Acute severe paediatric asthma: Study protocol for the development of a core outcome set, a Pediatric Emergency Research Networks (PERN) study

Craig, Simon; Babl, Franz E.; Dalziel, Stuart R.; Gray, Charmaine; Powell, Colin; Al Ansari, Khalid; Lyttle, Mark D.; Roland, Damian; Benito, Javier; Velasco, Roberto; Hoeffe, Julia; Moldovan, Diana; Thompson, Graham; Schuh, Suzanne; Zorc, Joseph J.; Kwok, Maria; Mahajan, Prashant; Johnson, Michael D.; Sapien, Robert; Khanna, Kajal; Rino, Pedro; Prego, Javier; Yock, Adriana; Fernandes, Ricardo M.; Santhanam, Indumathy; Cheema, Baljit; Ong, Gene; Chong, Shu-Ling; Graudins, Andis

Abstract

Background
Acute severe childhood asthma is an infrequent, but potentially life-threatening emergency condition. There is a wide range of different approaches to this condition, with very little supporting evidence, leading to significant variation in practice. To improve knowledge in this area, there must first be consensus on how to conduct clinical trials, so that valid comparisons can be made between future studies. We have formed an international working group comprising paediatricians and emergency physicians from North America, Europe, Asia, the Middle East, Africa, South America, Central America, Australasia and the United Kingdom.

Methods/design
A 5-stage approach will be used: (1) a comprehensive list of outcomes relevant to stakeholders will be compiled through systematic reviews and qualitative interviews with patients, families, and clinicians; (2) Delphi methodology will be applied to reduce the comprehensive list to a core outcome set; (3) we will review current clinical practice guidelines, existing clinical trials, and literature on bedside assessment of asthma severity. We will then identify practice differences in tne clinical assessment of asthma severity, and determine whether further prospective work is needed to achieve agreement on inclusion criteria for clinical trials in acute paediatric asthma in the emergency department (ED) setting; (4) a retrospective chart review in Australia and New Zealand will identify the incidence of serious clinical complications such as intubation, ICU admission, and death in children hospitalized with acute severe asthma. Understanding the incidence of such outcomes will allow us to understand how common (and therefore how feasible) particular outcomes are in asthma in the ED setting; and finally (5) a meeting of the Pediatric Emergency Research Networks (PERN) asthma working group will be held, with invitation of other clinicians interested in acute asthma research, and patients/families. The group will be asked to achieve consensus on a core set of outcomes and to make recommendations for the conduct of clinical trials in acute severe asthma. If this is not possible, the group will agree on a series of prioritized steps to achieve this aim.

Discussion
The development of an international consensus on core outcomes is an important first step towards the development of consensus guidelines and standardised protocols for randomized controlled trials (RCTs) in this population. This will enable us to better interpret and compare future studies, reduce risks of study heterogeneity and outcome reporting bias, and improve the evidence base for the management of this important condition.

• Journal Article Type: Article
• Acceptance Date: Oct 9, 2019
• Online Publication Date: Jan 13, 2020
• Publication Date: Jan 13, 2020
• Deposit Date: Jul 13, 2020
• Publicly Available Date: Jul 13, 2020
• Journal: Trials
• Print ISSN: 1745-6215
• Electronic ISSN: 1745-6215
• Publisher: BioMed Central
• Peer Reviewed: Peer Reviewed
• Volume: 21
• Article Number: 72 (2020)
• DOI: https://doi.org/10.1186/s13063-019-3785-6
• Keywords: Medicine (miscellaneous); Pharmacology (medical)
• Public URL: https://uwe-repository.worktribe.com/output/6137042
• Additional Information: Received: 11 August 2019; Accepted: 9 October 2019; First Online: 13 January 2020; : The patient interview study has been approved by the Monash Health Human Research Ethics Committee (RES-18-0000-530A), whilst the clinician interview study has been approved by the Women’s and Children’s Health Network Human Research Ethics Committee (HREC/18/WCHN/120). Informed consent will be obtained from all study subjects.The retrospective chart review project has been approved by the Monash Health Human Research Ethics Committee (RES-17-0000-238 L), with a waiver of consent to collect de-identified data from existing medical records. The clinical guidelines study has been approved as a quality assurance activity by the Monash Health Human Research Ethics Committee (RES-18-0000-525Q); no individual patient consent is required for this study, as we are collecting institutional clinical guidelines only.; : Not applicable.; : The authors declare that they have no competing interests.