Metaxotou-Mavromati A, Kattamis C, Matathia L, Tzetis M, Kanavakis E
Department of Pediatrics, Athens University, St Sophie's Children's Hospital, Greece.
J Med Genet. 1988 Mar;25(3):195-9. doi: 10.1136/jmg.25.3.195.
The clinical and haematological phenotype as well as chain synthesis data were studied in 35 doubly heterozygous patients with either normal Hb A2 and Hb F, type 2 beta thalassaemia and beta (high A2) thalassaemia (26 patients), or type 2 and other rare beta or delta beta variants (nine patients). Patients doubly heterozygous for type 2 and beta zero or delta beta zero thalassaemia variants had no detectable Hb A, indicating that the type 2 normal A2 beta thalassaemia is primarily the result of a beta zero gene. The clinical phenotype varied from severe thalassaemia major to mild thalassaemia intermedia, and was mainly related to the thalassaemia variant with which the type 2 normal A2 beta thalassaemia was combined, and the proportion of Hb A produced in beta + thalassaemia patients. Haematological and chain synthesis data were similar in heterozygotes with type 2 and beta zero or beta + (high A2) thalassaemia. Hb A2 levels were within the normal range (2.3 to 3.6%) though absolute values (Hb A2 per RBC) ranged from low normal (0.5 pg/RBC) to increased levels (1.0 pg/RBC.) The variation of Hb A2, as well as the presence of Hb A2 in a type 2/delta beta high F patient and the complete absence of HbA2 in a homozygous type 2 patient, indicate that there are at least two genotypes of type 2, one beta zero and the other delta beta zero. This has been recently proven by gene mapping studies. For clinicians, routine haematological and family studies are sufficient for the proper treatment and prevention of doubly heterozygous type 2 patients.
对35例双重杂合子患者的临床和血液学表型以及链合成数据进行了研究,这些患者具有正常的Hb A2和Hb F、2型β地中海贫血和β(高A2)地中海贫血(26例),或2型与其他罕见的β或δβ变异(9例)。2型与β0或δβ0地中海贫血变异的双重杂合子患者未检测到Hb A,这表明2型正常A2β地中海贫血主要是β0基因的结果。临床表型从重型地中海贫血到中间型地中海贫血不等,主要与2型正常A2β地中海贫血合并的地中海贫血变异以及β+地中海贫血患者产生的Hb A比例有关。2型与β0或β+(高A2)地中海贫血杂合子的血液学和链合成数据相似。Hb A2水平在正常范围内(2.3%至3.6%),尽管绝对值(每个红细胞的Hb A2)范围从低正常(0.5 pg/红细胞)到升高水平(1.0 pg/红细胞)。Hb A2的变化,以及2型/δβ高F患者中Hb A2的存在和纯合子2型患者中HbA2的完全缺失,表明至少有两种2型基因型,一种是β0,另一种是δβ0。这一点最近已通过基因定位研究得到证实。对于临床医生来说,常规血液学和家族研究足以对双重杂合子2型患者进行适当的治疗和预防。