"You can't always get what you want": from doctrine to practicability of study designs for clinical investigation in endometriosis
Open accessAbstract
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.
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Integrity signals
Registry
Cites
Statistics
Claims the paper makes
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
Many women would leave surgery as the second choice, only in case medications are ineffective or not tolerated.
“many women would leave surgery as the second choice, only in case medications are ineffective or not tolerated (9).”
From A stepped-care approach to symptomatic endometriosis management: A participatory research initiative (2018) · cites this paper as [9]
Self-check
Percentages, sums and reported differences re-computed from the paper’s own numbers.
The self-check needs the paper’s full text.
Cites
Randomized, Controlled Trials, Observational Studies, and the Hierarchy of Research Designs
Concato, J., Shah, N., & Horwitz, R. I. (2000) · New England Journal of Medicine · doi:10.1056/nejm200006223422507 · Cited by 3,563 · see citing works
A comparison of observational studies and randomized, controlled trials.
Benson, K., & Hartz, A. J. (2000) · New England Journal of Medicine · doi:10.1056/nejm200006223422506 · Cited by 2,347 · see citing works
Endometriosis: pathogenesis and treatment
Vercellini, P., Viganò, P., Somigliana, E., & Fedele, L. (2014) · Nature Reviews Endocrinology · doi:10.1038/nrendo.2013.255 · Cited by 1,940 · see citing works
Self-citationSurgical vs Nonoperative Treatment for Lumbar Disk Herniation
Weinstein, J. N., Tosteson, T. D., Lurie, J. D., Tosteson, A. N. A., Hanscom, B. S., Skinner, J., Abdu, W. A., Hilibrand, A. S., Boden, S. D., & Deyo, R. A. (2006) · JAMA · doi:10.1001/jama.296.20.2441 · Cited by 1,108 · see citing works
The Use and Interpretation of Quasi-Experimental Studies in Medical Informatics
Harris, A. D., McGregor, J. C., Perencevich, E. N., Furuno, J. P., Zhu, J., Peterson, D. E., & Finkelstein, J. (2006) · Journal of the American Medical Informatics Association · doi:10.1197/jamia.m1749 · Cited by 1,023 · see citing works
Surgical vs Nonoperative Treatment for Lumbar Disk Herniation
Weinstein, J. N., Lurie, J. D., Tosteson, T. D., Skinner, J., Hanscom, B. S., Tosteson, A. N. A., Herkowitz, H. N., Fischgrund, J. S., Cammisa, F. P., Albert, T. J., et al. (2006) · JAMA · doi:10.1001/jama.296.20.2451 · Cited by 764 · see citing works
Systematic review and meta-analysis of psychological therapies for children with chronic pain
Fisher, E., Heathcote, L., Palermo, T. M., de C Williams, A. C., Lau, J., & Eccleston, C. (2014) · Journal of Pediatric Psychology · doi:10.1093/jpepsy/jsu008 · Cited by 710 · see citing works
Holm, J., Nowicka, P., Farpour‐Lambert, N. J., O’Malley, G., Hassapidou, M. N., Weiss, R., & Baker, J. L. (2014) · Obesity Facts · doi:10.1159/000365773 · Cited by 596 · see citing works
Patient preferences and randomised clinical trials.
Brewin, C. R., & Bradley, C. (1989) · BMJ · doi:10.1136/bmj.299.6694.313 · Cited by 503 · see citing works
A comparison of observational studies and randomized, controlled trials
Benson, K., & Hartz, A. J. (2000) · American Journal of Ophthalmology · doi:10.1016/s0002-9394(00)00754-6 · Cited by 456 · see citing works
Treatment of pelvic pain associated with endometriosis: a committee opinion
(2014) · Fertility and Sterility · doi:10.1016/j.fertnstert.2014.02.012 · Cited by 397 · see citing works
Let the patient revolution begin
Richards, T., Montori, V. M., Godlee, F., Lapsley, P., & Paul, D. (2013) · BMJ · doi:10.1136/bmj.f2614 · Cited by 325 · see citing works
Endometriomas: their ultrasound characteristics
Holsbeke, C. V., CALSTER, B. V., Guerriero, S., Savelli, L., Paladini, D., Lissoni, A., Czekierdowski, A., Fischerová, D., Zhang, J., Mestdagh, G., et al. (2010) · Ultrasound in Obstetrics and Gynecology · doi:10.1002/uog.7668 · Cited by 298 · see citing works
Piketty, M., Chopin, N., Dousset, B. E., Millischer, A., Roseau, G., Leconte, M., Borghese, B., & Chapron, C. (2008) · Human Reproduction · doi:10.1093/humrep/den405 · Cited by 284 · see citing works
Hudelist, G., English, J., Thomas, A. E., Tinelli, A., Singer, C. F., & Keckstein, J. (2011) · Ultrasound in Obstetrics & Gynecology · doi:10.1002/uog.8858 · Cited by 270 · see citing works
