Department of Hematology, Hospital Nacional de Niños "Dr. Carlos Sáenz Herrera,", San José, Costa Rica.
Pediatr Blood Cancer. 2014 May;61(5):803-9. doi: 10.1002/pbc.24911. Epub 2013 Dec 24.
Five Asociación de Hemato-Oncología de Centroamérica (AHOPCA) countries have used an adapted BFM-based protocol for childhood acute lymphoblastic leukemia (ALL).
In the AHOPCA-ALL 2008 protocol, patients were stratified by age, white blood cell count, immunophenotype, central nervous system involvement, day 8 prednisone response, and morphologic bone marrow response to induction therapy. Patients at Standard Risk (SR) received a three-drug induction regimen, a reinduction phase, and maintenance with protracted intrathecal therapy. Those at Intermediate (IR) and High Risk (HR) received, in addition, daunorubicin during induction therapy, a consolidation phase and two or three reinduction phases respectively.
From August 2008 through July 2012, 1,313 patients were enrolled: 353 in SR, 548 in IR, 412 in HR. During induction therapy, 3.0% of patients died, 2.7% abandoned treatment, 1.1% had resistant ALL, and 93.2% achieved morphological complete remission (CR). Deaths and abandonment in first CR occurred in 2.7% and in 7.0% of patients, respectively. The relapse rate at a median observation time of 2.1 years was 15.0%. At 3 years, the event-free survival (EFS) and overall survival (OS), with abandonment considered as an event, were 59.4% (SE 1.7) and 68.2% (SE 1.6). Three-year EFS was 68.5% (SE 3.0), 62.1% (SE 2.6), and 47.8% (SE 3.2) for SR, IR, and HR groups. Adolescents had a significantly higher relapse rate (P = 0.001).
This experience shows that common international studies are feasible in lower-middle income countries. Toxic deaths, abandonment of treatment, and relapses remain major obstacles to the successful treatment. Alternative treatment strategies may be beneficial.
五个中美洲血液肿瘤协会（AHOPCA）国家使用改良的 BFM 为基础的方案治疗儿童急性淋巴细胞白血病（ALL）。
在 AHOPCA-ALL 2008 方案中，患者按年龄、白细胞计数、免疫表型、中枢神经系统受累、第 8 天泼尼松反应和诱导治疗后的形态骨髓反应进行分层。标准风险（SR）患者接受三药诱导治疗、再诱导期和延长鞘内治疗的维持治疗。中危（IR）和高危（HR）患者除诱导治疗期间接受柔红霉素外，还分别接受巩固治疗和两到三个再诱导治疗期。
2008 年 8 月至 2012 年 7 月，共纳入 1313 例患者：SR 组 353 例，IR 组 548 例，HR 组 412 例。诱导治疗期间，3.0%的患者死亡，2.7%的患者放弃治疗，1.1%的患者出现耐药 ALL，93.2%的患者获得形态学完全缓解（CR）。首次 CR 时的死亡和放弃治疗率分别为 2.7%和 7.0%。中位观察时间为 2.1 年时，复发率为 15.0%。3 年时，考虑放弃治疗为事件时的无事件生存率（EFS）和总生存率（OS）分别为 59.4%（SE 1.7）和 68.2%（SE 1.6）。SR、IR 和 HR 组 3 年 EFS 分别为 68.5%（SE 3.0）、62.1%（SE 2.6）和 47.8%（SE 3.2）。青少年的复发率明显较高（P=0.001）。
这一经验表明，常见的国际研究在中低收入国家是可行的。毒性死亡、放弃治疗和复发仍然是成功治疗的主要障碍。替代治疗策略可能有益。
