Department of Pediatric Solid Tumors, Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands.
Institut Curie, SIREDO Oncology Center (Care, Innovation and Research for Children and AYA with Cancer), PSL Research University, Paris, France.
Pediatr Blood Cancer. 2019 Oct;66(10):e27833. doi: 10.1002/pbc.27833. Epub 2019 Jun 26.
Malignant peripheral nerve sheath tumors (MPNST) are rare tumors of childhood. The role of standard chemotherapy in unresectable MPNST is still unclear. We report the outcome and prognostic factors in the EpSSG risk-adapted prospective study for localized pediatric MPNST.
Patients were stratified into four treatment groups defined by surgical resection, tumor size, and tumor grade (G): (a) surgery-only group-resected tumors G1; (b) adjuvant radiotherapy group-R0/R1, G2 tumors; (c) adjuvant chemotherapy group-R0/R1, G3 tumors; and (d) neoadjuvant chemotherapy group-R2 resected tumors and/or nodal involvement. Chemotherapy consisted of four courses of ifosfamide-doxorubicin and two courses of ifosfamide concomitant with radiotherapy (50.4-54 Gy).
Overall, the study included 51 patients. The 5-year event-free survival (EFS) and overall survival (OS) were 52.9% (95% confidence interval, 38.1-65.8) and 62.1% (46.7-74.3), respectively. The 5-year EFS was 92% (56.6-98.9) for treatment group 1 (N = 13), 33% (0.9-77.4) for treatment group 2 (N = 4), 29% (4.1-61.2) for treatment group 3 (N = 7), and 42% (23.1-60.1) for treatment group 4 (N = 27). Response rate to chemotherapy (partial response + complete response) in patients with measurable disease was 46%. The presence of neurofibromatosis type 1 (NF1; 51% of patients) was an independent poor prognostic factor for OS and EFS.
The outcome for patients with resectable MPNST was excellent. Standard ifosfamide-doxorubicin for unresectable MPNST rendered the best reported outcome. Children with NF1 disease seem to have worse prognosis.
恶性外周神经鞘瘤（MPNST）是儿童罕见的肿瘤。标准化疗在不可切除的 MPNST 中的作用仍不清楚。我们报告了 EpSSG 风险适应前瞻性研究中局部小儿 MPNST 的结果和预后因素。
患者根据手术切除、肿瘤大小和肿瘤分级（G）分为 4 个治疗组：（a）仅手术组-切除的 G1 肿瘤；（b）辅助放疗组-R0/R1，G2 肿瘤；（c）辅助化疗组-R0/R1，G3 肿瘤；和（d）新辅助化疗组-R2 切除的肿瘤和/或淋巴结受累。化疗包括四个疗程的异环磷酰胺-阿霉素和两个疗程的异环磷酰胺联合放疗（50.4-54 Gy）。
总体而言，该研究共纳入 51 例患者。5 年无事件生存率（EFS）和总生存率（OS）分别为 52.9%（95%置信区间，38.1-65.8）和 62.1%（46.7-74.3）。治疗组 1（N=13）的 5 年 EFS 为 92%（56.6-98.9），治疗组 2（N=4）为 33%（0.9-77.4），治疗组 3（N=7）为 29%（4.1-61.2），治疗组 4（N=27）为 42%（23.1-60.1）。可测量疾病患者化疗的缓解率（部分缓解+完全缓解）为 46%。神经纤维瘤病 1 型（NF1；51%的患者）的存在是 OS 和 EFS 的独立不良预后因素。
可切除 MPNST 患者的预后良好。标准的异环磷酰胺-阿霉素治疗不可切除的 MPNST 可获得最佳报道结果。NF1 疾病患儿预后似乎较差。