The Optimal Practice of Evidence-Based Medicine
Montori, V. M., Brito, J. P., & Murad, M. H. (2013) · JAMA · doi:10.1001/jama.2013.281422 · Cited by 268 · see citing works
Dosage aspects of danazol therapy in endometriosis: Short-term and long-term effectiveness
Biberoglu, K. O., & Behrman, S. J. (1981) · American Journal of Obstetrics and Gynecology · doi:10.1016/0002-9378(81)90478-6 · Cited by 267 · see citing works
Braunstein, J. B., Sherber, N., Schulman, S. P., Ding, E. L., & Powe, N. R. (2008) · Medicine · doi:10.1097/md.0b013e3181625d78 · Cited by 256 · see citing works
Getting the methods right--the foundation of patient-centered outcomes research
Gabriel, S. E., & Normand, S.-L. T. (2012) · New England Journal of Medicine · doi:10.1056/nejmp1207437 · Cited by 252 · see citing works
A systematic review of the accuracy of ultrasound in the diagnosis of endometriosis
Moore, J. A., Copley, S. J., Morris, J., Lindsell, D. R., Golding, S. J., & Kennedy, S. (2002) · Ultrasound in Obstetrics and Gynecology · doi:10.1046/j.1469-0705.2002.00862.x · Cited by 244 · see citing works
Crowther, C. A., Dodd, J. M., Hiller, J. E., Haslam, R. R., Robinson, J. S., & on behalf of the Birth After Caesarean Study Group (2012) · PLoS Medicine · doi:10.1371/journal.pmed.1001192 · Cited by 197 · see citing works
Equipoise and the Dilemma of Randomized Clinical Trials
Miller, F. G., & Joffe, S. (2011) · New England Journal of Medicine · doi:10.1056/nejmsb1011301 · Cited by 189 · see citing works
Specific barriers to the conduct of randomized trials
Duley, L., Antman, K. H., Arena, J. P., Avezum, Á., Blumenthal, M., Bosch, J., Chrolavicius, S., Li, T., Ôunpuu, S., Pérez, A. C., et al. (2008) · Clinical Trials · doi:10.1177/1740774507087704 · Cited by 173 · see citing works
King, M., Nazareth, I., Lampe, F. C., Bower, P., Chandler, M., Morou, M., Sibbald, B., & Lai, R. (2005) · Health Technology Assessment · doi:10.3310/hta9350 · Cited by 164 · see citing works
Hudelist, G., Fritzer, N., Stättner, S., Tammaa, A., Tinelli, A., Sparić, R., & Keckstein, J. (2013) · Ultrasound in Obstetrics and Gynecology · doi:10.1002/uog.12431 · Cited by 149 · see citing works
Vercellini, P., Parazzini, F., Pietropaolo, G., Cipriani, S., Frattaruolo, M., & Fedele, L. (2012) · BJOG: An International Journal of Obstetrics & Gynaecology · doi:10.1111/j.1471-0528.2012.03466.x · Cited by 134 · see citing works
Self-citationVercellini, P., Somigliana, E., Consonni, D., Frattaruolo, M. P., De Giorgi, O., & Fedele, L. (2012) · Human Reproduction · doi:10.1093/humrep/des313 · Cited by 129 · see citing works
Self-citationRandomized Clinical Trials — Removing Unnecessary Obstacles
Reith, C. A., Landray, M., Devereaux, P. J., Bosch, J., Granger, C. B., Baigent, C., Califf, R. M., Collins, R., & Yusuf, S. (2013) · New England Journal of Medicine · doi:10.1056/nejmsb1300760 · Cited by 123 · see citing works
Holland, T., Cutner, A. S., Sarıdoğan, E., Mavrelos, D., Pateman, K., & Jurkovic, D. (2013) · BMC Women s Health · doi:10.1186/1472-6874-13-43 · Cited by 118 · see citing works
Comparison of contraceptive ring and patch for the treatment of symptomatic endometriosis
Vercellini, P., Barbàra, G., Somigliana, E., Bianchi, S., Abbiati, A., & Fedele, L. (2009) · Fertility and Sterility · doi:10.1016/j.fertnstert.2009.01.071 · Cited by 112 · see citing works
Self-citationSnow, R., Humphrey, C., & Sandall, J. (2013) · BMJ Open · doi:10.1136/bmjopen-2013-003583 · Cited by 103 · see citing works
The pathogenesis of bladder detrusor endometriosis
Vercellini, P., Frontino, G., Pisacreta, A. M., Giorgi, O. D., Cattaneo, M., & Crosignani, P. G. (2002) · American Journal of Obstetrics and Gynecology · doi:10.1067/mob.2002.124286 · Cited by 99 · see citing works
Self-citationDiagnostic accuracy and potential limitations of transvaginal sonography for bladder endometriosis
Savelli, L., Manuzzi, L., Pollastri, P., et al. (2009) · Ultrasound in Obstetrics and Gynecology · doi:10.1002/uog.7356 · Cited by 97 · see citing works
Clinical trials bureaucracy: unintended consequences of well-intentioned policy
Califf, R. M. (2006) · Clinical Trials · doi:10.1177/1740774506073173 · Cited by 91 · see citing works
Before and after study designs
Sedgwick, P. M. (2014) · BMJ · doi:10.1136/bmj.g5074 · Cited by 91 · see citing works
The imperative of overcoming barriers to the conduct of large, simple trials.
