Department of Dermatology, Hospital Clínic of Barcelona, University of Barcelona, Spain.
Department of Haematology, Hospital Clínic of Barcelona, University of Barcelona, Spain.
Br J Dermatol. 2018 Aug;179(2):486-490. doi: 10.1111/bjd.15927. Epub 2018 Mar 2.
Congenital erythropoietic porphyria is a rare autosomal recessive disease caused by a deficiency of uroporphyrinogen III synthase, owing to mutations in UROS in chromosome 10. Occasionally, patients show a mild, late-onset disease, without germline UROS mutations, associated with haematological malignancies. We report a 65-year-old patient with photosensitivity, overexcretion of porphyrins and thrombocytopenia. Bone marrow analysis gave a diagnosis of myelodysplastic syndrome (MDS) with the presence of a derivative chromosome 3, possibly due to an inversion including 3q21 and 3q26 break points. After allogeneic stem-cell transplantation, complete remission of MDS and uroporphyria was achieved. To our knowledge, this is the first reported case of acquired erythropoietic uroporphyria associated with MDS, with chromosome 3 alterations.
先天性红细胞生成性卟啉症是一种罕见的常染色体隐性疾病,由尿卟啉原 III 合酶缺乏引起,这是由于 10 号染色体上的 UROS 基因突变所致。偶尔,患者会出现无胚系 UROS 突变、与血液恶性肿瘤相关的轻度、迟发性疾病。我们报告了一例 65 岁的患者,表现为光敏感、卟啉过度排泄和血小板减少。骨髓分析诊断为骨髓增生异常综合征(MDS),存在衍生染色体 3,可能是由于包括 3q21 和 3q26 断裂点在内的倒位所致。异基因造血干细胞移植后,MDS 和尿卟啉症完全缓解。据我们所知,这是首例报道的与 MDS 相关的获得性红细胞生成性卟啉症,伴有 3 号染色体改变的病例。