The relationship among multiple patient-reported outcomes measures for patients with ulcerative colitis receiving treatment with MMX ® formulated delayed-release mesalamine

Aaron Yarlas, Linnette Yen, Paul Hodgkins, Aaron Yarlas, Linnette Yen, Paul Hodgkins

Abstract

Purpose: Ulcerative colitis (UC) is associated with impaired health-related quality of life (HRQL) and work-related outcomes (WRO). This analysis examined correspondences among measures of HRQL and WRO in patients with UC, as well as the magnitude of each measure's responsiveness to disease activity and treatment.

Methods: An open-label, prospective trial of delayed-release mesalamine tablets formulated with MMX(®) technology included 8 weeks of treatment for patients with active mild-to-moderate UC (n = 137) and 12 months of maintenance treatment for patients with quiescent UC (n = 206). Spearman correlations (ρ) measured inter-domain associations across measures of generic HRQL [12-item Short-Form Health Survey (SF-12v2)], disease-specific HRQL [Short Inflammatory Bowel Disease Questionnaire (SIBDQ)], and disease-specific WRO [Work Productivity and Activity Impairment for Specific Health Problems (WPAI:SHP)]. Responsiveness to disease activity and treatment was assessed for each instrument.

Results: Changes in scores from baseline to week 8 were moderately correlated across all instrument domains: 65 of 80 (81 %) between-instrument inter-domain correlations were of moderate magnitude (0.30 < ρ < 0.70), with an average magnitude of 0.42 [95 % confidence interval (CI) 0.38-0.46]. Associations between symptom measures were stronger for SIBDQ (|average ρ| = 0.41; 95 % CI 0.34-0.48) and WPAI:SHP (0.40; 0.30-0.47) than SF-12v2 (0.30; 0.27-0.34). SIBDQ was most sensitive to treatment [effect size (d z ) for change from baseline to week 8 = 0.62; 95 % CI 0.35-0.89], followed by WPAI:SHP (d z = 0.43; 0.32-0.54) and SF-12v2 (d z = 0.33; 0.27-0.39).

Conclusion: While the SIBDQ showed the greatest overall responsiveness to disease activity and treatment, all three patient-reported outcomes instruments provided complementary interpretive information regarding the impact of UC treatment.

Trial registration: ClinicalTrials.gov NCT00446849.

Figures

Fig. 1
Fig. 1
Flowchart of study design. UC ulcerative colitis, MMX Multi Matrix System, PRO patient-reported outcome

