更新于：2024-11-01

Respiratory Distress Syndrome in Premature Infants

早产儿呼吸窘迫综合征

更新于：2024-11-01

基本信息

别名

HYALINE MEMBRANE DISEASE, FORMERLY、RDS OF PREMATURITY、RDS Of Prematurity

+ [3]

简介-

关联

项与早产儿呼吸窘迫综合征相关的药物

Beractant

贝雷克坦

靶点-

作用机制-

在研机构

AbbVie, Inc. [+1]

原研机构

AbbVie, Inc.

在研适应症

早产儿呼吸窘迫综合征 [+1]

非在研适应症-

最高研发阶段批准上市

首次获批国家/地区

日本

首次获批日期1987-10-02

Elifactant

靶点-

作用机制

细胞膜通透性增强剂

在研机构

Chiesi USA, Inc

原研机构

CHIESI Farmaceutici SpA

在研适应症

新生儿呼吸窘迫综合征

非在研适应症

早产儿呼吸窘迫综合征

最高研发阶段临床2期

首次获批国家/地区-

首次获批日期1800-01-20

Colfosceril Palmitate

棕榈胆磷

靶点-

作用机制-

在研机构-

原研机构

Glaxo Group Ltd.

在研适应症-

非在研适应症

早产儿呼吸窘迫综合征

最高研发阶段撤市

首次获批国家/地区

美国

首次获批日期1990-08-06

项与早产儿呼吸窘迫综合征相关的临床试验

NCT04056741 / Suspended临床2期

A Supraglottic Instillation Device for Administration of Surfactant in Neonates- A Pilot Study

A pilot study to explore and determine feasibility and safety of the administration of surfactant through a new supraglottic device in patients from 500 to 5000 grams with RDS.

开始日期2025-02-07

申办/合作机构

ONY, Inc.

NCT06554522 / Not yet recruiting临床4期IIT

Pragmatic Evaluation of Therapies to Enhance Respiratory Management in Preterm Infants in Africa

The goal of this pragmatic clinical trial is to learn if the drug surfactant given by a less invasive technique works to treat respiratory distress in preterm infants in low- and middle-income African countries where invasive ventilators are unavailable. It will also learn about the safety of the less invasive surfactant administration (LISA) technique. The main questions it aims to answer are:
Does surfactant given by a less invasive surfactant administration technique improve survival in preterm infants in low- and middle-income countries? What medical problems do participants have when receiving surfactant given by the less invasive surfactant administration technique?
Researchers will implement the less invasive surfactant administration technique and see if it works to treat respiratory distress in preterm infants compared to preterm who did not receive surfactant.
Participants with respiratory distress who are being treated with continuous positive airway pressure and caffeine citrate will:
Receive surfactant replacement therapy by the less invasive surfactant administration technique.
Be monitored for complications Be followed throughout their hospitalization to determine their survival rate.

开始日期2024-10-01

申办/合作机构

Indiana University

[+1]

ACTRN12624000292572 / RecruitingN/AIIT

Investigating inter-rater reliability for the use of lung ultrasound by physiotherapists in premature infants with Respiratory Distress Syndrome

开始日期2023-07-01

申办/合作机构-

100 项与早产儿呼吸窘迫综合征相关的临床结果

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100 项与早产儿呼吸窘迫综合征相关的转化医学

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0 项与早产儿呼吸窘迫综合征相关的专利（医药）

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项与早产儿呼吸窘迫综合征相关的文献（医药）

2022-11-01·Indian Journal of Pediatrics

Natural Versus Synthetic Surfactant Therapy in Respiratory Distress Syndrome of Prematurity

Article

作者: Shah, Mili ; Patel, Chintan L ; Patil, Karamchand ; Nimbalkar, Somashekhar M ; Bansal, Satvik C ; Patel, Dipen V

