促结缔组织增生性小圆细胞肿瘤：多模态治疗和新的危险因素。
Desmoplastic small round cell tumors: Multimodality treatment and new risk factors.
机构信息
Pediatrics 5, Olgahospital, Klinikum Stuttgart, Stuttgart, Germany.
Kiel Peadiatric Tumour Registry, Department of Pediatric Pathology, University Hospital Kiel, Germany.
出版信息
Cancer Med. 2019 Feb;8(2):527-542. doi: 10.1002/cam4.1940. Epub 2019 Jan 16.
BACKGROUND
To evaluate optimal therapy and potential risk factors.
METHODS
Data of DSRCT patients <40 years treated in prospective CWS trials 1997-2015 were analyzed.
RESULTS
Median age of 60 patients was 14.5 years. Male:female ratio was 4:1. Tumors were abdominal/retroperitoneal in 56/60 (93%). 6/60 (10%) presented with a localized mass, 16/60 (27%) regionally disseminated nodes, and 38/60 (63%) with extraperitoneal metastases. At diagnosis, 23/60 (38%) patients had effusions, 4/60 (7%) a thrombosis, and 37/54 (69%) elevated CRP. 40/60 (67%) patients underwent tumor resection, 21/60 (35%) macroscopically complete. 37/60 (62%) received chemotherapy according to CEVAIE (ifosfamide, vincristine, actinomycin D, carboplatin, epirubicin, etoposide), 15/60 (25%) VAIA (ifosfamide, vincristine, adriamycin, actinomycin D) and, 5/60 (8%) P6 (cyclophosphamide, doxorubicin, vincristine, ifosfamide, etoposide). Nine received high-dose chemotherapy, 6 received regional hyperthermia, and 20 received radiotherapy. Among 25 patients achieving complete remission, 18 (72%) received metronomic therapies. Three-year event-free (EFS) and overall survival (OS) were 11% (±8 confidence interval [CI] 95%) and 30% (±12 CI 95%), respectively, for all patients and 26.7% (±18.0 CI 95%) and 56.9% (±20.4 CI 95%) for 25 patients achieving remission. Extra-abdominal site, localized disease, no effusion or ascites only, absence of thrombosis, normal CRP, complete tumor resection, and chemotherapy with VAIA correlated with EFS in univariate analysis. In multivariate analysis, significant factors were no thrombosis and chemotherapy with VAIA. In patients achieving complete remission, metronomic therapy with cyclophosphamide/vinblastine correlated with prolonged time to relapse.
CONCLUSION
Pleural effusions, venous thrombosis, and CRP elevation were identified as potential risk factors. The VAIA scheme showed best outcome. Maintenance therapy should be investigated further.
背景
评估最佳治疗方法和潜在风险因素。
方法
分析了 1997 年至 2015 年在前瞻性 CWS 试验中接受治疗的<40 岁 DSRCT 患者的数据。
结果
60 例患者的中位年龄为 14.5 岁。男女比例为 4:1。56/60（93%）例肿瘤位于腹部/腹膜后。6/60（10%）例患者表现为局限性肿块，16/60（27%）例区域性播散性淋巴结，38/60（63%）例患者存在腹膜外转移。诊断时，23/60（38%）例患者存在胸腔积液，4/60（7%）例患者存在血栓，37/54（69%）例患者 CRP 升高。40/60（67%）例患者接受了肿瘤切除术，21/60（35%）例为肉眼完全切除。37/60（62%）例患者根据 CEVAIE（异环磷酰胺、长春新碱、放线菌素 D、卡铂、表柔比星、依托泊苷）方案接受化疗，15/60（25%）例患者接受 VAIA（异环磷酰胺、长春新碱、阿霉素、放线菌素 D）方案，5/60（8%）例患者接受 P6（环磷酰胺、多柔比星、长春新碱、异环磷酰胺、依托泊苷）方案。9 例患者接受了大剂量化疗，6 例患者接受了区域高温治疗，20 例患者接受了放疗。在 25 例达到完全缓解的患者中，18 例（72%）接受了节拍化疗。所有患者的 3 年无事件生存（EFS）和总生存（OS）率分别为 11%（±8 置信区间 [CI] 95%）和 30%（±12 CI 95%），25 例达到缓解的患者的 3 年 EFS 和 OS 率分别为 26.7%（±18.0 CI 95%）和 56.9%（±20.4 CI 95%）。非腹腔部位、局限性疾病、无胸腔积液或腹水、无血栓形成、CRP 正常、完全肿瘤切除和接受 VAIA 化疗与无事件生存呈单因素相关。多因素分析显示，无血栓形成和接受 VAIA 化疗是显著相关因素。在达到完全缓解的患者中，环磷酰胺/长春新碱节拍化疗与复发时间延长相关。
结论
胸腔积液、静脉血栓形成和 CRP 升高被认为是潜在的风险因素。VAIA 方案显示出最佳的结果。维持治疗应进一步研究。
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