Baĭdil'dina D D, Maschan M A, Skorobogatova E V, Dubrovina M E, Rumiantseva Iu V, Maschan A A, Rumiantsev A G, Samochatova E V
Ter Arkh. 2010;82(7):20-5.
To analyze the specific features of recurrences of acute promyelocytic leukemia (APL) in children after standard therapy with daunorubicin, cytosine arabinoside (Ara-C), all-trans retinoic acid (ATRA) and to develop further programmed treatment policy.
The study included 9 patients with recurrent APL. The recurrences developed significantly more frequently in a very high-risk group (patients with minimal residual disease being preserved after the intensive therapy phase). Induction used arsenic trioxide (ATO) and/or standard chemotherapy + ATRA; ATO monotherapy was in consolidation. CD34+ cells were mobilized until molecular remission was achieved with high-dose Ara-C and granulocyte colony-stimulating factor. Pretransplantation conditioning involved melfalan as a basic drug in combination with high-dose AraC (5 pts), treosulfan (1 pt) or bisulfan (1 pt). Six patients received gemtusumab ozogamicin, 3-9 mg/m2, at different stages of therapy.
Before therapy one patient died; 8 patients achieved the second molecular remission; CD34+ cell mobilization and sampling were effective in 7 cases. Five patients were in long-term molecular remission after autologous hemopoietic stem cell transplantation (autoHSCT). Follow-up was 23-40 months. One patient is being prepared for transplantation. Following autoHSCT, another patient with a developed repeat recurrence died from complications due to related partially compatible transplantation. Visceral, including cardiological, toxicity of therapy was insignificant. In the APL-2003 protocol, overall and event-free survival rates were 93 +/- 3 and 76 +/- 6%, respectively. CONCLUSION; The application of ATO and autoHSCT in recurrent APL makes it possible to achieve and preserve the second molecular remission in case of insignificant extrahematological toxicity. Russian clinics should have access to ATO.
分析儿童急性早幼粒细胞白血病（APL）经柔红霉素、阿糖胞苷（Ara-C）、全反式维甲酸（ATRA）标准治疗后复发的特点，并制定进一步的程序化治疗策略。
本研究纳入9例复发APL患者。复发在极高危组（强化治疗阶段后仍残留微小残留病的患者）中发生得更为频繁。诱导治疗采用三氧化二砷（ATO）和/或标准化疗+ATRA；巩固治疗采用ATO单药治疗。使用大剂量Ara-C和粒细胞集落刺激因子动员CD34+细胞，直至达到分子缓解。预处理以美法仑为基础药物，联合大剂量AraC（5例）、曲奥舒凡（1例）或白消安（1例）。6例患者在治疗的不同阶段接受了吉妥单抗奥唑米星，剂量为3-9mg/m²。
治疗前1例患者死亡；8例患者实现第二次分子缓解；7例患者的CD34+细胞动员和采集有效。5例患者在自体造血干细胞移植（autoHSCT）后处于长期分子缓解状态。随访时间为23-40个月。1例患者正在准备移植。autoHSCT后，另1例出现复发的患者因部分相合移植相关并发症死亡。治疗的内脏毒性，包括心脏毒性不明显。在APL-2003方案中，总生存率和无事件生存率分别为93±3%和76±6%。结论：在复发APL中应用ATO和autoHSCT，在血液学外毒性不明显的情况下，有可能实现并维持第二次分子缓解。俄罗斯诊所应能获得ATO。
