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复发或难治性Ph(-) B前体急性淋巴细胞白血病的管理与治疗:一项针对欧盟临床医生的基于网络的双盲调查

Management and treatment of relapsed or refractory Ph(-) B-precursor ALL: a web-based, double-blind survey of EU clinicians.

作者信息

Saltman Deborah, Barlev Arie, Seshagiri Divyagiri, Katsoulis Ioannis, Lin Vincent, Barber Beth

机构信息

PRMA Consulting, Fleet, UK.

School of Public Health, Imperial College, London, UK.

出版信息

BMC Cancer. 2015 Oct 24;15:771. doi: 10.1186/s12885-015-1745-4.

Abstract

BACKGROUND

The prognosis for adult patients with Ph(-) B-precursor acute lymphoblastic leukaemia (ALL) who are refractory to treatment or experience relapse (R/R), is poor; over 90% of these patients die from the disease, typically within a few months. While there are some national guidelines published for the treatment of adult patients with ALL, and local working group recommendations do exist, there is very little detail and no preferred treatment regimens for adult patients with R/R Ph(-) B-precursor ALL. The aim of this study was to describe current real-world clinical practice in Europe for the management and treatment of adult R/R Ph(-) B-precursor ALL.

METHODS

A web-based, double-blind survey was conducted in November/December 2013 in France, Germany, Italy, Spain, and the UK. The survey was developed following consultation with specialist clinicians and a critical review of published literature. Eligible clinicians (15 per country) were board-certified in haemato-oncology or haematology; had at least 4 years of experience in their current role and had treated at least five patients with adult ALL in the 36 months before the survey, including at least one with R/R Ph(-) B-precursor ALL.

RESULTS

Clinicians across the five countries consulted 16 guidelines and local working group recommendations for the diagnosis and treatment of R/R Ph(-) B-precursor ALL. Thirty three regimens for salvage therapy were reported; the most frequently cited was augmented hyper-CVAD (15%), with vincristine the most commonly used agent. Salvage therapy regimens involved a range of agents, and most respondents reported using at least one cytotoxic agent; across respondents 10 different cytotoxic agents were cited. All respondents reported that toxicity was common for the regimens they used to treat R/R Ph(-) B-precursor ALL.

CONCLUSIONS

This study provides evidence of current management and treatment patterns of R/R Ph(-) B-precursor ALL in the real-world clinical practice in Europe. The approach to the treatment of R/R Ph(-) B-precursor ALL is heterogeneous, reflecting the lack of any clearly superior chemotherapeutic option, thus it appears that clinicians are trying a wide variety of therapies. These findings show a clear need for effective, tolerable treatments for R/R Ph(-) B-precursor ALL.

摘要

背景

成人 Ph(-) B 前体急性淋巴细胞白血病(ALL)患者若对治疗难治或复发(R/R),预后较差;超过 90%的此类患者死于该疾病,通常在数月内。虽然已发布了一些针对成人 ALL 患者治疗的国家指南,且确实存在当地工作组的建议,但对于成人 R/R Ph(-) B 前体 ALL 患者,细节很少且没有首选治疗方案。本研究的目的是描述欧洲目前针对成人 R/R Ph(-) B 前体 ALL 的管理和治疗的实际临床实践。

方法

2013 年 11 月/12 月在法国、德国、意大利、西班牙和英国进行了一项基于网络的双盲调查。该调查是在咨询专科临床医生并对已发表文献进行严格审查后制定的。符合条件的临床医生(每个国家 15 名)均为血液肿瘤学或血液学委员会认证;在其当前职位上至少有 4 年经验,并且在调查前的 36 个月内至少治疗过 5 例成人 ALL 患者,包括至少 1 例 R/R Ph(-) B 前体 ALL 患者。

结果

五个国家的临床医生查阅了 16 份关于 R/R Ph(-) B 前体 ALL 诊断和治疗的指南及当地工作组建议。报告了 33 种挽救治疗方案;最常被提及的是强化 Hyper-CVAD(15%),长春新碱是最常用的药物。挽救治疗方案涉及一系列药物,大多数受访者报告至少使用了一种细胞毒性药物;在所有受访者中提到了 10 种不同的细胞毒性药物。所有受访者均报告,他们用于治疗 R/R Ph(-) B 前体 ALL 的方案中,毒性很常见。

结论

本研究提供了欧洲实际临床实践中 R/R Ph(-) B 前体 ALL 当前管理和治疗模式的证据。R/R Ph(-) B 前体 ALL 的治疗方法各异,反映出缺乏任何明显更优的化疗选择,因此临床医生似乎在尝试多种疗法。这些发现表明,对于 R/R Ph(-) B 前体 ALL,迫切需要有效且可耐受的治疗方法。

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