Rollón Noelia, Fernández-Jiménez María Cristina, Moreno-Carralero María Isabel, Murga-Fernández María José, Morán-Jiménez María Josefa
Department of Hematology, Complejo Hospitalario de Toledo, Hospital Virgen de la Salud, Avenida de Barber, 30, 45004, Toledo, Spain,
Int J Hematol. 2015 May;101(5):514-9. doi: 10.1007/s12185-014-1723-7. Epub 2014 Dec 30.
Sideroblastic anemias are a heterogeneous group of disorders characterized by anemia of varying severity and the presence of ringed sideroblasts in bone marrow. The most common form of inherited sideroblastic anemia is X-linked sideroblastic anemia (XLSA). In many XLSA patients, anemia responds variably to supplementation with pyridoxine (vitamin B6). We describe the case of a pregnant female with XLSA who had a novel mutation on the ALAS2 gene (c.1218G > T, p.Leu406Phe). Oral chelation therapy was contraindicated and high-dose vitamin B6 would have possible side effects in pregnancy. Serum hepcidin level was very low, indicating increased absorption of iron secondary to ineffective erythropoiesis. Therapy was begun with a low dose of pyridoxine that was increased post-partum. The patient's liver showed moderate iron deposits. During a subsequent 3-month period of pyridoxine supplementation, serum ferritin level and transferrin saturation decreased, hemoglobin content and serum hepcidin level normalized, and morphologic red cell abnormalities improved markedly. The patient responded well to treatment, showing the pyridoxine responsiveness of this novel ALAS2 mutation. The baby girl had the same mutation heterozygously, and although she was neither anemic nor showed abnormalities in a peripheral blood smear, she had a mild increment in RDW and her condition is now being followed.
铁粒幼细胞性贫血是一组异质性疾病,其特征为不同严重程度的贫血以及骨髓中出现环形铁粒幼细胞。遗传性铁粒幼细胞性贫血最常见的形式是X连锁铁粒幼细胞性贫血(XLSA)。在许多XLSA患者中,贫血对补充吡哆醇(维生素B6)的反应各不相同。我们描述了一例患有XLSA的孕妇病例,其ALAS2基因存在一种新的突变(c.1218G>T,p.Leu406Phe)。口服螯合疗法存在禁忌,高剂量维生素B6在孕期可能有副作用。血清铁调素水平非常低,表明继发于无效红细胞生成的铁吸收增加。治疗从低剂量的吡哆醇开始,产后增加剂量。患者肝脏显示有中度铁沉积。在随后3个月的吡哆醇补充期,血清铁蛋白水平和转铁蛋白饱和度下降,血红蛋白含量和血清铁调素水平恢复正常,红细胞形态异常明显改善。患者对治疗反应良好,表明这种新的ALAS2突变对吡哆醇有反应。女婴为该突变的杂合子,尽管她既不贫血,外周血涂片也无异常,但红细胞分布宽度轻度增加,目前正在对其病情进行随访。