Zeltzer P M, Boyett J M, Finlay J L, Albright A L, Rorke L B, Milstein J M, Allen J C, Stevens K R, Stanley P, Li H, Wisoff J H, Geyer J R, McGuire-Cullen P, Stehbens J A, Shurin S B, Packer R J
University of California at Irvine Medical Center, Orange, USA.
J Clin Oncol. 1999 Mar;17(3):832-45. doi: 10.1200/JCO.1999.17.3.832.
From 1986 to 1992, "eight-drugs-in-one-day" (8-in-1) chemotherapy both before and after radiation therapy (XRT) (54 Gy tumor/36 Gy neuraxis) was compared with vincristine, lomustine (CCNU), and prednisone (VCP) after XRT in children with untreated, high-stage medulloblastoma (MB).
Two hundred three eligible patients with an institutional diagnosis of MB were stratified by local invasion and metastatic stage (Chang T/M) and randomized to therapy. Median time at risk from study entry was 7.0 years.
Survival and progression-free survival (PFS) +/- SE at 7 years were 55%+/-5% and 54%+/-5%, respectively. VCP was superior to 8-in-1 chemotherapy, with 5-year PFS rates of 63%+/-5% versus 45%+/-5%, respectively (P = .006). Upon central neuropathology review, 188 patients were confirmed as having MB and were the subjects for analyses of prognostic factors. Children aged 1.5 to younger than 3 years had inferior 5-year estimates of PFS, compared with children 3 years old or older (P = .0014; 32%+/-10% v 58%+/-4%, respectively). For MB patients 3 years of age or older, the prognostic effect of tumor spread (MO v M1 v M2+) on PFS was powerful (P = .0006); 5-year PFS rates were 70%+/-5%, 57%+/-10%, and 40%+/-8%, respectively. PFS distributions at 5 years for patients with M0 tumors with less than 1.5 cm2 of residual tumor, versus > or = 1.5 cm2 of residual tumor by scan, were significantly different (P = .023; 78%+/-6% v 54%+/-11%, respectively).
VCP plus XRT is a superior adjuvant combination compared with 8-in-1 chemotherapy plus XRT. For patients with M0 tumors, residual tumor bulk (not extent of resection) is a predictor for PFS. Patients with M0 tumors, > or = 3 years with < or = 1.5 cm2 residual tumor, had a 78%+/-6% 5-year PFS rate. Children younger than 3 years old who received a reduced XRT dosage had the lowest survival rate.
1986年至1992年，对未经治疗的高分期髓母细胞瘤（MB）患儿在放射治疗（XRT）（肿瘤54 Gy/神经轴36 Gy）前后采用“一日八药”（8-in-1）化疗与XRT后采用长春新碱、洛莫司汀（CCNU）和泼尼松（VCP）化疗进行了比较。
203例经机构诊断为MB的合格患者按局部侵犯和转移分期（Chang T/M）分层并随机接受治疗。从研究入组开始的中位风险时间为7.0年。
7年时的总生存率和无进展生存率（PFS）±标准误分别为55%±5%和54%±5%。VCP优于8-in-1化疗，5年PFS率分别为63%±5%和45%±5%（P = 0.006）。经中枢神经病理学复查，188例患者确诊为MB，作为预后因素分析的对象。1.5岁至未满3岁的儿童5年PFS估计值低于3岁及以上的儿童（P = 0.0014；分别为32%±10%和58%±4%）。对于3岁及以上的MB患者，肿瘤播散（M0 vs M1 vs M2+）对PFS的预后影响显著（P = 0.0006）；5年PFS率分别为70%±5%、57%±10%和40%±8%。扫描显示残留肿瘤面积小于1.5 cm²的M0肿瘤患者与残留肿瘤面积≥1.5 cm²的患者5年PFS分布有显著差异（P = 0.023；分别为78%±6%和54%±11%）。
与8-in-1化疗加XRT相比，VCP加XRT是一种更优的辅助联合治疗方案。对于M0肿瘤患者，残留肿瘤体积（而非切除范围）是PFS的预测指标。残留肿瘤面积≤1.5 cm²且年龄≥3岁的M0肿瘤患者5年PFS率为78%±6%。接受减量XRT剂量的3岁以下儿童生存率最低。
