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大多数产前诊断为成骨不全且预后不良的婴儿存活:一家四级医疗成骨不全中心的经验。

Most infants with prenatal osteogenesis imperfecta diagnosis and poor prognosis survive: experience of a quaternary care osteogenesis imperfecta center.

作者信息

Carroll Ricki S, Little Sarah, McGreal Tina, Bonner Shannon, Willis Daria, Franzone Jeanne M, Campbell Jeffery, Chou Margaret, Raymond Megan B, Schelhaas Andrea, Costa Joanna, Jain Mahim

机构信息

Division of Orthogenetics, Department of Pediatrics, Nemours Children's Hospital, Wilmington, DE 19803, United States.

Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia, PA 19107, United States.

出版信息

JBMR Plus. 2025 Feb 2;9(4):ziaf022. doi: 10.1093/jbmrpl/ziaf022. eCollection 2025 Apr.

Abstract

Osteogenesis imperfecta is a genetic condition with improperly or inadequately produced Type I collagen. Manifestations include bowing deformities, fractures, hydrocephalus, respiratory insufficiency, and feeding difficulty. Moderate or severe OI is often diagnosed prenatally based on ultrasound findings and genetic testing may be labeled as lethal. Here, we present 18 infants with moderate to severely presenting OI who received neonatal care at a single center over a 5 yr period, 10 of which were delivered at our institution. All 18 infants survived to neonatal discharge, with 7 infants requiring respiratory support and 9 infants requiring feeding support at discharge. Through Fisher Exact Test, Mann-Whitney U Test and backward elimination regression, we do not observe that lethal or possibly lethal diagnoses prenatally were correlated with medically relevant outcomes such as need for respiratory support at discharge or need for feeding support at discharge. Sixteen of the eighteen individuals are alive, with a minority requiring either respiratory or feeding support. With a multidisciplinary team approach to neonatal care, outcomes may be optimized. Infants formerly diagnosed with lethal OI may survive. Given our findings, and lack of correlation of prenatal assessments with survival and other medical outcomes, we recommend all families be given the option to pursue medical interventions.

摘要

成骨不全症是一种因I型胶原蛋白产生不当或不足而导致的遗传性疾病。其表现包括弓形畸形、骨折、脑积水、呼吸功能不全和喂养困难。中度或重度成骨不全症通常在产前根据超声检查结果进行诊断,基因检测可能会将其标记为致死性疾病。在此,我们报告了18例中度至重度成骨不全症患儿,他们在5年期间于单一中心接受新生儿护理,其中10例在我们机构分娩。所有18例婴儿均存活至新生儿出院,7例婴儿在出院时需要呼吸支持,9例婴儿在出院时需要喂养支持。通过Fisher精确检验、Mann-Whitney U检验和向后逐步回归分析,我们未观察到产前的致死性或可能致死性诊断与出院时对呼吸支持的需求或出院时对喂养支持的需求等医学相关结局存在相关性。18例中有16例存活,少数需要呼吸或喂养支持。采用多学科团队方法进行新生儿护理,结局可能会得到优化。以前被诊断为致死性成骨不全症的婴儿可能存活。鉴于我们的研究结果,以及产前评估与生存及其他医学结局缺乏相关性,我们建议给予所有家庭选择进行医学干预的机会。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c52b/11894799/f7b097d598d0/ziaf022f1.jpg

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