References

    1. Bernklev T, Jahnsen J, Aadland E, Sauar J, Schulz T, Lygren I, et al. Health-related quality of life in patients with inflammatory bowel disease five years after the initial diagnosis. Scandinavian Journal of Gastroenterology. 2004;39:365–373. doi: 10.1080/00365520310008386.
    1. Han SW, McColl E, Barton JR, James P, Steen IN, Welfare MR. Predictors of quality of life in ulcerative colitis: The importance of symptoms and illness representations. Inflammatory Bowel Diseases. 2005;11:24–34. doi: 10.1097/00054725-200501000-00004.
    1. Janke KH, Klump B, Gregor M, Meisner C, Haeuser W. Determinants of life satisfaction in inflammatory bowel disease. Inflammatory Bowel Diseases. 2005;11:272–286. doi: 10.1097/01.MIB.0000160809.38611.f7.
    1. Larsson K, Loof L, Ronnblom A, Nordin K. Quality of life for patients with exacerbation in inflammatory bowel disease and how they cope with disease activity. Journal of Psychosomatic Research. 2008;64:139–148. doi: 10.1016/j.jpsychores.2007.10.007.
    1. Lix LM, Graff LA, Walker JR, Clara I, Rawsthorne P, Rogala L, et al. Longitudinal study of quality of life and psychological functioning for active, fluctuating, and inactive disease patterns in inflammatory bowel disease. Inflammatory Bowel Diseases. 2008;14:1575–1584. doi: 10.1002/ibd.20511.
    1. Poole CD, Connolly MP, Nielsen SK, Currie CJ, Marteau P. A comparison of physician-rated disease severity and patient reported outcomes in mild to moderately active ulcerative colitis. Journal of Crohn’s and Colitis. 2010;4:275–282. doi: 10.1016/j.crohns.2009.11.010.
    1. Zahn A, Hinz U, Karner M, Ehehalt R, Stremmel W. Health-related quality of life correlates with clinical and endoscopic activity indexes but not with demographic features in patients with ulcerative colitis. Inflammatory Bowel Diseases. 2006;12:1058–1067. doi: 10.1097/01.mib.0000234134.35713.d2.
    1. Bernklev T, Jahnsen J, Lygren I, Henriksen M, Vatn M, Moum B. Health-related quality of life in patients with inflammatory bowel disease measured with the Short Form-36: Psychometric assessments and a comparison with general population norms. Inflammatory Bowel Diseases. 2005;11:909–918. doi: 10.1097/01.mib.0000179467.01748.99.
    1. Boonen A, Dagnelie PC, Feleus A, Hesselink MA, Muris JW, Stockbrugger RW, et al. The impact of inflammatory bowel disease on labor force participation: Results of a population sampled case–control study. Inflammatory Bowel Diseases. 2002;8:382–389. doi: 10.1097/00054725-200211000-00002.
    1. Cohen BL, Zoega H, Shah SA, Leleiko N, Lidofsky S, Bright R, et al. Fatigue is highly associated with poor health-related quality of life, disability and depression in newly-diagnosed patients with inflammatory bowel disease, independent of disease activity. Alimentary Pharmacology & Therapeutics. 2014;39:811–822. doi: 10.1111/apt.12659.
    1. Gibson PR, Vaizey C, Black CM, Nicholls R, Weston AR, Bampton P, et al. Relationship between disease severity and quality of life and assessment of health care utilization and cost for ulcerative colitis in Australia: A cross-sectional, observational study. Journal of Crohn’s and Colitis. 2014;8:598–606. doi: 10.1016/j.crohns.2013.11.017.
    1. Gibson TB, Ng E, Ozminkowski RJ, Wang S, Burton WN, Goetzel RZ, et al. The direct and indirect cost burden of Crohn’s disease and ulcerative colitis. Journal of Occupational and Environmental Medicine. 2008;50:1261–1272. doi: 10.1097/JOM.0b013e318181b8ca.
    1. Hoivik ML, Moum B, Solberg IC, Henriksen M, Cvancarova M, Bernklev T. Work disability in inflammatory bowel disease patients 10 years after disease onset: Results from the IBSEN Study. Gut. 2013;62:368–375. doi: 10.1136/gutjnl-2012-302311.
    1. Neovius M, Arkema EV, Blomqvist P, Ekbom A, Smedby KE. Patients with ulcerative colitis miss more days of work than the general population, even following colectomy. Gastroenterology. 2013;144:536–543. doi: 10.1053/j.gastro.2012.12.004.
