Bassel H Al Wattar1,2, Helena Teede3,4, Rhonda Garad3,4, Steve Franks5, Adam Balen6, Priya Bhide1,7, Terhi Piltonen8, Daniela Romualdi9,10, Joop Laven11, Mala Thondan12, Aurora Bueno-Cavanillas13,14,15, Ngawai Moss1, Caroline Andrews16, Rachel Hawkes16, Ben W Mol17, Khalid S Khan1, Shakila Thangaratinam1. 1. Barts Research Centre for Women's Health (BARC), Women's Health Research Unit, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, London, UK. 2. Warwick Medical School, University of Warwick, Coventry, UK. 3. National Health and Medical Research Council Centre for Research Excellence in PCOS, Monash Centre for Health Research and Implementation, Monash University, Melbourne, VIC, Australia. 4. Endocrine and Diabetes Units, Monash Health, Melbourne, VIC, Australia. 5. Imperial College School of Medicine, Institute of Reproductive and Developmental Biology, Hammersmith Hospital, London, UK. 6. Leeds Fertility, Seacroft Hospital, Leeds Teaching Hospitals NHS Trust, Leeds, UK. 7. Reproductive medicine, Homerton University Hospital NHS Foundation Trust, London, UK. 8. Department of Obstetrics and Gynecology, PEDEGO Research Unit, Medical Research Centre, Oulu University Hospital, University of Oulu, Oulu, Finland. 9. Department of Woman and Child Health and Public Health, Woman Health Area, Fondazione Policlinico Universitario A. Gemelli, Rome, Italy. 10. Department of Woman and Child Health, Azienda Ospedaliera Card. Panico, Tricase, Italy. 11. Div Reproductive Endocrinology and Infertility, Dept of Obstetrics and Gynecology, Erasmus University Medical Centre, Rotterdam, The Netherlands. 12. Harp Family Medical Centre, Melbourne, VIC, Australia. 13. Department of Preventive Medicine and Public Health, University of Granada, Granada, Spain. 14. Consortium for Biomedical Research in Epidemiology and Public Health, (CIBER Epidemiología y Salud Pública-CIBERESP), Madrid, Spain. 15. Ibs Granada, Instituto de Investigación Biosanitaria de Granada, Granada, Spain. 16. Verity, The PCOS Self Help Group, Surry, UK. 17. Department of Obstetrics and Gynaecology, University of Monash, Melbourne, Australia.
Abstract
STUDY QUESTION: What are the key core outcomes to be reported in studies on polycystic ovary syndrome (PCOS)? SUMMARY ANSWER: We identified 3 generic and 30 specific core outcomes in 6 specialist domains: metabolic (8), reproductive (7), pregnancy (10), oncological (1), psychological (1) and long-term outcomes (1). WHAT IS KNOWN ALREADY: Research reporting PCOS is heterogeneous with high variation in outcome selection, definition and quality. STUDY DESIGN, SIZE, DURATION: Evidence synthesis and a modified Delphi method with e-surveys were used as well as a consultation meeting. PARTICIPANTS/MATERIALS, SETTING, METHODS: Overall, 71 health professionals and 123 lay consumers (women with lived experience of PCOS and members of advocacy and peer support groups) from 17 high-, middle- and low-income countries were involved in this analysis. MAIN RESULTS AND THE ROLE OF CHANCE: The final core outcome set included 3 generic outcomes (BMI, quality of life, treatment satisfaction) that are applicable to all studies on women with PCOS and 30 specific outcomes that were categorised into six specialist domains: 8 metabolic outcomes (waist circumference, type 2 diabetes, insulin resistance, impaired glucose tolerance, hypertension, coronary heart disease, lipid profile, venous thromboembolic disease); 7 reproductive outcomes [viable pregnancy (confirmed by ultrasound including singleton, twins and higher multiples), clinical and biochemical hyperandrogenism, menstrual regularity, reproductive hormonal profile, chronic anovulation, ovulation stimulation success including the number of stimulated follicles ≥ 12 mm, incidence and severity of ovarian hyperstimulation syndrome]; 10 pregnancy outcomes (live birth, miscarriage, stillbirth, neonatal mortality, gestational weight gain, gestational diabetes, preterm birth, hypertensive disease in pregnancy, baby birth weight, major congenital abnormalities); 3 psychological outcomes (depression, anxiety, eating disorders); 1 oncological (abnormal endometrial proliferation including atypical endometrial hyperplasia and endometrial cancer); and 1 outcome in the long-term domain (long-term offspring metabolic and developmental outcomes). LIMITATIONS, REASONS FOR CAUTION: We involved lay consumers in all stages of study through e-surveys but not through focus groups, thereby limiting our understanding of their choices. We did not address the variations in the definitions and measurement tools for some of the core outcomes. WIDER IMPLICATIONS OF THE FINDINGS: Implementing this core outcome set in future studies on women with PCOS will improve the quality of reporting and aid evidence synthesis. STUDY FUNDING/COMPETING INTEREST(S): Evidence synthesis was funded through the Australian government, National Health and Medical Research Council (NHMRC) Centre for Research Excellence in PCOS, and H.T. is funded through an NHMRC fellowship. B.H.A. is funded through an NIHR lectureship. All authors have no competing interest to declare.
