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儿童急性淋巴细胞白血病患者的临床特征与总生存期的关联

Association of the clinical profile and overall survival of pediatric patients with acute lymphoblastic leukemia.

作者信息

Correa Bruno Terra, Silva Gabriela Sales Serra, Mendes Webert Joaquim Silva, Maia Amanda de Moraes, Aleluia Augusto Cezar Magalhães, Fonseca Teresa Cristina Cardoso, da Guarda Caroline Conceição, Gonçalves Marilda de Souza, Aleluia Milena Magalhães

机构信息

Departamento de Ciências Biológicas, Laboratório de Patologia Aplicada e Genética, Universidade Estadual de Santa Cruz, Ilhéus, Bahia, Brasil.

Departamento de Medicina, UNEX, Itabuna, Bahia, Brasil.

出版信息

Front Pediatr. 2023 Sep 7;11:1223889. doi: 10.3389/fped.2023.1223889. eCollection 2023.

Abstract

INTRODUCTION

The clarification of etiopathology, the improvement of chemotherapy regimens and their risk stratifications, and the improvement in treatment support have increased the survival of children and adolescents affected by Acute Lymphoblastic Leukemia (ALL) past few years. This study aimed to estimate overall survival (OS) and event-free survival (EFS) in an onco-hematology treatment center in Brazil, reports the main clinical-laboratory characteristics of patients at diagnosis, verify the frequency of treatment-related adverse effects and the main causes of death.

MATERIAL AND METHODS

Retrospective analysis involving patients diagnosed with ALL, treated with the protocol of the Brazilian Group for Treatment of Leukemias in Childhood (GBTLI), between 2010 and 2020 was carried out; the outcomes (relapse, deaths, development of new neoplasms) were analyzed SPSS® software was used for the statistical analyses, and the -value was considered significant when less than 0.05 for all analyses.

RESULTS

109 patients were included in the study; the median age was 5 years, with a slight predominance of males. Sixty-six patients were classified as high-risk (HR) group and 43 patients were classified as low-risk (LR) group. After 5 years of diagnosis, the OS was 71.5%, and the EFS was 65%. No statistical difference was found between the HR and LR groups for OS and EFS, while leukocyte counts were statistically associated with the outcome of death (= 0.028). Among the patients, 28 (25.6%) died due to infection accounting 46.4% of death causes. Among the 34 patients with unfavorable outcomes (death and/or relapse), 32 had no research for the minimal residual disease at the end of remission induction, and 25 were not investigated for the presence of chromosomal abnormalities. The most reported complications and treatment-related adverse effects were increased liver transaminases (85.9%), airway infection (79.4%), oral mucositis (67.2%), febrile neutropenia (64.4%), and diarrhea (36.4%).

CONCLUSIONS

The rates of OS and EFS obtained in this cohort are similar to those obtained in the few previous similar studies in Brazil and lower than those carried out in developed countries. The unavailability of prognostic tests may have hindered risk stratification and influenced the results obtained.

摘要

引言

在过去几年中,急性淋巴细胞白血病(ALL)病因病理学的阐明、化疗方案及其风险分层的改进以及治疗支持的改善提高了受其影响的儿童和青少年的生存率。本研究旨在评估巴西一家肿瘤血液学治疗中心的总生存期(OS)和无事件生存期(EFS),报告患者诊断时的主要临床实验室特征,核实治疗相关不良反应的发生率以及主要死亡原因。

材料与方法

对2010年至2020年间诊断为ALL并按照巴西儿童白血病治疗组(GBTLI)方案进行治疗的患者进行回顾性分析;分析结局(复发、死亡、新发肿瘤);使用SPSS®软件进行统计分析,所有分析中P值小于0.05时被认为具有统计学意义。

结果

109例患者纳入研究;中位年龄为5岁,男性略占优势。66例患者被归类为高危(HR)组,43例患者被归类为低危(LR)组。诊断后5年,OS为71.5%,EFS为65%。HR组和LR组在OS和EFS方面未发现统计学差异,而白细胞计数与死亡结局具有统计学关联(P = 0.028)。患者中,28例(25.6%)死于感染,占死亡原因的46.4%。在34例预后不良(死亡和/或复发)的患者中,32例在缓解诱导结束时未进行微小残留病检测,25例未进行染色体异常检测。报告最多的并发症和治疗相关不良反应为肝转氨酶升高(85.9%)、气道感染(79.4%)、口腔黏膜炎(67.2%)、发热性中性粒细胞减少(64.4%)和腹泻(36.4%)。

结论

该队列获得的OS和EFS率与巴西之前少数类似研究的结果相似,但低于发达国家的研究结果。预后检测的不可用可能阻碍了风险分层并影响了所得结果。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7ad3/10513176/a76c53ada879/fped-11-1223889-g001.jpg

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