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      Core outcomes in neonatology: development of a core outcome set for neonatal research

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          Abstract

          Background

          Neonatal research evaluates many different outcomes using multiple measures. This can prevent synthesis of trial results in meta-analyses, and selected outcomes may not be relevant to former patients, parents and health professionals.

          Objective

          To define a core outcome set (COS) for research involving infants receiving neonatal care in a high-income setting.

          Design

          Outcomes reported in neonatal trials and qualitative studies were systematically reviewed. Stakeholders were recruited for a three-round international Delphi survey. A consensus meeting was held to confirm the final COS, based on the survey results.

          Participants

          Four hundred and fourteen former patients, parents, healthcare professionals and researchers took part in the eDelphi survey; 173 completed all three rounds. Sixteen stakeholders participated in the consensus meeting.

          Results

          The literature reviews identified 104 outcomes; these were included in round 1. Participants proposed 10 additional outcomes; 114 outcomes were scored in rounds 2 and 3. Round 1 scores showed different stakeholder groups prioritised contrasting outcomes. Twelve outcomes were included in the final COS: survival, sepsis, necrotising enterocolitis, brain injury on imaging, general gross motor ability, general cognitive ability, quality of life, adverse events, visual impairment/blindness, hearing impairment/deafness, retinopathy of prematurity and chronic lung disease/bronchopulmonary dysplasia.

          Conclusions and relevance

          A COS for clinical trials and other research studies involving infants receiving neonatal care in a high-income setting has been identified. This COS for neonatology will help standardise outcome selection in clinical trials and ensure these are relevant to those most affected by neonatal care.

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          Most cited references46

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          Global, regional, and national disability-adjusted life-years (DALYs) for 359 diseases and injuries and healthy life expectancy (HALE) for 195 countries and territories, 1990–2017: a systematic analysis for the Global Burden of Disease Study 2017

          Summary Background How long one lives, how many years of life are spent in good and poor health, and how the population’s state of health and leading causes of disability change over time all have implications for policy, planning, and provision of services. We comparatively assessed the patterns and trends of healthy life expectancy (HALE), which quantifies the number of years of life expected to be lived in good health, and the complementary measure of disability-adjusted life-years (DALYs), a composite measure of disease burden capturing both premature mortality and prevalence and severity of ill health, for 359 diseases and injuries for 195 countries and territories over the past 28 years. Methods We used data for age-specific mortality rates, years of life lost (YLLs) due to premature mortality, and years lived with disability (YLDs) from the Global Burden of Diseases, Injuries, and Risk Factors Study (GBD) 2017 to calculate HALE and DALYs from 1990 to 2017. We calculated HALE using age-specific mortality rates and YLDs per capita for each location, age, sex, and year. We calculated DALYs for 359 causes as the sum of YLLs and YLDs. We assessed how observed HALE and DALYs differed by country and sex from expected trends based on Socio-demographic Index (SDI). We also analysed HALE by decomposing years of life gained into years spent in good health and in poor health, between 1990 and 2017, and extra years lived by females compared with males. Findings Globally, from 1990 to 2017, life expectancy at birth increased by 7·4 years (95% uncertainty interval 7·1–7·8), from 65·6 years (65·3–65·8) in 1990 to 73·0 years (72·7–73·3) in 2017. The increase in years of life varied from 5·1 years (5·0–5·3) in high SDI countries to 12·0 years (11·3–12·8) in low SDI countries. Of the additional years of life expected at birth, 26·3% (20·1–33·1) were expected to be spent in poor health in high SDI countries compared with 11·7% (8·8–15·1) in low-middle SDI countries. HALE at birth increased by 6·3 years (5·9–6·7), from 57·0 years (54·6–59·1) in 1990 to 63·3 years (60·5–65·7) in 2017. The increase varied from 3·8 years (3·4–4·1) in high SDI countries to 10·5 years (9·8–11·2) in low SDI countries. Even larger variations in HALE than these were observed between countries, ranging from 1·0 year (0·4–1·7) in Saint Vincent and the Grenadines (62·4 years [59·9–64·7] in 1990 to 63·5 years [60·9–65·8] in 2017) to 23·7 years (21·9–25·6) in Eritrea (30·7 years [28·9–32·2] in 1990 to 54·4 years [51·5–57·1] in 2017). In most countries, the increase in HALE was smaller than the increase in overall life expectancy, indicating more years lived in poor health. In 180 of 195 countries and territories, females were expected to live longer than males in 2017, with extra years lived varying from 1·4 years (0·6–2·3) in Algeria to 11·9 years (10·9–12·9) in Ukraine. Of the extra years gained, the proportion spent in poor health varied largely across countries, with less than 20% of additional years spent in poor health in Bosnia and Herzegovina, Burundi, and Slovakia, whereas in Bahrain all the extra years were spent in poor health. In 2017, the highest estimate of HALE at birth was in Singapore for both females (75·8 years [72·4–78·7]) and males (72·6 years [69·8–75·0]) and the lowest estimates were in Central African Republic (47·0 years [43·7–50·2] for females and 42·8 years [40·1–45·6] for males). Globally, in 2017, the five leading causes of DALYs were neonatal disorders, ischaemic heart disease, stroke, lower respiratory infections, and chronic obstructive pulmonary disease. Between 1990 and 2017, age-standardised DALY rates decreased by 41·3% (38·8–43·5) for communicable diseases and by 49·8% (47·9–51·6) for neonatal disorders. For non-communicable diseases, global DALYs increased by 40·1% (36·8–43·0), although age-standardised DALY rates decreased by 18·1% (16·0–20·2). Interpretation With increasing life expectancy in most countries, the question of whether the additional years of life gained are spent in good health or poor health has been increasingly relevant because of the potential policy implications, such as health-care provisions and extending retirement ages. In some locations, a large proportion of those additional years are spent in poor health. Large inequalities in HALE and disease burden exist across countries in different SDI quintiles and between sexes. The burden of disabling conditions has serious implications for health system planning and health-related expenditures. Despite the progress made in reducing the burden of communicable diseases and neonatal disorders in low SDI countries, the speed of this progress could be increased by scaling up proven interventions. The global trends among non-communicable diseases indicate that more effort is needed to maximise HALE, such as risk prevention and attention to upstream determinants of health. Funding Bill & Melinda Gates Foundation.
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            An Experimental Application of the DELPHI Method to the Use of Experts

