Department of Pediatrics, Pulmonary Division, University of Michigan Medical School, 1500 E. Medical Center Drive, Ann Arbor, MI, USA.
Department of Medical and Molecular Genetics, Indiana University School of Medicine, Indianapolis, IN, USA.
Orphanet J Rare Dis. 2022 Sep 9;17(1):351. doi: 10.1186/s13023-022-02515-2.
Heterotaxy (HTX) is a rare condition of abnormal thoraco-abdominal organ arrangement across the left-right axis of the body. The pathogenesis of HTX includes a derangement of the complex signaling at the left-right organizer early in embryogenesis involving motile and non-motile cilia. It can be inherited as a single-gene disorder, a phenotypic feature of a known genetic syndrome or without any clear genetic etiology. Most patients with HTX have complex cardiovascular malformations requiring surgical intervention. Surgical risks are relatively high due to several serious comorbidities often seen in patients with HTX. Asplenia or functional hyposplenism significantly increase the risk for sepsis and therefore require antimicrobial prophylaxis and immediate medical attention with fever. Intestinal rotation abnormalities are common among patients with HTX, although volvulus is rare and surgical correction carries substantial risk. While routine screening for intestinal malrotation is not recommended, providers and families should promptly address symptoms concerning for volvulus and biliary atresia, another serious morbidity more common among patients with HTX. Many patients with HTX have chronic lung disease and should be screened for primary ciliary dyskinesia, a condition of respiratory cilia impairment leading to bronchiectasis. Mental health and neurodevelopmental conditions need to be carefully considered among this population of patients living with a substantial medical burden. Optimal care of children with HTX requires a cohesive team of primary care providers and experienced subspecialists collaborating to provide compassionate, standardized and evidence-based care. In this statement, subspecialty experts experienced in HTX care and research collaborated to provide expert- and evidence-based suggestions addressing the numerous medical issues affecting children living with HTX.
内脏转位(HTX)是一种罕见的体轴左右胸腔-腹腔器官排列异常的疾病。HTX 的发病机制包括胚胎早期左右组织者复杂信号的紊乱,涉及能动和非能动纤毛。它可以作为一种单基因疾病遗传,也可以作为已知遗传综合征的一种表型特征,或者没有明确的遗传病因。大多数 HTX 患者有复杂的心血管畸形需要手术干预。由于 HTX 患者常伴有多种严重的合并症,因此手术风险相对较高。无脾或功能性脾功能减退症显著增加了败血症的风险,因此需要进行抗菌预防,并在发热时立即进行医疗干预。HTX 患者中肠道旋转异常很常见,尽管扭转很少见,且手术矫正有很大风险。虽然不建议常规筛查肠旋转不良,但提供者和家属应及时处理疑似扭转和胆道闭锁的症状,这是 HTX 患者另一种更为常见的严重并发症。许多 HTX 患者有慢性肺部疾病,应筛查原发性纤毛运动障碍,这是一种呼吸道纤毛功能障碍导致的支气管扩张症。在这个有大量医疗负担的患者群体中,需要仔细考虑心理健康和神经发育状况。HTX 患儿的最佳治疗需要由初级保健提供者和经验丰富的专科医生组成的协作团队,共同提供富有同情心、标准化和基于证据的治疗。在本声明中,有 HTX 治疗和研究经验的专科专家合作提供了专家和基于证据的建议,以解决影响 HTX 患儿的众多医疗问题。