Casertano Alberto, Fontana Paolo, Hennekam Raoul C, Tartaglia Marco, Genesio Rita, Dieber Tina Barbaro, Ortega Lucia, Nitsch Lucio, Melis Daniela
Department of Translational Medical Sciences, Section of Pediatrics, Federico II University, Naples, Italy.
Department of Molecular Medicine and Medical Biotechnology, Federico II University, Naples, Italy.
Am J Med Genet A. 2017 Jul;173(7):1896-1902. doi: 10.1002/ajmg.a.38124. Epub 2017 Apr 30.
Primrose syndrome is characterized by unusual facial features, macrocephaly, intellectual disability, enlarged, and calcified external ears, sparse body hair, and distal muscle wasting. Nine patients have been described in the literature. The disorder is due to missense mutations in ZBTB20. Here we describe one newly diagnosed 18-month-old patient and provide 10 year follow-up of an earlier reported patient, highlighting the progression and complexity of the disorder. Metabolic studies showed reduced glucose tolerance with prevalence of amino acids and fatty acids catabolism, ketogenesis, and gluconeogenesis, resulting in a Krebs cycle reversion.
报春花综合征的特征为特殊面容、巨头畸形、智力残疾、外耳增大及钙化、体毛细少和远端肌肉萎缩。文献中已报道9例患者。该疾病由ZBTB20基因的错义突变所致。在此,我们描述1例新诊断的18个月大患者,并对1例先前报道患者进行了10年随访,突出了该疾病的进展和复杂性。代谢研究显示葡萄糖耐量降低,伴有氨基酸和脂肪酸分解代谢、生酮作用和糖异生作用,导致三羧酸循环逆转。