OBJECTIVESTo compare the clinical efficacy and the cost of treatment between the newborns who received either a natural or a protein-free synthetic surfactant for respiratory distress syndrome (RDS) of prematurity.METHODSThis is a retrospective analytical study incorporating comparisons of clinical parameters and cost in newborns having RDS of prematurity who received either Survanta (bovine lung extract), a natural surfactant or Surfact (protein-free colfosceril palmitate), a synthetic surfactant.RESULTSThere were 100 newborns who received either of the natural (n = 52) or synthetic (n = 48) surfactant with mean (SD) gestational age and mean (SD) birth weight of 31.5 (2.6) wk, 1425 (461) g and 32.2 (2.2) wk, 1519 (413) g, respectively. Majority of the newborns (> 90%) received endotracheal surfactant within the first 24 h of life and had similar baseline characteristics in either group. No differences were noted in ventilator settings on admission and 24 h after surfactant/admission. Oxygen requirement, extubation age, complications, hospital stay, and mortality were similar across groups, except that the necrotizing enterocolitis was noted only in natural surfactant group. There was a significant pharmacy cost savings in synthetic surfactant group.CONCLUSIONSynthetic surfactant was comparable to natural surfactant with regard to outcomes, like ventilator settings, hospital stay, and mortality. Pharmacy cost was less in synthetic surfactant group.

2016-10-01·American Journal of Respiratory Cell and Molecular Biology1区 · 医学

Endothelial Monocyte-Activating Polypeptide II Mediates Macrophage Migration in the Development of Hyperoxia-Induced Lung Disease of Prematurity

1区 · 医学

Article

作者: Lal, Charitharth V. ; Awasthi, Niranjan ; Lowe, Chinn-Woan ; Schwarz, Anna M. ; Persad, Elizabeth A. ; Schwarz, Roderich E. ; Schwarz, Margaret A. ; Lee, Daniel D.

Myeloid cells are key factors in the progression of bronchopulmonary dysplasia (BPD) pathogenesis. Endothelial monocyte-activating polypeptide II (EMAP II) mediates myeloid cell trafficking. The origin and physiological mechanism by which EMAP II affects pathogenesis in BPD is unknown. The objective was to determine the functional consequences of elevated EMAP II levels in the pathogenesis of murine BPD and to investigate EMAP II neutralization as a therapeutic strategy. Three neonatal mouse models were used: (1) BPD (hyperoxia), (2) EMAP II delivery, and (3) BPD with neutralizing EMAP II antibody treatments. Chemokinic function of EMAP II and its neutralization were assessed by migration in vitro and in vivo. We determined the location of EMAP II by immunohistochemistry, pulmonary proinflammatory and chemotactic gene expression by quantitative polymerase chain reaction and immunoblotting, lung outcome by pulmonary function testing and histological analysis, and right ventricular hypertrophy by Fulton's Index. In BPD, EMAP II initially is a bronchial club-cell-specific protein-derived factor that later is expressed in galectin-3⁺ macrophages as BPD progresses. Continuous elevated expression corroborates with baboon and human BPD. Prolonged elevation of EMAP II levels recruits galectin-3⁺ macrophages, which is followed by an inflammatory state that resembles a severe BPD phenotype characterized by decreased pulmonary compliance, arrested alveolar development, and signs of pulmonary hypertension. In vivo pharmacological EMAP II inhibition suppressed proinflammatory genes Tnfa, Il6, and Il1b and chemotactic genes Ccl2 and Ccl9 and reversed the severe BPD phenotype. EMAP II is sufficient to induce macrophage recruitment, worsens BPD progression, and represents a targetable mechanism of BPD development.

2015-10-01·Biochemical Society Transactions2区 · 生物学

ABCA3, a key player in neonatal respiratory transition and genetic disorders of the surfactant system

2区 · 生物学

Review

作者: Boldrini, Renata ; Masotti, Andrea ; Cutrera, Renato ; Peca, Donatella ; Danhaive, Olivier

Genetic disorders of the surfactant system are rare diseases with a broad range of clinical manifestations, from fatal respiratory distress syndrome (RDS) in neonates to chronic interstitial lung disease (ILD) in children and adults. ABCA3 [ATP-binding cassette (ABC), subfamily A, member 3] is a lung-specific phospholipid transporter critical for intracellular surfactant synthesis and storage in lamellar bodies (LBs). Its expression is developmentally regulated, peaking prior to birth under the influence of steroids and transcription factors. Bi-allelic mutations of the ABCA3 gene represent the most frequent cause of congenital surfactant deficiency, indicating its critical role in lung function. Mutations affect surfactant lipid and protein processing and LBs’ morphology, leading to partial or total surfactant deficiency. Approximately 200 mutations have been reported, most of which are unique to individuals and families, which makes diagnosis and prognosis challenging. Various types of mutations, affecting different domains of the protein, account in part for phenotype diversity. Disease-causing mutations have been reported in most coding and some non-coding regions of the gene, but tend to cluster in the first extracellular loop and the second nucleotide-binding domain (NBD), leading to defective glycosylation and trafficking defects and interfering with ATP binding and hydrolysis respectively. Mono-allelic damaging and benign variants are often subclinical but may act as disease modifiers in lung diseases such as RDS of prematurity or associate with mutations in other surfactant-related genes. Diagnosis is complex but essential and should combine pathology and ultrastructure studies on lung biopsy with broad-spectrum genetic testing of surfactant-related genes, made possible by recent technology advances in the massive parallel sequencing technology.

