High-dose chemotherapy for Ewing sarcoma and Rhabdomyosarcoma: A systematic review by the Australia and New Zealand sarcoma association clinical practice guidelines working party

IF 11.3 1区化学 Q1 CHEMISTRY, PHYSICAL ACS Catalysis Pub Date : 2024-02-02 DOI:10.1016/j.ctrv.2024.102694

Ashika Ramamurthy , Elizabeth A Connolly , Jasmine Mar , Jeremy Lewin , Vivek A Bhadri , Marianne B Phillips , Mark Winstanley , Lisa M Orme , Peter Grimison , Joanna Connor , Smaro Lazarakis , Angela M Hong , Natacha Omer , Julie Cayrol

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Abstract

Introduction

Patients with high-risk or metastatic Ewing sarcoma (ES) and rhabdomyosarcoma (RMS) have a guarded prognosis. High-dose chemotherapy (HDT) with autologous stem cell transplant (ASCT) has been evaluated as a treatment option to improve outcomes. However, survival benefits remain unclear, and treatment is associated with severe toxicities.

Methods

A systematic review was conducted, using the population, intervention, comparison outcome (PICO) model, to evaluate whether utilization of HDT/ASCT impacts the outcome of patients with ES and RMS compared to standard chemotherapy alone, as part of first line treatment or in the relapse setting. Medline, Embase and Cochrane Central were queried for publications from 1990 to October 2022 that evaluated event-free survival (EFS), overall survival (OS), and toxicities. Each study was screened by two independent reviewers for suitability. A qualitative synthesis of the results was performed.

Results

Of 1,172 unique studies screened, 41 studies were eligible for inclusion with 29 studies considering ES, 10 studies considering RMS and 2 studies considering both. In ES patients with high-risk localised disease who received HDT/ASCT after VIDE chemotherapy, consolidation with melphalan-based HDT/ASCT as first line therapy conveyed an EFS and OS benefit over standard chemotherapy consolidation. Efficacy of HDT/ASCT using a VDC/IE backbone, which is now standard care, has not been established. Survival benefits are not confirmed for ES patients with metastatic disease at initial diagnosis. For relapsed/refractory ES, four retrospective studies report improvement in outcomes with HDT/ASCT with the greatest evidence in patients who demonstrate a treatment response before HDT, and in patients under the age of 14. In RMS, there is no proven survival benefit of HDT/ASCT in primary localised, metastatic or relapsed disease.

Conclusion

Prospective randomised trials are required to determine the utility of HDT/ASCT in ES and RMS. Selected patients with relapsed ES could be considered for HDT/ASCT.

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尤文肉瘤和横纹肌肉瘤的大剂量化疗：澳大利亚和新西兰肉瘤协会临床实践指南工作组的系统性综述

导言高危或转移性尤文肉瘤（ES）和横纹肌肉瘤（RMS）患者的预后堪忧。高剂量化疗（HDT）联合自体干细胞移植（ASCT）已被评估为一种可改善预后的治疗方案。方法采用人群、干预、比较结果（PICO）模型进行系统综述，以评估作为一线治疗的一部分或在复发情况下，与单纯标准化疗相比，使用HDT/ASCT是否会影响ES和RMS患者的预后。研究人员在 Medline、Embase 和 Cochrane Central 查询了 1990 年至 2022 年 10 月期间发表的评估无事件生存率 (EFS)、总生存率 (OS) 和毒性的论文。每项研究均由两名独立审稿人进行筛选，以确定是否合适。结果在筛选出的 1172 项独特研究中，有 41 项研究符合纳入条件，其中 29 项研究考虑了 ES，10 项研究考虑了 RMS，2 项研究同时考虑了两者。在VIDE化疗后接受HDT/ASCT的高危局部疾病ES患者中，以美法仑为基础的HDT/ASCT作为一线疗法的巩固治疗与标准化疗巩固治疗相比，具有EFS和OS获益。以VDC/IE为骨干的HDT/ASCT目前已成为标准疗法，但其疗效尚未确定。初次诊断时患有转移性疾病的 ES 患者的生存获益尚未得到证实。对于复发/难治性 ES，有四项回顾性研究报告称，HDT/ASCT 可改善预后，其中证据最多的是 HDT 前已出现治疗反应的患者和 14 岁以下的患者。对于 RMS，HDT/ASCT 对原发局部疾病、转移性疾病或复发疾病的生存获益尚未得到证实。部分复发的 ES 患者可考虑接受 HDT/ASCT。

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来源期刊

ACS Catalysis CHEMISTRY, PHYSICAL-

CiteScore

20.80

自引率

6.20%

发文量

1253

审稿时长

1.5 months

期刊介绍： ACS Catalysis is an esteemed journal that publishes original research in the fields of heterogeneous catalysis, molecular catalysis, and biocatalysis. It offers broad coverage across diverse areas such as life sciences, organometallics and synthesis, photochemistry and electrochemistry, drug discovery and synthesis, materials science, environmental protection, polymer discovery and synthesis, and energy and fuels. The scope of the journal is to showcase innovative work in various aspects of catalysis. This includes new reactions and novel synthetic approaches utilizing known catalysts, the discovery or modification of new catalysts, elucidation of catalytic mechanisms through cutting-edge investigations, practical enhancements of existing processes, as well as conceptual advances in the field. Contributions to ACS Catalysis can encompass both experimental and theoretical research focused on catalytic molecules, macromolecules, and materials that exhibit catalytic turnover.