研究动态
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在儿童和青少年急性淋巴细胞白血病中,分子最小残留病变的前瞻性应用于风险分层:奥地利AIEOP-BFM ALL 2000临床试验的长期结果。

Prospective use of molecular minimal residual disease for risk stratification in children and adolescents with acute lymphoblastic leukemia : Long-term results of the AIEOP-BFM ALL 2000 trial in Austria.

发表日期:2023 Aug 03
作者: Leila Ronceray, Michael Dworzak, Karin Dieckmann, Georg Ebetsberger-Dachs, Evgenia Glogova, Oskar A Haas, Neil Jones, Karin Nebral, Reinhard Moser, Thomas Lion, Bernhard Meister, Renate Panzer-Grümayer, Sabine Strehl, Christina Peters, Ulrike Pötschger, Christian Urban, Georg Mann, Andishe Attarbaschi,
来源: GENES & DEVELOPMENT

摘要:

自1979年起,奥地利的儿童和青少年急性淋巴细胞性白血病(ALL)采用柏林-法兰克福-明斯特(BFM)研究小组的协议进行治疗。Associazione Italiana di Ematologia e Oncologia Pediatrica和BFM(AIEOP-BFM) ALL 2000研究旨在通过在患者早期疾病过程中的两个时间点进行微小残留病(MRD)研究,将患者分层为三个风险组。通过应用基于定量聚合酶链反应的免疫球蛋白和T细胞受体基因的克隆特异性重排检测技术,监测MRD水平。在AIEOP-BFM 2000研究组中,对1999年6月至2009年12月期间接受治疗的608名奥地利患者的7年事件无进展生存率(EFS)和总生存率分别为84±2%和91±1%,观察时间中位数为6.58年。前体B-细胞和T-细胞ALL患者的事件无进展生存率分别为84±2%(n=521)和84±4%(n=87;p=0.460)。94%的患者可进行MRD评估,甚至可以在临床相关亚组的基础上确定低、中、高风险复发的前体B-细胞ALL患者。由于患者和事件数量较少,无法在T-ALL患者中找到与MRD相关性相似的结果。自此关键的国际AIEOP-BFM ALL 2000试验以来,分子治疗反应不断被用于AIEOP-BFM ALL研究组的所有后续试验中,通过进一步改进,将患者分层为不同的风险组。© 2023. The Author(s), under exclusive licence to Springer-Verlag GmbH Austria, part of Springer Nature.
Since 1979 Austrian children and adolescents with acute lymphoblastic leukemia (ALL) have been treated according to protocols of the Berlin-Frankfurt-Münster (BFM) study group. The Associazione Italiana di Ematologia e Oncologia Pediatrica and BFM (AIEOP-BFM) ALL 2000 study was designed to prospectively study patient stratification into three risk groups using minimal residual disease (MRD) on two time points during the patient's early disease course. The MRD levels were monitored by detection of clone-specific rearrangements of the immunoglobulin and T‑cell receptor genes applying a quantitative polymerase chain reaction-based technique. The 7‑year event-free survival (EFS) and overall survival rates for all 608 Austrian patients treated between June 1999 and December 2009 within the AIEOP-BFM 2000 study were 84 ± 2% and 91 ± 1%, respectively, with a median observation time of 6.58 years. Event-free survival for patients with precursor B‑cell and T‑cell ALL were 84 ± 2% (n = 521) and 84 ± 4% (n = 87; p = 0.460), respectively. The MRD assessment was feasible in 94% of the patients and allowed the definition of precursor B‑cell ALL patients with a low, intermediate or high risk of relapse even on top of clinically relevant subgroups. A similar finding with respect to MRD relevance in T‑ALL patients was not possible due to the small number of patients and events. Since this pivotal international AIEOP-BFM ALL 2000 trial, molecular response to treatment has been continuously used with additional refinements to stratify patients into different risk groups in all successive trials of the AIEOP-BFM ALL study group.© 2023. The Author(s), under exclusive licence to Springer-Verlag GmbH Austria, part of Springer Nature.