• 文献检索
  • 文档翻译
  • 深度研究
  • 学术资讯
  • Suppr Zotero 插件Zotero 插件
  • 邀请有礼
  • 套餐&价格
  • 历史记录
应用&插件
Suppr Zotero 插件Zotero 插件浏览器插件Mac 客户端Windows 客户端微信小程序
定价
高级版会员购买积分包购买API积分包
服务
文献检索文档翻译深度研究API 文档MCP 服务
关于我们
关于 Suppr公司介绍联系我们用户协议隐私条款
关注我们

Suppr 超能文献

核心技术专利:CN118964589B侵权必究
粤ICP备2023148730 号-1Suppr @ 2026

文献检索

告别复杂PubMed语法,用中文像聊天一样搜索,搜遍4000万医学文献。AI智能推荐,让科研检索更轻松。

立即免费搜索

文件翻译

保留排版,准确专业,支持PDF/Word/PPT等文件格式,支持 12+语言互译。

免费翻译文档

深度研究

AI帮你快速写综述,25分钟生成高质量综述,智能提取关键信息,辅助科研写作。

立即免费体验

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.

DOI:10.1093/jbmrpl/ziaf022
PMID:40070560
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11894799/
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
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c52b/11894799/f7b097d598d0/ziaf022f1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c52b/11894799/f7b097d598d0/ziaf022f1.jpg

相似文献

1
Most infants with prenatal osteogenesis imperfecta diagnosis and poor prognosis survive: experience of a quaternary care osteogenesis imperfecta center.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。
JBMR Plus. 2025 Feb 2;9(4):ziaf022. doi: 10.1093/jbmrpl/ziaf022. eCollection 2025 Apr.
2
Current Overview of Osteogenesis Imperfecta.成骨不全症概述。
Medicina (Kaunas). 2021 May 10;57(5):464. doi: 10.3390/medicina57050464.
3
Osteogenesis Imperfecta: Multidisciplinary and Goal-Centered Care.成骨不全症:多学科及以目标为中心的护理
AJP Rep. 2022 Sep 29;12(3):e144-e147. doi: 10.1055/s-0042-1757481. eCollection 2022 Jul.
4
Osteogenesis Imperfecta: Multidisciplinary and Goal-Centered Care.成骨不全症:多学科与以目标为中心的护理
AJP Rep. 2022 Sep 30;12(3):e144-e147. doi: 10.1055/a-1911-3755. eCollection 2022 Jul.
5
Osteogenesis imperfecta: mode of delivery and neonatal outcome.成骨不全症:分娩方式与新生儿结局
Obstet Gynecol. 2001 Jan;97(1):66-9. doi: 10.1016/s0029-7844(00)01100-5.
6
A Sporadic Case of Osteogenesis Imperfecta: From Prenatal Diagnosis to Outcomes in Infancy-Case Report and Literature Review.一例散发型成骨不全症:从产前诊断到婴儿期结局——病例报告与文献复习。
Genes (Basel). 2023 Nov 10;14(11):2062. doi: 10.3390/genes14112062.
7
[Lethal osteogenesis imperfecta: antenatal diagnosis].[致死性成骨不全:产前诊断]
Pan Afr Med J. 2016 Oct 17;25:88. doi: 10.11604/pamj.2016.25.88.5871. eCollection 2016.
8
Comprehensive pain management strategy for infants with moderate to severe osteogenesis imperfecta in the perinatal period.围生期中度至重度成骨不全婴儿的综合疼痛管理策略
Paediatr Neonatal Pain. 2021 Dec 4;3(4):156-162. doi: 10.1002/pne2.12066. eCollection 2021 Dec.
9
Rare diseases: a challenge in paediatric dentistry.罕见病:儿科牙科的挑战。
Eur J Paediatr Dent. 2024 Sep 3;25(3):171-171. doi: 10.23804/ejpd.2024.25.03.01. Epub 2024 Sep 1.
10
Distinct type I collagen alterations cause intrinsic lung and respiratory defects of variable severity in mouse models of osteogenesis imperfecta.不同类型 I 型胶原的改变导致成骨不全症小鼠模型中肺和呼吸系统缺陷的严重程度不同。
J Physiol. 2023 Jan;601(2):355-379. doi: 10.1113/JP283452. Epub 2022 Nov 9.

本文引用的文献

1
Using MRI-derived observed-to-expected total fetal lung volume to predict lethality in fetal skeletal dysplasia.利用 MRI 衍生的观察到的与预期的胎儿总肺体积来预测胎儿骨骼发育不良的致死性。
Pediatr Radiol. 2024 Jan;54(1):43-48. doi: 10.1007/s00247-023-05825-0. Epub 2023 Dec 5.
2
MRI prediction of fetal lung volumes and the impact on counselling.MRI 预测胎儿肺容积及其对咨询的影响。
Clin Radiol. 2023 Dec;78(12):955-959. doi: 10.1016/j.crad.2023.09.006. Epub 2023 Sep 25.
3
Nosology of genetic skeletal disorders: 2023 revision.遗传骨骼疾病分类学:2023 修订版。
Am J Med Genet A. 2023 May;191(5):1164-1209. doi: 10.1002/ajmg.a.63132. Epub 2023 Feb 13.
4
Comprehensive pain management strategy for infants with moderate to severe osteogenesis imperfecta in the perinatal period.围生期中度至重度成骨不全婴儿的综合疼痛管理策略
Paediatr Neonatal Pain. 2021 Dec 4;3(4):156-162. doi: 10.1002/pne2.12066. eCollection 2021 Dec.
5
Revisiting Skeletal Dysplasias in the Newborn.重新审视新生儿骨骼发育不良。
Neoreviews. 2021 Apr;22(4):e216-e229. doi: 10.1542/neo.22-4-e216.
6
Health-related quality of life in adults with osteogenesis imperfecta.成骨不全症成人的健康相关生活质量。
Clin Genet. 2021 Jun;99(6):772-779. doi: 10.1111/cge.13939. Epub 2021 Feb 22.
7
Osteogenesis imperfecta.成骨不全症。
Nat Rev Dis Primers. 2017 Aug 18;3:17052. doi: 10.1038/nrdp.2017.52.
8
Evaluating skeletal dysplasias on prenatal ultrasound: an emphasis on predicting lethality.产前超声评估骨骼发育不良:重点在于预测致死性。
Pediatr Radiol. 2017 Feb;47(2):134-145. doi: 10.1007/s00247-016-3725-5. Epub 2016 Dec 1.
9
What If the Prenatal Diagnosis of a Lethal Anomaly Turns Out to Be Wrong?如果产前诊断出的致命异常结果是错误的呢?
Pediatrics. 2016 May;137(5). doi: 10.1542/peds.2015-4514. Epub 2016 Apr 1.
10
The psychosocial experience of individuals living with osteogenesis imperfecta: a mixed-methods systematic review.成骨不全症患者的心理社会经历:一项混合方法的系统评价
Qual Life Res. 2016 Aug;25(8):1877-96. doi: 10.1007/s11136-016-1247-0. Epub 2016 Feb 19.