Frydman M, Vardimon D, Shalev E, Orlin J B
Department of Pediatrics, Hasharon Hospital, Petah Tiqva, Israel.
Prenat Diagn. 1996 Mar;16(3):266-9. doi: 10.1002/(SICI)1097-0223(199603)16:3<266::AID-PD845>3.0.CO;2-#.
Rambam-Hasharon syndrome (RHS) is a newly recognized autosomal recessive inborn error in fucose metabolism. Mental retardation, short stature, coarse facies, and recurrent infections are the main clinical findings. Several fucosilated proteoglycans are deficient in these patients. Leukocyte adhesion deficiency type 2 is associated with lack of the membrane glycoprotein sialyl-Lewisx (CD15s). In the red blood cells (RBCs), lack of the membrane glycoprotein H is manifested as a Bombay (Oh) blood type. Two consecutive pregnancies it risk for RHS were monitored during mid-trimester by cordocentesis. One fetus expressed H substance and her blood phenotype was O Rh+. The second fetus, a female, was 2 weeks smaller than expected by dates and had the Bombay blood type. The placenta of the affected fetus was small and irregular. This is the first prenatal diagnosis of this syndrome and the first case found in a female. The documentation of the syndrome in patients of both sexes and the parental consanguinity support an autosomal recessive inheritance. Two apparent recombinations between fucosyl-transferase 1 (FUT1, the H gene) and fucosyl-transferase 2 (secretor) are suggestive of non-allelic heterogeneity. We believe that the Bombay phenotype in this family is caused by a mutated gene, other than FUT1, which is causing multiple deficiencies of fucosilated proteoglycans.
兰巴姆-哈沙龙综合征(RHS)是一种新发现的常染色体隐性遗传性岩藻糖代谢先天性缺陷疾病。智力发育迟缓、身材矮小、面容粗糙和反复感染是主要临床症状。这些患者体内几种岩藻糖化蛋白聚糖缺乏。2型白细胞黏附缺陷与膜糖蛋白唾液酸化路易斯x(CD15s)缺乏有关。在红细胞(RBC)中,膜糖蛋白H缺乏表现为孟买(Oh)血型。在孕中期通过脐血穿刺监测了两例有RHS风险的连续妊娠。一个胎儿表达H物质,其血型为O Rh+。第二个胎儿为女性,比预期孕周小2周,血型为孟买型。患病胎儿的胎盘小且形态不规则。这是该综合征的首例产前诊断,也是首例在女性中发现的病例。该综合征在男女患者中的记录以及父母的近亲关系支持常染色体隐性遗传。岩藻糖基转移酶1(FUT1,即H基因)和岩藻糖基转移酶2(分泌型)之间的两次明显重组提示非等位基因异质性。我们认为,这个家族中的孟买血型是由FUT1以外的一个突变基因引起的,该基因导致多种岩藻糖化蛋白聚糖缺乏。