PDF(Pubmed)
Abstract：
Autologous hematopoietic stem cell transplantation (SCT) is not a standard treatment option for Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ALL); however, its position has been reassessed since the introduction of tyrosine kinase inhibitors (TKIs). We prospectively analyzed the efficacy and safety of autologous peripheral blood SCT (auto-PBSCT) for Ph+ALL patients aged between 55 and 70 years who had achieved complete molecular remission. Melphalan, cyclophosphamide, etoposide, and dexamethasone were used for conditioning. A total of 12 courses of maintenance therapy, including dasatinib, were performed. The required number of CD34+ cells was harvested in all five patients. No patient died within 100 days after auto-PBSCT, and no unexpected serious adverse events were observed. Although 1-year event-free survival was 100%, hematological relapse was observed in three patients at a median of 801 days (range, 389-1088 days) after auto-PBSCT. Molecular progressive disease was observed in the other two patients, although they maintained their first hematological remission at the last visit. Auto-PBSCT can be safely performed for Ph+ALL with TKIs. A limitation of auto-PBSCT was suggested, despite the increase in the intensity of a single treatment. The development of long-term therapeutic strategies by including new molecular targeted drugs is warranted to maintain long-term molecular remission.
摘要：
自体造血干细胞移植(SCT)不是费城染色体阳性急性淋巴细胞白血病(Ph+ALL)的标准治疗选择;然而,自酪氨酸激酶抑制剂（TKIs）引入以来，其地位已被重新评估。我们前瞻性分析了自体外周血SCT（auto-PBSCT）对年龄在55至70岁之间，已实现分子完全缓解的PhALL患者的疗效和安全性。Melphalan,环磷酰胺,依托泊苷,和地塞米松用于调理。共12个疗程的维持治疗,包括达沙替尼,被执行了。在所有五名患者中收集所需数量的CD34+细胞。自动PBSCT后100天内没有患者死亡，未观察到意外的严重不良事件.尽管1年无事件生存率为100%,在中位数为801天的三名患者中观察到血液学复发（范围，自动PBSCT后389-1088天）。在另外两名患者中观察到分子进行性疾病，尽管他们在最后一次就诊时保持了第一次血液学缓解。自动PBSCT可以安全地执行与TKI的Ph+ALL。提出了自动PBSCT的局限性，尽管单一治疗的强度增加。通过包括新的分子靶向药物来开发长期治疗策略是必要的，以维持长期的分子缓解。
