Design of Treatment Trials for Functional Gastrointestinal Disorders

Gastroenterology - Tập 130 - Trang 1538-1551 - 2006
E. Jan Irvine1, William E. Whitehead2, William D. Chey3, Kei Matsueda4, Michael Shaw5, Nicholas J. Talley6,7, Sander J.O. Veldhuyzen van Zanten8
1Division of Gastroenterology, St. Michael’s Hospital and University of Toronto, Toronto, Ontario, Canada
2Division of Gastroenterology, University of North Carolina–Chapel Hill, Chapel Hill, North Carolina
3Division of Gastroenterology, University of Michigan, Ann Arbor, Michigan
4Division of Gastroenterology, NCNP, Ichakawa City, Japan
5Division of Gastroenterology, Park Nicollet Clinic and University of Minnesota, Minneapolis, Minnesota
6Division of Gastroenterology, Mayo Clinic College of Medicine and Division of Gastroenterology and Hepatology, Rochester, Minnesota
7Department of Medicine, University of Sydney, Sydney, Australia
8Division of Gastroenterology, Dalhousie University, Halifax, Nova Scotia, Canada

Tài liệu tham khảo

Irvine, 2006, Design of treatment trials for functional gastrointestinal disorders Veldhuyzen van Zanten, 1999, Design of treatment trials for functional gastrointestinal disorders, Gut, 45, II69, 10.1136/gut.45.2008.ii69 Talley, 1993, The irritable bowel syndrome, Gastroenterology International, 189 Camilleri, 2000, Efficacy and safety of alosetron in women with irritable bowel syndrome, Lancet, 355, 1035, 10.1016/S0140-6736(00)02033-X Muller-Lissner, 2001, Tegaserod, a 5-HT(4) receptor partial agonist, relieves symptoms in irritable bowel syndrome patients with abdominal pain, bloating and constipation, Aliment Pharmacol Ther, 15, 1655, 10.1046/j.1365-2036.2001.01094.x Fass, 2001, Evidence- and consensus-based practice guidelines for the diagnosis of irritable bowel syndrome, Arch Intern Med, 161, 2081, 10.1001/archinte.161.17.2081 Cash, 2002, The utility of diagnostic tests in irritable bowel syndrome patients, Am J Gastroenterol, 97, 2812, 10.1111/j.1572-0241.2002.07027.x Jones, 1999, Likely impacts of recruitment site and methodology on characteristics of enrolled patient population, Am J Med, 107, 85S, 10.1016/S0002-9343(99)00085-6 Talley, 1998, Efficacy of omeprazole in functional dyspepsia, Aliment Pharmacol Ther, 12, 1055, 10.1046/j.1365-2036.1998.00410.x Longstreth, 2001, Characteristics of patients with irritable bowel syndrome recruited from three sources, Aliment Pharmacol Ther, 15, 959, 10.1046/j.1365-2036.2001.01010.x Camilleri, 2002, Serotonin-transporter polymorphism pharmacogenetics in diarrhea-predominant irritable bowel syndrome, Gastroenterology, 123, 425, 10.1053/gast.2002.34780 Guthrie, 2004, Changes in tolerance to rectal distension correlate with changes in psychological state in patients with severe irritable bowel syndrome, Psychosom Med, 66, 578, 10.1097/01.psy.0000128899.22514.c0 Spiller, 1999, Problems and challenges in the design of irritable bowel syndrome clinical trials, Am J Med, 107, 91S, 10.1016/S0002-9343(99)00086-8 Veldhuyzen van Zanten, 1996, Drug treatment of functional dyspepsia, Am J Gastroenterol, 91, 660 Hahn, 1998, Irritable bowel syndrome symptom patterns, Dig Dis Sci, 43, 2715, 10.1023/A:1026663613695 Drossman, 1992, The irritable bowel syndrome, Ann Intern Med, 116, 1009, 10.7326/0003-4819-116-12-1009 Whitehead, 2004, Control groups appropriate for behavioral interventions, Gastroenterol, 126, S159, 10.1053/j.gastro.2003.10.038 FDA updates warnings for cisapride. FDA Talk Paper T00-6. Rockville, MD, 2000. Camilleri, 2002, Safety concerns about alosetron, Arch Intern Med, 162, 100, 10.1001/archinte.162.1.100-a Sackett, 1979, Bias in analytic research, J Chronic Dis, 32, 51, 10.1016/0021-9681(79)90012-2 Spilker, 1991, 15 Altman, 1991, Randomisation, Br Med J, 302, 1481, 10.1136/bmj.302.6791.1481 Altman, 1985, Comparability of randomised groups, The Statistician, 34, 125, 10.2307/2987510 Spilker, 