Ehrich E, Aranoff G, Johnson W G
Am J Med Genet. 1987 Mar;26(3):637-44. doi: 10.1002/ajmg.1320260319.
We report on two brothers with achalasia, adrenocortical insufficiency, alacrima, short stature, microcephaly, ataxia, optic atrophy, and developmental delay. The parents and three sibs are unaffected. Achalasia, adrenocortical insufficiency, and alacrima comprise a recently characterized familial multisystem disorder of unknown cause. Achalasia has also been described in association with microcephaly and mental retardation in one family and ataxia, optic atrophy, and mental retardation in another. The above reports and these sibs may represent variants of a single pleiotropic recessive gene. We suggest that abnormalities of the central nervous system are a manifestation of the achalasia, adrenocortical insufficiency, alacrima syndrome.
我们报告了两兄弟,他们患有贲门失弛缓症、肾上腺皮质功能不全、无泪症、身材矮小、小头畸形、共济失调、视神经萎缩和发育迟缓。其父母和三个兄弟姐妹未受影响。贲门失弛缓症、肾上腺皮质功能不全和无泪症构成了一种最近才被描述的病因不明的家族性多系统疾病。在一个家族中,贲门失弛缓症还与小头畸形和智力迟钝有关;在另一个家族中,则与共济失调、视神经萎缩和智力迟钝有关。上述报告以及这两兄弟的情况可能代表了单一多效性隐性基因的不同变体。我们认为中枢神经系统异常是贲门失弛缓症、肾上腺皮质功能不全、无泪症综合征的一种表现。