Abstract

Background

Previous pilot studies have shown the feasibility of preoperative chemotherapy in patients with medulloblastoma, but benefits and risks compared with initial surgery have not been assessed.

Methods

Two therapeutic strategies were retrospectively compared in 92 patients with metastatic medulloblastoma treated at Gustave Roussy between 2002 and 2015: surgery at diagnosis (n = 54, group A) and surgery delayed after carboplatin and etoposide-based neoadjuvant therapy (n = 38, group B). Treatment strategies were similar in both groups.

Results

The rate of complete tumor excision was significantly higher in group B than in group A (93.3% vs 57.4%, P = 0.0013). Postoperative complications, chemotherapy-associated side effects, and local progressions were not increased in group B. Neoadjuvant chemotherapy led to a decrease in the primary tumor size in all patients; meanwhile 4/38 patients experienced a distant progression. The histological review of 19 matched tumor pairs (before and after chemotherapy) showed that proliferation was reduced and histological diagnosis feasible and accurate even after neoadjuvant chemotherapy. The 5-year progression-free and overall survival rates were comparable between groups. Comparison of the longitudinal neuropsychological data showed that intellectual outcome tended to be better in group B (the mean predicted intellectual quotient value was 6 points higher throughout the follow-up).

Conclusion

Preoperative chemotherapy is a safe and efficient strategy for metastatic medulloblastoma. It increases the rate of complete tumor excision and may improve the neuropsychological outcome without jeopardizing survival.

Key Points

1. Preoperative chemotherapy increases the rate of complete tumor removal.2. No additional risk (toxic or disease progression) is linked to the delayed surgery.3. Preoperative chemotherapy could have a positive impact on the neuropsychological outcome of patients.

中文翻译：

---

新辅助化疗在转移性髓母细胞瘤中的作用：92例儿童的比较研究

摘要

背景

先前的先导研究表明，在髓母细胞瘤患者中进行术前化学疗法是可行的，但尚未评估与初次手术相比的益处和风险。

方法

回顾性分析2002年至2015年在Gustave Roussy治疗的92例转移性髓母细胞瘤患者的两种治疗策略：诊断时的手术（n = 54，A组）和卡铂和依托泊苷为基础的新辅助治疗后延迟手术（n = 38，B组） ）。两组的治疗策略相似。

结果

B组的完全肿瘤切除率显着高于A组（93.3％vs 57.4％，P= 0.0013）。B组的术后并发症，与化疗相关的副作用和局部进展没有增加。新辅助化疗导致所有患者的原发肿瘤大小减小。同时，有4/38的患者进展较远。对19个匹配的肿瘤对（在化疗前后）的组织学检查表明，即使在新辅助化疗后，增殖也减少，组织学诊断可行且准确。两组之间的5年无进展生存率和总生存率相当。纵向神经心理学数据的比较表明，B组的智力结局趋向于更好（在整个随访过程中，平均预测的智力商值平均高6点）。

结论

术前化疗是转移性髓母细胞瘤的安全有效策略。它增加了肿瘤的完全切除率，并且可以改善神经心理学结果，而不会危及生存。

关键点

1.术前化学疗法可提高肿瘤完全切除率。2。没有额外的风险（毒性或疾病进展）与延迟手术有关3。术前化疗可能会对患者的神经心理结局产生积极影响。