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高剂量疗法及自体造血干细胞移植治疗复发性或难治性儿童霍奇金病:结果与预后指标

High-dose therapy and autologous hematopoietic stem-cell transplantation for recurrent or refractory pediatric Hodgkin's disease: results and prognostic indices.

作者信息

Lieskovsky YeeYie E, Donaldson Sarah S, Torres Mylin A, Wong Ruby M, Amylon Michael D, Link Michael P, Agarwal Rajni

机构信息

Department of Radiation Oncology, Stanford University Medical Center, Stanford, CA 94305-5847, USA.

出版信息

J Clin Oncol. 2004 Nov 15;22(22):4532-40. doi: 10.1200/JCO.2004.02.121.

Abstract

PURPOSE

To evaluate the outcome of pediatric patients with refractory or relapsed Hodgkin's disease (HD) who undergo high-dose therapy and autologous hematopoietic stem-cell transplantation (AHSCT).

PATIENTS AND METHODS

From 1989 to 2001, 41 pediatric patients with relapsed or primary refractory HD underwent high-dose therapy followed by AHSCT according to one of four autologous transplantation protocols at Stanford University Medical Center (Stanford, CA). Pretreatment factors were analyzed by univariate and multivariate analysis for prognostic significance for 5-year overall survival (OS), event-free survival (EFS), and progression-free survival (PFS).

RESULTS

At a median follow-up of 4.2 years (range, 0.7 to 11.9 years), the 5-year OS, EFS, and PFS rates were 68%, 53%, and 63%, respectively. Multivariate analysis determined the following three factors to be significant predictors of poor OS and EFS: extranodal disease at first relapse, presence of mediastinal mass at time of AHSCT, and primary induction failure. Two of these factors also predicted for poor PFS (extranodal disease at time of first relapse and presence of mediastinal mass at time of transplantation).

CONCLUSION

More than half of children with relapsed or refractory HD can be successfully treated with the combination of high-dose therapy and AHSCT, confirming the efficacy of this approach. Further investigation is now required to determine the optimal timing of AHSCT, as well as to develop alternative regimens for those patients with factors prognostic for poor outcome after AHSCT.

摘要

目的

评估接受大剂量治疗及自体造血干细胞移植(AHSCT)的难治性或复发性霍奇金淋巴瘤(HD)患儿的治疗结果。

患者与方法

1989年至2001年,41例复发性或原发性难治性HD患儿在斯坦福大学医学中心(加利福尼亚州斯坦福)按照四种自体移植方案之一接受了大剂量治疗,随后进行AHSCT。通过单因素和多因素分析对预处理因素进行分析,以确定其对5年总生存(OS)、无事件生存(EFS)和无进展生存(PFS)的预后意义。

结果

中位随访4.2年(范围0.7至11.9年),5年OS、EFS和PFS率分别为68%、53%和63%。多因素分析确定以下三个因素是OS和EFS不良的显著预测因素:首次复发时的结外病变、AHSCT时纵隔肿块的存在以及初次诱导失败。其中两个因素也预测了PFS不良(首次复发时的结外病变和移植时纵隔肿块的存在)。

结论

超过一半的复发性或难治性HD患儿可通过大剂量治疗和AHSCT联合成功治疗,证实了该方法的有效性。现在需要进一步研究以确定AHSCT的最佳时机,以及为那些AHSCT后预后不良因素的患者制定替代方案。

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