Ellis Anthony, Risk Janet M, Maruthappu Thiviyani, Kelsell David P
Department of Gastroenterology, Royal Liverpool University Hospital, Prescot Street, Liverpool, L7 8XP, UK.
Department of Molecular & Clinical Cancer Medicine, Institute of Translational Medicine, University of Liverpool, L69 3BX, Liverpool, UK.
Orphanet J Rare Dis. 2015 Sep 29;10:126. doi: 10.1186/s13023-015-0346-2.
Tylosis (hyperkeratosis palmaris et plantaris) is characterised by focal thickening of the skin of the hands and feet and is associated with a very high lifetime risk of developing squamous cell carcinoma of the oesophagus. This risk has been calculated to be 95% at the age of 65 in one large family, however the frequency of the disorder in the general population is not known and is likely to be less than one in 1,000,000. Oesophageal lesions appear as small (2-5 mm), white, polyploid lesions dotted throughout the oesophagus and oral leukokeratosis has also been described. Although symptoms of oesophageal cancer can include dysphagia, odynophagia, anorexia and weight loss, there may be an absence of symptoms in early disease, highlighting the importance of endoscopic surveillance in these patients. Oesophageal cancer associated with tylosis usually presents in middle to late life (from mid-fifties onwards) and shows no earlier development than the sporadic form of the disease. Tylosis with oesophageal cancer is inherited as an autosomal dominant trait with complete penetrance of the cutaneous features, usually by 7 to 8 years of age but can present as late as puberty. Mutations in RHBDF2 located on 17q25.1 have recently been found to be causative. A diagnosis of tylosis with oesophageal cancer is made on the basis of a positive family history, characteristic clinical features, including cutaneous and oesophageal lesions, and genetic analysis for mutations in RHBDF2. The key management goal is surveillance for early detection and treatment of oesophageal dysplasia. Surveillance includes annual gastroscopy with biopsy of any suspicious lesion together with quadratic biopsies from the upper, middle and lower oesophagus. This is coupled with dietary and lifestyle modification advice and symptom education. Symptomatic management of the palmoplantar keratoderma includes regular application of emollients, specialist footwear and early treatment of fissures and super-added infection, particularly tinea pedis. More specific treatment for the thick skin is available in the form of oral retinoids, which are very effective but commonly produce side effects, including nasal excoriation and bleeding, hypercholesterolaemia, and abnormal liver function tests. Genetic counselling can be offered to patients and family members once a family history has been established. The prognosis of tylosis with oesophageal cancer is difficult to determine due to the limited number of affected individuals. In the last 40 years of surveillance, five out of six cases of squamous oesophageal cancer in the Liverpool family were detected endoscopically and were surgically removed. Four of five patients had stage 1 disease at presentation and remain alive and well more than 8 years later. This suggests that the presence of a screening program improves prognosis for these patients.
掌跖角化症(掌跖过度角化症)的特征是手脚皮肤局部增厚,且终生患食管鳞状细胞癌的风险极高。据计算,在一个大家庭中,65岁时患此病的风险为95%,然而,该疾病在普通人群中的发病率尚不清楚,可能低于百万分之一。食管病变表现为小的(2 - 5毫米)、白色、多倍体病变,散布于整个食管,口腔黏膜白斑病也有相关描述。尽管食管癌的症状可能包括吞咽困难、吞咽痛、厌食和体重减轻,但早期疾病可能没有症状,这凸显了对这些患者进行内镜监测的重要性。与掌跖角化症相关的食管癌通常在中年至老年(五十多岁起)出现,且不比散发性食管癌发病更早。伴有食管癌的掌跖角化症作为常染色体显性性状遗传,皮肤特征通常在7至8岁时完全显现,但也可能直到青春期才出现。最近发现位于17q25.1的RHBDF2基因突变是致病原因。伴有食管癌的掌跖角化症的诊断基于阳性家族史、特征性临床特征(包括皮肤和食管病变)以及对RHBDF2基因突变的基因分析。关键的管理目标是监测以便早期发现和治疗食管发育异常。监测包括每年进行胃镜检查,对任何可疑病变进行活检,并对食管上、中、下部位进行二次活检。同时还会提供饮食和生活方式调整建议以及症状教育。掌跖角化病的对症治疗包括定期涂抹润肤剂、穿特制鞋子以及早期治疗皲裂和继发感染,尤其是足癣。对于增厚皮肤更具特异性的治疗方法是口服维甲酸类药物,这类药物非常有效,但通常会产生副作用,包括鼻黏膜擦伤和出血、高胆固醇血症以及肝功能检查异常。一旦确定家族史,可为患者及其家庭成员提供遗传咨询。由于受影响个体数量有限,伴有食管癌的掌跖角化症的预后难以确定。在过去40年的监测中,利物浦家族的6例食管鳞状细胞癌中有5例通过内镜检查发现并手术切除。5名患者中有4名在就诊时处于1期疾病,8年多后仍存活且状况良好。这表明筛查项目的存在改善了这些患者的预后。