1991, 69 Temple, 1997, When are clinical trials of a given agent vs. placebo no longer appropriate or feasible?, Control Clin Trials, 18, 613, 10.1016/S0197-2456(97)00058-5 Guthrie, 1991, A controlled trial of psychological treatment for the irritable bowel syndrome, Gastroenterology, 100, 450, 10.1016/0016-5085(91)90215-7 Borkovec, 1972, Credibility of analogue therapy rationales, J Behav Ther Exp Psychiatry, 3, 257, 10.1016/0005-7916(72)90045-6 Drossman, 2003, Cognitive-behavioral therapy versus education and desipramine versus placebo for moderate to severe functional bowel disorders, Gastroenterology, 125, 19, 10.1016/S0016-5085(03)00669-3 Thompson, 2000, Placebos, Am J Gastroenterol, 95, 1637, 10.1111/j.1572-0241.2000.02179.x Bland, 1994, Some examples of regression towards the mean, BMJ, 309, 780, 10.1136/bmj.309.6957.780 Pitz, 2005, Defining the predictors of the placebo response in irritable bowel syndrome, Clin Gastroenterol Hepatol, 3, 237, 10.1016/S1542-3565(04)00626-3 Howarth, 1999, Double-blind, placebo-controlled study comparing the efficacy and safety of fexofenadine hydrochloride (120 and 180 mg once daily) and cetirizine in seasonal allergic rhinitis, J Allergy Clin Immunol, 104, 927, 10.1016/S0091-6749(99)70070-9 Bachert, 1998, Mizolastine therapy also has an effect on nasal blockade in perennial allergic rhinoconjunctivitis, Allergy, 53, 969, 10.1111/j.1398-9995.1998.tb03798.x Berger, 2003, Direct effect on validity of response run-in selection in clinical trials, Control Clin Trials, 24, 156, 10.1016/S0197-2456(02)00316-1 Hills, 2004, The two-period cross-over clinical trial, Br J Clin Pharmacol, 58, S703, 10.1111/j.1365-2125.2004.02275.x 1998 Cleophas, 2000, Limitations of randomized clinical trials. Proposed alternative designs, Clin Chem Lab Med, 38, 1217, 10.1515/CCLM.2000.192 2003 Tack, 2005, A randomised controlled trial assessing the efficacy and safety of repeated tegaserod therapy in women with irritable bowel syndrome with constipation (IBS-C), Gut, 54, 1707, 10.1136/gut.2005.070789 Bardhan, 2003, Intermittent and on-demand use of proton pump inhibitors in the management of symptomatic gastroesophageal reflux disease, Am J Gastroenterol, 98, S40, 10.1016/S0002-9270(03)00014-5 Thompson, 2000, Functional bowel disorders and functional abdominal pain, 351 Tfelt-Hansen, 2000, Guidelines for controlled trials of drugs in migraine. 2nd ed, Cephalalgia, 20, 765, 10.1046/j.1468-2982.2000.00117.x Tytgat, 2003, Contemporary understanding and management of reflux and constipation in the general population and pregnancy, Aliment Pharmacol Ther, 18, 291, 10.1046/j.1365-2036.2003.01679.x Tinmouth, 2004, Are claims of equivalency in digestive diseases trials supported by the evidence?, Gastroenterology, 126, 1700, 10.1053/j.gastro.2004.03.005 Tillisch, 2005, Characterization of the alternating bowel habit subtype in patients with irritable bowel syndrome, Am J Gastroenterol, 100, 896, 10.1111/j.1572-0241.2005.41211.x Talley, 1992, Onset and disappearance of gastrointestinal symptoms and functional gastrointestinal disorders, Am J Epidemiol, 136, 165, 10.1093/oxfordjournals.aje.a116483 1979 Von, 2001, Stepped care for back pain, Ann Intern Med, 134, 911, 10.7326/0003-4819-134-9_Part_2-200105011-00016 Means, 1989 Sandha, 1999, A systematic overview of the use of diary cards, quality-of-life questionnaires, and psychometric tests in treatment trials of Helicobacter pylori-positive and -negative non-ulcer dyspepsia, Scand J Gastroenterol, 34, 244, 10.1080/00365529950173636 Stone, 2002, Patient non-compliance with paper diaries, BMJ, 324, 1193, 10.1136/bmj.324.7347.1193 Harding, 1997, Use of a novel electronic data collection system in multicenter studies of irritable bowel syndrome, Aliment Pharmacol Ther, 11, 1073, 10.1046/j.1365-2036.1997.00256.x Bardhan, 2000, A double-blind, randomized, placebo-controlled dose-ranging study