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成人软组织肉瘤的造血干细胞移植:来自欧洲血液和骨髓移植学会的分析。

Haematopoietic stem cell transplantation in adult soft-tissue sarcoma: an analysis from the European Society for Blood and Marrow Transplantation.

机构信息

Department of Translational Medical Oncology (TMO), National Center for Tumor Diseases (NCT) and German Cancer Research Center (DKFZ), Heidelberg, Germany; German Cancer Consortium (DKTK), Heidelberg, Baden-Württemberg, Germany; Department of Medicine V, University Hospital Heidelberg, Heidelberg, Baden-Württemberg, Germany.

EBMT Paris study office, Department of Haematology, Hospital Saint-Antoine, Paris, Île-de-France, France.

出版信息

ESMO Open. 2020 Oct;5(5):e000860. doi: 10.1136/esmoopen-2020-000860.

Abstract

BACKGROUND

The role of high-dose chemotherapy with autologous stem cell transplantation (ASCT) in the treatment of soft-tissue sarcoma (STS) remains an unsettled issue. Prospective clinical trials failed to prove a benefit of the procedure but were limited by small and heterogeneous patient cohorts. Thus, it is unknown if ASCT may be a valuable treatment option in specific patient subgroups.

METHODS

The purpose of this study was to investigate the value of ASCT according to histological subtype in STS patients who were registered in the European Society for Blood and Marrow Transplantation database between 1996 and 2016.

RESULTS

Median progression-free (PFS) and overall survival (OS) in the entire cohort of 338 patients were 8.3 and 19.8 months, respectively, and PFS and OS at 5 years were 13% and 25%, respectively. Analysis of outcomes in different subgroups showed that younger age, better remission status before transplantation and melphalan-based preparative regimen were predictive of benefit from ASCT, whereas histology and grading had no statistically significant impact.

CONCLUSIONS

Outcomes after ASCT compared favorably to those of recent trials on conventional chemotherapies and targeted therapies in STS, including histology-tailored approaches. ASCT, thus, should be reinvestigated in clinical trials focusing on defined patient subgroups.

摘要

背景

大剂量化疗联合自体造血干细胞移植(ASCT)在软组织肉瘤(STS)治疗中的作用仍存在争议。前瞻性临床试验未能证明该方法的益处,但受到患者队列小且异质性的限制。因此,尚不清楚 ASCT 是否可能成为特定患者亚组的有价值的治疗选择。

方法

本研究旨在根据组织学亚型调查 ASCT 在 1996 年至 2016 年间登记在欧洲血液和骨髓移植学会数据库中的 STS 患者中的价值。

结果

338 例患者的中位无进展生存期(PFS)和总生存期(OS)分别为 8.3 个月和 19.8 个月,5 年 PFS 和 OS 分别为 13%和 25%。不同亚组的结果分析表明,年龄较小、移植前缓解状态较好以及基于美法仑的预处理方案是 ASCT 获益的预测因素,而组织学和分级则没有统计学意义上的影响。

结论

ASCT 后的结果与 STS 中最近的常规化疗和靶向治疗试验(包括针对特定组织学的方法)相比具有优势。因此,应在针对特定患者亚组的临床试验中重新评估 ASCT。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/57ed/7590345/2c3b0445e513/esmoopen-2020-000860f01.jpg

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