Sukarova-Angelovska E, Kocova M, Sabolich V, Palcevska S, Angelkova N
Pediatric Clinic, Medical Faculty, Skopje, Republic of Macedonia.
Balkan J Med Genet. 2014 Dec 11;17(1):23-30. doi: 10.2478/bjmg-2014-0021. eCollection 2014 Jun.
Wolf-Hirschhorn syndrome (WHS) is a rare chromosomal disorder caused by terminal deletion of the short arm of chromosome 4. The clinical picture includes growth retardation, severe mental retardation, characteristic "Greek helmet" like face, seizures and midline defects in the brain, heart, palate and genitalia. Recently-used molecular techniques increase the number of diagnosed cases due to the detection of smaller deletions. The severity of the clinical presentation is variable depending on the haploinsufficiency of genes in a deleted region. We present six children with WHS with variable clinical appearance. The assessment of several elements (facial dysmorphism, mental retardation, additional congenital anomalies) provided classification into minor, mild or severe forms. Three of the children had a visible cytogenetic deletion on chromosome 4p, two had microdeletions detected with fluorescent in situ hybridization (FISH), and one child with a less characteristic clinical picture had a mosaic type of the deletion. Correlation between the clinical presentation and the length of the deleted region was confirmed.
沃尔夫-赫希霍恩综合征(WHS)是一种罕见的染色体疾病,由4号染色体短臂末端缺失引起。临床表现包括生长发育迟缓、严重智力障碍、具有特征性的“希腊头盔”样面容、癫痫发作以及脑、心脏、腭和生殖器的中线缺陷。由于能够检测到更小的缺失,最近使用的分子技术增加了确诊病例的数量。临床表现的严重程度因缺失区域内基因的单倍剂量不足而有所不同。我们介绍了6例临床表现各异的WHS患儿。通过对几个要素(面部畸形、智力障碍、其他先天性异常)的评估,将其分为轻度、中度或重度形式。其中3名患儿在4号染色体短臂上有可见的细胞遗传学缺失,2名患儿通过荧光原位杂交(FISH)检测到微缺失,1名临床表现不太典型的患儿具有嵌合型缺失。临床表现与缺失区域长度之间的相关性得到了证实。