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首页> 美国卫生研究院文献>Cancer Medicine >Atypical teratoid rhabdoid tumor: improved long-term survival with an intensive multimodal therapy and delayed radiotherapy. The Medical University of Vienna Experience 1992–2012
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Atypical teratoid rhabdoid tumor: improved long-term survival with an intensive multimodal therapy and delayed radiotherapy. The Medical University of Vienna Experience 1992–2012

机译：非典型性teratoid横纹肌瘤：采用密集的多式联运疗法和延迟放疗可改善长期生存。维也纳医科大学经验1992–2012

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Atypical teratoid rhabdoid tumors (ATRTs) are recently defined highly aggressive embryonal central nervous system tumors with a poor prognosis and no definitive guidelines for treatment. We report on the importance of an initial correct diagnosis and disease-specific therapy on outcome in 22 consecutive patients and propose a new treatment strategy. From 1992 to 2012, nine patients initially diagnosed correctly as ATRT (cohort A, median age 24 months) were treated according to an intensive multimodal regimen (MUV-ATRT) consisting of three 9-week courses of a dose-dense regimen including doxorubicin, cyclophosphamide, vincristine, ifosfamide, cisplatin, etoposide, and methotrexate augmented with intrathecal therapy, followed by high-dose chemotherapy (HDCT) and completed with local radiotherapy. Thirteen patients were treated differently (cohort B, median age 30 months) most of whom according to protocols in use for their respective diagnoses. As of July 2013, 5-year overall survival (OS) and event-free survival (EFS) for all 22 consecutive patients was 56.3 ± 11.3% and 52.9 ± 11.0%, respectively. For MUV-ATRT regimen-treated patients (cohort A) 5-year OS was 100% and EFS was 88.9 ± 10.5%. For patients treated differently (cohort B) 5-year OS and EFS were 28.8 ± 13.1%. All nine MUV-ATRT regimen-treated patients are alive for a median of 76 months (range: 16–197), eight in first complete remission. Our results compare favorably to previously published data. The drug combination and sequence used in the proposed MUV-ATRT regimen appear to be efficacious in preventing early relapses also in young children with M1–M3 stage disease allowing postponement of radiotherapy until after HDCT.
机译：非典型性类畸形横纹肌瘤（ATRT）最近被定义为高度侵袭性的胚胎中枢神经系统肿瘤，预后较差，尚无明确的治疗指南。我们报告了在22位连续患者中进行初始正确诊断和针对疾病的治疗的重要性，并提出了一种新的治疗策略。从1992年到2012年，按照强化多式联运方案（MUV-ATRT）对9例最初被正确诊断为ATRT的患者（队列A，中位年龄为24个月）进行了治疗，该方案包括3个为期9周的剂量密集方案，包括阿霉素，鞘内治疗可增强环磷酰胺，长春新碱，异环磷酰胺，顺铂，依托泊苷和甲氨蝶呤，然后进行大剂量化疗（HDCT），并通过局部放疗完成。 13例患者接受了不同的治疗（队列B，中位年龄30个月），其中大多数根据各自诊断所用的方案进行治疗。截至2013年7月，连续22例患者的5年总生存率（OS）和无事件生存率（EFS）分别为56.3±11.3％和52.9±11.0％。对于接受MUV-ATRT方案治疗的患者（队列A），其5年OS为100％，EFS为88.9±10.5％。对于不同治疗的患者（队列B），其5年OS和EFS为28.8±13.1％。所有9名接受MUV-ATRT方案治疗的患者平均存活76个月（16-197个月），其中8例首次完全缓解。我们的结果与以前发布的数据相比具有优势。拟议的MUV-ATRT方案中使用的药物组合和顺序在预防M1–M3期疾病的幼儿中也可有效预防早期复发，从而允许放疗推迟至HDCT之后。

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