Diamantidis Michael D, Pitsava Stefania, Zayed Omar, Argyrakouli Ioanna, Karapiperis Konstantinos, Chatzoulis Christos, Alexiou Evangelos, Manafas Achilles, Tsangalas Evangelos, Karakoussis Konstantinos
Thalassemia and Sickle Cell Disease Unit, Department of Hematology, General Hospital of Larissa, 41221 Larissa, Greece.
Radiology Department, General Hospital of Larissa, 41221 Larissa, Greece.
Hematol Rep. 2023 Aug 8;15(3):483-490. doi: 10.3390/hematolrep15030050.
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).
血红蛋白(Hb)阿格里尼奥是一种罕见的非缺失型α-珠蛋白突变,几乎仅在希腊、西班牙或其他地中海家族中观察到。单一Hb阿格里尼奥突变携带者(单杂合子)的临床表现取决于β、α或其他修饰基因中其他突变或变异的伴存情况。我们报告了一名希腊患者,其携带Hb阿格里尼奥变异体以及--地中海α缺失等位基因,患有罕见的严重α地中海贫血,这与典型的α地中海贫血患者不同,需要定期进行红细胞(RBC)输血和螯合治疗。我们还提供了关于α地中海贫血血红蛋白变异体及其分子和临床组合的简要文献综述。一项由我们中心参与的2期、双盲、随机、安慰剂对照、多中心临床试验,旨在确定鲁司泊西(BMS-986346/ACE-536)治疗成人α地中海贫血贫血的疗效和安全性,目前正在招募患者(NCT05664737)。