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美国转移性去分化脂肪肉瘤患者的真实世界生存结果及MDM2患病率

Real-world survival outcomes and MDM2 prevalence in US patients with metastatic dedifferentiated liposarcoma.

作者信息

Robinson Steven, Klein Alyssa B, Stanhope Stephen A, Evans Kathryn, Agulnik Mark

机构信息

Division of Medical Oncology, Mayo Clinic, Rochester, MN, USA.

Global Integrated Evidence, Boehringer Ingelheim Pharmaceuticals Inc, Ridgefield, CT, USA.

出版信息

Future Oncol. 2025 Jun;21(14):1797-1807. doi: 10.1080/14796694.2025.2502319. Epub 2025 Jun 4.

Abstract

BACKGROUND

Limited data exist regarding dedifferentiated liposarcoma (DDLPS) treatment, biomarker frequency, and clinical outcomes. Additional epidemiological data are needed to inform clinical trial design for testing novel therapeutics.

MATERIALS AND METHODS

Retrospective data from a US-based deidentified clinico-genomic database were analyzed for patients treated for metastatic DDLPS between 2011 and 2021.

RESULTS

Overall survival (OS), real-world progression-free survival (rwPFS), and time to next treatment (TTNT) were described in the overall cohort ( = 51) and in a subgroup of patients with murine double minute 2 () amplification and wild-type tumor protein p53 ( WT) ( = 38, 74.5%). Patients had a median age of 64.8 years, and 62.7% were male. The most common first-line treatment was doxorubicin with olaratumab (23.5%). From time of first-line (1 L) treatment, median OS for the entire cohort and -amplified, WT subgroup was 12.6 and 11.7 months, respectively; median rwPFS was 2.5 months for both. Median TTNT was 3.9 months for the full cohort and 4.8 months for the -amplified, WT subgroup.

CONCLUSIONS

The descriptive analysis here contributes real-world data describing treatment patterns, biomarker status, and clinical outcomes for patients with DDLPS, an aggressive and poorly characterized form of LPS with limited treatment options.

摘要

背景

关于去分化脂肪肉瘤(DDLPS)的治疗、生物标志物频率和临床结果的数据有限。需要更多的流行病学数据来为测试新型疗法的临床试验设计提供信息。

材料与方法

分析了一个美国匿名临床基因组数据库中的回顾性数据,该数据库涉及2011年至2021年间接受转移性DDLPS治疗的患者。

结果

描述了整个队列(n = 51)以及小鼠双微体2(MDM2)扩增且肿瘤蛋白p53野生型(p53 WT)患者亚组(n = 38,74.5%)的总生存期(OS)、真实世界无进展生存期(rwPFS)和下次治疗时间(TTNT)。患者的中位年龄为64.8岁,62.7%为男性。最常见的一线治疗是多柔比星联合奥拉单抗(23.5%)。从一线(1L)治疗开始,整个队列以及MDM2扩增、p53 WT亚组的中位OS分别为12.6个月和11.7个月;两者的中位rwPFS均为2.5个月。整个队列的中位TTNT为3.9个月,MDM2扩增、p53 WT亚组为4.8个月。

结论

本描述性分析提供了真实世界数据,描述了DDLPS患者的治疗模式、生物标志物状态和临床结果,DDLPS是一种侵袭性强且特征不明的脂肪肉瘤形式,治疗选择有限。

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