项与早产儿呼吸窘迫综合征相关的新闻（医药）

2022-08-25

·医药观澜

超7000万美元！李氏大药厂引进儿童用药全球权利

▎药明康德内容团队报道近日消息，李氏大药厂宣布从Windtree Therapeutics取得Surfaxin（KL4表面活性剂）、Surfaxin LS（冻干KL4表面活性剂）、AEROSURF及任何其他含有合成KL4表面活性剂的医药组成物的独家全球开发、生产及商业化权利。协议中的3款产品主要用于改善早产婴儿呼吸窘迫综合征的管理。为此，Windtree公司将获得高达7890万美元的开发、监管和商业里程碑付款以及特许权使用费。呼吸窘迫综合征（RDS）为婴儿出生后不久出现呼吸窘迫并进行性加重的临床综合征，多见于早产儿，胎龄越小，患病率越高。研究发现，早产儿发生RDS的原因是其肺部无法产生足够的表面活性剂。表面活性剂是一种存在于肺组织内部的液体，在呼吸时可降低肺泡气-液界面的表面张力，在静息跨肺压时可稳定肺泡塌陷。如果肺部缺乏足够的表面活性剂，会导致呼吸困难。Surfaxin（lucinactant）及Surfaxin LS是Windtree公司（前身为Discovery Laboratories）开发的试验性合成肽，均含有为改善早产婴儿的呼吸窘迫症候群管理而开发的药物——KL4表面活性剂。这种产品可以通过补偿表面活性剂的不足，恢复早产儿肺部的表面活性而发挥药效。AEROSURF（吸入式视光活性剂）是以非入侵性方式释放雾化KL4表面活性剂而设计的药物/器械组合，有望有效减少使用具入侵性的气管插管及机械式呼吸器。此前，美国FDA已就治疗呼吸窘迫症候群授予AEROSURF快速通道资格，而Surfaxin则获得孤儿药资格。 2012年3月，Surfaxin获得FDA批准上市，用于RDS高风险早产儿的RDS预防。临床研究结果证实，与对照组相比，Surfaxin治疗组婴儿的24小时内RDS改善情况和14日内RDS相关死亡率都明显优于前者，包括候选药在内的外源性表面活性药物可快速影响肺的顺应性和氧合作用。研究中观察到，与Surfaxin相关的常见不良反应包括心动过缓、氧饱和度下降、药物回流至气管内导管（ETT）以及气道ETT阻塞。此前，Windtree公司曾向李氏大药厂及其子公司兆科药业授予KL4和AEROSURF在大中华区的区域许可。此次双方签订新协议，将合作区域扩展到全球。根据许可协议，李氏大药厂将获得Surfaxin 、冻干lucinactant和AEROSURF用于任何潜在适应症和应用的开发和商业化全球许可，并将负责资助所有开发、制造和商业化活动。参考文献：[1] 李氏大药厂從WINDTREE THERAPEUTICS, INC.取得SURFAXIN™、 SURFAXIN LS™及 AEROSURF™的全球許可. Retrieved Aug 23, 2022, from https://pdf.dfcfw.com/pdf/H2_AN202208231577518411_1.pdf?1661299367000.pdf[2] WINDTREE THERAPEUTICS ANNOUNCES KL4 SURFACTANT AND AEROSURF® GLOBAL LICENSE AGREEMENT . Retrieved Aug 25, 2022, from https://ir.windtreetx.com/news-releases/news-release-details/windtree-therapeutics-announces-kl4-surfactant-and-aerosurfr[3]夏训明（编译）."美国FDA批准Surfaxin(lucinactant)用于治疗早产儿呼吸窘迫综合征."广东药学院学报 .(2012):155-155.Print.本文由药明康德内容团队根据公开资料整理编辑，欢迎个人转发至朋友圈。转发授权请在「医药观澜」微信公众号留言联系我们。其他合作需求，请联系wuxi_media@wuxiapptec.com。免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。

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