Translational Pediatrics Laboratory, Experimental Research Center, Hospital de Clínicas de Porto Alegre, Porto Alegre, RS, Brazil.
Graduate Program in Child and Adolescent Health, Universidade Federal do Rio Grande do Sul, Porto Alegre, RS, Brazil.
Br J Haematol. 2021 Jul;194(1):168-173. doi: 10.1111/bjh.17494. Epub 2021 May 15.
Our group recently showed that the (ASNase) formulation available in Brazil from 2017 to 2018 when used at the same dose and frequency as the formulation provided previously did not reach the activity considered therapeutic. Based on these, our goal was to assess the impact of these facts on the prognosis of children with ALL at different oncology centers. A multicentre retrospective observational study followed by a prospective follow-up. Patients aged >1 and <18 years in first-line treatment followed up at 10 referral centres, between 2014 and 2018 who received the formulation Leuginase were identified (Group B). For each patient, the centre registered 2 patients who received ASNase in the presentation of Aginasa exclusively (Group A). Data collection was registered using (Redcap ). A total of 419 patients were included; 282 in Group A and 137 in B. Group A had a 3-year OS and EFS of 91·8% and 84·8% respectively, while Group B had a 3-year OS of 83·8% (P = 0·003) and EFS of 76·1% (P = 0·008). There was an impact on 3-year OS and EFS of children who received a formulation. This result highlights the importance of evaluating ASNase and monitoring its activity.
我们的研究小组最近表明，2017 年至 2018 年在巴西使用的与之前提供的制剂相同剂量和频率的制剂（天冬酰胺酶）没有达到治疗活性。基于这些发现，我们的目标是评估这些因素对不同肿瘤中心 ALL 患儿预后的影响。这是一项多中心回顾性观察研究，随后进行前瞻性随访。我们确定了 2014 年至 2018 年间在 10 个转诊中心接受 Leuginase 治疗的年龄>1 岁和<18 岁的一线治疗患者（B 组）。每个中心还登记了 2 名仅接受过 ASNase 制剂治疗的患者（A 组）。数据收集使用（Redcap）进行登记。共纳入 419 例患者，A 组 282 例，B 组 137 例。A 组 3 年 OS 和 EFS 分别为 91.8%和 84.8%，B 组 3 年 OS 为 83.8%（P=0.003）和 EFS 为 76.1%（P=0.008）。接受制剂治疗的儿童 3 年 OS 和 EFS 受到影响。这一结果强调了评估 ASNase 并监测其活性的重要性。
