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急性淋巴细胞白血病(ALL)对 FDA 批准靶向治疗和免疫治疗反应的预测

英文原题:Prediction of Response to FDA-Approved Targeted Therapy and Immunotherapy in Acute Lymphoblastic Leukemia (ALL).

PubMed 2024/08/05(内容时间) Curr Treat Options Oncol Q1 · IF 5.8(JCR 2025)

研究概要

急性淋巴细胞白血病(ALL)是儿童人群中最主要的癌症,但在成人中相对罕见。

中文摘要

急性淋巴细胞白血病(ALL)是儿童人群中最主要的恶性肿瘤,但在成人中相对罕见。治疗前风险分层对于预测预后至关重要。评估的重要因素包括患者年龄、诊断时的白细胞(WBC)计数、髓外受累、免疫表型和细胞遗传学异常。微小残留病(MRD)主要在缓解后通过流式细胞术评估,在指导管理方案中发挥重要作用。在过去十年中,ALL的结局取得了显著进展。常规化疗显著降低了死亡率;然而,其强化性质引发了安全性担忧,并导致治疗耐药病例的出现及复发的再次发生。因此,美国食品药品监督管理局(FDA)已批准几种用于复发/难治性ALL的新型治疗,因其已证实的疗效,表现为完全缓解率和生存率的改善。这些治疗包括酪氨酸激酶抑制剂(TKIs)、抗CD19单克隆抗体blinatumomab、抗CD22 inotuzumab ozogamicin、抗CD20 rituximab和嵌合抗原受体(CAR)T细胞疗法。识别影响治疗决策的变量是迫切的必要需求,以便根据异质性患者特征制定个体化治疗。在各种观察性研究和临床试验中确定的关键预测因素包括淋巴细胞清除前疾病负荷、复杂遗传学异常和MRD。此外,可通过预测模型预判治疗后严重不良事件的发生,从而考虑采取适当的预防措施。最终目标是通过有效的预测平台将精准医学理念融入ALL领域,以促进选择最合适的治疗方法。

展开英文摘要原文

Acute lymphoblastic leukemia (ALL) represents the predominant cancer in pediatric populations, though its occurrence in adults is relatively rare. Pre-treatment risk stratification is crucial for predicting prognosis. Important factors for assessment include patient age, white blood cell (WBC) count at diagnosis, extramedullary involvement, immunophenotype, and cytogenetic aberrations. Minimal residual disease (MRD), primarily assessed by flow cytometry following remission, plays a substantial role in guiding management plans. Over the past decade, significant advancements in ALL outcomes have been witnessed. Conventional chemotherapy has remarkably reduced mortality rates; however, its intensive nature raises safety concerns and has led to the emergence of treatment-resistant cases with recurrence of relapses. Consequently, The U.S. Food and Drug Administration (FDA) has approved several novel treatments for relapsed/refractory ALL due to their demonstrated efficacy, as indicated by improved complete remission and survival rates. These treatments include tyrosine kinase inhibitors (TKIs), the anti-CD19 monoclonal antibody blinatumomab, anti-CD22 inotuzumab ozogamicin, anti-CD20 rituximab, and chimeric antigen receptor (CAR) T-cell therapy. Identifying the variables that influence treatment decisions is a pressing necessity for tailoring therapy based on heterogeneous patient characteristics. Key predictive factors identified in various observational studies and clinical trials include prelymphodepletion disease burden, complex genetic abnormalities, and MRD. Furthermore, the development of serious adverse events following treatment could be anticipated through predictive models, allowing for appropriate prophylactic measures to be considered. The ultimate aim is to incorporate the concept of precision medicine in the field of ALL through valid prediction platform to facilitate the selection of the most suitable treatment approach.

论文信息

作者
Khawaji ZY、Khawaji NY、Alahmadi MA、Elmoneim AA
单位
College of Medicine, Taibah University, Madinah, Kingdom of Saudi Arabia. Zakaria.khawaji1@gmail.com.Saudi Arabia
文献类型
综述
期刊
Current treatment options in oncology2024 Sep
原文标识
PubMed 39102166 · DOI 10.1007/s11864-024-01237-w