Costa T, Azouz E M, Fitzpatrick J, Kamel-Reid S, Smith C R, Silver M M
Department of Pediatrics and Genetics, Hospital for Sick Children, Toronto, Canada.
Am J Med Genet. 1998 Mar 5;76(2):125-32.
We describe 3 new cases of a rare form of dwarfism (so-called "lethal skeletal dysplasia with gracile bones" or "osteocraniostenosis"), a condition characterized by thin, brittle bones and death in late gestation or early neonatal life. The first was a 37-week gestation female who died at delivery. She had facial anomalies and positional abnormalities of the hands and feet. The others were male stillborn sibs, who died in utero in the third trimester. Their mother had an undiagnosed dwarfing condition associated with body asymmetry, microcephaly, and unusual facial appearance. Both fetuses were documented by ultrasound to have short limbs and probable long bone fractures late in the second trimester. At autopsy, one fetus had no spleen and the other a hypoplastic spleen. Radiographically, all three cases had very thin diaphyses, diaphyseal fractures, and thin ribs and clavicles. In contrast to the first case who had a normally mineralized calvaria, the sibs had grossly deficient calvarial mineralization. Microscopically, endochondral ossification was qualitatively normal but quantitatively deficient in all three cases. The long bones, especially those of the sibs, lacked the well-defined outer cortex in the mid-shaft normally seen by the third trimester. This failure of organization into the cortex and medulla suggests a failure of bone remodelling. Given the variable presentation in these cases, "lethal skeletal dysplasia with gracile bones" is probably a heterogeneous disorder. The recurrence in one family suggests that the mother has somatic/germline mosaicism of a lethal gene, expressed clinically as growth failure and asymmetry.
我们描述了3例罕见类型侏儒症(所谓的“致死性骨纤细性骨骼发育不良”或“颅骨狭窄症”)的新病例,这种病症的特征是骨骼纤细、脆弱,在妊娠晚期或新生儿早期死亡。第一例是一名妊娠37周的女性,出生时死亡。她有面部异常以及手足位置异常。另外两例是男性死产同胞,在妊娠晚期死于子宫内。他们的母亲有一种未被诊断出的侏儒症,伴有身体不对称、小头畸形和异常面容。超声检查显示,两个胎儿均四肢短小,在妊娠中期晚期可能发生了长骨骨折。尸检发现,一个胎儿没有脾脏,另一个胎儿脾脏发育不全。影像学检查显示,所有3例均有非常纤细的骨干、骨干骨折以及纤细的肋骨和锁骨。与第一例颅骨矿化正常的病例不同,这对同胞的颅骨矿化严重不足。显微镜检查显示,所有3例的软骨内成骨在质量上正常,但在数量上不足。长骨,尤其是这对同胞的长骨,在妊娠晚期通常应有的中轴部位缺乏清晰的外层皮质。这种未能组织形成皮质和髓质的情况提示骨重塑失败。鉴于这些病例表现各异,“致死性骨纤细性骨骼发育不良”可能是一种异质性疾病。一个家族中的复发表明,母亲存在致死基因的体细胞/生殖细胞嵌合体,临床上表现为生长发育迟缓及不对称。