Vetter B, Schwarz C, Kohne E, Kulozik A E
Department of General Paediatrics, Charité-Virchow Medical Centre, Humboldt University, Berlin, Germany.
Br J Haematol. 1997 May;97(2):266-72. doi: 10.1046/j.1365-2141.1997.342674.x.
In Germany homozygous beta-thalassaemia mainly occurs in the immigrant population from endemic regions. In non-immigrants beta-thalassaemia is rare. Heterozygous beta-thalassaemia minor, however, is more common and must be considered in the differential diagnosis of hypochromic anaemia. The clinical and molecular data of 221 homozygous patients and 256 non-immigrant German heterozygous individuals are presented. Clinically, 87% (n = 192) of the homozygotes are classified as thalassaemia major (TM) and the other 13% as thalassaemia intermedia (TI). There is a wide spectrum of 39 thalassaemia mutations which occur with relatively low frequencies in individual cases. In 17/29 TI patients 'mild' mutations have been found and in 16/29 there are mutations that are associated with increased gamma-globin gene activity. alpha-Thalassaemia is rare and found only in 3/29. In the 256 Germans with heterozygous beta-thalassaemia there are 27 different thalassaemia mutations. The three most common are Mediterranean, together accounting for 61%. Also relatively common (5%) is an otherwise rare frameshift mutation of codon 83 (FS83 deltaG). The other mutations occur in < 10 individuals. Two mutations described here are novel. One of them affects position -2 of the intron 1 splice acceptor site (IVSI-129 A-G) and the other is a deletion of a single G in codon 15/16 (FS 15/16 deltaG). These data suggest that beta-thalassaemia in Germans was introduced from the Mediterranean in about two-thirds of cases and that the remaining third has probably originated locally.
在德国,纯合子β地中海贫血主要发生在来自流行地区的移民群体中。在非移民人群中,β地中海贫血较为罕见。然而,杂合子β地中海贫血轻型更为常见,在低色素性贫血的鉴别诊断中必须予以考虑。本文呈现了221例纯合子患者和256例非移民德国杂合子个体的临床及分子数据。临床上,87%(n = 192)的纯合子被归类为重型地中海贫血(TM),另外13%为中间型地中海贫血(TI)。存在39种地中海贫血突变,其在个别病例中的发生频率相对较低。在17/29例TI患者中发现了“轻度”突变,16/29例中有与γ珠蛋白基因活性增加相关的突变。α地中海贫血罕见,仅在3/29例中发现。在256例患有杂合子β地中海贫血的德国人中,有27种不同的地中海贫血突变。最常见的三种是地中海型,共占61%。同样相对常见(5%)的是密码子83的一种罕见移码突变(FS83 deltaG)。其他突变发生在少于10个人中。这里描述的两种突变是新的。其中一种影响内含子1剪接受体位点的-2位置(IVSI-129 A-G),另一种是密码子15/16处单个G的缺失(FS 15/16 deltaG)。这些数据表明,德国人中的β地中海贫血约三分之二是从地中海地区传入的,其余三分之一可能起源于本地。