Roda Ângela, Travassos Ana Rita, Soares-de-Almeida Luís, Has Cristina
Centro Hospitalar Lisboa Norte EPE, Hospital de Santa Maria, Serviço de Dermatologia, Lisboa, Portugal.
Dermatol Online J. 2018 Mar 15;24(3):13030/qt4k08r7x4.
Kindler syndrome is a rare, autosomal recessive genodermatosis, caused by mutations in the FERMT1 gene. It is thought to be primarily a skin disease, but other organs may also be involved. We report a case of a novel mutation of FERMT1 gene in a patient with a probable new phenotype of Kindler syndrome, including colitis and primary sclerosing cholangitis. A 42-year-old man, born to first cousin parents, was referred to our outpatient dermatology clinic with an unknown dermatosis since birth. He presented with neonatal blistering and developed photosensitivity and changes in skin pigmentation during childhood. Since the age of 20, he has had regular follow-up in the gastroenterology clinic, owing to esophageal stenosis, ulcerative colitis, and primary sclerosing cholangitis. Clinical examination revealed jaundice, poikiloderma, diffuse cigarette paper-like atrophy on dorsal surfaces of the hands, and palmoplantar hyperkeratosis. Skin biopsy showed epidermal atrophy covered by orthokeratotic hyperkeratosis. DNA molecular analysis revealed FERMT1 homozygous mutation c.1179G>A, p.W393X, which has not been reported before. The intestinal phenotype of Kindler syndrome has already been defined previously. However, to the best of our knowledge, no other case of primary sclerosing cholangitis in a patient with Kindler syndrome has been reported.
Kindler综合征是一种罕见的常染色体隐性遗传性皮肤病,由FERMT1基因突变引起。它被认为主要是一种皮肤病,但其他器官也可能受累。我们报告了一例FERMT1基因新突变的患者,其可能具有Kindler综合征的新表型,包括结肠炎和原发性硬化性胆管炎。一名42岁男性,父母为近亲结婚,自出生以来因不明皮肤病被转诊至我们的皮肤科门诊。他出生时即出现新生儿水疱,儿童期出现光敏性和皮肤色素沉着改变。20岁起,由于食管狭窄、溃疡性结肠炎和原发性硬化性胆管炎,他一直在胃肠病科定期随访。临床检查发现黄疸、皮肤异色症、双手背弥漫性卷烟纸样萎缩以及掌跖角化过度。皮肤活检显示表皮萎缩,其上覆盖正角化性过度角化。DNA分子分析显示FERMT1基因纯合突变c.1179G>A,p.W393X,此前未见报道。Kindler综合征的肠道表型此前已有定义。然而,据我们所知,尚未有Kindler综合征患者合并原发性硬化性胆管炎的其他病例报道。