Abeyagunawardena Ishanya, Mayura Sinnathurai, Samarasingha Piyum, Nawinne Achala, Karunatilake Harindra
Internal Medicine, National Hospital of Sri Lanka, Colombo, LKA.
Hematology, National Hospital of Sri Lanka, Colombo, LKA.
Cureus. 2024 Dec 24;16(12):e76335. doi: 10.7759/cureus.76335. eCollection 2024 Dec.
Hereditary hemochromatosis occurs due to genetic mutations, namely, cysteine-to-tyrosine substitution at amino acid 282 (C282Y) and histidine-to-aspartic acid substitution at 63 (H63D) mutations. The role of H63D mutation in hemochromatosis is less clear, and its penetrance is low even in homozygotes. Therefore, iron overload in H63D heterozygotes is extremely rare and scarcely reported. We report the case of an asymptomatic Sinhalese man, previously unscreened, who was found to have elevated liver enzymes and hemoglobin in a routine medical check-up. His ferritin was 1272 (ng/ml) (22-322) with a transferrin saturation of 61% (15-50%). MRI of the abdomen for iron content revealed primary early iron deposition in the liver and pancreas with sparing of the spleen. Genetic studies detected H63D heterozygous homeostatic iron regulator (HFE) gene mutation with a normal C282Y gene. In his erythrocytosis workup, his erythropoietin level was suppressed. However, bone marrow biopsy did not reveal morphology suggestive of a clonal disorder, and he was negative for JAK2 V617F mutation, MPL gene, JAK2 exon 12 mutations, and calreticulin gene. He was diagnosed with H63D heterozygous hereditary hemochromatosis with iron overload and erythrocytosis and commenced on venesections as treatment for both conditions, with a good response. This case report highlights the rare possibility of developing clinically significant iron overload in H63D heterozygous hereditary hemochromatosis. Furthermore, several studies have reported the detection of HFE mutations in patients previously diagnosed with 'idiopathic' erythrocytosis. Hence, this case report calls attention to the need to suspect the presence of HFE gene mutations in patients with erythrocytosis with a negative workup for clonal red cell disorders.
遗传性血色素沉着症是由基因突变引起的,即第282位氨基酸的半胱氨酸被酪氨酸取代(C282Y)以及第63位的组氨酸被天冬氨酸取代(H63D)突变。H63D突变在血色素沉着症中的作用尚不清楚,即使在纯合子中其外显率也很低。因此,H63D杂合子中的铁过载极为罕见,鲜有报道。我们报告了一例无症状的僧伽罗族男性病例,此前未接受过筛查,在常规体检中发现其肝酶和血红蛋白升高。他的铁蛋白为1272(ng/ml)(22 - 322),转铁蛋白饱和度为61%(15 - 50%)。腹部MRI检查铁含量显示肝脏和胰腺有原发性早期铁沉积,脾脏未受累。基因研究检测到H63D杂合性稳态铁调节因子(HFE)基因突变,C282Y基因正常。在对其红细胞增多症的检查中,他的促红细胞生成素水平受到抑制。然而,骨髓活检未发现提示克隆性疾病的形态学改变,他的JAK2 V617F突变、MPL基因、JAK2外显子12突变和钙网蛋白基因均为阴性。他被诊断为H63D杂合性遗传性血色素沉着症伴铁过载和红细胞增多症,并开始进行静脉放血治疗这两种病症,反应良好。本病例报告强调了H63D杂合性遗传性血色素沉着症发生具有临床意义的铁过载的罕见可能性。此外,多项研究报告了在先前被诊断为“特发性”红细胞增多症的患者中检测到HFE突变。因此,本病例报告提醒人们注意,对于红细胞增多症且克隆性红细胞疾病检查结果为阴性的患者,需要怀疑存在HFE基因突变。