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心面肢综合征中的纤维结构不良:病例报告及文献复习。

Fibrous dysplasia in cardio-facio-cutaneous syndrome: A case report and review of literature.

机构信息

Department of Paediatrics, KK Women's and Children's Hospital, Singapore.

Department of Diagnostic and Interventional Radiology, KK Women's and Children's Hospital, Singapore.

出版信息

Am J Med Genet A. 2022 Sep;188(9):2732-2737. doi: 10.1002/ajmg.a.62879. Epub 2022 Jul 7.

Abstract

Cardio-facio-cutaneous (CFC) syndrome (OMIM #:115150, 615278, 615279, 615280) is a rare genetic condition caused by variants in the RAS/mitogen-activated protein kinase (MAPK) signal transduction pathway. Up to 75% of cases are caused by mutations in the BRAF gene, whereas KRAS gene mutation has only been reported in <2% of cases. CFC syndrome is characterized by cardiac abnormalities, distinctive craniofacial dysmorphism, and various cutaneous abnormalities. Musculoskeletal and orthopedic manifestations are also prevalent in patients with CFC syndrome, among which the most common are skeletal deformities and joint laxities. Dysplastic bone disorders, on the other hand, have not been reported in CFC syndrome before. We report on a case of symmetrical polyostotic fibrous dysplasia (FD) in a patient with CFC syndrome with the KRAS(NM_004985.5):c.57G>C; p.Leu19Phe variant. The FDs were incidentally picked up, and patient was conservatively managed and remained asymptomatic on follow-up. The same variant was reported previously in a patient with Oculoectodermal Syndrome (OES), who developed polyostotic non-ossifying fibroma (NOF). This case explores FD as a possible new clinical feature of CFC syndrome, and when linked to the historical case of OES, explores whether the KRAS(NM_004985.5):c.57G>C; p.Leu19Phe mutation may potentially contribute to the development of dysplastic bone lesions in patients with this particular mutation.

摘要

心面颌皮肤(CFC)综合征(OMIM#:115150、615278、615279、615280)是一种罕见的遗传疾病,由 RAS/丝裂原活化蛋白激酶(MAPK)信号转导通路中的变异引起。多达 75%的病例是由 BRAF 基因突变引起的,而 KRAS 基因突变仅在<2%的病例中报道过。CFC 综合征的特征是心脏异常、独特的颅面发育不良和各种皮肤异常。骨骼肌肉和矫形表现也常见于 CFC 综合征患者,其中最常见的是骨骼畸形和关节松弛。然而,CFC 综合征以前没有报道过发育不良性骨疾病。我们报告了一例 CFC 综合征伴 KRAS(NM_004985.5):c.57G>C;p.Leu19Phe 变异的对称性多骨性纤维发育不良(FD)病例。FD 是偶然发现的,患者接受了保守治疗,随访时无症状。同样的变异以前也在眼外胚层发育不良(OES)患者中报道过,该患者发生了多骨性非骨化性纤维瘤(NOF)。该病例探讨了 FD 作为 CFC 综合征的一种可能的新临床特征,并结合 OES 的历史病例,探讨了 KRAS(NM_004985.5):c.57G>C;p.Leu19Phe 突变是否可能导致具有该特定突变的患者发育不良性骨病变的发展。

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