Centre for Rare Diseases and Transition, Department of Woman and Child Health and Public Health, Fondazione Policlinico Universitario A. Gemelli IRCCS, 00168 Rome, Italy.
Department of Life Sciences and Public Health, Faculty of Medicine and Surgery, Catholic University of Sacred Heart, 00168 Rome, Italy.
Genes (Basel). 2024 Aug 23;15(9):1109. doi: 10.3390/genes15091109.
Feeding difficulties are constantly present in patients with Crisponi/cold-induced sweating syndrome type 1 (CS/CISS1). The aim of our study was to describe their prevalence and evolution from birth to adult age. We performed an observational study at the Department of Life Sciences and Public Health, Rome. Fourteen patients were included in this study (six M; mean age: 18 years; SD: 10.62 years; median age: 15 years; age range: 6-44 years); six were adults (43%). Data on oral motor abilities from birth were collected. Meal duration, presence of swallowing reflex, dysphagia symptoms, difficulty chewing, and drooling management were assessed. At birth, all patients needed enteral feeding. Introduction of solid food was postponed beyond the age of 18 months in 43% of patients. During childhood and adolescence, mealtime was characterized by increased duration (43%) accompanied by fatigue during chewing (43%), food spillage from the nasal cavities (21%), sialorrhea (86%), and poor/reduced appetite (57%). A mature rotatory chewing skill was never achieved. This report expands the phenotype description of CS/CISS1 and also improves the overall management and prevention of complications in this ultra-rare disease.
患有 Crisponi/冷诱导出汗综合征 1 型(CS/CISS1)的患者经常存在喂养困难。我们的研究目的是描述从出生到成年的患病率和演变情况。我们在罗马生命科学与公共卫生系进行了一项观察性研究。本研究纳入了 14 名患者(6 名男性;平均年龄:18 岁;标准差:10.62 岁;中位数年龄:15 岁;年龄范围:6-44 岁);其中 6 名成年患者(43%)。收集了从出生开始的口腔运动能力数据。评估了进餐时间、吞咽反射存在情况、吞咽困难症状、咀嚼困难以及流涎管理情况。出生时,所有患者均需要肠内喂养。43%的患者固体食物的引入推迟到 18 个月以后。在儿童和青少年时期,进餐时间延长(43%),咀嚼时感到疲劳(43%),食物从鼻腔溢出(21%),流涎(86%)以及食欲减退或不佳(57%)。从未达到成熟的旋转咀嚼技能。本报告扩展了 CS/CISS1 的表型描述,还改善了这种超罕见疾病的整体管理和并发症预防。