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Cell-based therapy for bronchopulmonary dysplasia in preterm infants

机译:早产儿的细胞基治疗肺飞机发育不良

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摘要

Bronchopulmonary dysplasia (BPD) is the most common complication of extreme prematurity. Currently, there is no specific treatment available. Preclinical studies support cell therapy as a promising therapy for BPD in preterm infants. A successful translation to a safe and effective clinical intervention depends on multiple factors including the perspective of neonatal health care providers. A 2-hour workshop with 40 Canadian neonatologists was held to enhance the design of a phase II trial of stem cells for babies at risk for BPD, with a focus on the population to target and the outcomes to measure in such a trial. The consensus was that infants recruited in an early trial of stem cells should be the ones with the highest risk of developing severe BPD. This risk should be established based on known antenatal, perinatal, and postnatal risk factors. The primary outcome in a phase II trial will be focussed on a non-clinical outcome (e.g., a dose-finding study or a safety study). With other aspects of a translational study discussed, this workshop contributed to accelerate the design of a first Canadian clinical cell-therapy study for BPD in preterm infants.
机译:支气管扩张发育不良(BPD)是极端早产的最常见并发症。目前,没有可用的特定治疗方法。临床前研究支持细胞疗法作为早产儿对BPD的有希望的疗法。成功翻译到安全有效的临床干预措施取决于包括新生儿保健提供者的视角的多个因素。举行了一名2小时的加拿大新生儿学家的2小时车间,以增强对BPD风险的婴儿干细胞的II期试验的设计,重点是目标的人口和在此类试验中衡量的结果。共识是,在干细胞早期试验中募集的婴儿应该是发育严重BPD的最高风险的婴儿。应基于已知的产前,围产期和产后风险因素来建立这种风险。 II期试验中的主要结果将侧重于非临床结果(例如剂量寻找研究或安全研究)。讨论了翻译研究的其他方面,这次研讨会有助于加速对早产儿的BPD加拿大临床细胞治疗研究的设计。

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