Afifi H H, Zaki M S, El-Gerzawy A M S, Kayed H F
Clinical Genetics Department, National Research Centre, Cairo, Egypt.
Genet Couns. 2008;19(1):47-58.
Jacobsen syndrome is a rare disorder, caused by segmental monosomy for the distal end of the long arm of chromosome 11 with variable phenotypic expressivity. We report on the first male (6 years old) and female (3 years old) sibs with clinical and cytogenetics characterization of Jacobsen syndrome. Their karyotypes showed deletion 11q23.3-qter. Patients presented with growth and psychomotor retardation, facial dysmorphism, eye anomalies, and congenital heart disease (variable degrees of septal defect). Family history revealed a clinically similar brother, who died at 2 months old from cardiac anomalies in the form of single ventricle without being subjected to further investigations. Chromosomal analysis of the parents was normal. Karyotyping for the 2 patients and their parents was confirmed by fluorescence in situ hybridization analysis (FISH) using whole chromosome painting probes for 11 (WCP 11). Relevant investigations for both sibs showed mild thrombocytopenia with normal platelets morphology and striking periventricular demyelination on neuroimaging. Inguinal small testicles as well as focal epileptiform dysfunction were recorded in the male patient only. Abdominal ultrasound, hearing test, and DEXA scan were normal in both patients. Due to of the presence of apparently 3 affected offspring and normal parental karyotypes, an inherited predisposition was highly suspected. The large size of the distal deleted 11q segment in our patients support the recent hypothesis, that Jacobsen syndrome is a chromosomal deletion syndrome with genetic predisposition, due to expansion of p(CCG)n trinucleotide in the folate-sensitive fragile site FRA11B, at breakpoint 11q23.3. In conclusion, identification and further delineation of more similar patients will contribute to understanding the genetic basis of the 11q phenotype.
雅各布森综合征是一种罕见的疾病,由11号染色体长臂末端的节段性单体性引起,具有可变的表型表达。我们报告了首例具有雅各布森综合征临床和细胞遗传学特征的男性(6岁)和女性(3岁)同胞。他们的核型显示11q23.3-qter缺失。患者表现出生长发育和精神运动发育迟缓、面部畸形、眼部异常和先天性心脏病(不同程度的间隔缺损)。家族史显示有一个临床症状相似的兄弟,2个月大时死于单心室形式的心脏异常,未进行进一步检查。父母的染色体分析正常。使用11号全染色体涂染探针(WCP 11)进行荧光原位杂交分析(FISH),证实了这2例患者及其父母的核型分析结果。对这两名同胞的相关检查显示轻度血小板减少,血小板形态正常,神经影像学检查显示明显的脑室周围脱髓鞘。仅在男性患者中记录到腹股沟小睾丸以及局灶性癫痫样功能障碍。两名患者的腹部超声、听力测试和双能X线吸收法扫描均正常。由于明显有3名患病后代且父母核型正常,高度怀疑存在遗传易感性。我们患者中11q远端缺失片段的大尺寸支持了最近的假说,即雅各布森综合征是一种具有遗传易感性的染色体缺失综合征,这是由于叶酸敏感脆性位点FRA11B处的p(CCG)n三核苷酸在11q23.3断点处扩增所致。总之,识别和进一步描述更多相似患者将有助于理解11q表型的遗传基础。
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