Morgenstern Daniel A, Gibson Sian, Brown Tanya, Sebire Neil J, Anderson John
Unit of Molecular Haematology and Cancer Biology, UCL Institute of Child Health, London, UK.
Pediatr Blood Cancer. 2010 Jan;54(1):29-34. doi: 10.1002/pbc.22231.
Malignant rhabdoid tumours (MRT) and their central nervous system (CNS) counterparts atypical teratoid/rhabdoid tumours (ATRT) are rare, highly aggressive malignant neoplasms of childhood. Although there are isolated reports of long-term survival with intensive, multimodal therapy, outcomes are generally poor.
We conducted a retrospective review of all patients diagnosed with MRT/ATRT at Great Ormond Street Hospital over the 20 years from 1989 to 2009. All cases were subjected to expert pathological review including INI-1 immunostaining.
In a final cohort of 34 cases, overall survival was 17.4%, with median survival 10.1 months. Outcome in patients aged <3 years was significantly worse (median survival 6.2 months vs. 19.2 months). Data demonstrated a statistically significant benefit of radiotherapy (median survival 14.9 months vs. 6.6 months), although this analysis is confounded by the impact of patient age. There were four long-term survivors (>30 months), all of whom received chemotherapy with or without surgical resection or radiotherapy. In the present study, immunohistochemistry revealed no significant staining for either c-Erb or c-Met in any case, suggesting that targeting these molecules is unlikely to be of benefit in treating MRT/ATRT.
In view of poor outcomes, there is a clear need for new treatment strategies and the identification of novel molecular targets for MRT/ATRT.
恶性横纹肌样瘤（MRT）及其中枢神经系统（CNS）对应物非典型畸胎样/横纹肌样瘤（ATRT）是儿童罕见的、高度侵袭性的恶性肿瘤。尽管有关于强化多模式治疗后长期生存的个别报道，但总体预后通常较差。
我们对1989年至2009年20年间在大奥蒙德街医院诊断为MRT/ATRT的所有患者进行了回顾性研究。所有病例均接受了包括INI-1免疫染色在内的专家病理检查。
在最终的34例队列中，总生存率为17.4%，中位生存期为10.1个月。年龄<3岁患者的预后明显更差（中位生存期6.2个月对19.2个月）。数据显示放疗有统计学显著益处（中位生存期14.9个月对6.6个月），尽管该分析因患者年龄的影响而受到混淆。有4例长期存活者（>30个月），他们均接受了化疗，部分还接受了手术切除或放疗。在本研究中，免疫组化显示所有病例中c-Erb或c-Met均无明显染色，这表明靶向这些分子对治疗MRT/ATRT不太可能有益。
鉴于预后较差，显然需要新的治疗策略并确定MRT/ATRT的新分子靶点。
