Hussein M A, Juturi J V, Rybicki L, Lutton S, Murphy B R, Karam M A
Multiple Myeloma Research Program, The Cleveland Clinic Foundation, 9500 Euclid Ave, Cleveland, Ohio 44195, United States of America.
Med Oncol. 2003;20(3):283-90. doi: 10.1385/MO:20:3:283.
No effective treatment exists for primary amyloidosis, a plasma cell dyscrasia characterized by deposition of amyloid fibrils consisting of monoclonal light chains in various organs. TNF-alpha has been implicated in other amyloid disorders; therefore, we used etanercept to treat patients with advanced amyloidosis who had failed other therapies or were ineligible for other treatment regimens. Sixteen patients with amyloidosis that included patients with severe cardiac or multiple organ involvement were treated with etanercept and evaluated every 4-6 wk for evidence of toxicity and clinical response. Patients were treated with etanercept for a median of 42 wk. Eight of 16 patients (50%) experienced objective improvements and 14 patients (88%) experienced subjective improvements in symptoms. Only one patient experienced an adverse effect attributable to etanercept. For the entire group, improvement in performance status was statistically significant (p = 0.001), estimated median survival is 24.2 mo, 8 of whom are still alive with a median survival is 26.6 mo. The 12 patients with any cardiac involvement had an estimated median survival of 24.2 mo. Six of those 12 patients are still alive, with a median survival is 26.6 mo. The group of eight patients with severe cardiac involvement showed an estimated median survival of 13.2 mo, three of whom are still alive with a median survival is 25.9 mo. The clinical observations in this group of advanced and relapsed/refractory patients are highly encouraging. For the group as a whole, median survival was 24.2 mo and improvement in performance status was highly significant. Median survival for the patients with severe cardiac involvement was 13.2 mo with 3/8 patients are alive with a median survival of 25+ mo. Moreover, there was a statistically significant improvement in patients' performance status. These results, even though in a small group of patients, suggest that etanercept may provide a new therapeutic option for the management of amyloidosis that should be studied further.
原发性淀粉样变性病尚无有效的治疗方法，它是一种浆细胞发育异常疾病，其特征是由单克隆轻链构成的淀粉样原纤维在各器官沉积。肿瘤坏死因子-α（TNF-α）与其他淀粉样变性疾病有关；因此，我们使用依那西普治疗其他治疗无效或不符合其他治疗方案条件的晚期淀粉样变性病患者。16例淀粉样变性病患者，包括有严重心脏受累或多器官受累的患者，接受了依那西普治疗，并每4 - 6周评估一次毒性证据和临床反应。患者接受依那西普治疗的中位时间为42周。16例患者中有8例（50%）出现客观改善，14例患者（88%）症状有主观改善。只有1例患者出现了与依那西普有关的不良反应。对于整个组而言，体能状态的改善具有统计学意义（p = 0.001），估计中位生存期为24.2个月，其中8例仍存活，中位生存期为26.6个月。12例有任何心脏受累的患者估计中位生存期为24.2个月。这12例患者中有6例仍存活，中位生存期为26.6个月。8例有严重心脏受累的患者组估计中位生存期为13.2个月，其中3例仍存活，中位生存期为25.9个月。这组晚期和复发/难治性患者的临床观察结果非常令人鼓舞。对于整个组来说，中位生存期为24.2个月，体能状态改善非常显著。有严重心脏受累的患者中位生存期为13.2个月，8例中有3例存活，中位生存期超过25个月。此外，患者的体能状态有统计学意义的改善。这些结果，尽管是在一小部分患者中得出的，但表明依那西普可能为淀粉样变性病的治疗提供一种新的治疗选择，值得进一步研究。
