Gertz M A, Lacy M Q, Dispenzieri A, Ansell S M, Elliott M A, Gastineau D A, Inwards D J, Micallef I N M, Porrata L F, Tefferi A, Litzow M R
Division of Hematology, Mayo Clinic, 200 First Street SW, Rochester, MN 55905, USA.
Bone Marrow Transplant. 2004 Dec;34(12):1025-31. doi: 10.1038/sj.bmt.1704691.
High-dose chemotherapy and autologous stem cell transplantation are used increasingly to treat patients with light-chain-related amyloidosis (AL). Treatment-related mortality is approximately 15%. To enable more patients to undergo stem cell transplantation, a risk-adapted strategy has been developed to treat with lower chemotherapy doses those patients who are at excessive risk. It is unclear whether reducing the chemotherapy dose in patients at excessive risk of treatment toxicity reduces the overall response. We retrospectively reviewed 171 AL patients who underwent conditioning chemotherapy with stem cell transplantation. The patients comprised two groups: those receiving standard high-dose melphalan and those receiving intermediate-dose melphalan. Responses were categorized as hematologic response, which used criteria for myeloma response. The two groups showed statistically significant differences; the overall response rates were 75% in the high-dose group and 53% in the intermediate-dose group although treatment-related mortality was the same in both groups. Reducing the melphalan dose appeared to render more AL patients eligible for stem cell transplantation but sacrificed an element of response. Methods are needed to reduce treatment-related toxicity so that more patients can receive full-dose conditioning chemotherapy.
大剂量化疗和自体干细胞移植越来越多地用于治疗轻链相关性淀粉样变性（AL）患者。治疗相关死亡率约为15%。为了使更多患者能够接受干细胞移植，已制定了一种风险适应性策略，对那些风险过高的患者采用较低化疗剂量进行治疗。尚不清楚降低治疗毒性风险过高患者的化疗剂量是否会降低总体缓解率。我们回顾性分析了171例接受干细胞移植预处理化疗的AL患者。这些患者分为两组：接受标准大剂量美法仑的患者和接受中等剂量美法仑的患者。缓解情况分为血液学缓解，采用骨髓瘤缓解标准。两组显示出统计学上的显著差异；高剂量组的总体缓解率为75%，中等剂量组为53%，尽管两组的治疗相关死亡率相同。降低美法仑剂量似乎使更多AL患者有资格接受干细胞移植，但牺牲了一定的缓解率。需要采取方法降低治疗相关毒性，以便更多患者能够接受全剂量预处理化疗。
