Harmonising research outcomes for polycystic ovary syndrome: an international multi-stakeholder core outcome set

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.

Contributors

Bassel H.AlWattar1,2,*,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, BenW.Mol17, Khalid S. Khan1, and
Shakila Thangaratinam1

1Barts Research Centre forWomen?s Health (BARC),Women?s Health Research Unit, Barts and the London School of Medicine and
Dentistry, Queen Mary University of London, London, UK 2Warwick Medical School, University of Warwick, Coventry, UK 3National
Health and Medical Research Council Centre for Research Excellence in PCOS, Monash Centre for Health Research and Implementation,
Monash University, Melbourne, VIC, Australia 4Endocrine and Diabetes Units, Monash Health, Melbourne, VIC, Australia 5Imperial College
School of Medicine, Institute of Reproductive and Developmental Biology, Hammersmith Hospital, London, UK 6Leeds Fertility, Seacroft
Hospital, Leeds Teaching Hospitals NHS Trust, Leeds, UK 7Reproductive medicine, Homerton University Hospital NHS Foundation Trust,
London, UK 8Department of Obstetrics and Gynecology, PEDEGO Research Unit, Medical Research Centre, Oulu University Hospital,
University of Oulu, Oulu, Finland 9Department of Woman and Child Health and Public Health,Woman Health Area, Fondazione Policlinico
Universitario A. Gemelli, Rome, Italy 10Department of Woman and Child Health, Azienda Ospedaliera Card. Panico, Tricase, Italy 11Div
Reproductive Endocrinology and Infertility, Dept of Obstetrics and Gynecology, Erasmus University Medical Centre, Rotterdam, The
Netherlands 12Harp Family Medical Centre, Melbourne, VIC, Australia 13Department of Preventive Medicine and Public Health, University
of Granada, Granada, Spain 14Consortium for Biomedical Research in Epidemiology and Public Health, (CIBER Epidemiolog?a y Salud
P?blica-CIBERESP), Madrid, Spain 15Ibs Granada, Instituto de Investigaci?n Biosanitaria de Granada, Granada, Spain 16Verity, The PCOS Self
Help Group, Surry, UK 17Department of Obstetrics and Gynaecology, University of Monash, Melbourne, Australia

Publication

Journal: Human Reproduction
Volume:
Issue:
Pages: -
Year: 2020
DOI: https://doi.org/10.1093/humrep/dez272

Further Study Information

Current Stage: Completed
Date: February 2018 - August 2018
Funding source(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.


Health Area

Disease Category: Gynaecology

Disease Name: Polycystic ovary syndrome

Target Population

Age Range: 18 - 55

Sex: Female

Nature of Intervention: Any

Stakeholders Involved

- Charities
- Clinical experts
- Consumers (patients)
- Patient/ support group representatives
- Researchers

Study Type

- COS for clinical trials or clinical research

Method(s)

- Consensus meeting
- Delphi process
- Literature review

Evidence synthesis and a modified Delphi method with e-surveys were used as well as a consultation meeting.

Linked Studies

    No related studies


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