Pazopanib in the real-world setting in soft tissue sarcomas: data from the Italian national registry

IF 7.1 2区医学 Q1 ONCOLOGY

ESMO Open Pub Date : 2024-11-27 DOI:10.1016/j.esmoop.2024.103995

B. Vincenzi , P.P. Olimpieri , S. Celant , A. Mazzocca , A. Cortellini , A. Comandone , L. Tomassini , S. Di Segni , P. Russo , P.G. Casali

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引用次数: 0

Abstract

Background

Pazopanib is part of the therapeutic armamentarium for the treatment of patients with advanced non-adipocytic soft tissue sarcomas (STS) who have received prior chemotherapy, but its optimal use in STS histologies is still left to be further defined.

Design and methods

Data on STS patients treated with pazopanib in Italy have been prospectively collected from July 2013 to December 2019 through a drug monitoring registry managed by the Italian Medicines Agency (AIFA). This nationwide observational cohort study included patients with advanced STS who received pazopanib. Clinicians were mandatorily requested to fill in the AIFA monitoring registry in order to prescribe pazopanib.

Patients were recorded on the basis of their clinical characteristics, histological subtype captured at the time of treatment start, and clinical outcome. Primary outcome was time to treatment discontinuation (TTD). Secondary outcomes recorded were frequency of dose reduction and time to first dose reduction.

Results

We analyzed data from 1964 sarcoma patients. The most represented histological subtypes were leiomyosarcoma (44.7%), undifferentiated sarcomas/not otherwise specified (11.5%), and synovial sarcoma (8.1%). Overall, the median TTD was 106 days. The variables significantly associated to shorter TTD were Eastern Cooperative Oncology Group performance status (1-2 versus 0), the number of previous lines of treatment (2-4 versus 0-1) and prescribed dose (200 mg or 400 mg versus 800 mg, all once daily). Among the most represented (>20 patients) histological subtypes, we also observed longer TTD in patients with histological diagnosis of malignant solitary fibrous tumor if compared with undifferentiated sarcoma not otherwise specified.

Conclusions

In this nationwide observational real-world study, the outcomes are similar to those reported in the pivotal trial (PALETTE study). Our study includes a significant number of patients with rare/ultra-rare sarcoma subtypes and underlines possible differences in treatment duration among these histologies.

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帕唑帕尼在软组织肉瘤实际治疗中的应用：来自意大利国家登记处的数据

背景帕唑帕尼是治疗既往接受过化疗的晚期非脂肪细胞软组织肉瘤（STS）患者的治疗药物之一，但其在STS组织学中的最佳应用仍有待进一步明确。设计与方法从2013年7月至2019年12月，通过意大利药品管理局（AIFA）管理的药物监测登记处前瞻性地收集了意大利接受帕唑帕尼治疗的STS患者的数据。这项全国性的观察性队列研究纳入了接受帕唑帕尼治疗的晚期 STS 患者。临床医生被强制要求填写AIFA监测登记表，以便开具帕唑帕尼处方。患者的临床特征、开始治疗时捕获的组织学亚型和临床结果均被记录在案。主要结果是停药时间（TTD）。记录的次要结果是减量频率和首次减量时间。最常见的组织学亚型为亮肌肉瘤（44.7%）、未分化肉瘤/未另作说明的肉瘤（11.5%）和滑膜肉瘤（8.1%）。总体而言，中位TTD为106天。与较短的TTD明显相关的变量是东部合作肿瘤学组的表现状态（1-2对0）、之前的治疗次数（2-4对0-1）和处方剂量（200毫克或400毫克对800毫克，均为每日一次）。在最具代表性的组织学亚型（20 例患者）中，我们还观察到，组织学诊断为恶性单发纤维瘤的患者与未分化肉瘤（未另作说明）相比，TTD 更长。我们的研究包括了大量罕见/超罕见肉瘤亚型患者，并强调了这些组织类型在治疗时间上可能存在的差异。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

ESMO Open Medicine-Oncology

CiteScore

11.70

自引率

2.70%

发文量

255

审稿时长

10 weeks

期刊介绍： ESMO Open is the online-only, open access journal of the European Society for Medical Oncology (ESMO). It is a peer-reviewed publication dedicated to sharing high-quality medical research and educational materials from various fields of oncology. The journal specifically focuses on showcasing innovative clinical and translational cancer research. ESMO Open aims to publish a wide range of research articles covering all aspects of oncology, including experimental studies, translational research, diagnostic advancements, and therapeutic approaches. The content of the journal includes original research articles, insightful reviews, thought-provoking editorials, and correspondence. Moreover, the journal warmly welcomes the submission of phase I trials and meta-analyses. It also showcases reviews from significant ESMO conferences and meetings, as well as publishes important position statements on behalf of ESMO. Overall, ESMO Open offers a platform for scientists, clinicians, and researchers in the field of oncology to share their valuable insights and contribute to advancing the understanding and treatment of cancer. The journal serves as a source of up-to-date information and fosters collaboration within the oncology community.