Peking University People's Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Peking University, Beijing, China.
Peking-Tsinghua Center for Life Sciences, Academy for Advanced Interdisciplinary Studies, Peking University, Beijing, China.
Br J Haematol. 2024 Aug;205(2):510-516. doi: 10.1111/bjh.19464. Epub 2024 Apr 26.
There is an urgent need for an oral, efficient and safe regimen for high-risk APL under the pandemic of COVID-19. We retrospectively analysed 60 high-risk APL patients. For induction therapy (IT), in addition to all-trans retinoic acid (ATRA) and oral arsenic (RIF), 22 patients received oral etoposide (VP16) as cytotoxic chemotherapy (CC), and 38 patients received intravenous CC as historical control group. The median dose of oral VP16 was 1000 mg [interquartile rage (IQR), 650-1250]. One patient died during IT in the control group, 59 evaluable patients (100%) achieved complete haematological remission (CHR) after IT and complete molecular remission (CMR) after consolidation therapy. The median time to CHR and CMR was 36 days (33.8-44) versus 35 days (32-42; p = 0.75) and 3 months (0.8-3.5) versus 3.3 months (2.4-3.7; p = 0.58) in the oral VP16 group and in the control group. Two (9.1%) and 3 (7.9%) patients experienced molecular relapse in different group respectively. The 2-year estimated overall survival and event-free survival were 100% versus 94.7% (p = 0.37) and 90.9% versus 89.5% (p = 0.97) respectively. A completely oral, efficient and safe induction regimen including oral VP16 as cytoreductive chemotherapy combined with ATRA and RIF is more convenient to administer for patients with high-risk APL.
在 COVID-19 大流行期间，高危 APL 急需一种口服、有效且安全的治疗方案。我们回顾性分析了 60 例高危 APL 患者。在诱导治疗（IT）中，除全反式维甲酸（ATRA）和口服砷剂（RIF）外，22 例患者接受口服依托泊苷（VP16）作为细胞毒性化疗（CC），38 例患者接受静脉 CC 作为历史对照组。口服 VP16 的中位剂量为 1000mg[四分位间距（IQR），650-1250]。对照组 1 例患者在 IT 期间死亡，59 例可评估患者（100%）在 IT 后达到完全血液学缓解（CHR），在巩固治疗后达到完全分子缓解（CMR）。CHR 和 CMR 的中位时间分别为 36 天（33.8-44）与 35 天（32-42;p=0.75）和 3 个月（0.8-3.5）与 3.3 个月（2.4-3.7;p=0.58）在口服 VP16 组和对照组。两组分别有 2 例（9.1%）和 3 例（7.9%）患者出现分子复发。2 年估计总生存率和无事件生存率分别为 100%与 94.7%（p=0.37）和 90.9%与 89.5%（p=0.97）。一种完全口服、有效且安全的诱导方案，包括口服 VP16 作为细胞减灭化疗，联合 ATRA 和 RIF，更便于治疗高危 APL 患者。
