TY - JOUR
T1 - Research Priorities in Pediatric Asthma
T2 - Results of a Global Survey of Multiple Stakeholder Groups by the Pediatric Asthma in Real Life (PeARL) Think Tank
AU - Mathioudakis, Alexander G.
AU - Custovic, Adnan
AU - Deschildre, Antoine
AU - Ducharme, Francine M.
AU - Kalayci, Omer
AU - Murray, Clare
AU - Garcia, Antonio Nieto
AU - Phipatanakul, Wanda
AU - Price, David
AU - Sheikh, Aziz
AU - Agache, Ioana
AU - Bacharier, Leonard
AU - Bonini, Matteo
AU - Castro-Rodriguez, Jose A.
AU - De Carlo, Giuseppe
AU - Craig, Timothy
AU - Diamant, Zuzana
AU - Feleszko, Wojciech
AU - Ierodiakonou, Despo
AU - Gern, James E.
AU - Grigg, Jonathan
AU - Hedlin, Gunilla
AU - Hossny, Elham M.
AU - Jartti, Tuomas
AU - Kaplan, Alan
AU - Lemanske, Robert F.
AU - Le Souef, Peter
AU - Makela, Mika J.
AU - Matricardi, Paolo M.
AU - Miligkos, Michael
AU - Morais-Almeida, Mário
AU - Pite, Helena
AU - Pitrez, Paulo M.C.
AU - Pohunek, Petr
AU - Roberts, Graham
AU - Sanchez-Garcia, Sylvia
AU - Tsiligianni, Ioanna
AU - Turner, Steve
AU - Winders, Tonya A.
AU - Wong, Gary
AU - Xepapadaki, Paraskevi
AU - Zar, Heather J.
AU - Papadopoulos, Nikolaos G.
N1 - Funding Information:
This study was supported by the Respiratory Effectiveness Group (REG). REG has received support from AstraZeneca, Novartis, and Sanofi for continued work on PeARL. A. G. Mathioudakis was supported by the National Institute of Health Research Manchester Biomedical Research Centre (NIHR Manchester BRC).Conflicts of interest: None of the authors had any conflicts of interest directly related to this work. A. G. Mathioudakis reports grants from Boehringer Ingelheim outside the submitted work. A. Custovic reports personal fees from Novartis, Regeneron/Sanofi, Thermo Fisher Scientific, Boehringer-Ingelheim, and Philips, outside the submitted work. A. Deschildre reports grants and personal fees from Stallergenes Greer; and personal fees from Novartis, ALK, TEVA, GSK, MEDA-MYLAN, CHIESI, Aimmune, DBV technologies, and Astra Zeneca, outside the submitted work. F. M. Ducharme reports grants from Thorasys Inc.; personal fees from Jean-Coutu Pharmaceuticals; and nonfinancial support from Novartis Canada and Trudell Medical, outside the submitted work. C. Murray reports personal fees from Novartis, GSK, Astra Zeneca, Thermo Fisher, and Boehringer-Ingelheim outside the submitted work. W. Phipatanakul reports grants from NIH; grants and personal fees from Genentech/Novartis, Sanofi/Regeneron; personal fees from GSK; and nonfinancial support from Thermo Fisher, Lincoln Diagnostics, ALK-Abello, and Monaghen, outside the submitted work. D. Price reports grants from AKL Research and Development Ltd, British Lung Foundation, Respiratory Effectiveness Group, and the UK National Health Service; grants and personal fees from Boehringer Ingelheim, Chiesi, Circassia, Mylan, Mundipharma, Napp, Novartis, Pfizer, Regeneron Pharmaceuticals, Sanofi Genzyme, Teva, Theravance, and Zentiva (Sanofi Generics); personal fees from Cipla, GlaxoSmithKline, Kyorin, and Merck; nonfinancial support from Efficacy and Mechanism Evaluation program, Health Technology Assessment, outside the submitted work; stock/stock options from AKL Research and Development Ltd that produces phytopharmaceuticals; and owns 74% of the social enterprise Optimum Patient Care Ltd (Australia and UK) and 74% of Observational and Pragmatic Research Institute Pte Ltd (Singapore), outside the submitted work. L. Bacharier reports personal fees from Aerocrine, GlaxoSmithKline, Genentech/Novartis, Merck, DBV Technologies, TEVA, Boehringer-Ingelheim, AstraZeneca, WebMD/Medscape, Sanofi/Regeneron, Vectura, and Circassia outside the submitted work. T. Craig reports grants and personal fees CSL Behring, Dyax, Takeda, BioCryst, Pharming; personal fees from Grifols; and grants and nonfinancial support from GSK, Regeneron, and Novartis/Genentech outside the submitted work. Z. Diamant reports personal fees from academic