disappointing. Intensive chemotherapy regimens, with high-dose melphalan followed by peripheral blood stem cell transplantation, lead to increased response rates and improved survival, but are complicated by severe short term morbidity and mortality. These regimens offer the best clinical perspectives for selected patients, until the introduction of innovative agents capable of interfering with amyloid fibril formation.
轻链型淀粉样变性是一种浆细胞疾病,其特征为免疫球蛋白轻链在组织中蓄积。轻链型淀粉样变性的预后较差,中位生存期不足两年。导致该疾病临床表现的淀粉样沉积物,在淀粉样前体生成受到抑制后至少可部分消退。因此,目前轻链型淀粉样变性的治疗策略旨在使用多发性骨髓瘤的化疗方案减少浆细胞克隆。美法仑和泼尼松联合使用是轻链型淀粉样变性的标准疗法,但其效果仍不尽人意。强化化疗方案,即大剂量美法仑后进行外周血干细胞移植,可提高缓解率并改善生存率,但会伴有严重的短期发病率和死亡率。在能够干扰淀粉样纤维形成的创新药物出现之前,这些方案为特定患者提供了最佳的临床前景。