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              The COMET Handbook: version 1.0

              The selection of appropriate outcomes is crucial when designing clinical trials in order to compare the effects of different interventions directly. For the findings to influence policy and practice, the outcomes need to be relevant and important to key stakeholders including patients and the public, health care professionals and others making decisions about health care. It is now widely acknowledged that insufficient attention has been paid to the choice of outcomes measured in clinical trials. Researchers are increasingly addressing this issue through the development and use of a core outcome set, an agreed standardised collection of outcomes which should be measured and reported, as a minimum, in all trials for a specific clinical area. Accumulating work in this area has identified the need for guidance on the development, implementation, evaluation and updating of core outcome sets. This Handbook, developed by the COMET Initiative, brings together current thinking and methodological research regarding those issues. We recommend a four-step process to develop a core outcome set. The aim is to update the contents of the Handbook as further research is identified. Electronic supplementary material The online version of this article (doi:10.1186/s13063-017-1978-4) contains supplementary material, which is available to authorized users.
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                Author and article information

                Journal
                Arch Dis Child Fetal Neonatal Ed
                Arch. Dis. Child. Fetal Neonatal Ed
                fetalneonatal
                fnn
                Archives of Disease in Childhood. Fetal and Neonatal Edition
                BMJ Publishing Group (BMA House, Tavistock Square, London, WC1H 9JR )
                1359-2998
                1468-2052
                July 2020
                15 November 2019
                : 105
                : 4
                : 425-431
                Affiliations
                [1 ] departmentAcademic Neonatal Medicine , Imperial College London , London, UK
                [2 ] departmentNuffield Department of Primary Care Health Sciences , University of Oxford , Oxford, Oxfordshire, UK
                [3 ] departmentNeonatal Unit , Rosie Hospital , Cambridge, Cambridgeshire, UK
                [4 ] departmentThe Neonatal Unit , Royal Glamorgan Hospital , Llantrisant, Rhondda Cynon Taf, UK
                [5 ] UCL Institute of Education Centre for Longitudinal Studies , London, UK
                [6 ] departmentDepartment of Women and Children's Health, School of Life Sciences, Faculty of Life Sciences and Medicine , King's College London , London, UK
                [7 ] departmentPaediatric Surgery , Southampton General Hospital , Southampton, UK
                [8 ] National Perinatal Epidemiology Unit , Oxford, UK
                [9 ] departmentSchool of Nursing and Midwifery, Faculty of Health, Education and Society , Plymouth University , Plymouth, Devon, UK
                [10 ] departmentSchool of Nursing and Midwifery, Faculty of Health Sciences , Curtin University , Perth, Western Australia, Australia
                [11 ] Bliss , London, UK
                [12 ] departmentInstitute for Women's Health , University College London , London, UK
                [13 ] Parent of Neonatal Patient , London, UK
                [14 ] departmentNeonatal Unit , Birmingham City Hospital , Birmingham, UK
                [15 ] Former neonatal patient , London, UK
                [16 ] departmentNeonatal Medicine , Imperial College London , London, UK
                Author notes
                [Correspondence to ] Dr James William Harrison Webbe, Academic Neonatal Medicine, Imperial College London, London, UK; j.webbe@ 123456imperial.ac.uk
                Author information
                http://orcid.org/0000-0001-8546-3212
                http://orcid.org/0000-0002-8672-5349
                http://orcid.org/0000-0001-8570-9374
                http://orcid.org/0000-0002-1984-4575
                http://orcid.org/0000-0001-5890-2953
                http://orcid.org/0000-0002-2093-0681
                http://orcid.org/0000-0003-0707-876X
                Article
                fetalneonatal-2019-317501
                10.1136/archdischild-2019-317501
                7363790
                31732683
                30efc398-a2a5-41c5-b762-a7e820b5afca
                © Author(s) (or their employer(s)) 2020. Re-use permitted under CC BY. Published by BMJ.

                This is an open access article distributed in accordance with the Creative Commons Attribution 4.0 Unported (CC BY 4.0) license, which permits others to copy, redistribute, remix, transform and build upon this work for any purpose, provided the original work is properly cited, a link to the licence is given, and indication of whether changes were made. See: https://creativecommons.org/licenses/by/4.0/.

                History
                : 01 May 2019
                : 01 October 2019
                : 30 October 2019
                Funding
                Funded by: Mason Medical Research Foundation;
                Funded by: The Portland Hospital;
                Funded by: FundRef http://dx.doi.org/10.13039/501100000265, Medical Research Council;
                Award ID: MR/N008405/1
                Categories
                Original Research
                1506
                Custom metadata
                unlocked

                Neonatology
                neonatology,outcomes research,evidence based medicine
                Neonatology
                neonatology, outcomes research, evidence based medicine

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