Department of Medical Oncology, The Cancer Institute Hospital of the Japanese Foundation for Cancer Research, Tokyo, Japan.
Department of Orthopedic Oncology, The Cancer Institute Hospital of the Japanese Foundation for Cancer Research, Tokyo, Japan.
PLoS One. 2023 Mar 1;18(3):e0280508. doi: 10.1371/journal.pone.0280508. eCollection 2023.
Trabectedin is a therapeutic option for patients with advanced sarcoma. While a randomized trial demonstrated its prolonged progression-free survival (PFS), the reported PFS was <6 months. Some patients can achieve long-term disease control with this treatment. However, the reference information is insufficient. Herein, we retrospectively reviewed 51 sarcoma patients who received trabectedin. We analyzed the clinicopathological features, trabectedin dose, administration schedule, and clinical outcomes, including the overall response rate (ORR) and PFS. Among them, we assessed the detailed data of patients who achieved long-term disease control (PFS >1 year). The ORR in the 49 evaluable patients was 8%, and the median PFS in 51 patients was 7.5 months. Six patients (12%) achieved PFS of >1 year. Five of the six patients had metastatic lesions at trabectedin initiation. The pathological subtypes were myxoid liposarcoma (n = 2), leiomyosarcoma (n = 2), synovial sarcoma (n = 1), and Ewing sarcoma (n = 1). The final administration dose was the minimum dose (0.8 mg/m2) in two patients who continued the treatment over 20 cycles. The best radiological response was partial response (PR) in two myxoid liposarcoma patients and stable disease in four. The durations from trabectedin initiation to the first response in the two PR cases were 163 and 176 days, respectively. Our results support the validity of continuing trabectedin at a sustainable dose and interval in patients who can tolerate it. These results may be useful when considering the clinical application of trabectedin.
曲贝替定是晚期肉瘤患者的一种治疗选择。虽然一项随机试验证明了其无进展生存期(PFS)延长,但报告的 PFS 不足 6 个月。一些患者可以通过这种治疗实现长期疾病控制。然而,参考信息不足。在此,我们回顾性分析了 51 例接受曲贝替定治疗的肉瘤患者。我们分析了临床病理特征、曲贝替定剂量、给药方案和临床结局,包括总缓解率(ORR)和 PFS。其中,我们评估了长期疾病控制(PFS>1 年)患者的详细数据。49 例可评估患者的 ORR 为 8%,51 例患者的中位 PFS 为 7.5 个月。6 例(12%)患者 PFS>1 年。6 例患者中,有 5 例在曲贝替定开始时就有转移病灶。病理亚型为黏液样脂肪肉瘤(n=2)、平滑肌肉瘤(n=2)、滑膜肉瘤(n=1)和尤因肉瘤(n=1)。5 例患者持续治疗超过 20 个周期,最终给予最小剂量(0.8mg/m2)。2 例黏液样脂肪肉瘤患者的最佳影像学反应为部分缓解(PR),4 例为疾病稳定。2 例 PR 患者从曲贝替定开始到首次反应的时间分别为 163 天和 176 天。我们的结果支持在能够耐受的患者中以可持续剂量和间隔继续使用曲贝替定的有效性。这些结果在考虑曲贝替定的临床应用时可能有用。