to evaluate the efficacy of alosetron in the treatment of irritable bowel syndrome, Aliment Pharmacol Ther, 14, 23, 10.1046/j.1365-2036.2000.00684.x Payne, 1995, A controlled comparison of cognitive therapy and self-help support groups in the treatment of irritable bowel syndrome, J Consult Clin Psychol, 63, 779, 10.1037/0022-006X.63.5.779 Whitehead WE, Palsson OS, Levy RL, Feld AD, Von Korff M, Turner M. Reports of “satisfactory relief” by IBS patients receiving usual medical care are confounded by baseline symptom severity and do not accurately reflect symptom improvement. Am J Gastroenterol (In press). Camilleri, 1999, Improvement in pain and bowel function in female irritable bowel patients with alosetron, a 5-HT3 receptor antagonist, Aliment Pharmacol Ther, 13, 1149, 10.1046/j.1365-2036.1999.00610.x Camilleri, 2001, A randomized controlled clinical trial of the serotonin type 3 receptor antagonist alosetron in women with diarrhea-predominant irritable bowel syndrome, Arch Intern Med, 161, 1733, 10.1001/archinte.161.14.1733 Chey, 2004, Long-term safety and efficacy of alosetron in women with severe diarrhea-predominant irritable bowel syndrome, Am J Gastroenterol, 99, 2195, 10.1111/j.1572-0241.2004.30509.x Kellow, 2003, An Asia-Pacific, double blind, placebo controlled, randomised study to evaluate the efficacy, safety, and tolerability of tegaserod in patients with irritable bowel syndrome, Gut, 52, 671, 10.1136/gut.52.5.671 Nyhlin, 2004, A double-blind, placebo-controlled, randomized study to evaluate the efficacy, safety and tolerability of tegaserod in patients with irritable bowel syndrome, Scand J Gastroenterol, 39, 119, 10.1080/00365520310006748 Wyrwich, 2004, A blueprint for symptom scales and responses, Gut, 53, iv45, 10.1136/gut.2003.034348 Fallone, 2004, Do physicians correctly assess patient symptom severity in gastro-oesophageal reflux disease?, Aliment Pharmacol Ther, 20, 1161, 10.1111/j.1365-2036.2004.02257.x Guyatt, 1993, Measuring health-related quality of life, Ann Intern Med, 118, 622, 10.7326/0003-4819-118-8-199304150-00009 Mangel, 1998, Adequate relief as an endpoint in clinical trials in irritable bowel syndrome, J Int Med Res, 26, 76, 10.1177/030006059802600203 Dunger-Baldauf, 2003, Subject’s global assessment of satisfactory relief as a measure to assess treatment effect in clinical trials in irritable bowel syndrome (IBS), Am J Gastroenterol, 98, S269, 10.1016/S0002-9270(03)01581-8 Blanchard, 1992, Prediction of outcome from cognitive-behavioral treatment of irritable bowel syndrome, Behav Res Ther, 30, 647, 10.1016/0005-7967(92)90011-5 Wiklund, 1998, Quality of Life in Reflux and Dyspepsia patients. Psychometric documentation of a new disease-specific questionnaire (QOLRAD), Eur J Surg Suppl, 583, 41 Drossman, 1995, Functional bowel disorders. A multicenter comparison of health status and development of illness severity index, Dig Dis Sci, 40, 986, 10.1007/BF02064187 Francis, 1997, The irritable bowel severity scoring system, Aliment Pharmacol Ther, 11, 395, 10.1046/j.1365-2036.1997.142318000.x Gonsalkorale, 2003, Long term benefits of hypnotherapy for irritable bowel syndrome, Gut, 52, 1623, 10.1136/gut.52.11.1623 Whitehead, 2004, Usual medical care for irritable bowel syndrome, Aliment Pharmacol Ther, 20, 1305, 10.1111/j.1365-2036.2004.02256.x Corazziari, 2003, Clinical trial guidelines for pharmacological treatment of irritable bowel syndrome, Aliment Pharmacol Ther, 18, 569, 10.1046/j.1365-2036.2003.01709.x Twisk, 2003 Snijders, 1999 Fraser, 2005, Symptom-based outcome measures for dyspepsia and GERD trials, Am J Gastroenterol, 100, 442, 10.1111/j.1572-0241.2005.40122.x El-Omar, 1996, The Glasgow Dyspepsia Severity Score—a tool for the global measurement of dyspepsia, Eur J Gastroenterol Hepatol, 8, 967, 10.1097/00042737-199610000-00006 Veldhuyzen van Zanten, 1993, Can severity of symptoms be used as an