Dimitrov Boyan, Balikova Irina, Jekova Nely, Vakrilova Lilija, Fryns Jean-Pierre, Simeonov Emil
Center for Human Genetics, University Hospital Gasthuisberg, University of Leuven, Herestraat 49, 3000 Leuven, Belgium.
Am J Med Genet A. 2005 May 15;135(1):81-5. doi: 10.1002/ajmg.a.30673.
The acrofacial dysostoses (AFD) are a clinically and causally heterogeneous group of conditions characterized by mandibulofacial dysostosis and a variety of limb anomalies. Several abnormalities affecting different internal organs and the central nervous system (CNS) have been described. Depending on the type of limb defects, two major groups have been delineated: (1) with predominantly pre-axial anomalies, Nager type AFD, and (2) with predominantly post-axial involvement, Genee-Wiedemann form of AFD, also known as POADS, respectively. Other forms of "true AFD" have been described as Kelly, Reynolds, Arens (also Tel Aviv form), Rodríguez (or Madrid form), Richieri-Costa, and Patterson-Stevenson-Fontaine types. However, whether they are distinct entities or represent variants of the same condition remains unclear. Rodríguez AFD was described as a new lethal form of AFD in three affected sibs with severe mandibular hypoplasia, severe predominantly pre-axial limb deficiencies, absent fibulae and ribs, and internal organ anomalies, the most remarkable of which are arrhinencephaly and abnormal lung lobulation. We present a newborn girl with Rodríguez type of AFD, who died a few days after the birth due to respiratory failure. The phenotype and the cause of this condition are discussed.
肢端颜面发育不全(AFD)是一组临床和病因各异的病症,其特征为下颌面部发育不全以及多种肢体异常。已经描述了一些影响不同内脏器官和中枢神经系统(CNS)的异常情况。根据肢体缺陷的类型,可分为两大组:(1)主要为轴前异常的纳格尔型AFD,以及(2)主要为轴后受累的吉内-维德曼型AFD,也分别称为POADS。其他形式的“真性AFD”已被描述为凯利、雷诺兹、阿伦斯(也称为特拉维夫型)、罗德里格斯(或马德里型)、里希耶里-科斯塔型以及帕特森-史蒂文森-方丹型。然而,它们是不同的实体还是同一病症的变体仍不清楚。罗德里格斯型AFD被描述为一种新的致死性AFD形式,见于三名患病同胞,他们患有严重的下颌发育不全、严重的主要为轴前肢体缺陷、腓骨和肋骨缺失以及内脏器官异常,其中最显著的是无脑回畸形和肺叶异常。我们报告一名患有罗德里格斯型AFD的新生女婴,她出生几天后因呼吸衰竭死亡。本文讨论了这种病症的表型和病因。