Department of Medicine, Boston University Medical Center, Boston, MA 02118, USA.
Bone Marrow Transplant. 2011 Jul;46(7):976-80. doi: 10.1038/bmt.2010.239. Epub 2010 Oct 18.
High-dose melphalan and auto-SCT (HDM/SCT) induces hematological complete responses (HCRs) in 40% of patients with immunoglobulin light chain (AL) amyloidosis. However, relapses occur in 8% of patients who initially achieve HCR. We conducted a study to explore the feasibility and efficacy of a second HDM/SCT in this setting. Eleven patients were enrolled. Five patients underwent repeat stem cell mobilization with G-CSF; the others had stem cells cryopreserved from the first mobilization. Six patients received 200 mg/m(2) HDM; five patients received modified HDM at 140 mg/m(2). Engraftment occurred at a median of 10 days for neutrophils and 12 days for platelets. There was no treatment-related mortality or death within the first year, but significant grade III/IV non-hematological toxicities occurred. In all, 4 of 11 patients (36%) achieved HCR at 1 year. Two of these patients are in continuous remission at 3 and 6 years; the other two relapsed at 2 and 3 years. Of the four patients who achieved partial hematological response at 1 year, three have relapsed at a median of 3 years. Three patients died of progressive disease at 1-2 years. In conclusion, one-third of patients with AL amyloidosis who relapse after HDM/SCT can achieve HCR with a second SCT.
高剂量马法兰和自体造血干细胞移植（HDM/SCT）可诱导 40%的免疫球蛋白轻链（AL）淀粉样变性患者达到血液学完全缓解（HCR）。然而，8%最初达到 HCR 的患者会出现复发。我们进行了一项研究，以探讨在此情况下进行第二次 HDM/SCT 的可行性和疗效。共纳入 11 例患者。5 例患者进行了 G-CSF 重复干细胞动员；其他患者则使用第一次动员时冷冻的干细胞。6 例患者接受 200mg/m² HDM；5 例患者接受 140mg/m² 的改良 HDM。中性粒细胞和血小板的中位植入时间分别为 10 天和 12 天。无治疗相关死亡或一年内死亡，但发生了显著的 III/IV 级非血液学毒性。所有患者中，11 例中有 4 例（36%）在 1 年内达到 HCR。其中 2 例患者在 3 年和 6 年内持续缓解；另外 2 例患者在 2 年和 3 年内复发。在 1 年内达到部分血液学缓解的 4 例患者中，3 例在中位时间 3 年内复发。3 例患者在 1-2 年内因疾病进展而死亡。总之，HDM/SCT 后复发的 AL 淀粉样变性患者中有三分之一可以通过第二次 SCT 达到 HCR。
