Department of Hematology, University of Groningen and University Medical Center Groningen, Groningen, The Netherlands.
Blood. 2011 Dec 1;118(23):6037-42. doi: 10.1182/blood-2011-07-370247. Epub 2011 Sep 27.
We report the results of a prospective, randomized phase 3 trial evaluating autologous peripheral blood stem cell transplantation (ASCT) versus intensive consolidation chemotherapy in newly diagnosed AML patients in complete remission (CR1). Patients with AML (16-60 years) in CR1 after 2 cycles of intensive chemotherapy and not eligible for allogeneic SCT were randomized between intensive chemotherapy with etoposide and mitoxantrone or ASCT ater high-dose cyclophosphamide and busulfan. Of patients randomized (chemotherapy, n = 259; ASCT, n = 258), more than 90% received their assigned treatment. The 2 groups were comparable with regard to prognostic factors. The ASCT group showed a markedly reduced relapse rate (58% vs 70%, P = .02) and better relapse-free survival at 5 years (38% vs 29%, P = .065, hazard ratio = 0.82; 95% confidence interval, 0.66-1.1) with nonrelapse mortality of 4% versus 1% in the chemotherapy arm (P = .02). Overall survival was similar (44% vs 41% at 5 years, P = .86) because of more opportunities for salvage with second-line chemotherapy and stem cell transplantation in patients relapsing on the chemotherapy arm. This large study shows a relapse advantage for ASCT as postremission therapy but similar survival because more relapsing patients on the chemotherapy arm were salvaged with a late transplantation for relapse. This trial is registered at www.trialregister.nl as #NTR230 and #NTR291.
我们报告了一项前瞻性、随机的 3 期临床试验结果，该试验评估了自体外周血干细胞移植（ASCT）与强化巩固化疗在新诊断的完全缓解（CR1）AML 患者中的疗效。CR1 后接受 2 周期强化化疗且不符合异基因 SCT 条件的 AML 患者（16-60 岁）被随机分为接受依托泊苷和米托蒽醌强化化疗或 ASCT 组，后者接受大剂量环磷酰胺和白消安预处理。接受随机分组的患者（化疗组 259 例，ASCT 组 258 例）中，超过 90%的患者接受了其指定的治疗方案。两组在预后因素方面具有可比性。ASCT 组的复发率明显降低（58% vs 70%，P =.02），5 年无复发生存率更好（38% vs 29%，P =.065，危险比 = 0.82；95%置信区间，0.66-1.1），而化疗组的非复发死亡率为 4%vs1%（P =.02）。由于化疗组复发患者有更多机会接受二线化疗和干细胞移植挽救治疗，因此总生存率相似（5 年时为 44% vs 41%，P =.86）。这项大型研究表明，ASCT 作为缓解后治疗具有复发优势，但生存率相似，因为化疗组更多的复发患者通过延迟移植挽救了复发。该试验在 www.trialregister.nl 上注册，编号为#NTR230 和#NTR291。
