Ou-Yang Mei-Chen, Yang San-Nan, Hsu Yung-Ming, Ou-Yang Mei-Hui, Haung Hsin-Chun, Lee Shin-Yi, Hsieh Wu-Shiun, Su Yi-Ning, Liu Chieh-An
Division of Neonatology, Department of Pediatrics, Chang Gung Memorial Hospital-Kaohsiung Medical Center, Chang Gung University College of Medicine, Hsien, Taiwan.
J Pediatr Surg. 2007 Feb;42(2):e9-11. doi: 10.1016/j.jpedsurg.2006.10.022.
Hirschsprung's disease (HSCR) is characterized by the absence of intramural ganglion cells in the distal gut, resulting in bowel obstruction shortly after birth. Congenital central hypoventilation syndrome (CCHS) results in hypoventilation, most pronounced during sleep, with relative insensitivity to hypercarbia and reduced insensitivity to hypoxia. Congenital central hypoventilation syndrome with HSCR is a rare condition with variable severity. Both CCHS and HSCR are uncommon and their co-occurrence may suggest a common etiology, probably involving a fault of neural crest development. Recent reports have identified the paired-like homeobox 2B (PHOX2B) gene as the major gene for CCHS and HSCR. We report here an identified PHOX2B gene in a newborn baby who had concurrence of CCHS and total colonic aganglionosis with proximal small bowel involvement. Management of this rare disorder is challenging not only because it presents in newborn stage but also because it has extensive HSCR. Considering the issue of medical futility, the therapeutic and ethical dilemma of this infant was discussed.
先天性巨结肠症(HSCR)的特征是远端肠道壁内神经节细胞缺失,导致出生后不久出现肠梗阻。先天性中枢性低通气综合征(CCHS)导致通气不足,在睡眠期间最为明显,对高碳酸血症相对不敏感,对低氧血症的敏感性降低。伴有HSCR的先天性中枢性低通气综合征是一种罕见疾病,严重程度不一。CCHS和HSCR都不常见,它们的同时出现可能提示存在共同病因,可能涉及神经嵴发育缺陷。最近的报告已确定配对样同源盒2B(PHOX2B)基因是CCHS和HSCR的主要基因。我们在此报告在一名患有CCHS且全结肠无神经节症累及近端小肠的新生儿中发现的PHOX2B基因。这种罕见疾病的治疗具有挑战性,不仅因为它在新生儿期出现,还因为它存在广泛的HSCR。考虑到医疗无效性问题,讨论了该婴儿的治疗和伦理困境。