Nikolaos Spyrou, John E. Levine, James L.M. Ferrara
{"title":"Acute GVHD: New approaches to clinical trial monitoring","authors":"Nikolaos Spyrou, John E. Levine, James L.M. Ferrara","doi":"10.1016/j.beha.2022.101400","DOIUrl":null,"url":null,"abstract":"<div><p><span>Acute GVHD occurs in nearly 50% of patients receiving hematopoietic </span>cell transplantation<span><span><span> (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 </span>treatment. We then present preliminary data on the feasibility of a surrogate </span>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.</span></p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":null,"pages":null},"PeriodicalIF":2.2000,"publicationDate":"2022-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"2","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Best Practice & Research Clinical Haematology","FirstCategoryId":"3","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S152169262200055X","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q3","JCRName":"HEMATOLOGY","Score":null,"Total":0}
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.
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.