University Medical Center Hamburg-Eppendorf, Department of Pediatric Hematology and Oncology, Hamburg, Germany.
J Clin Oncol. 2010 Nov 20;28(33):4961-8. doi: 10.1200/JCO.2010.30.2299. Epub 2010 Oct 12.
To assess the prognostic role of clinical parameters and histology in early childhood medulloblastoma.
Clinical and histologic data from 270 children younger than age 5 years diagnosed with medulloblastoma between March 1987 and July 2004 and treated within prospective trials of five national study groups were centrally analyzed.
Two hundred sixty children with medulloblastoma and specified histologic subtype were eligible for analysis (median age, 1.89 years; median follow-up, 8.0 years). Rates for 8-year event-free survival (EFS) and overall survival (OS) were 55% and 76%, respectively, in 108 children with desmoplastic/nodular medulloblastoma (DNMB) or medulloblastoma with extensive nodularity (MBEN); 27% and 42%, respectively, in 145 children with classic medulloblastoma (CMB); and 14% and 14%, respectively, in seven children with large-cell/anaplastic (LC/A) medulloblastoma (P < .001). Histology (DNMB/MBEN: hazard ratio [HR], 0.44; 95% CI, 0.31 to 0.64; LC/A medulloblastoma: HR, 2.27; 95% CI, 0.95 to 5.54; P < .001 compared with CMB), incomplete resection and metastases (M0R1: HR, 1.86; 95% CI, 1.29 to 2.80; M+: HR, 2.28; 95% CI, 1.50 to 3.46; P < .001 compared with M0R0), and national group were independent prognostic factors for EFS, and OS. The HRs for OS ranged from 0.14 for localized M0 and DNMB/MBEN to 13.67 for metastatic LC/A medulloblastoma in different national groups.
Our results confirm the high frequency of desmoplastic variants of medulloblastomas in early childhood and histopathology as a strong independent prognostic factor. A controlled de-escalation of treatment may be appropriate for young children with DNMB and MBEN in future clinical trials.
评估临床参数和组织学在儿童早期髓母细胞瘤中的预后作用。
对 1987 年 3 月至 2004 年 7 月期间在五个国家研究组的前瞻性试验中诊断为髓母细胞瘤且年龄小于 5 岁的 270 名患儿的临床和组织学数据进行了中心分析。
260 名具有明确组织学亚型的髓母细胞瘤患儿符合分析条件（中位年龄为 1.89 岁；中位随访时间为 8.0 年）。108 例促纤维增生性/结节性髓母细胞瘤（DNMB）或广泛结节性髓母细胞瘤（MBEN）患儿的 8 年无事件生存率（EFS）和总生存率（OS）分别为 55%和 76%；145 例经典髓母细胞瘤（CMB）患儿分别为 27%和 42%；7 例大细胞/间变性（LC/A）髓母细胞瘤患儿分别为 14%和 14%（P<0.001）。组织学（DNMB/MBEN：危险比 [HR]，0.44；95%CI，0.31 至 0.64；LC/A 髓母细胞瘤：HR，2.27；95%CI，0.95 至 5.54；P<0.001 与 CMB 相比）、不完全切除和转移（M0R1：HR，1.86；95%CI，1.29 至 2.80；M+：HR，2.28；95%CI，1.50 至 3.46；P<0.001 与 M0R0 相比）和国家组是 EFS 的独立预后因素，OS 的 HR 范围从局部 M0 和 DNMB/MBEN 的 0.14 到不同国家组中转移性 LC/A 髓母细胞瘤的 13.67。
我们的结果证实了儿童早期促纤维增生性变异型髓母细胞瘤的高发生率和组织病理学作为一个强有力的独立预后因素。在未来的临床试验中，对于 DNMB 和 MBEN 的年轻患儿，可能需要适当控制治疗强度。
