Duker Angela L, Niiler Tim, Eldridge Grant, Brereton Nga H, Braverman Nancy E, Bober Michael B
Division of Medical Genetics, Nemours/Alfred I. duPont Hospital for Children, Wilmington, Delaware.
Gait Laboratory, Nemours/Alfred I. duPont Hospital for Children, Wilmington, Delaware.
Am J Med Genet A. 2017 Jan;173(1):108-113. doi: 10.1002/ajmg.a.37961. Epub 2016 Sep 12.
Rhizomelic chondrodysplasia punctata (RCDP) is a class of peroxisomal disorders characterized by defective plasmalogen biosynthesis. There are multiple recognized types of RCDP, all of which have autosomal recessive inheritance, and their associated genes are known: RCDP type 1 with PEX7, RCDP type 2 with GNPAT, RCDP type 3 with AGPS, RCDP type 4 with FAR1, and RCDP type 5 with PEX5. Among other medical/developmental issues, plasmalogen deficiency has a direct effect on bone growth and results in postnatal growth failure, the severity of which corresponds to the degree of plasmalogen deficiency. In order to document growth in patients with RCDP, we present detailed growth curves for length, weight, and head circumference derived from retrospective data from 23 individuals with RCDP types 1 and 2 confirmed by molecular and/or biochemical studies. We stratified growth curves by age as well as by plasmalogen level, with those with higher plasmalogens grouped as "non-classic." The growth charts presented here provide guidance to families and physician caretakers on the natural course of growth in individuals with RCDP during infancy into early childhood, and thus will have particular utility in setting expectations and guiding optimal feeding interventions in this population.© 2016 Wiley Periodicals, Inc.
肢根型点状软骨发育不良(RCDP)是一类过氧化物酶体疾病,其特征为缩醛磷脂生物合成存在缺陷。RCDP有多种已被认可的类型,均为常染色体隐性遗传,且其相关基因已为人所知:1型RCDP与PEX7相关,2型RCDP与GNPAT相关,3型RCDP与AGPS相关,4型RCDP与FAR1相关,5型RCDP与PEX5相关。在其他医学/发育问题中,缩醛磷脂缺乏对骨骼生长有直接影响,并导致出生后生长发育迟缓,其严重程度与缩醛磷脂缺乏程度相对应。为了记录RCDP患者的生长情况,我们展示了23例经分子和/或生化研究确诊为1型和2型RCDP患者的回顾性数据得出的详细身长、体重和头围生长曲线。我们按年龄以及缩醛磷脂水平对生长曲线进行了分层,缩醛磷脂水平较高的患者被归为“非典型”组。此处展示的生长图表为家庭和医生护理人员提供了关于RCDP患者从婴儿期到幼儿期生长自然进程的指导,因此在设定预期和指导该人群的最佳喂养干预方面将具有特殊用途。© 2016威利期刊公司