Eapen, Z. J., Lauer, M. S., & Temple, R. J. (2014) · JAMA · doi:10.1001/jama.2014.1030 · Cited by 89 · see citing works
Subjects no more: what happens when trial participants realize they hold the power?
Wicks, P. J., Vaughan, T. E., & Heywood, J. (2014) · BMJ · doi:10.1136/bmj.g368 · Cited by 75 · see citing works
The unintended consequences of clinical trials regulations
McMahon, A. D., Conway, D. I., MacDonald, T. M., & McInnes, G. T. (2009) · PLoS Medicine · doi:10.1371/journal.pmed.1000131 · Cited by 73 · see citing works
Assessing Participant-Centered Outcomes to Improve Clinical Research
Kost, R. G., Lee, L. M., Yessis, J. L., WESLEY, R. A., Henderson, D. K., & Coller, B. S. (2013) · New England Journal of Medicine · doi:10.1056/nejmp1311461 · Cited by 60 · see citing works
An overview of the current status of clinical trials on endometriosis: issues and concerns
Guo, S. (2013) · Fertility and Sterility · doi:10.1016/j.fertnstert.2013.08.050 · Cited by 54 · see citing works
Holsbeke, C. V., Zhang, J., Belle, V. V., et al. (2009) · Ultrasound in Obstetrics and Gynecology · doi:10.1002/uog.7537 · Cited by 47 · see citing works
Impediments to Clinical Research in the United States
Kramer, J. M., Smith, P. B., & Califf, R. M. (2012) · Clinical Pharmacology & Therapeutics · doi:10.1038/clpt.2011.341 · Cited by 46 · see citing works
Lack of Transparency of Clinical Trials on Endometriosis
Guo, S., & Evers, J. L. H. (2013) · Obstetrics and Gynecology · doi:10.1097/aog.0b013e318291f299 · Cited by 40 · see citing works
Sculpher, M. J. (1998) · Health Economics · doi:10.1002/(sici)1099-1050(199803)7:2 · Cited by 32 · see citing works
Jackson, C., Dixon‐Woods, M., Eborall, H., Kenyon, S. L., Toozs-Hobson, P. M., & Tincello, D. G. (2010) · Clinical Trials · doi:10.1177/1740774510381286 · Cited by 29 · see citing works
Urodynamics prior to treatment as an intervention: A pilot study
Majumdar, A., Latthe, P. M., & Toozs-Hobson, P. M. (2009) · Neurourology and Urodynamics · doi:10.1002/nau.20810 · Cited by 28 · see citing works
Krumholz, H. M., & Selby, J. V. (2012) · Annals of Internal Medicine · doi:10.7326/0003-4819-157-6-201209180-00519 · Cited by 24 · see citing works
Oseltamivir: the real world data
Freemantle, N., Shallcross, L. J., Kyte, D. G., Rader, T., & Calvert, M. (2014) · BMJ · doi:10.1136/bmj.g2371 · Cited by 22 · see citing works
Partially randomised patient preference trials
Brocklehurst, P. (1997) · BJOG An International Journal of Obstetrics & Gynaecology · doi:10.1111/j.1471-0528.1997.tb10999.x · Cited by 21 · see citing works
What is a patient preference trial?
