Acute Promyelocytic Leukemia
Acute Promyelocytic Leukemia
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DOI：
10.1182/ashimagebank-2002-100364
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发表时间：
2002-06
期刊：
影响因子：
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通讯作者：
Peter Maslak
中科院分区：
文献类型：
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作者：
Peter Maslak
。过去几十年来，针对发病机制中涉及的特定遗传畸变的分化剂的引入，例如全反式视黄酸 (ATRA) 和三氧化二砷 (ATO)，已将急性早幼粒细胞白血病 (APL) 转变为最可治愈的急性白血病，其长期存活率接近 90%。 1 然而，5-20% 的患者在初次缓解后仍然复发。 1 目前复发性 APL 的治疗指南指出，在初始基于 ATO 的治疗后早期复发时使用标准化疗，相反，在 ATO 方案后未曾暴露、早期复发（6 个月）的情况下使用 ATO。 2 在接受 ATO 治疗的大量复发患者中，血液学 CR 率为 86%，完全分子 CR 为 52%，估计早期死亡率为 7%，估计 24 个月总生存 (OS) 范围在 50% 至 81% 之间。 3 一项荟萃分析比较了 ATO 作为单一药物与 ATO 加 ATRA 的效果，显示了组合在 CR 和分子 CR 方面的优势，且不会增加早期死亡率。 4 对于接受 ATO 再诱导再治疗的患者来说，作为巩固治疗的最佳选择仍然是一个讨论问题：最近的一项研究显示，与基于 ATO 的维持治疗 (58.6%) 相比，接受自体干细胞移植 (90.3%) 的患者 5 年后 OS 具有优势。 5 ATO 与硼替佐米联合进行 II 期试验，分子 CR 率为 86%：该组合通过 p62 依赖性自噬途径清除 PML/RARa。 6 口服 ATO 也是一种可能的选择：中位随访时间为 94 个月
. The introduction over the last decades of differentiating agents targeting the specific genetic aberration involved in pathogenesis, such as all-trans retinoic acid (ATRA) and arsenic trioxide (ATO), has turned acute promyelocytic leukemia (APL) into the most curable acute leukemia with almost 90% long-term survivors. 1 However, 5-20% of patients still relapsed after an initial remission. 1 Current guidelines of treatment in relapsed APL indicated the use of standard chemotherapy for early relapses after initial ATO-based treatment and, on the contrary, the use of ATO in case of no prior exposure, early relapse (6 months) after ATO regimen. 2 In a large series of relapsed patients treated with ATO, the hematological CR rate was 86%, the complete molecular CR was 52%, the estimated early death rate was 7%, and the estimated overall survival (OS) ranged between 50 and 81% at 24 months. 3 A meta-analysis compared the effect of ATO as single agent versus ATO plus ATRA, showing an advantage of the combination in terms of CR, and molecular CR, without increasing early death rate. 4 Is still a matter of discussion the best choice as consolidation treatment in patients re-treated with ATO reinduction: a recent study showed an advantage in OS after 5 years of patients who received autologous stem cell transplant (90.3%) compared to ATO-based maintenance (58.6%). 5 ATO was combined with bortezomib in a phase II trial with a molecular CR rate of 86%: PML/RARa is cleared by this combination through p62-dependent autophagy pathway. 6 Oral ATO could be also a possible option: at a median follow-up of 94 months