Advancing Therapeutic Development for Pulmonary Morbidities Associated with Preterm Birth.
Advancing Therapeutic Development for Pulmonary Morbidities Associated with Preterm Birth.
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DOI：
10.1007/s43441-020-00153-z
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发表时间：
2020-11
影响因子：
1.5
通讯作者：
McCune, Susan
中科院分区：
文献类型：
--
作者：
Sheehan, Sarah;Baer, Gerri;Romine, Morgan;Hudson, Lynn;Lim, Robert;Papadopoulos, Elektra;Campbell, Michelle;Daniel, Gregory;McCune, Susan
关键词：
与早产相关的慢性肺部和呼吸系统疾病特征不完全，使长期治疗和开发更有效的治疗方法复杂化。利益相关者在开发有效的、有临床意义的终点方面面临挑战，这些终点可以充分衡量早产儿的发病率，并预测或代表早产儿的健康结局。我们在本文中提出了一个研究议程，根据我们在2018年召开的关于该主题的研讨会上专家的意见，以推进终点和治疗的发展。我们讨论了进一步评估现有终点和改进疾病内型表征的必要性。我们还讨论了开发优化的短期和长期终点的关键步骤，这些终点可以与有意义的健康结果相关联。最后，我们讨论了在数据收集和新临床试验终点的应用中限制可变性的重要性，以及多方利益相关者合作对促进这一弱势患者群体的治疗开发的关键性质。
Chronic pulmonary and respiratory conditions associated with preterm birth are incompletely characterized, complicating long-term treatment and development of more effective therapies. Stakeholders face challenges in the development of validated, clinically meaningful endpoints that adequately measure morbidities and predict or represent health outcomes for preterm neonates. We propose in this paper a research agenda, informed by the input of experts from a 2018 workshop we convened on this topic, to advance endpoint and treatment development. We discuss the necessity of further evaluation of existing endpoints and the improved characterization of disease endotypes. We also discuss key steps to the development of optimized short- and long-term endpoints that can be linked to meaningful health outcomes. Finally, we discuss the importance of limiting variability in data collection and the application of new clinical trial endpoints as well as the critical nature of multi-stakeholder collaboration to advancing therapeutic development for this vulnerable patient population.
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DOI：
10.1164/rccm.201412-2142pp
发表时间：
2015-07-15
影响因子：
24.7
作者：
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影响因子：
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通讯作者：
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