affiliations; acts as Executive and Scientific Medical Director at a phase I/II pharmacological unit (QPS-NL), which performs clinical studies for pharmaceutical companies; and reports personal fees from AstraZeneca, ALK, Aquilon, Boehringer-Ingelheim, CSL, HAL Allergy, MSD, and Sanofi-Genzyme outside the submitted work. J. E. Gern reports grants from National Institutes of Health (NIH)/National Institute of Allergy and Infectious Diseases (NIAID); personal fees from Regeneron, Ena Therapeutics, and MedImmune outside the submitted work; and personal fees and stock options from Meissa Vaccines Inc. outside the submitted work. J. Grigg reports personal fees from GSK, Vifor Pharmaceuticals, Novartis, BV Pharma, and AstraZeneca outside the submitted work. A. Kaplan reports personal fees from Astra Zeneca, Behring, Boehringer-Ingelheim, Covis, GSK, Novo Nordisk, Novartis, Griffols, Pfizer, Sanofi, TEVA, and Trudel, outside the submitted work. R. F. Lemanske reports grants from NIH; nonfinancial support from GlaxoSmithKline, Boehringer-Ingelheim, Merck, TEVA, American Academy of Allergy Asthma and Immunology; grants from Clinical and Translational Science Award (NIH), Childhood Origins of ASThma (COAST) grant, AsthmaNet; and personal fees from LSU, Elsevier, UpToDate, the University of Kentucky, Thermo Fisher, and Food Allergy Research and Education (FARE) Network, outside the submitted work. P. M. C. Pitrez reports grants from Astra Zeneca, Chiesi, and TEVA; and personal fees from Astra Zeneca, TEVA, Novartis, Mundipharma, S&D Pharma, and GlaxoSmithKline outside the submitted work. G. Roberts reports personal fees from ALK, Allergen Therapeutics, Meda Plus, Merck; and a patent for the use of sublingual immunotherapy to prevent the development of allergy in at-risk infants, outside the submitted work. I. Tsiligianni reports personal fees from Novartis, GSK, Boehringer-Ingelheim, and Astra Zeneca; and grants from GSK Hellas, outside the submitted work. P. Xepapadaki reports personal fees from Nutricia, Nestle, Friesland, Uriach, Novartis Pharma AG, and GlaxoSmithKline outside the submitted work. N. G. Papadopoulos reports personal fees from ALK, Novartis, Nutricia, HAL, Menarini/FAES Farma, Sanofi, Mylan/MEDA, Biomay, AstraZeneca, GSK, MSD, ASIT BIOTECH, and Boehringer-Ingelheim; and grants from Gerolymatos International SA and Capricare outside the submitted work. The rest of the authors declare that they have no relevant conflicts of interest.
Funding Information:
This study was supported by the Respiratory Effectiveness Group (REG). REG has received support from AstraZeneca , Novartis , and Sanofi for continued work on PeARL. A. G. Mathioudakis was supported by the National Institute of Health Research Manchester Biomedical Research Centre (NIHR Manchester BRC).
Publisher Copyright:
© 2020 American Academy of Allergy, Asthma & Immunology
PY - 2020/6
Y1 - 2020/6
N2 - Background: Pediatric asthma remains a public health challenge with enormous impact worldwide. Objective: 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. Methods: 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. Results: 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. Conclusions: 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.
AB - Background: Pediatric asthma remains a public health challenge with enormous impact worldwide. Objective: 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. Methods: 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. Results: 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. Conclusions: 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.
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U2 - 10.1016/j.jaip.2020.01.059
DO - 10.1016/j.jaip.2020.01.059
M3 - Article
C2 - 32146166
AN - SCOPUS:85081199992
SN - 2213-2198
VL - 8
SP - 1953-1960.e9
JO - Journal of Allergy and Clinical Immunology: In Practice
JF - Journal of Allergy and Clinical Immunology: In Practice
IS - 6
ER -