{"paper_id":"582b6f9e-864f-45a8-ab71-f78da1f699d1","body_text":"Improving outcomes for women with endometriosis\nItem Status\nEmbargo End Date\nDate\nAuthors\nAbstract\nBackground\n“Endometriosis is a chronic hormone-dependent inflammatory disorder that affects 6-\n10% of women of reproductive age. It is defined by the presence of endometrial-like\ntissue (lesions) found outside the uterine cavity, usually within the pelvis. The most\nwidely reported symptoms of endometriosis are chronic pelvic pain and infertility.\nEndometriosis-associated pain can have a significant and debilitating impact on\nquality of life. In the United Kingdom, the impact on society of endometriosis is\nestimated at £8.2 billion in healthcare/treatment costs coupled with lost productivity.\nThe exact aetiology of endometriosis is unknown. Standard treatments such as\nanalgesic regimens, drugs that induce hormone suppression or invasive surgery can\nbe associated with unacceptable side effects or complications. Notably many medical\ntreatments are contraceptive, and endometriosis-associated symptoms typically recur\nafter women stop taking drug treatments or after surgery. New therapies are therefore\nneeded that: (i) can reduce painful symptoms associated with the condition (ii) are\ndisease modifying (reduce lesion size) (iii) preserve the patients’ ability to conceive\nwhilst on medication (iv) have no, or limited, side effects and (v) are acceptable to\nwomen with endometriosis.”\nObjectives\n“The two aims of my thesis were (a) to identify research priorities for endometriosis\nand (b) to determine the feasibility of a future multi-centre, large randomised\ncontrolled trial (RCT) to evaluate the effectiveness of Omega-3 purified fatty acids\n(PUFA) for endometriosis-associated pain. To achieve these objectives, I have (a)\nengaged with experts in the management of endometriosis, women with\nendometriosis and prepared a report of the research uncertainties, using a template\nprovided by the James Lind Alliance (JLA) and (b) undertaken a two-arm, double-blind\npilot randomised controlled clinical trial.”\n(a) Identifying research priorities for endometriosis\n“The JLA brings patients, carers and clinicians together in Priority Setting\nPartnerships (PSPs) to identify and prioritise the ‘top 10’ uncertainties, or\nunanswered questions, about the effects of treatments. An endometriosis PSP was\nlaunched in 2016 with the formation of a steering group, I prepared a survey where\nresearch questions were invited from members of the public (this included women\nwith endometriosis and their families) and health care professionals (HCPs). Priority\nsetting was carried out using data from (a) online survey complemented by clinical\nguidelines and systematic reviews (b) online voting and (c) a facilitated workshop of\nequal numbers of women with endometriosis and HCPs. In the first online survey,\n4767 research questions were submitted by 1237 UK and Ireland respondents (67.7%\nwomen with endometriosis and 15.8% HCPs). A further 111 research uncertainties\nwere identified from literature searches. After removing questions already addressed\nby systematic reviews or ongoing research and merging similar questions, 72 were\nlisted in a second survey allowing participants to vote and rank the questions, 1380\nparticipants from the UK and Ireland voted. From the 30 uncertainties which\nreceived most votes, 10 research priorities were agreed during a final face to face\nworkshop comprising of equal numbers of women with endometriosis and HCPs.”\n(b) A pilot trial to evaluate the feasibility of performing a future RCT of the\neffectiveness of Omega-3 PUFA for endometriosis-associated pain (PurFECT)\n“In human trials on painful inflammatory conditions, other than endometriosis,\nOmega-3 PUFA have been shown to have minimal side effects with no obvious\neffects on fertility and may therefore be an acceptable treatment for endometriosis-associated\nsymptoms. I performed a pilot two-armed double-blind RCT in NHS\nLothian, comparing Omega-3 PUFA to a comparator (olive oil), to assess the\nfeasibility of a future large-scale RCT for treatment of women with endometriosis-associated\npain. The primary objectives were to determine recruitment and retention\nrates. The secondary objectives were to evaluate the acceptability of the methods of\nrecruitment, randomisation, interventions and assessment tools and any signals of\neffectiveness of the interventions. The analyses by treatment group were by\nintention-to-treat. Between June 2016 and June 2017, of the 92 women who were\nreferred to the study, 73 (79.3%) were eligible, 42 (57.5%) recruited and 33 (81.8%)\nwomen randomised. These 33 women all of whom had endometriosis-associated\npain were randomised double-blindly and given either Omega-3 PUFA or the\ncomparator. The effects of the interventions were assessed by validated pain scores\nand physical/emotional functioning questionnaires, completed at baseline and at the\nend of the study. The acceptability of the questionnaires was recorded as data\ncompletion and the absence of missing values. The majority of participants described\ntheir trial experience favourably. The recruitment rate was 57.5% (i.e. > 50%), the\nretention rate was 81.8% (considered a satisfactory rate), and the primary outcome\nwas achieved. For most of the assessment tools, it appeared that there is no evidence\nthat the treatment is better than control.”\nConclusions\nThe top 10 priorities identified by the PSP provide a platform for researchers, funding\nbodies and the pharmaceutical industry to ensure that future research activities focus\non questions that are important to both women with endometriosis and HCPs. The\nPurFECT trial shows that a future RCT is feasible, but also highlights the need for a\nlarger appropriately powered trial to assess the effectiveness of Omega-3 PUFA as\ntreatment for endometriosis-associated pain.\nThis item appears in the following Collection(s)","source_license":"CC0","license_restricted":false}