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引用次数: 0
摘要
血红蛋白(Hb) aginio是一种罕见的非缺失性a-球蛋白突变,几乎只在希腊,西班牙或其他地中海家庭中观察到。单个Hb aginio突变(单杂合性)携带者的临床表现取决于β、α或其他修饰基因中是否同时存在其他突变或变异。我们报告了一名希腊患者,其携带Hb aginio变异加上- - med α缺失等位基因,与典型的α地中海贫血患者相比,患有罕见的严重形式的α地中海贫血,需要定期输注红细胞(RBC)和螯合治疗。我们也提供了关于α地中海贫血血红蛋白变异及其分子和临床组合的简明文献综述。一项2期、随机、安慰剂对照、多中心临床试验,旨在确定luspatercept (BMS-986346/ACE-536)治疗成人α型地中海贫血的有效性和安全性,目前正在招募患者(NCT05664737)。
Concomitant Presence of Hb Agrinio and - -Med Deletion in a Greek Male Patient with Hemoglobinopathy H: More Severe Phenotype and Literature Review.
Hemoglobin (Hb) Agrinio is a rare non-deletional a-globin mutation observed almost exclusively in Greek, Spanish or other Mediterranean families. The clinical manifestations of a carrier of a single Hb Agrinio mutation (single heterozygosity) depend on the concomitant presence or absence of other mutations or variants in the beta, alpha or other modifying genes. We present a Greek patient harboring a Hb Agrinio variant plus the - -Med alpha deletional allele, having an infrequent severe form of alpha thalassemia, in contrast to the typical alpha thalassemic patient and requiring regular red blood cell (RBC) transfusions and chelation treatment. We also provide a concise literature review regarding alpha thalassemic hemoglobin variants and their molecular and clinical combinations. A phase 2, double-blind, randomized, placebo-controlled, multicenter clinical trial to determine the efficacy and safety of luspatercept (BMS-986346/ACE-536) for the treatment of anemia in adults with alpha thalassemia with the participation of our center is currently recruiting patients (NCT05664737).
期刊介绍:
Accounts of Chemical Research presents short, concise and critical articles offering easy-to-read overviews of basic research and applications in all areas of chemistry and biochemistry. These short reviews focus on research from the author’s own laboratory and are designed to teach the reader about a research project. In addition, Accounts of Chemical Research publishes commentaries that give an informed opinion on a current research problem. Special Issues online are devoted to a single topic of unusual activity and significance.
Accounts of Chemical Research replaces the traditional article abstract with an article "Conspectus." These entries synopsize the research affording the reader a closer look at the content and significance of an article. Through this provision of a more detailed description of the article contents, the Conspectus enhances the article's discoverability by search engines and the exposure for the research.