    1. Siebert U, Wurm J, Gothe RM, Arvandi M, Vavricka SR, von Känel R, et al. Predictors of temporary and permanent work disability in patients with inflammatory bowel disease: Results of the swiss inflammatory bowel disease cohort study. Inflammatory Bowel Diseases. 2013;19:847–855. doi: 10.1097/MIB.0b013e31827f278e.
    1. van der Valk ME, Mangen MJ, Leenders M, Dijkstra G, van Bodegraven AA, Fidder HH, et al. Risk factors of work disability in patients with inflammatory bowel disease—A Dutch nationwide web-based survey: Work disability in inflammatory bowel disease. Journal of Crohn’s and Colitis. 2014;8:580–597.
    1. Connolly MP, Poole CD, Currie CJ, Marteau P, Nielsen SK. Quality of life improvements attributed to combination therapy with oral and topical mesalazine in mild-to-moderately active ulcerative colitis. Digestion. 2009;80:241–246. doi: 10.1159/000235916.
    1. Feagan BG, Reinisch W, Rutgeerts P, Sandborn WJ, Yan S, Eisenberg D, et al. The effects of infliximab therapy on health-related quality of life in ulcerative colitis patients. The American Journal of Gastroenterology. 2007;102:794–802. doi: 10.1111/j.1572-0241.2007.01094.x.
    1. Hodgkins P, Yen L, Yarlas A, Karlstadt R, Solomon D, Kane S. The impact of MMX mesalamine on improvement and maintenance of health-related quality of life in patients with ulcerative colitis. Inflammatory Bowel Diseases. 2013;19:386–396. doi: 10.1002/ibd.23022.
    1. Irvine EJ, Yeh CH, Ramsey D, Stirling AL, Higgins PD. The effect of mesalazine therapy on quality of life in patients with mildly and moderately active ulcerative colitis. Alimentary Pharmacology & Therapeutics. 2008;28:1278–1286. doi: 10.1111/j.1365-2036.2008.03854.x.
    1. Reinisch W, Sandborn WJ, Rutgeerts P, Feagan BG, Rachmilewitz D, Hanauer SB, et al. Long-term infliximab maintenance therapy for ulcerative colitis: The ACT-1 and -2 extension studies. Inflammatory Bowel Diseases. 2012;18:201–211. doi: 10.1002/ibd.21697.
    1. Solomon D, Yarlas A, Hodgkins P, Karlstadt R, Yen L, Kane S. The impact of MMX mesalazine on disease-specific health-related quality of life in ulcerative colitis patients. Alimentary Pharmacology & Therapeutics. 2012;35:1386–1396. doi: 10.1111/j.1365-2036.2012.05107.x.
    1. Reinisch W, Sandborn WJ, Bala M, Yan S, Feagan BG, Rutgeerts P, et al. Response and remission are associated with improved quality of life, employment and disability status, hours worked, and productivity of patients with ulcerative colitis. Inflammatory Bowel Diseases. 2007;13:1135–1140. doi: 10.1002/ibd.20165.
    1. Langhorst J, Mueller T, Luedtke R, Franken U, Paul A, Michalsen A, et al. Effects of a comprehensive lifestyle modification program on quality-of-life in patients with ulcerative colitis: A twelve-month follow-up. Scandinavian Journal of Gastroenterology. 2007;42:734–745. doi: 10.1080/00365520601101682.
    1. McColl E, Han SW, Barton JR, Welfare MR. A comparison of the discriminatory power of the Inflammatory Bowel Disease Questionnaire and the SF-36 in people with ulcerative colitis. Quality of Life Research. 2004;13:805–811. doi: 10.1023/B:QURE.0000021701.28467.57.
    1. Bernklev T, Jahnsen J, Henriksen M, Lygren I, Aadland E, Sauar J, et al. Relationship between sick leave, unemployment, disability, and health-related quality of life in patients with inflammatory bowel disease. Inflammatory Bowel Diseases. 2006;12:402–412. doi: 10.1097/01.MIB.0000218762.61217.4a.
    1. Kane S, Katz S, Jamal MM, Safdi M, Dolin B, Solomon D, et al. Strategies in Maintenance for Patients Receiving Long-Term Therapy (SIMPLE): A study of MMX® mesalamine for the long-term maintenance of quiescent ulcerative colitis. Inflammatory Bowel Diseases. 2012;18:1026–1033. doi: 10.1002/ibd.21841.
    1. Maruish M. User’s Manual for the SF-12v2 Health Survey. 3. Lincoln, RI: QualityMetric, Incorporated; 2012.