outcome measure in trials of non-ulcer dyspepsia and Helicobacter pylori associated gastritis?, J Clin Epidemiol, 46, 273, 10.1016/0895-4356(93)90075-C Moayyedi, 1998, The Leeds Dyspepsia Questionnaire, Aliment Pharmacol Ther, 12, 1257, 10.1046/j.1365-2036.1998.00404.x Rabeneck, 2002, Reliability, validity, and responsiveness of severity of dyspepsia assessment (SODA) in a randomized clinical trial of a COX-2-specific inhibitor and traditional NSAID therapy, Am J Gastroenterol, 97, 32, 10.1111/j.1572-0241.2002.05419.x Talley, 1999, Validity of a new quality of life scale for functional dyspepsia, Am J Gastroenterol, 94, 2390, 10.1111/j.1572-0241.1999.01363.x Guyatt, 1987, Measuring change over time, J Chronic Dis, 40, 171, 10.1016/0021-9681(87)90069-5 Ioannidis, 2004, Better reporting of harms in randomized trials, Ann Intern Med, 141, 781, 10.7326/0003-4819-141-10-200411160-00009 Creed, 2003, The cost-effectiveness of psychotherapy and paroxetine for severe irritable bowel syndrome, Gastroenterology, 124, 303, 10.1053/gast.2003.50055 Calvert, 2002, Long-term improvement in functional dyspepsia using hypnotherapy, Gastroenterology, 123, 1778, 10.1053/gast.2002.37071 Whitehead, 1996, Impact of irritable bowel syndrome on quality of life, Dig Dis Sci, 41, 2248, 10.1007/BF02071408 Talley, 1995, Impact of functional dyspepsia on quality of life, Dig Dis Sci, 40, 584, 10.1007/BF02064375 Bergner, 1981, The Sickness Impact Profile, Med Care, 19, 787, 10.1097/00005650-198108000-00001 Stewart, 1988, The MOS short-form general health survey. Reliability and validity in a patient population, Med Care, 26, 724, 10.1097/00005650-198807000-00007 Dimenas, 1995, Well-being and gastrointestinal symptoms among patients referred to endoscopy owing to suspected duodenal ulcer, Scand J Gastroenterol, 30, 1046, 10.3109/00365529509101605 Patrick, 1998, Quality of life in persons with irritable bowel syndrome, Dig Dis Sci, 43, 400, 10.1023/A:1018831127942 Borgaonkar, 2000, Quality of life measurement in gastrointestinal and liver disorders, Gut, 47, 444, 10.1136/gut.47.3.444 Watson, 2001, Alosetron improves quality of life in women with diarrhea-predominant irritable bowel syndrome, Am J Gastroenterol, 96, 455, 10.1111/j.1572-0241.2001.03525.x Moher, 2001, The CONSORT statement, JAMA, 285, 1987, 10.1001/jama.285.15.1987 Bossuyt, 2003, Towards complete and accurate reporting of studies of diagnostic accuracy, BMJ, 326, 41, 10.1136/bmj.326.7379.41 Moher, 1999, Improving the quality of reports of meta-analyses of randomised controlled trials: the QUOROM statement. Quality of Reporting of Meta-analyses, Lancet, 354, 1896, 10.1016/S0140-6736(99)04149-5 Perneger, 1998, What’s wrong with Bonferroni adjustments, BMJ, 316, 1236, 10.1136/bmj.316.7139.1236 Guyatt, 1995, Basic statistics for clinicians: 2. Interpreting study results: confidence intervals, CMAJ, 152, 169 Nuovo, 2002, Reporting number needed to treat and absolute risk reduction in randomized controlled trials, JAMA, 287, 2813, 10.1001/jama.287.21.2813 Gore, 1992, The Lancet’s statistical review process, Lancet, 340, 100, 10.1016/0140-6736(92)90409-V Katz, 2003, Multivariable analysis, Ann Intern Med, 138, 644, 10.7326/0003-4819-138-8-200304150-00012 Altman, 2001, The revised CONSORT statement for reporting randomized trials, Ann Intern Med, 134, 663, 10.7326/0003-4819-134-8-200104170-00012 DeMets, 1999, The agonising negative trend in monitoring of clinical trials, Lancet, 354, 1983, 10.1016/S0140-6736(99)03464-9 Campbell, 1995, Estimating sample sizes for binary, ordered categorical, and continuous outcomes in two group comparisons, BMJ, 311, 1145, 10.1136/bmj.311.7013.1145 De Angelis, 2004, Clinical trial registration, N Engl J Med, 351, 1250, 10.1056/NEJMe048225 Bero, 1995, Preparing, maintaining, and disseminating systematic reviews of the effects of health care, JAMA, 274, 1935, 10.1001/jama.274.24.1935