Center for Cancer and Blood Disorders, Connecticut Children's Medical Center, Hartford, Connecticut.
Division of Pediatric Hematology/Oncology, University of California San Francisco, Benioff Children's Hospital, San Francisco, California.
Pediatr Blood Cancer. 2019 Feb;66(2):e27524. doi: 10.1002/pbc.27524. Epub 2018 Oct 30.
Patients with recurrent or refractory osteosarcoma have a poor prognosis with less than 30% surviving two years. Eribulin is a synthetic analog of halichondrin B, has a novel mechanism of action when compared with other microtubule inhibitors, and may have antitumor activity in osteosarcoma.
A prospective study was designed to assess the disease control success at four months and objective response rates in patients with recurrent or refractory osteosarcoma treated with eribulin. Eligible patients were between 12 and 50 years of age, had measurable tumor, and met standard organ function requirements. Patients were given eribulin 1.4 mg/m /dose on days 1 and 8 of each 3-week cycle for up to 24 months if there was no progressive disease. Response to therapy was assessed using RECIST 1.1 criteria after cycles 2 and 5 and every fourth cycle thereafter.
Nineteen patients enrolled on the AOST1322 study. The median age of enrollment was 16 years (range, 12-25 years). Twelve patients were male and seven female. Eribulin was well tolerated, with neutropenia identified as the most common toxicity. The median progression-free survival was 38 days and no patients reached the four-month time point without progression. No objective responses were seen in any patient.
This study rapidly assessed the clinical activity of a novel agent in this patient population. Eribulin was well tolerated, but there were no patients who demonstrated objective response, and all patients had progression prior to four months.
复发性或难治性骨肉瘤患者的预后较差，两年生存率低于 30%。艾立布林是海鞘素 B 的合成类似物，与其他微管抑制剂相比具有新颖的作用机制，并且可能在骨肉瘤中有抗肿瘤活性。
设计了一项前瞻性研究，以评估接受艾立布林治疗的复发性或难治性骨肉瘤患者在四个月时疾病控制成功率和客观缓解率。符合条件的患者年龄在 12 至 50 岁之间，有可测量的肿瘤，并且符合标准器官功能要求。如果没有进行性疾病，患者在每个 3 周周期的第 1 天和第 8 天接受 1.4 mg/m /剂量的艾立布林，最多可接受 24 个月的治疗。在第 2 周期和第 5 周期以及此后每四个周期后使用 RECIST 1.1 标准评估治疗反应。
19 名患者参加了 AOST1322 研究。入组的中位年龄为 16 岁（范围为 12-25 岁）。12 名患者为男性，7 名女性。艾立布林耐受良好，中性粒细胞减少症是最常见的毒性。中位无进展生存期为 38 天，没有患者在四个月时无进展。没有患者出现客观缓解。
这项研究快速评估了新型药物在该患者人群中的临床活性。艾立布林耐受良好，但没有患者表现出客观缓解，所有患者在四个月前均出现进展。