STUDY QUESTION: What are the key core outcomes to be reported in studies on polycystic ovary syndrome (PCOS)? SUMMARY ANSWER: We identified 3 generic and 30 specific core outcomes in 6 specialist domains: metabolic (8), reproductive (7), pregnancy (10), oncological (1), psychological (1) and long-term outcomes (1). WHAT IS KNOWN ALREADY: Research reporting PCOS is heterogeneous with high variation in outcome selection, definition and quality. STUDY DESIGN, SIZE, DURATION: Evidence synthesis and a modified Delphi method with e-surveys were used as well as a consultation meeting. PARTICIPANTS/MATERIALS, SETTING, METHODS: Overall, 71 health professionals and 123 lay consumers (women with lived experience of PCOS and members of advocacy and peer support groups) from 17 high-, middle- and low-income countries were involved in this analysis. MAIN RESULTS AND THE ROLE OF CHANCE: The final core outcome set included 3 generic outcomes (BMI, quality of life, treatment satisfaction) that are applicable to all studies on women with PCOS and 30 specific outcomes that were categorised into six specialist domains: 8 metabolic outcomes (waist circumference, type 2 diabetes, insulin resistance, impaired glucose tolerance, hypertension, coronary heart disease, lipid profile, venous thromboembolic disease); 7 reproductive outcomes [viable pregnancy (confirmed by ultrasound including singleton, twins and higher multiples), clinical and biochemical hyperandrogenism, menstrual regularity, reproductive hormonal profile, chronic anovulation, ovulation stimulation success including the number of stimulated follicles ≥ 12 mm, incidence and severity of ovarian hyperstimulation syndrome]; 10 pregnancy outcomes (live birth, miscarriage, stillbirth, neonatal mortality, gestational weight gain, gestational diabetes, preterm birth, hypertensive disease in pregnancy, baby birth weight, major congenital abnormalities); 3 psychological outcomes (depression, anxiety, eating disorders); 1 oncological (abnormal endometrial proliferation including atypical endometrial hyperplasia and endometrial cancer); and 1 outcome in the long-term domain (long-term offspring metabolic and developmental outcomes). LIMITATIONS, REASONS FOR CAUTION: We involved lay consumers in all stages of study through e-surveys but not through focus groups, thereby limiting our understanding of their choices. We did not address the variations in the definitions and measurement tools for some of the core outcomes. WIDER IMPLICATIONS OF THE FINDINGS: Implementing this core outcome set in future studies on women with PCOS will improve the quality of reporting and aid evidence synthesis. STUDY FUNDING/COMPETING INTEREST(S): Evidence synthesis was funded through the Australian government, National Health and Medical Research Council (NHMRC) Centre for Research Excellence in PCOS, and H.T. is funded through an NHMRC fellowship. B.H.A. is funded through an NIHR lectureship. All authors have no competing interest to declare.
Authors: Krystle Y Chong; Sarah Solangon; James Kemper; Kurt Barnhart; Pamela Causa Andrieu; Perrine Capmas; Carolina Chacon; George Condous; Liesl de Waard; James M N Duffy; Andrew Horne; Maria Memtsa; Femke Mol; Munira Oza; Annika Strandell; Madelon van Wely; Janneke Van't Hooft; Lan N Vuong; Jian Zhang; Davor Jurkovic; Ben W Mol Journal: Trials Date: 2021-11-17 Impact factor: 2.279
Authors: Erica Musgrove; Loretta Gasparini; Katie McBain; Susan A Clifford; Simon A Carter; Helena Teede; Melissa Wake Journal: Pediatr Res Date: 2021-12-17 Impact factor: 3.953
Authors: Michael P Rimmer; Ruth A Howie; Richard A Anderson; Christopher L R Barratt; Kurt T Barnhart; Yusuf Beebeejaun; Ricardo Pimenta Bertolla; Siladitya Bhattacharya; Lars Björndahl; Pietro Bortoletto; Robert E Brannigan; Astrid E P Cantineau; Ettore Caroppo; Barbara L Collura; Kevin Coward; Michael L Eisenberg; Christian De Geyter; Dimitrios G Goulis; Ralf R Henkel; Vu N A Ho; Alayman F Hussein; Carin Huyser; Jozef H Kadijk; Mohan S Kamath; Shadi Khashaba; Yoshitomo Kobori; Julia Kopeika; Tansu Kucuk; Saturnino Luján; Thabo Christopher Matsaseng; Raj S Mathur; Kevin McEleny; Rod T Mitchell; Ben W Mol; Alfred M Murage; Ernest H Y Ng; Allan Pacey; Antti H Perheentupa; Stefan Du Plessis; Nathalie Rives; Ippokratis Sarris; Peter N Schlegel; Majid Shabbir; Maciej Śmiechowski; Venkatesh Subramanian; Sesh K Sunkara; Basil C Tarlarzis; Frank Tüttelmann; Andy Vail; Madelon van Wely; Mónica H Vazquez-Levin; Lan N Vuong; Alex Y Wang; Rui Wang; Armand Zini; Cindy M Farquhar; Craig Niederberger; James M N Duffy Journal: Hum Reprod Open Date: 2022-03-16