Pazopanib in advanced desmoplastic small round cell tumours: a multi-institutional experience
Pazopanib in advanced desmoplastic small round cell tumours: a multi-institutional experience
复制标题
DOI：
10.1186/2045-3329-4-7
复制
发表时间：
2014-01-01
影响因子：
--
通讯作者：
Hayward, Larry
中科院分区：
文献类型：
--
作者：
Frezza, Anna Maria;Benson, Charlotte;Hayward, Larry
背景：我们回顾性回顾了9例接受帕唑帕尼治疗的转移性结缔组织增生小圆细胞瘤（DSRCT）患者的数据。患者和方法：3名患者接受了EORTC II期62043,3名患者接受了EORTC III期62072,3名患者接受了英国命名患者项目。结果：从数据库中检索到9例患者，中位年龄30岁（范围21 ~ 47岁），均为男性。所有人之前都接受过化疗。治疗开始时，4例（44%）患者ECOG PS 0、4 (44%)PS 1、1 (11%)PS 2。最佳缓解是2/9（22%）患者部分缓解（PR）， 5/9（56%）患者病情稳定（SD）， 2/9（22%）患者病情进展（PD），临床获益率（PR + SD bb0 12周）为78%。中位PFS和OS分别为9.2 （95% CI: 0-23.2）和15.4 （95% CI: 1.5-29.3）个月。中位随访时间为20个月，2/9（22%）患者仍然存活，全部进展。最常见的毒性包括中性粒细胞减少（G1-2 45%; G3-4 11%）、贫血（G1-2 45%）、疲劳（G1-2 67%）、腹泻（G1-2 45%; G3-4 11%）、恶心（G1-2 45%）、高血压（G1-2 45%）和肝酶升高（G1-2 34%; G3-4 11%）。3名患者（34%）需要减少剂量。一名患者因总胆红素水平持续升高而停止治疗，一名患者因患者选择而停止治疗。结论：在这个系列中，帕唑帕尼在既往化疗后进展的DSRCT患者中显示出有趣的活性，没有重大毒性。
Background: We retrospectively reviewed data from nine pre-treated metastatic desmoplastic small round cell tumour (DSRCT) patients who received pazopanib.Patients and methods: Three patients received pazopanib within the EORTC phase II 62043, three in the EORTC phase III 62072, and three in the context of UK named patient program.Results: Nine patients were retrieved from the databases, the median age was 30 years (range: 21-47), they were all males. All had received prior chemotherapy. At the time of treatment start, 4 patients (44%) had ECOG PS 0, 4 (44%) PS 1, 1 (11%) PS 2. Best response was partial response (PR) in 2/9 (22%) patients, stable disease (SD) in 5/9 (56%) and progressive disease (PD) in 2/9 (22%) with a clinical benefit rate (PR + SD > 12 weeks) of 78%. Median PFS and OS were 9.2 (95% CI: 0-23.2) and 15.4 (95% CI: 1.5-29.3) months respectively. With a median follow-up of 20 months, 2/9 (22%) patients are still alive, all progressed. The most common toxicities included neutropenia (G1-2 45%; G3-4 11%), anaemia (G1-2 45%), fatigue (G1-2 67%), diarrhoea (G1-2 45%; G3-4 11%), nausea (G1-2 45%), hypertension (G1-2 45%) and increase in liver enzymes (G1-2 34%; G3-4 11%). Three patients (34%) required a dose reduction. One of the patients discontinued treatment because of persistent increase in total bilirubin level, one due to patient's choice.Conclusion: In this series, pazopanib showed interesting activity in DSRCT patients who progressed after prior chemotherapy without major toxicity.