Guillot Mireille, Offringa Martin, Lacaze-Masmonteil Thierry, Thébaud Bernard
a Children's Hospital of Eastern Ontario, Ottawa, ON K1H 8L1, Canada.
b The Ottawa Hospital, General Campus, Ottawa, ON K1H 8L6, Canada.
Can J Physiol Pharmacol. 2019 Mar;97(3):232-234. doi: 10.1139/cjpp-2018-0342. Epub 2018 Oct 5.
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.
支气管肺发育不良（BPD）是极早产儿最常见的并发症。目前，尚无特效治疗方法。临床前研究支持细胞疗法作为治疗早产儿BPD的一种有前景的疗法。成功转化为安全有效的临床干预措施取决于多个因素，包括新生儿医疗保健提供者的观点。举办了一场有40位加拿大新生儿科医生参加的两小时研讨会，以完善针对有BPD风险婴儿的干细胞II期试验设计，重点是此类试验的目标人群和测量结果。达成的共识是，早期干细胞试验招募的婴儿应是发生严重BPD风险最高的婴儿。这种风险应根据已知的产前、围产期和产后风险因素来确定。II期试验的主要结果将集中在非临床结果（例如剂量探索研究或安全性研究）上。随着转化研究其他方面的讨论，本次研讨会有助于加快加拿大首个针对早产儿BPD的临床细胞疗法研究的设计。
