The authors declare that they have no competing interest.
Conception and design: PV, and ES. Acquisition of data: BB, LdB and DD. Analysis and interpretation of data: PV, ES, IC, and SM. Drafting the article: PV. Critical revision of the article for intellectual content: all authors. All the authors approved the final version of the manuscript.
1. Department of Clinical Sciences and Community Health, Università degli Studi and Fondazione Ca’ Granda - Ospedale Maggiore Policlinico, Via Commenda 12, 20122 Milan, Italy; PV is Associate Professor of Obstetrics and Gynecology; BB and LdB are residents in Obstetrics and Gynecology; DD is research fellow in Obstetrics and Gynecology.
2. Infertility Unit, Fondazione Ca’ Granda - Ospedale Maggiore Policlinico, Via Manfredo Fanti 6, 20122 Milan, Italy; ES is Director of the Infertility Unit.
3. Unit of Medical Statistics and Biometry, Department of Clinical Sciences and Community Health, Università degli Studi, Via Vanzetti 5, 20133 Milan, Italy. IC is Researcher in Medical Statistics; SM is Full Professor of Medical Statistics.
Patients, now generally well informed through dedicated websites and support organizations, are beginning to look askance at clinical experimentation. We conducted a survey investigation to verify whether women with endometriosis would still accept to participate in a randomized controlled trial (RCT) on treatment for pelvic pain.
A total of 500 patients consecutively self-referring to an academic outpatient endometriosis clinic, were asked to compile two questionnaires focused on hypothetical comparisons between a new drug and a standard drug, and between medical and surgical treatment, for endometriosis-associated pelvic pain. The main outcome measure was the percentage of patients willing to participate in a theoretical RCT.
A total of 239 (48 %) women would decline participation in a comparative study on a new drug and a standard drug, as 204 (41 %) would prefer the former medication, and 35 (7 %) the latter. Fifty women (10 %) would participate in a RCT, but only 24 (5 %) would accept blinding. The most frequently chosen option was the patient preference trial (211; 42 %). No significant differences were observed in demographic and clinical characteristics between the 50 women who would accept and the 450 who would decline to be enrolled in a RCT. A total of 229 women (46 %) would decline participation in a comparative study on medical versus surgical treatment, as 186 (37 %) would prefer pharmacological therapy and 43 (9 %) a surgical procedure. Only 11 (2 %) women would participate in such a RCT. More than half of the women (260; 52 %) selected the patient preference trial. No significant variations in distributions of answers were observed between women who did or did not undergo a previous surgical procedure.
Only a small minority of the women included in our study sample would accept randomization, and even less so blinding. Patient preference appears to play a central role when planning interventional trials on endometriosis-associated pelvic pain. Adequately designed observational analytic studies could be considered when recruitment in a RCT appears cumbersome.
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- "You can't always get what you want": from doctrine to practicability of study designs for clinical investigation in endometriosis
Lucrezia de Braud
- BioMed Central
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