Division of Hematology, Department of Internal Medicine, University Hospital of Basel, Basel, Switzerland.
Bone Marrow Transplant. 2011 May;46(5):636-40. doi: 10.1038/bmt.2010.181. Epub 2010 Aug 9.
Treatment strategies for relapsed/refractory AML are limited and disappointing. Recently, high-dose melphalan (HDM) chemotherapy and autologous hematopoietic SCT (HSCT) has been proposed for AML re-induction. We investigated the impact of HDM remission induction in highly advanced relapsed/refractory AML patients planned for allogeneic HSCT. A total of 23 patients with relapsed/refractory AML were prospectively scheduled for HDM with or without stem cell support followed by myeloablative allogeneic HSCT. Patients included nine individuals with a history of previous HSCT (seven allogeneic, two autologous). A total of 18 patients (78%) achieved a leukemia-free state and an additional four had substantial reduction of the initial leukemia burden warranting treatment continuation. There were no differences between patients with or without immediate stem cell support regarding mucositis or other organ toxicity. A total of 20 patients proceeded to myeloablative allogeneic HSCT. Outcome of allogeneic HSCT was poor: 11 patients (55%) relapsed, 7 patients (35%) died from TRM and only 2 patients (10%) were alive at the last follow-up. Our study shows that HDM is effective in inducing a leukemia-free state in patients with highly advanced relapsed/refractory AML. Leukemia burden reduction with HDM, however, did not translate into improved OS.
复发/难治性 AML 的治疗策略有限且令人失望。最近，高剂量美法仑（HDM）化疗和自体造血干细胞移植（HSCT）已被提议用于 AML 的再诱导。我们研究了 HDM 缓解诱导对计划进行异基因 HSCT 的高度进展性复发/难治性 AML 患者的影响。共有 23 例复发/难治性 AML 患者前瞻性地计划接受 HDM 联合或不联合干细胞支持，随后进行清髓性异基因 HSCT。患者包括 9 例有既往 HSCT 史的个体（7 例异基因，2 例自体）。共有 18 例患者（78%）达到无白血病状态，另外 4 例患者初始白血病负担显著减少，需要继续治疗。有无即刻干细胞支持的患者在粘膜炎或其他器官毒性方面无差异。共有 20 例患者接受了清髓性异基因 HSCT。异基因 HSCT 的结果较差：11 例患者（55%）复发，7 例患者（35%）因 TRM 死亡，只有 2 例患者（10%）在最后一次随访时存活。我们的研究表明，HDM 可有效诱导高度进展性复发/难治性 AML 患者无白血病状态。然而，HDM 降低白血病负担并未转化为改善 OS。
