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贝林妥欧单抗治疗复发或难治性霍奇金淋巴瘤患儿的真实世界研究结果。

Brentuximab vedotin in the treatment of paediatric patients with relapsed or refractory Hodgkin's lymphoma: Results of a real-life study.

机构信息

Paediatric Hematology, Oncology and Stem Cell Transplant Division, Padova University Hospital, Padua, Italy.

Maternal and Child Health Department, Padova University Hospital, Padua, Italy.

出版信息

Pediatr Blood Cancer. 2022 Oct;69(10):e29801. doi: 10.1002/pbc.29801. Epub 2022 Jun 2.

Abstract

BACKGROUND

Brentuximab vedotin (BV) is an antibody drug-conjugated anti-CD30 approved for the treatment of adult classical Hodgkin's lymphoma (HL), whereas it is considered as off-label indication in paediatrics. The aim of the study was to evaluate the safety and efficacy of BV to treat patients aged less than 18 years with refractory/relapsed HL.

MATERIALS AND METHODS

In this multicentre, retrospective study, 68 paediatric patients who received at least one dose of BV between November 2011 and August 2020 were enrolled. A median of nine doses of BV were administered as monotherapy (n = 31) or combined with other therapies (n = 37). BV was administrated alone as consolidation therapy after stem cell transplantation (SCT) in 12 patients, before SCT in 18 patients, whereas in 15 patients it was used before and after SCT as consolidation therapy. Median follow-up was 2.8 years (range: 0.6-8.9 years).

RESULTS

The best response was observed in the 86% of patients; the overall response rate was 66%. The 3-year progression-free survival was 58%, whereas the overall survival was 75%. No statistically significant differences between patients treated with BV monotherapy or combination were highlighted. In multivariate analysis, patients with non-nodular sclerosis HL and not transplanted had an increased risk of failure. Overall, 46% of patients had grade 3-4 adverse events that led to BV discontinuation in five of them.

CONCLUSION

In conclusion, our study confirms that BV was a safe and effective drug, able to induce complete remission, either as monotherapy or in association with standard therapy.

摘要

背景

本妥昔单抗维布妥昔单抗(BV)是一种抗 CD30 的抗体药物偶联物,获批用于治疗成人经典霍奇金淋巴瘤(HL),而在儿科则被认为是超适应证用药。本研究旨在评估 BV 治疗年龄小于 18 岁的难治/复发 HL 患者的安全性和有效性。

材料和方法

在这项多中心、回顾性研究中,纳入了 2011 年 11 月至 2020 年 8 月期间至少接受过一次 BV 治疗的 68 例儿科患者。31 例患者接受单药 BV 治疗(n=31),37 例患者接受 BV 联合其他治疗。12 例患者在干细胞移植(SCT)后单独接受 BV 巩固治疗,18 例患者在 SCT 前接受 BV 治疗,15 例患者在 SCT 前后接受 BV 巩固治疗。中位随访时间为 2.8 年(范围:0.6-8.9 年)。

结果

86%的患者获得最佳缓解,总体缓解率为 66%。3 年无进展生存率为 58%,总生存率为 75%。BV 单药或联合治疗的患者之间未观察到统计学差异。多变量分析显示,非结节性硬化型 HL 患者和未接受移植的患者复发风险增加。总体而言,46%的患者发生 3-4 级不良事件,其中 5 例患者因不良事件停止使用 BV。

结论

综上所述,本研究证实 BV 是一种安全有效的药物,无论是单药治疗还是联合标准治疗,均能诱导完全缓解。

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