OBJECTIVES: PCOS is the most common endocrinopathy affecting reproductive aged women yet recent evidence-based treatment guidelines have been noted as inadequate. This study utilized real word data (RWD) to identify first-line treatments for PCOS anovulatory infertility and assessed the one-year pregnancy rates for each treatment.

METHODS: Female patients aged 18-45 with at least two PCOS documented visits (ICD-10 E28.2) during January 1, 2014 – January 1, 2017 were identified in TriNetX Research utilizing a longitudinal dataset of linked primary care data, medical and pharmacy claims of 190M patients. Patients had at least 1 year of medical history prior to and after first diagnosis of PCOS. Treatment Pathways analytic calculated percentage of first-line patients on specified treatments. Cohorts of patients on highest yielding single or combination treatments but no other treatment exposure were used to assess risk of pregnancy (ICD-10 Z33 or CPT O00-O9A) within 12 months from first exposure by measures of association and survival analysis conducted via Kaplan-Meier estimator.

RESULTS: The study included 111,144 PCOS patients (median age 32.4y). Top first-line treatments identified were metformin (67.9%), dietary counseling and surveillance (13.8%), clomiphene+gondatropins (5.4%), letrozole (4.1%), laparoscopic ovarian surgery (3.7%), gonadotropins (1.9%), and in vitro fertilization (0.9%). Pregnancy outcomes were highest for IVF-treated patients (risk 69.90%; survival probability 24.92%) followed by G (46.07%; 44/77%), CG (39.82%; 57.10%), L (39.70%; 54/31%), DCS (10.80%; 87.57%), LOS (10.65%; 87.32%), and M (8.95%; 9.57%).

CONCLUSIONS: This study provides RWD based insights on the prevalence of first-line treatments utilized by PCOS females and rate of pregnancy within first year of each treatment. Further research is warranted to investigate pregnancy outcomes in second- and third-line patients.