    1. Irvine EJ, Zhou Q, Thompson AK. The Short Inflammatory Bowel Disease Questionnaire: A quality of life instrument for community physicians managing inflammatory bowel disease. CCRPT Investigators. Canadian Crohn’s Relapse Prevention Trial. The American Journal of Gastroenterology. 1996;91:1571–1578.
    1. Han SW, Gregory W, Nylander D, Tanner A, Trewby P, Barton R, et al. The SIBDQ: Further validation in ulcerative colitis patients. The American Journal of Gastroenterology. 2000;95:145–151. doi: 10.1111/j.1572-0241.2000.01676.x.
    1. Jowett SL, Seal CJ, Barton JR, Welfare MR. The short inflammatory bowel disease questionnaire is reliable and responsive to clinically important change in ulcerative colitis. The American Journal of Gastroenterology. 2001;96:2921–2928. doi: 10.1111/j.1572-0241.2001.04682.x.
    1. Lam MY, Lee H, Bright R, Korzenik JR, Sands BE. Validation of interactive voice response system administration of the Short Inflammatory Bowel Disease Questionnaire. Inflammatory Bowel Diseases. 2009;15:599–607. doi: 10.1002/ibd.20803.
    1. Reilly MC, Zbrozek AS, Dukes EM. The validity and reproducibility of a work productivity and activity impairment instrument. PharmacoEconomics. 1993;4:353–365. doi: 10.2165/00019053-199304050-00006.
    1. Wahlqvist P, Carlsson J, Stalhammar NO, Wiklund I. Validity of a Work Productivity and Activity Impairment questionnaire for patients with symptoms of gastro-esophageal reflux disease (WPAI-GERD)—results from a cross-sectional study. Value in Health. 2002;5:106–113. doi: 10.1046/j.1524-4733.2002.52101.x.
    1. Wahlqvist P, Guyatt GH, Armstrong D, Degl’innocenti A, Heels-Ansdell D, El-Dika S, et al. The Work Productivity and Activity Impairment Questionnaire for Patients with Gastroesophageal Reflux Disease (WPAI-GERD): Responsiveness to change and English language validation. PharmacoEconomics. 2007;25:385–396. doi: 10.2165/00019053-200725050-00003.
    1. Reilly M, Gerlier L, Brabant Y, Brown M. Validity, reliability, and responsiveness of the work productivity and activity impairment questionnaire in Crohn’s disease. Clinical Therapeutics. 2008;30:393–404. doi: 10.1016/j.clinthera.2008.02.016.
    1. Reilly MC, Bracco A, Ricci JF, Santoro J, Stevens T. The validity and accuracy of the Work Productivity and Activity Impairment questionnaire—Irritable bowel syndrome version (WPAI:IBS) Alimentary Pharmacology & Therapeutics. 2004;20:459–467. doi: 10.1111/j.1365-2036.2004.02091.x.
    1. Sutherland LR, Martin F, Greer S, Robinson M, Greenberger N, Saibil F, et al. 5-Aminosalicylic acid enema in the treatment of distal ulcerative colitis, proctosigmoiditis, and proctitis. Gastroenterology. 1987;92:1894–1898.
    1. Schroeder KW, Tremaine WJ, Ilstrup DM. Coated oral 5-aminosalicylic acid therapy for mildly to moderately active ulcerative colitis. A randomized study. The New England Journal of Medicine. 1987;317:1625–1629. doi: 10.1056/NEJM198712243172603.
    1. Lewis JD, Chuai S, Nessel L, Lichtenstein GR, Aberra FN, Ellenberg JH. Use of the noninvasive components of the Mayo score to assess clinical response in ulcerative colitis. Inflammatory Bowel Diseases. 2008;14:1660–1666. doi: 10.1002/ibd.20520.
    1. Fisher RA. Frequency distribution of the values of the correlation coefficient in samples of an indefinitely large population. Biometrika. 1915;10:507–521.
    1. Corey DM, Dunlap WP, Burke MJ. Averaging correlations: Expected values and bias in combined Pearson rs and Fisher’s z transformations. The Journal of General Psychology. 1998;125:245–261. doi: 10.1080/00221309809595548.
    1. Silver NC, Dunlap WP. Averaging correlation coefficients: Should Fisher’s z transformation be used? Journal of Applied Psychology. 1987;72:146–148. doi: 10.1037/0021-9010.72.1.146.
    1. Fisher RA. On the “probable error” of a coefficient of correlation deduced from a small sample. Metron. 1921;1:3–32.
    1. Cohen J. Statistical power analysis for the behavioral sciences. Hillsdale, NJ: L. Erlbaum Associates; 1988.

Source: PubMed

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