Vincenzi B, Olimpieri P P, Celant S, Mazzocca A, Cortellini A, Comandone A, Tomassini L, Di Segni S, Russo P, Casali P G
Department of Medical Oncology, Università Campus Bio-Medico di Roma, Rome, Italy; Fondazione Policlinico Universitario Campus Bio-Medico, Rome, Italy.
Italian Medicines Agency, Rome, Italy.
ESMO Open. 2024 Dec;9(12):103995. doi: 10.1016/j.esmoop.2024.103995. Epub 2024 Nov 27.
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.
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.
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.
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.
帕唑帕尼是用于治疗接受过先前化疗的晚期非脂肪细胞性软组织肉瘤（STS）患者的治疗药物之一，但其在STS组织学类型中的最佳应用仍有待进一步明确。
2013年7月至2019年12月期间，通过意大利药品管理局（AIFA）管理的药物监测登记系统，前瞻性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。临床医生必须填写AIFA监测登记系统以开具帕唑帕尼。根据患者的临床特征、治疗开始时记录的组织学亚型以及临床结局进行记录。主要结局是治疗中断时间（TTD）。记录的次要结局是剂量减少频率和首次剂量减少时间。
我们分析了1964例肉瘤患者的数据。最常见的组织学亚型是平滑肌肉瘤（44.7%）、未分化肉瘤/未另行规定（11.5%）和滑膜肉瘤（8.1%）。总体而言，中位TTD为106天。与较短TTD显著相关的变量包括东部肿瘤协作组体能状态（1 - 2对比0）、先前治疗线数（2 - 4对比0 - 1）和规定剂量（200毫克或400毫克对比800毫克，均为每日一次）。在最常见（>20例患者）的组织学亚型中，与未另行规定的未分化肉瘤相比，组织学诊断为恶性孤立性纤维瘤的患者TTD也更长。
在这项全国性观察性真实世界研究中，结果与关键试验（PALETTE研究）报告的结果相似。我们的研究纳入了大量罕见/超罕见肉瘤亚型患者，并强调了这些组织学类型在治疗持续时间上可能存在的差异。
