Pappo A S, Anderson J R, Crist W M, Wharam M D, Breitfeld P P, Hawkins D, Raney R B, Womer R B, Parham D M, Qualman S J, Grier H E
Intergroup Rhabdomyosarcoma Study Group, Rochester, MN 55905-0001, USA.
J Clin Oncol. 1999 Nov;17(11):3487-93. doi: 10.1200/JCO.1999.17.11.3487.
Despite advances in therapy, nearly 30% of children with rhabdomyosarcoma experience progressive or relapsed disease, which is often fatal.
To facilitate the development of a retrieval therapy protocol, we studied potential risk factors that were predictive of survival after first relapse in 605 children who were enrolled onto three consecutive Intergroup Rhabdomyosarcoma Study Group protocols.
The median survival time from first recurrence was 0.8 years; the estimated percentage of patients who survived 5 years from first recurrence was 17% +/- 2% (mean +/- SD). Univariate analysis showed that tumor histology was an important predictor of 5-year survival (P <.001): the 5-year survival rate was 64% for patients with botryoid tumors (n = 19), 26% for patients with embryonal tumors (n = 313), and 5% for patients with alveolar or undifferentiated sarcoma (n = 273). Further analysis identified prognostic factors within histologic subtypes (P <.001). For patients with embryonal tumors, the estimated 5-year survival rate was 52% for patients who initially presented with stage 1 or group I disease, 20% for those with stage 2/3 or group II/III disease, and 12% for those with group IV disease. For patients with stage 1/group I disease, estimated 5-year survival rates were higher for patients with local (72%) or regional (50%) recurrence than for those with distant (30%) recurrence. Among patients with alveolar or undifferentiated sarcoma, only the disease group predicted outcome: the 5-year survival estimate was 40% for group I versus 3% for groups II through IV. We identified a "favorable risk" group (approximately 20% of patients) whose 5-year estimated survival rate was near 50%; for all other patients, the estimated survival was near 10%.
This analysis demonstrates that the probability of 5-year survival after relapse for rhabdomyosarcoma is dependent on several factors at the time of initial diagnosis, including histologic subtype, disease group, and stage. These findings will form the basis of a multi-institutional risk-adapted relapse protocol for childhood rhabdomyosarcoma.
尽管治疗方法有所进步，但近30%的横纹肌肉瘤患儿仍经历疾病进展或复发，这往往是致命的。
为促进挽救治疗方案的制定，我们研究了605名连续参加三项横纹肌肉瘤国际协作组研究组方案的患儿首次复发后生存的潜在预测风险因素。
首次复发后的中位生存时间为0.8年；首次复发后存活5年的患者估计百分比为17%±2%（均值±标准差）。单因素分析显示肿瘤组织学是5年生存的重要预测因素（P<0.001）：葡萄状肿瘤患者（n=19）的5年生存率为64%，胚胎性肿瘤患者（n=313）为26%，肺泡型或未分化肉瘤患者（n=273）为5%。进一步分析确定了组织学亚型内的预后因素（P<0.001）。对于胚胎性肿瘤患者，最初表现为1期或I组疾病的患者估计5年生存率为52%，2/3期或II/III组疾病患者为20%，IV组疾病患者为12%。对于1期/I组疾病患者，局部（72%）或区域（50%）复发患者的估计5年生存率高于远处（30%）复发患者。在肺泡型或未分化肉瘤患者中，只有疾病分组可预测预后：I组的5年生存估计为40%，而II至IV组为3%。我们确定了一个“低危”组（约占患者的20%），其5年估计生存率接近50%；对于所有其他患者，估计生存率接近10%。
该分析表明，横纹肌肉瘤复发后5年生存概率取决于初始诊断时的几个因素，包括组织学亚型、疾病分组和分期。这些发现将构成儿童横纹肌肉瘤多机构风险适应性复发方案的基础。
