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.

IF 1.9 4区 医学 Q2 MEDICINE, GENERAL & INTERNAL
Wiener Klinische Wochenschrift Pub Date : 2024-07-01 Epub Date: 2023-08-03 DOI:10.1007/s00508-023-02249-6
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
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引用次数: 0

Abstract

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.

Abstract Image

前瞻性使用分子极小残留病对儿童和青少年急性淋巴细胞白血病患者进行风险分层:奥地利AIEOP-BFM ALL 2000试验的长期结果。
自 1979 年以来,奥地利儿童和青少年急性淋巴细胞白血病(ALL)患者一直按照柏林-法兰克福-明斯特(BFM)研究小组的方案进行治疗。意大利儿科肿瘤协会和 BFM(AIEOP-BFM)2000 年急性淋巴细胞白血病研究的目的是在患者早期病程的两个时间点上,利用最小残留病(MRD)将患者分为三个风险组进行前瞻性研究。通过应用定量聚合酶链式反应技术检测免疫球蛋白和T细胞受体基因的克隆特异性重排来监测MRD水平。在AIEOP-BFM 2000研究中,1999年6月至2009年12月间接受治疗的608名奥地利患者的7年无事件生存率(EFS)和总生存率分别为84±2%和91±1%,中位观察时间为6.58年。前体B细胞和T细胞ALL患者的无事件生存率分别为84±2%(n = 521)和84±4%(n = 87;p = 0.460)。MRD评估在94%的患者中可行,即使在临床相关亚组之上,也可定义复发风险低、中或高的前体B细胞ALL患者。由于患者人数和事件较少,无法就T-ALL患者的MRD相关性得出类似结论。自这项关键性的国际 AIEOP-BFM ALL 2000 试验以来,在 AIEOP-BFM ALL 研究小组的所有后续试验中,治疗的分子反应一直被用于将患者分为不同的风险组别,并进行了进一步的改进。
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来源期刊
Wiener Klinische Wochenschrift
Wiener Klinische Wochenschrift 医学-医学:内科
CiteScore
4.70
自引率
3.80%
发文量
110
审稿时长
4-8 weeks
期刊介绍: The Wiener klinische Wochenschrift - The Central European Journal of Medicine - is an international scientific medical journal covering the entire spectrum of clinical medicine and related areas such as ethics in medicine, public health and the history of medicine. In addition to original articles, the Journal features editorials and leading articles on newly emerging topics, review articles, case reports and a broad range of special articles. Experimental material will be considered for publication if it is directly relevant to clinical medicine. The number of international contributions has been steadily increasing. Consequently, the international reputation of the journal has grown in the past several years. Founded in 1888, the Wiener klinische Wochenschrift - The Central European Journal of Medicine - is certainly one of the most prestigious medical journals in the world and takes pride in having been the first publisher of landmarks in medicine.
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