Girnius S, Seldin D C, Meier-Ewert H K, Sloan J M, Quillen K, Ruberg F L, Berk J L, Doros G, Sanchorawala V
1] Department of Medicine, Amyloidosis Center, Boston University School of Medicine, Boston, MA, USA [2] Section of Hematology-Oncology, Department of Medicine, Boston Medical Center, Boston, MA, USA.
1] Department of Medicine, Amyloidosis Center, Boston University School of Medicine, Boston, MA, USA [2] Section of Cardiovascular Medicine, Department of Medicine, Boston Medical Center, Boston, MA, USA.
Bone Marrow Transplant. 2014 Mar;49(3):434-9. doi: 10.1038/bmt.2013.192. Epub 2013 Dec 9.
In Ig light chain (AL) amyloidosis, cardiac involvement is associated with worse prognosis and increased treatment-related complications. In this retrospective cohort study, we assessed survival, hematologic and cardiac responses to high-dose melphalan and auto-SCT (HDM/SCT) in patients with AL amyloidosis and cardiac involvement, stratified by cardiac biomarkers brain natriuretic peptide and Troponin I, analogous to the Mayo cardiac staging. Forty-seven patients underwent HDM/SCT based upon functional measures; six patients had modified cardiac stage I disease, seventeen had modified cardiac stage II disease and twenty-four had modified cardiac stage III disease. Treatment-related mortality was 4% for all patients and 8% for patients with stage III disease. Three-year survival was 88% and EFS was 47%; these did not differ by stage. By intention-to-treat analysis, 27% of patients achieved a hematologic complete response and 32% a very good partial response, of whom 70 and 45%, respectively, have not required additional therapy at 36 months. Cardiac response was achieved in 53% of patients. We conclude that with appropriate patient selection and a risk-adapted treatment approach, HDM/SCT is safe and effective in patients with AL amyloidosis and cardiac involvement.
在免疫球蛋白轻链（AL）淀粉样变性中，心脏受累与预后较差及治疗相关并发症增加有关。在这项回顾性队列研究中，我们评估了AL淀粉样变性合并心脏受累患者接受大剂量美法仑和自体造血干细胞移植（HDM/SCT）后的生存情况、血液学和心脏反应，并根据心脏生物标志物脑钠肽和肌钙蛋白I进行分层，类似于梅奥心脏分期。47例患者根据功能指标接受了HDM/SCT；6例患者为改良心脏I期疾病，17例为改良心脏II期疾病，24例为改良心脏III期疾病。所有患者的治疗相关死亡率为4%，III期疾病患者为8%。三年生存率为88%，无事件生存率为47%；这些在各阶段之间没有差异。根据意向性分析，27%的患者实现了血液学完全缓解，32%的患者实现了非常好的部分缓解，其中分别有70%和45%的患者在36个月时无需额外治疗。53%的患者实现了心脏反应。我们得出结论，通过适当的患者选择和风险适应性治疗方法，HDM/SCT对AL淀粉样变性合并心脏受累患者是安全有效的。
