Gitto Lorenzo, Stoppacher Robert, Richardson Timothy Eric, Serinelli Serenella
State University of New York, Upstate Medical University, Department of Pathology, Syracuse, NY, USA.
Autops Case Rep. 2020 Sep 2;10(3):e2020203. doi: 10.4322/acr.2020.203.
Dyskeratosis congenita (DC) is a genetic syndrome with progressive multisystem involvement classically characterized by the clinical triad of oral leukoplakia, nail dystrophy, and reticular hyperpigmentation. Frequent complications are bone marrow failure, increased rate of malignancy, lung and liver diseases. DC results from an anomalous progressive shortening of telomeres resulting in DNA replication problems inducing replicative senescence. We report a death due to DC in a 16-year-old male with bone marrow failure and multiple organ dysfunction. At autopsy, nail dystrophy and skin hypopigmentation were observed. Gross and microscopic examinations of the internal organs showed cardiac hypertrophy, multiple lung consolidations and prominent interstitial fibrosis, liver cirrhosis, and fibrosis. Multiple foci of extramedullary hematopoiesis were identified, including on the epidural surface of the dura, that is an infrequent location, mimicking a focal area of epidural hemorrhage. Only a few autopsy studies about DC are reported in the literature. Further research should be done to understand the pathophysiology of the disease and its complications.
先天性角化不良(DC)是一种遗传性综合征,会进行性累及多系统,其典型临床特征为口腔黏膜白斑、指甲营养不良和网状色素沉着三联征。常见并发症有骨髓衰竭、恶性肿瘤发生率增加、肺部和肝脏疾病。DC是由端粒异常进行性缩短导致DNA复制问题,进而引发复制性衰老所致。我们报告了一名16岁男性因DC合并骨髓衰竭和多器官功能障碍死亡的病例。尸检时,观察到指甲营养不良和皮肤色素减退。对内脏器官进行大体和显微镜检查发现心脏肥大、多处肺实变及显著的间质纤维化、肝硬化和纤维化。发现多个髓外造血灶,包括在硬脑膜的硬膜外表面,这是一个不常见的部位,类似硬膜外出血的局灶性区域。文献中仅报道了少数关于DC的尸检研究。应进一步开展研究以了解该疾病及其并发症的病理生理学。