Aflac Cancer Center and Blood Disorders Service/Children's Healthcare of Atlanta/Emory University, Atlanta, Georgia 30322, USA.
Cancer. 2012 Feb 1;118(3):761-9. doi: 10.1002/cncr.26190. Epub 2011 Jul 15.
The development of antigen-targeted therapies may provide additional options to improve outcomes in children with acute myeloid leukemia (AML). The Children's Oncology Group AAML03P1 trial sought to determine the safety of adding 2 doses of gemtuzumab ozogamicin, a humanized anti-CD33 antibody-targeted agent, to intensive chemotherapy during remission induction and postremission intensification for children with de novo AML.
AAML03P1 enrolled 350 children with previously untreated AML. Patients with a matched family donor received 3 courses of chemotherapy followed by hematopoietic stem cell transplantation; those without a matched family donor received 5 courses of chemotherapy. Gemtuzumab ozogamicin 3 mg/m(2)/dose was administered on Day 6 of Course 1 and Day 7 of Course 4.
Toxicities observed in all courses of therapy were typical of AML chemotherapy regimens, with infection being most common. Patients achieved a complete remission rate of 83% after 1 course and 87% after 2 courses. The mortality rate was 1.5% after the first gemtuzumab ozogamicin-containing induction course and 2.6% after 2 induction courses. The 3-year event-free survival and overall survival rates were 53 ± 6% and 66 ± 5%, respectively.
This trial determined that it is safe and feasible to include gemtuzumab ozogamicin in combination with intensive chemotherapy. The survival rates compare favorably with the recently published results of clinical trials worldwide.
抗原靶向治疗的发展可能为改善儿童急性髓系白血病（AML）的预后提供更多选择。儿童肿瘤组 AAML03P1 试验旨在确定在缓解诱导和缓解后强化阶段添加 2 剂吉妥珠单抗奥佐米星（一种人源化抗 CD33 抗体靶向药物）的安全性，用于治疗初治 AML 儿童。
AAAML03P1 纳入了 350 名未经治疗的 AML 儿童患者。有匹配家族供者的患者接受 3 个疗程的化疗，随后进行造血干细胞移植；无匹配家族供者的患者接受 5 个疗程的化疗。吉妥珠单抗奥佐米星 3mg/m2/剂量在第 1 疗程第 6 天及第 4 疗程第 7 天给药。
所有疗程的治疗毒性均为 AML 化疗方案的典型毒性，感染最常见。患者在第 1 个疗程后完全缓解率为 83%，第 2 个疗程后为 87%。第 1 个含吉妥珠单抗奥佐米星的诱导疗程后死亡率为 1.5%，第 2 个诱导疗程后死亡率为 2.6%。3 年无事件生存率和总生存率分别为 53±6%和 66±5%。
该试验确定了在强化化疗中联合使用吉妥珠单抗奥佐米星是安全可行的。生存率与最近发表的全球临床试验结果相比具有优势。
