Phenotyping Responses to Optimize the Management of Pain Treatment in Endometriosis

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PROJECT SUMMARY/ABSTRACT Endometriosis is a debilitating and progressive disease that affects 10% of reproductive-aged women. Characterized by the presence of endometrial lesions outside of the uterus, endometriosis is associated with chronic pelvic pain, bleeding, and infertility, with pain being the most debilitating symptom for which patients seek care. Endometriosis results in tremendous economic and healthcare burden and greatly impacts quality of life. Clinical practice guidelines for endometriosis recommend six months of conservative medical treatment for suppression of local or systemic estrogen levels; however, substantial variation exists in the degree of pain relief obtained from pharmacological therapy and some women subsequently require surgical removal of lesions, suggesting that heterogeneity in disease and symptom presentation may underlie treatment response. Further, the mechanisms that predict treatment failure and/or success are poorly characterized, thereby leaving providers in a position of long-term symptom management and with little guidance regarding therapies that may achieve the greatest efficacy. Quantifying this heterogeneity and identifying treatment response characteristics may be a crucial step toward individualizing therapeutic approaches for endometriosis to reduce overall pain burden. In line with the Precision Medicine Initiative, the aims of this prospective observational study are to explore mechanisms associated with treatment response in patients undergoing guideline-based conservative medical therapy for endometriosis, and to characterize associations between phenotypic profiles with pelvic pain severity and pain impact trajectories. Premenopausal women ages 18 to 45 years with clinically suspected endometriosis will be recruited and followed for a period of six months after undergoing conservative treatment according to the American College of Obstetricians and Gynecologists clinical practice guidelines. Heterogeneity in pre- treatment endometriosis symptom presentation will be quantified across the following measures: (1) sociodemographic, (2) clinical, (3) biomedical, (4) pain quality, (5) behavioral, (6) psychological, and (7) functional health. Patients will be followed monthly for six months to assess pelvic pain severity and pain impact. Collectively, these aims have the potential to advance our understanding of phenotypic mechanisms underlying conservative treatment response among women with endometriosis, effects which may serve as a foundation for reducing unnecessary and ineffective medical treatments and lessening the time to proper diagnosis and treatment.