Mathioudakis Alexander G, Custovic Adnan, Deschildre Antoine, Ducharme Francine M, Kalayci Omer, Murray Clare, Garcia Antonio Nieto, Phipatanakul Wanda, Price David, Sheikh Aziz, Agache Ioana, Bacharier Leonard, Bonini Matteo, Castro-Rodriguez Jose A, De Carlo Giuseppe, Craig Timothy, Diamant Zuzana, Feleszko Wojciech, Ierodiakonou Despo, Gern James E, Grigg Jonathan, Hedlin Gunilla, Hossny Elham M, Jartti Tuomas, Kaplan Alan, Lemanske Robert F, Le Souef Peter, Makela Mika J, Matricardi Paolo M, Miligkos Michael, Morais-Almeida Mário, Pite Helena, Pitrez Paulo M C, Pohunek Petr, Roberts Graham, Sanchez-Garcia Sylvia, Tsiligianni Ioanna, Turner Steve, Winders Tonya A, Wong Gary, Xepapadaki Paraskevi, Zar Heather J, Papadopoulos Nikolaos G
Division of Infection, Immunity and Respiratory Medicine, School of Biological Sciences, The University of Manchester, Manchester, United Kingdom; North West Lung Centre, Wythenshawe Hospital, Manchester University NHS Foundation Trust, Manchester Academic Health Science Centre, Manchester, United Kingdom.
Department of Paediatrics, Imperial College London, London, United Kingdom.
J Allergy Clin Immunol Pract. 2020 Jun;8(6):1953-1960.e9. doi: 10.1016/j.jaip.2020.01.059. Epub 2020 Mar 4.
Pediatric asthma remains a public health challenge with enormous impact worldwide.
The aim of this study was to identify and prioritize unmet clinical needs in pediatric asthma, which could be used to guide future research and policy activities.
We first identified unmet needs through an open-question survey administered to international experts in pediatric asthma who were members of the Pediatric Asthma in Real Life Think Tank. Prioritization of topics was then achieved through a second, extensive survey with global reach, of multiple stakeholders (leading experts, researchers, clinicians, patients, policy makers, and the pharmaceutical industry). Differences across responder groups were compared.
A total of 57 unmet clinical need topics identified by international experts were prioritized by 412 participants from 5 continents and 60 countries. Prevention of disease progression and prediction of future risk, including persistence into adulthood, emerged as the most urgent research questions. Stratified care, based on biomarkers, clinical phenotypes, the children's age, and demographics were also highly rated. The identification of minimum diagnostic criteria in different age groups, cultural perceptions of asthma, and best treatment by age group were priorities for responders from low-middle-income countries. There was good agreement across different stakeholder groups in all domains with some notable exceptions that highlight the importance of involving the whole range of stakeholders in formulation of recommendations.
Different stakeholders agree in the majority of research and strategic (eg, prevention, personalized approach) priorities for pediatric asthma. Stakeholder diversity is crucial for highlighting divergent issues that future guidelines should consider.
儿童哮喘仍是一项全球性的重大公共卫生挑战。
本研究旨在识别儿童哮喘中未满足的临床需求并确定其优先级，以指导未来的研究和政策活动。
我们首先通过对现实生活中儿童哮喘智库成员中的国际儿童哮喘专家进行开放式问卷调查来识别未满足的需求。然后通过对全球多个利益相关者（顶尖专家、研究人员、临床医生、患者、政策制定者和制药行业）进行的第二轮广泛调查来确定主题的优先级。比较了不同应答组之间的差异。
来自5个大洲60个国家的412名参与者对国际专家确定的57个未满足临床需求主题进行了优先级排序。预防疾病进展和预测未来风险，包括持续到成年期，成为最紧迫的研究问题。基于生物标志物、临床表型、儿童年龄和人口统计学的分层护理也得到了高度评价。确定不同年龄组的最低诊断标准、哮喘的文化认知以及按年龄组进行的最佳治疗是低收入和中等收入国家应答者的优先事项。在所有领域，不同利益相关者群体之间都有很好的一致性，但也有一些明显的例外，这突出了让所有利益相关者参与制定建议的重要性。
不同利益相关者在儿童哮喘的大多数研究和战略（如预防、个性化方法）优先事项上达成一致。利益相关者的多样性对于突出未来指南应考虑的不同问题至关重要。