Sedgwick, P. M. (2013) · BMJ · doi:10.1136/bmj.f5970 · Cited by 16 · see citing works
Response‐adaptive decision‐theoretic trial design: operating characteristics and ethics
Lipsky, A. M., & Lewis, R. (2013) · Statistics in Medicine · doi:10.1002/sim.5807 · Cited by 14 · see citing works
A GENERALIZED LINEAR MODEL FOR ANALYSING RECEIVER OPERATING CHARACTERISTIC CURVES
SMITH, P. J., Thompson, T. J., Engelgau, M. M., & Herman, W. H. (1996) · Statistics in Medicine · doi:10.1002/(sici)1097-0258(19960215)15:3 · Cited by 13 · see citing works
Culyer, A. J. (2014) · Cited by 12 · see citing works
The development of fair tests of treatments
Chalmers, I. (2014) · The Lancet · doi:10.1016/s0140-6736(14)60821-7 · Cited by 11 · see citing works
When Ethics Precludes Randomization: Put Prospective, Matched-Pair Observational Studies to Work
Kowalski, C. J. (2013) · Perspectives in biology and medicine · doi:10.1353/pbm.2013.0014 · Cited by 8 · see citing works
Endometriosis: the elusive gray area between evidence-based and evidence-biased medicine
Vercellini, P. (2014) · Fertility and Sterility · doi:10.1016/j.fertnstert.2013.09.023 · Cited by 7 · see citing works
Self-citation
Flagged first, then by citations
Cited by
Sorted by citations
Risk for and consequences of endometriosis: A critical epidemiologic review
Shafrir, A. L., Farland, L. V., Shah, D. K., Harris, H. R., Kvaskoff, M., Zondervan, K., & Missmer, S. A. (2018) · Best Practice & Research Clinical Obstetrics & Gynaecology · doi:10.1016/j.bpobgyn.2018.06.001 · Cited by 823 · see citing works
A stepped-care approach to symptomatic endometriosis management: A participatory research initiative
Vercellini, P., Donati, A., Ottolini, F., Frassineti, A., Fiorini, J., Nebuloni, V., Frattaruolo, M. P., Roberto, A., Mosconi, P., & Somigliana, E. (2018) · Fertility and Sterility · doi:10.1016/j.fertnstert.2018.01.037 · Cited by 64 · see citing works
Self-citationLessey, B. A., Gordts, S., Donnez, O., Somigliana, E., Chapron, C., Garcia-Velasco, J. A., & Donnez, J. (2018) · Fertility and Sterility · doi:10.1016/j.fertnstert.2018.10.003 · Cited by 48 · see citing works
Self-citationVercellini, P., Buggio, L., Borghi, A., Monti, E., Gattei, U., & Frattaruolo, M. P. (2018) · Acta Obstetricia et Gynecologica Scandinavica · doi:10.1111/aogs.13328 · Cited by 39 · see citing works
Self-citationManagement of endometriosis: Toward value-based, cost-effective, affordable care
Vercellini, P., Facchin, F., Buggio, L., Barbara, G., Berlanda, N., Frattaruolo, M. P., & Somigliana, E. (2018) · Journal of Obstetrics and Gynaecology Canada · doi:10.1016/j.jogc.2017.07.011 · Cited by 36 · see citing works
Self-citationAnogenital Distance and Endometriosis: Results of a Case–Control Study
Buggio, L., Somigliana, E., Sergenti, G., Ottolini, F., Dridi, D., & Vercellini, P. (2022) · Reproductive Sciences · doi:10.1007/s43032-022-01009-7 · Cited by 8 · see citing works
Self-citation
- Authors
- Vercellini, P., Somigliana, E., Cortinovis, I., Bracco, B., de Braud, L., Dridi, D., & Milani, S.
- Affiliations
- University of Milan (IT)
- Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico (IT)
- Corresponding author
- Vercellini, Paolo
- Venue
- BMC Women's Health
- Published
- 2015-10-22
- Volume
- 15
- Issue
- 1
- Pages
- 89-89
- ISSN
- 1472-6874 (ISSN-L)
- PMC
- PMC4618787
- OpenAlex
- W2163605459
- Cited by
- 22
- References
- 56
- Citations by year
- 2024: 1 · 2023: 1 · 2022: 7 · 2019: 2 · 2018: 7 · 2017: 2 · 2016: 2
- FWCI
- 4.19
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Licence
- Locations
- BMC Women s Health (publishedVersion, cc-by)
- Europe PMC (PubMed Central) (publishedVersion, cc-by)
- PubMed Central (publishedVersion, cc-by)
- PubMed (publishedVersion)
- Archivio Istituzionale della Ricerca (Universita Degli Studi Di Milano) (submittedVersion, other-oa)
- Funders
- Università degli Studi di Milano
- Topics
- Health Sciences › Medicine › Reproductive Medicine › Endometriosis Research and Treatment
- Health Sciences › Medicine › Public Health, Environmental and Occupational Health › Ethics in Clinical Research
- Physical Sciences › Mathematics › Statistics and Probability › Statistical Methods in Clinical Trials
- Keywords
- endometriosis, willingness to participate in clinical trials, randomized controlled trials, surgical versus medical treatment, trial design, patient recruitment
- MeSH terms
- Adult, Endometriosis, Female, Humans, Middle Aged, Prospective Studies, Research Design, Randomized Controlled Trials as Topic, Pelvic Pain
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review
