Gertz M A, Kyle R A, Greipp P R
Dysproteinemia Clinic, Mayo Clinic, Rochester, MN 55905.
Blood. 1991 Jan 15;77(2):257-62.
Patients (153) with biopsy-proven primary systemic amyloidosis (AL) were evaluated for their response rate to alkylating agent-based chemotherapy. Twenty-seven of the patients (18%) responded. The serum creatinine concentration had an adverse effect on response rate (P = .05). In patients with nephrotic syndrome, a normal serum creatinine value, and no echocardiographic evidence of cardiac amyloidosis, the response rate was 39% (12 of 31). Five of 34 patients with amyloid cardiomyopathy responded. Two of these five are alive 10 years after diagnosis. None of the 18 patients with amyloid peripheral neuropathy showed regression of their disease. The median time to achieve response was 11.7 months. The median survival of the 27 patients was 89.4 months and 21 of 27 survived 5 years (78%). Eight patients remain alive with a minimum follow-up of 90 months. Seven died of acute leukemia or dysmyelopoietic syndrome, a presumed complication of melphalan therapy. In the group of 126 patients who showed no response to alkylating agent-based therapy, the median survival was 14.7 months and 9 (7%) survived over 5 years. All 126 patients have died. Alkylating agent-based chemotherapy for AL is beneficial in a subset of patients and a trial of chemotherapy is strongly recommended. Those patients who do respond demonstrate survival benefit.
对153例经活检证实为原发性系统性淀粉样变性（AL）的患者进行了基于烷化剂化疗的缓解率评估。其中27例患者（18%）出现缓解。血清肌酐浓度对缓解率有不良影响（P = 0.05）。在患有肾病综合征、血清肌酐值正常且无心脏淀粉样变性超声心动图证据的患者中，缓解率为39%（31例中的12例）。34例淀粉样心肌病患者中有5例出现缓解。这5例中有2例在诊断后10年仍然存活。18例淀粉样周围神经病患者中无一例病情出现缓解。达到缓解的中位时间为11.7个月。27例患者的中位生存期为89.4个月，27例中有21例存活5年（78%）。8例患者仍然存活，最短随访时间为90个月。7例死于急性白血病或骨髓增生异常综合征，这被认为是美法仑治疗的并发症。在126例对基于烷化剂治疗无反应的患者组中，中位生存期为14.7个月，9例（7%）存活超过5年。所有126例患者均已死亡。基于烷化剂的化疗对部分AL患者有益，强烈建议进行化疗试验。那些有反应的患者显示出生存获益。
