Ortega J A, Douglass E C, Feusner J H, Reynolds M, Quinn J J, Finegold M J, Haas J E, King D R, Liu-Mares W, Sensel M G, Krailo M D
Division of Hematology/Oncology, Children's Hospital of Los Angeles, CA, USA. JORTEGA@
J Clin Oncol. 2000 Jul;18(14):2665-75. doi: 10.1200/JCO.2000.18.14.2665.
Previous studies demonstrated that chemotherapy with either cisplatin, vincristine, and fluorouracil (regimen A) or cisplatin and continuous infusion doxorubicin (regimen B) improved survival in children with hepatoblastoma. The current trial is a randomized comparison of these two regimens.
Patients (N = 182) were enrolled onto study between August 1989 and December 1992. After initial surgery, patients with stage I-unfavorable histology (UH; n = 43), stage II (n = 7), stage III (n = 83), and stage IV (n = 40) hepatoblastoma were randomized to receive regimen A (n = 92) or regimen B (n = 81). Patients with stage I-favorable histology (FH; n = 9) were treated with four cycles of doxorubicin alone.
There were no events among patients with stage I-FH disease. Five-year event-free survival (EFS) estimates were 57% (SD = 5%) and 69% (SD = 5%) for patients on regimens A and B, respectively (P =.09) with a relative risk of 1.54 (95% confidence interval, 0.93 to 2.5) for regimen A versus B. Toxicities were more frequent on regimen B. Patients with stage I-UH, stage II, stage III, or stage IV disease had 5-year EFS estimates of 91% (SD = 4%), 100%, 64% (SD = 5%), and 25% (SD = 7%), respectively. Outcome was similar for either regimen within disease stages. At postinduction surgery I, patients with stage III or IV disease who were found to be tumor-free had no events; those who had complete resections achieved a 5-year EFS of 83% (SD = 6%); other patients with stage III or IV disease had worse outcome.
Treatment outcome was not significantly different between regimen A and regimen B. Excellent outcome was achieved for patients with stage I-UH and stage II hepatoblastoma and for subsets of patients with stage III disease. New treatment strategies are needed for the majority of patients with advanced-stage hepatoblastoma.
既往研究表明，顺铂、长春新碱和氟尿嘧啶联合化疗（方案A）或顺铂与阿霉素持续输注联合化疗（方案B）可提高肝母细胞瘤患儿的生存率。本试验是对这两种方案进行随机对照比较。
1989年8月至1992年12月期间纳入182例患者进行研究。初始手术后，组织学类型为I期不良型（UH；n = 43）、II期（n = 7）、III期（n = 83）和IV期（n = 40）的肝母细胞瘤患者被随机分为接受方案A（n = 92）或方案B（n = 81）治疗。组织学类型为I期良好型（FH；n = 9）的患者仅接受4个周期的阿霉素治疗。
I期FH疾病患者未发生任何事件。方案A和方案B患者的5年无事件生存率（EFS）估计值分别为57%（标准差 = 5%）和69%（标准差 = 5%）（P = 0.09），方案A与方案B相比的相对风险为1.54（95%置信区间，0.93至2.5）。方案B的毒性反应更常见。I期UH、II期、III期或IV期疾病患者的5年EFS估计值分别为91%（标准差 = 4%）、100%、64%（标准差 = 5%）和25%（标准差 = 7%）。疾病分期内两种方案的结果相似。在诱导术后I期，III期或IV期疾病且肿瘤切除后无肿瘤残留的患者未发生任何事件；那些完全切除的患者5年EFS为83%（标准差 = 6%）；其他III期或IV期疾病患者的预后较差。
方案A和方案B的治疗结果无显著差异。I期UH和II期肝母细胞瘤患者以及部分III期疾病患者获得了良好的治疗结果。大多数晚期肝母细胞瘤患者需要新的治疗策略。
