Cargando…

"You can't always get what you want": from doctrine to practicability of study designs for clinical investigation in endometriosis

BACKGROUND: 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 controll...

Descripción completa

Detalles Bibliográficos
Autores principales: Vercellini, Paolo, Somigliana, Edgardo, Cortinovis, Ivan, Bracco, Benedetta, de Braud, Lucrezia, Dridi, Dhouha, Milani, Silvano
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2015
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4618787/
https://www.ncbi.nlm.nih.gov/pubmed/26490454
http://dx.doi.org/10.1186/s12905-015-0248-4
_version_ 1782396974250590208
author Vercellini, Paolo
Somigliana, Edgardo
Cortinovis, Ivan
Bracco, Benedetta
de Braud, Lucrezia
Dridi, Dhouha
Milani, Silvano
author_facet Vercellini, Paolo
Somigliana, Edgardo
Cortinovis, Ivan
Bracco, Benedetta
de Braud, Lucrezia
Dridi, Dhouha
Milani, Silvano
author_sort Vercellini, Paolo
collection PubMed
description BACKGROUND: 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. METHODS: 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. RESULTS: 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. CONCLUSION: 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.
format Online
Article
Text
id pubmed-4618787
institution National Center for Biotechnology Information
language English
publishDate 2015
publisher BioMed Central
record_format MEDLINE/PubMed
spelling pubmed-46187872015-10-25 "You can't always get what you want": from doctrine to practicability of study designs for clinical investigation in endometriosis Vercellini, Paolo Somigliana, Edgardo Cortinovis, Ivan Bracco, Benedetta de Braud, Lucrezia Dridi, Dhouha Milani, Silvano BMC Womens Health Research Article BACKGROUND: 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. METHODS: 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. RESULTS: 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. CONCLUSION: 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. BioMed Central 2015-10-22 /pmc/articles/PMC4618787/ /pubmed/26490454 http://dx.doi.org/10.1186/s12905-015-0248-4 Text en © Vercellini et al. 2015 Open AccessThis article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated.
spellingShingle Research Article
Vercellini, Paolo
Somigliana, Edgardo
Cortinovis, Ivan
Bracco, Benedetta
de Braud, Lucrezia
Dridi, Dhouha
Milani, Silvano
"You can't always get what you want": from doctrine to practicability of study designs for clinical investigation in endometriosis
title "You can't always get what you want": from doctrine to practicability of study designs for clinical investigation in endometriosis
title_full "You can't always get what you want": from doctrine to practicability of study designs for clinical investigation in endometriosis
title_fullStr "You can't always get what you want": from doctrine to practicability of study designs for clinical investigation in endometriosis
title_full_unstemmed "You can't always get what you want": from doctrine to practicability of study designs for clinical investigation in endometriosis
title_short "You can't always get what you want": from doctrine to practicability of study designs for clinical investigation in endometriosis
title_sort "you can't always get what you want": from doctrine to practicability of study designs for clinical investigation in endometriosis
topic Research Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4618787/
https://www.ncbi.nlm.nih.gov/pubmed/26490454
http://dx.doi.org/10.1186/s12905-015-0248-4
work_keys_str_mv AT vercellinipaolo youcantalwaysgetwhatyouwantfromdoctrinetopracticabilityofstudydesignsforclinicalinvestigationinendometriosis
AT somiglianaedgardo youcantalwaysgetwhatyouwantfromdoctrinetopracticabilityofstudydesignsforclinicalinvestigationinendometriosis
AT cortinovisivan youcantalwaysgetwhatyouwantfromdoctrinetopracticabilityofstudydesignsforclinicalinvestigationinendometriosis
AT braccobenedetta youcantalwaysgetwhatyouwantfromdoctrinetopracticabilityofstudydesignsforclinicalinvestigationinendometriosis
AT debraudlucrezia youcantalwaysgetwhatyouwantfromdoctrinetopracticabilityofstudydesignsforclinicalinvestigationinendometriosis
AT drididhouha youcantalwaysgetwhatyouwantfromdoctrinetopracticabilityofstudydesignsforclinicalinvestigationinendometriosis
AT milanisilvano youcantalwaysgetwhatyouwantfromdoctrinetopracticabilityofstudydesignsforclinicalinvestigationinendometriosis