Acute GVHD: New approaches to clinical trial monitoring

IF 2.2 4区 医学 Q3 HEMATOLOGY
Nikolaos Spyrou, John E. Levine, James L.M. Ferrara
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引用次数: 2

Abstract

Acute GVHD occurs in nearly 50% of patients receiving hematopoietic cell transplantation (HCT), and is the major driver of mortality. However, progress in the development of new acute GVHD therapeutics has been slow, in part due to heterogeneity in acute GVHD data collection and interpretation among centers. Herein, we first describe the methods used by the Mount Sinai Acute GVHD International Consortium (MAGIC) to standardize acute GVHD data collection and curation. We then review the utility of serum biomarkers, specifically the MAGIC Algorithm Probability (MAP) that combines two GI biomarkers (ST2 and REG3α) that has been shown to be more accurate than changes in clinical symptom severity after GVHD treatment. We then present preliminary data on the feasibility of a surrogate clinical trial endpoint that combines clinical response and MAP two weeks after treatment. This novel endpoint is an earlier and potentially better predictor of non-relapse mortality than the current gold standard of clinical response four weeks after treatment.

急性GVHD:临床试验监测的新方法
急性GVHD发生在近50%接受造血细胞移植(HCT)的患者中,是死亡率的主要驱动因素。然而,新的急性GVHD治疗方法的开发进展缓慢,部分原因是各中心在急性GVHD数据收集和解释方面存在异质性。在此,我们首先描述了西奈山急性GVHD国际联盟(MAGIC)用于规范急性GVHD数据收集和管理的方法。然后,我们回顾了血清生物标志物的效用,特别是MAGIC算法概率(MAP),它结合了两种GI生物标志物(ST2和REG3α),已被证明比GVHD治疗后临床症状严重程度的变化更准确。然后,我们提出了在治疗后两周结合临床反应和MAP的替代临床试验终点的可行性的初步数据。这个新的终点比目前治疗后4周临床反应的金标准更早、更有可能更好地预测非复发死亡率。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
CiteScore
4.20
自引率
0.00%
发文量
42
审稿时长
35 days
期刊介绍: Best Practice & Research Clinical Haematology publishes review articles integrating the results from the latest original research articles into practical, evidence-based review articles. These articles seek to address the key clinical issues of diagnosis, treatment and patient management. Each issue follows a problem-orientated approach which focuses on the key questions to be addressed, clearly defining what is known and not known, covering the spectrum of clinical and laboratory haematological practice and research. Although most reviews are invited, the Editor welcomes suggestions from potential authors.
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