U.O.C. Medicina Generale, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
Department of Internal Medicine, American University of Beirut Medical Center, PO Box 11-0236, Beirut, 11072020, Lebanon.
Ann Hematol. 2019 Apr;98(4):833-840. doi: 10.1007/s00277-018-3567-3. Epub 2018 Nov 30.
Beta-thalassemia can present with a wide spectrum of phenotypes determined by the coinheritance of α-thalassemia, hereditary persistence of fetal hemoglobin, and polymorphic variants in the BCL11A, HMIP, and HBB clusters. The codon 29 (cd29) mutation in the beta gene has been associated with a broad diversity of thalassemia phenotypes, possibly through genetic modifiers determining the genotype-phenotype relationship. In this study, we evaluated the effect of 10 single nucleotide polymorphisms (SNPs) on β-thalassemia severity in a group of 21 Lebanese patients bearing the cd29 mutation. Hematological parameters and clinical characteristics were evaluated according to transfusion dependence. The proportions and absolute concentrations of HbF were found to be higher in non-transfusion-dependent (NTD) patients than in transfusion-dependent (TD) ones. Iron parameters were found to be higher in TD patients. The SNPs that were evaluated included the XmnI-158 polymorphism in the HBG gene and SNPs in the BCL11A and HMIP loci. It was noted that individuals homozygous or heterozygous for the effect allele in the BCL11A and HMIP SNPs had higher HbF levels, lower ferritin concentrations, and lower liver iron content and were less likely to be transfusion dependent. Our results showed that HbF production variants may have an important impact on the severity of β-thalassemia, which might provide a severity prediction tool that can help in the anticipation of patients' phenotypes and therefore in future therapeutic decision making.
β-地中海贫血可表现出广泛的表型,这些表型由α-地中海贫血、胎儿血红蛋白遗传持续存在以及 BCL11A、HMIP 和 HBB 簇中的多态性变异共同决定。β 基因中的 29 号密码子(cd29)突变与多种地中海贫血表型相关,这可能是通过遗传修饰物来确定基因型-表型关系。在这项研究中,我们评估了 10 个单核苷酸多态性(SNP)对 21 名携带 cd29 突变的黎巴嫩患者β-地中海贫血严重程度的影响。根据输血依赖情况评估血液学参数和临床特征。非输血依赖(NTD)患者的 HbF 比例和绝对浓度高于输血依赖(TD)患者。TD 患者的铁参数较高。评估的 SNP 包括 HBG 基因中的 XmnI-158 多态性以及 BCL11A 和 HMIP 基因座中的 SNP。值得注意的是,BCL11A 和 HMIP SNP 中的效应等位基因纯合或杂合的个体具有更高的 HbF 水平、更低的铁蛋白浓度、更低的肝铁含量,并且不太可能依赖输血。我们的结果表明,HbF 产生变异可能对β-地中海贫血的严重程度有重要影响,这可能提供一种严重程度预测工具,有助于预测患者的表型,从而为未来的治疗决策提供参考。