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婴儿(<12 个月)和幼儿(12-36 个月)局限性横纹肌肉瘤在 EpSSG RMS 2005 研究中的治疗。

Localised rhabdomyosarcoma in infants (<12 months) and young children (12-36 months of age) treated on the EpSSG RMS 2005 study.

机构信息

Department of Oncology, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.

University College London Hospital NHS Foundation Trust, London, UK.

出版信息

Eur J Cancer. 2022 Jan;160:206-214. doi: 10.1016/j.ejca.2021.10.031. Epub 2021 Dec 2.

Abstract

BACKGROUND

Infants (<12 months) with rhabdomyosarcoma have historically had poorer outcome than the older age groups. We present outcomes for infants and young children aged 12-36 months with localised rhabdomyosarcoma with a particular emphasis on infants.

PATIENTS AND METHODS

All children less than 36 months of age enrolled on the EpSSG RMS 2005 study for localised disease are included. Treatment comprised chemotherapy, local surgery and/or radiation therapy adapted to risk group and age. Main outcome measures were event free survival (EFS) and overall survival (OS).

RESULTS

Outcome data were available for 485/490 patients aged less than 36 months, 110 were infants. Infants received chemotherapy according to the risk group with no toxic deaths. Radiotherapy was delivered to 33.6% of infants and 63.5% of 12-36 months old, with respectively 41.7% and 22.2% receiving brachytherapy. Radical surgery was performed in 62% of infants and 57.1% of 12-36 months old. Median follow up for patients who are alive (n = 393) was 72.7 months (range 6.9-158.2). Five-year OS for infants was 88.4% (95%CI 80.3-93.2), which is significantly better than the OS in 12-36 months old patients of 78.0% (95%CI 73.2-82.0; p = 0.0204). Five-year EFS for infants was 72.5% (95%CI 62.8-80.0) compared with 66.1% (95%CI 61.0-70.7; p = 0.2663) for 12-36 months old.

CONCLUSION

Infants treated on RMS 2005 achieved excellent EFS and OS. The EpSSG RMS 2005 chemotherapy regimen, combined with an increase in the application of adequate local therapy, improvements in imaging and supportive care and potentially favourable patients' characteristics may have contributed to these results.

摘要

背景

与年长儿童相比,患有横纹肌肉瘤的婴儿(<12 个月)的预后历来较差。我们报告了年龄在 12-36 个月的局限性横纹肌肉瘤婴儿和幼儿的结果,特别强调了婴儿。

患者和方法

所有年龄小于 36 个月且患有局限性疾病的 EpSSG RMS 2005 研究患儿均纳入本研究。治疗包括根据风险组和年龄调整的化疗、局部手术和/或放射治疗。主要观察指标为无事件生存率(EFS)和总生存率(OS)。

结果

485/490 名年龄小于 36 个月的患儿有结局数据,其中 110 名为婴儿。婴儿根据风险组接受化疗,无化疗相关死亡。33.6%的婴儿和 63.5%的 12-36 个月患儿接受了放疗,其中分别有 41.7%和 22.2%接受了近距离放疗。62%的婴儿和 57.1%的 12-36 个月患儿进行了根治性手术。存活患者(n=393)的中位随访时间为 72.7 个月(范围 6.9-158.2)。婴儿的 5 年 OS 为 88.4%(95%CI 80.3-93.2),明显优于 12-36 个月患儿的 78.0%(95%CI 73.2-82.0;p=0.0204)。婴儿的 5 年 EFS 为 72.5%(95%CI 62.8-80.0),而 12-36 个月患儿的 5 年 EFS 为 66.1%(95%CI 61.0-70.7;p=0.2663)。

结论

接受 RMS 2005 治疗的婴儿获得了极好的 EFS 和 OS。EpSSG RMS 2005 化疗方案,结合适当局部治疗的应用增加、成像和支持性治疗的改善以及可能有利的患者特征,可能促成了这些结果。

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