Sobczuk Paweł, Teterycz Paweł, Czarnecka Anna M, Świtaj Tomasz, Koseła-Paterczyk Hanna, Kozak Katarzyna, Falkowski Sławomir, Rutkowski Piotr
Department of Soft Tissue/Bone Sarcoma and Melanoma, Maria Sklodowska-Curie National Research Institute of Oncology, 02-781 Warsaw, Poland.
Department of Experimental and Clinical Physiology, Laboratory of Centre for Preclinical Research, Medical University of Warsaw, 02-097 Warsaw, Poland.
J Clin Med. 2020 Sep 29;9(10):3157. doi: 10.3390/jcm9103157.
Malignant peripheral nerve sheath tumor (MPNST) is a rare type of soft tissue sarcomas. The localized disease is usually treated with surgery along with perioperative chemo- or radiotherapy. However, up to 70% of patients can develop distant metastases. The study aimed to evaluate the modes and outcomes of systemic treatment of patients with diagnosed MPNST treated in a reference center. In total, 115 patients (56 female and 59 male) diagnosed with MPNST and treated due to unresectable or metastatic disease during 2000-2019 were included in the retrospective analysis. Schemes of systemic therapy and the outcomes-progression-free survival (PFS) and overall survival (OS)-were evaluated. The median PFS in the first line was 3.9 months (95% CI 2.5-5.4). Doxorubicin-based regimens were the most commonly used in the first line (50.4% of patients). There were no significant differences in PFS between chemotherapy regimens most commonly used in the first line ( = 0.111). The median OS was 15.0 months (95% CI 11.0-19.0) and the one-year OS rate was 63%. MPNST are resistant to the majority of systemic therapies, resulting in poor survival in advanced settings. Chemotherapy with doxorubicin and ifosfamide is associated with the best response and longest PFS. Future studies and the development of novel treatment options are necessary for the improvement of treatment outcomes.
恶性外周神经鞘瘤（MPNST）是一种罕见的软组织肉瘤。局限性疾病通常采用手术联合围手术期化疗或放疗进行治疗。然而，高达70%的患者会发生远处转移。本研究旨在评估在一个参考中心接受治疗的确诊MPNST患者的全身治疗模式和结果。共有115例（56例女性和59例男性）在2000年至2019年期间被诊断为MPNST并因不可切除或转移性疾病接受治疗的患者纳入回顾性分析。评估了全身治疗方案以及无进展生存期（PFS）和总生存期（OS）等结果。一线治疗的中位PFS为3.9个月（95%CI 2.5 - 5.4）。一线治疗中最常用的是以阿霉素为基础的方案（占患者的50.4%）。一线最常用的化疗方案之间的PFS无显著差异（ = 0.111）。中位OS为15.0个月（95%CI 11.0 - 19.0），一年OS率为63%。MPNST对大多数全身治疗耐药，导致晚期患者生存率较差。阿霉素和异环磷酰胺化疗的反应最佳，PFS最长。未来的研究和新治疗方案的开发对于改善治疗结果是必要的。
