阿扎胞苷
医学
骨髓增生异常综合症
国际预后积分系统
临床终点
肿瘤科
随机对照试验
内科学
威尼斯人
临床试验
低甲基化剂
人口
代理终结点
安慰剂
髓系白血病
重症监护医学
骨髓
白血病
病理
替代医学
DNA甲基化
慢性淋巴细胞白血病
化学
基因表达
基因
环境卫生
生物化学
作者
David A. Sallman,Zhuoer Xie
出处
期刊:Hematology
[American Society of Hematology]
日期:2023-12-08
卷期号:2023 (1): 65-72
被引量:1
标识
DOI:10.1182/hematology.2023000421
摘要
Abstract Although remarkable international efforts have been ongoing for over 17 years to improve upon azacitidine, representing the standard of care therapy for higher-risk myelodysplastic neoplasms (MDS), there still has not been a positive randomized trial in comparison to azacitidine. Real-world data from numerous trials have shown similar results with a median overall survival of 14-18 months, a 40%-50% overall response rate, and a complete remission rate close to 20%. Despite these outcomes, 6 randomized controlled trials have failed to improve outcomes in this patient population, although relevant issues in some of these studies included improper dose adjustments of the hypomethylating agent, lack of placebo- controlled studies, and lack of overall survival (OS) as a primary endpoint, among others. Critical updates in MDS management include the development of molecular prognostication models (eg, the molecular international prognostic scoring system), updates in classification systems highlighting significant overlap in patients with MDS-increased blasts and acute myeloid leukemia (most relevant to TP53 mutations), and refinement of response criteria. Although these paradigm-shifting studies have had great impact in MDS management, the current ongoing randomized phase 3 trials were initiated prior, and prognostic stratification remains via the revised international prognostic scoring system) and with bone marrow blast counts of <20%. Notably, azacitidine + venetoclax, azacitidine + sabatolimab, and azacitidine + magrolimab have shown exciting results in large, single-arm studies and have completed accrual in placebo-controlled, double-blind studies with OS as a primary endpoint. We all eagerly await the results of these studies.
科研通智能强力驱动
Strongly Powered by AbleSci AI