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高剂量化疗后自体造血干细胞移植治疗儿童、青少年和青年初发尤文肉瘤的研究。

High-dose chemotherapy followed by autologous haematopoietic cell transplantation for children, adolescents, and young adults with first recurrence of Ewing sarcoma.

机构信息

Princess Máxima Center for Pediatric Oncology, Utrecht, Netherlands.

Department of Paediatric Oncology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam, Netherlands.

出版信息

Cochrane Database Syst Rev. 2021 Sep 2;9(9):CD011406. doi: 10.1002/14651858.CD011406.pub2.

Abstract

BACKGROUND

Ewing sarcoma is a solid tumour, which is the second most common primary bone malignancy in children, often occurring in the long bones and pelvis. An incidence rate of 4.5 per million a year is reported, with a peak incidence of 11 per million at the age of 12 years. Despite more intensive chemotherapy, 30% to 40% of young people with Ewing sarcoma will have recurrence of the disease. Less than 30% of young people with a recurrence of Ewing sarcoma are alive at 24 months, and less than 10% are alive at 48 months. High-dose chemotherapy (HDC), followed by autologous haematopoietic cell transplantation (AHCT), is used in a variety of paediatric groups with diverse solid tumours. The hypothesis is that HDC regimens may overcome resistance to standard polychemotherapy, and this way may eradicate minimal residual disease, leading to improved survival after a first recurrence of disease.

OBJECTIVES

To assess the efficacy of HDC with AHCT versus conventional chemotherapy in improving event-free survival, overall survival, quality-adjusted survival, and progression-free survival in children, adolescents, and young adults with first recurrence of Ewing sarcoma, and to determine the toxicity of the treatment.

SEARCH METHODS

We searched CENTRAL, MEDLINE, Embase, conference proceedings from the SIOP, ASPHO, CTOS, ASBMT, EBMT, and EMSOS, and two trial registries in January 2020. We also searched reference lists of relevant articles and review articles.

SELECTION CRITERIA

We planned to include randomised controlled trials (RCTs) or (historical) controlled clinical trials (CCTs) comparing the effectiveness of HDC plus AHCT with conventional chemotherapy for children, adolescents, and young adults (up to 30 years old at the date of diagnostic biopsy) with a first recurrence of Ewing sarcoma.

DATA COLLECTION AND ANALYSIS

We used standard methodological procedures expected by Cochrane.

MAIN RESULTS

We did not identify any eligible studies.

AUTHORS' CONCLUSIONS: Since we did not identify any eligible studies, we are unable to draw any conclusions about the efficacy and toxicity of HDC with AHCT versus conventional chemotherapy in children, adolescents, and young adults with a first recurrence of Ewing sarcoma. Further high-quality research is urgently needed.

摘要

背景

尤文肉瘤是一种实体肿瘤,是儿童中第二常见的原发性骨恶性肿瘤,常发生在长骨和骨盆。据报道,其年发病率为每百万分之 4.5,12 岁时的发病率峰值为每百万分之 11。尽管采用了更强化疗,仍有 30%至 40%的尤文肉瘤患儿会出现疾病复发。在 24 个月时,复发尤文肉瘤的年轻人中不到 30%存活,在 48 个月时不到 10%存活。高剂量化疗(HDC),随后进行自体造血细胞移植(AHCT),用于治疗各种具有不同实体瘤的儿科人群。其假设是 HDC 方案可能克服对标准多化疗的耐药性,并且可以消灭微小残留疾病,从而在首次疾病复发后提高生存。

目的

评估 HDC 联合 AHCT 与常规化疗在改善首次复发的尤文肉瘤患儿、青少年和年轻成人的无事件生存、总生存、质量调整生存和无进展生存方面的疗效,并确定治疗的毒性。

检索方法

我们于 2020 年 1 月检索了 CENTRAL、MEDLINE、Embase、SIOP、ASPOH、CTOS、ASBMT、EBMT 和 EMSOS 的会议记录,以及两个试验注册处。我们还检索了相关文章和综述文章的参考文献列表。

选择标准

我们计划纳入比较 HDC 联合 AHCT 与常规化疗在首次复发的尤文肉瘤儿童、青少年和年轻成人(诊断性活检时年龄不超过 30 岁)中的有效性的随机对照试验(RCT)或(历史)对照临床试验(CCT)。

数据收集与分析

我们使用 Cochrane 预期的标准方法程序。

主要结果

我们没有发现任何符合条件的研究。

作者结论

由于我们没有发现任何符合条件的研究,因此我们无法就 HDC 联合 AHCT 与常规化疗在首次复发的尤文肉瘤儿童、青少年和年轻成人中的疗效和毒性得出任何结论。迫切需要进一步开展高质量的研究。

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