Differences in Outcomes Reported by Patients With Inflammatory Bowel Diseases vs Their Health Care Professionals.

BACKGROUND & AIMS: Inflammatory bowel disease (IBD) scoring systems combine patient-reported data with physicians' observations to determine patient outcomes, but these systems are believed to have limitations. We used real-world data from a large IBD cohort in Switzerland to compare results between patients and healthcare professionals' from scoring systems for Crohn's disease (CD) and ulcerative colitis (UC). METHODS: We collected data from the Swiss IBD cohort, beginning in 2006, using 2453 reports for 1385 patients (52% female, 58% with CD). During office visits, physicians asked patients about signs and symptoms and recorded their answers (health care professional-reported outcomes). On a later date, patients received a questionnaire at home (independently of the medical visit), complete it, and sent it back to the data center. Patients also completed the short form 36 and IBD quality of life (QoL) questionnaires. We calculated Cohen's kappa (κ) statistics to assess the level of agreement in scores between patients and health care professionals (Δt between reports collected less than 2 months apart). We used Spearman correlation coefficients (r) to compare general well-being (GWB) and QoL scores determined by patients vs health care professionals. Our primary aim was to investigate the overall and individual level of agreement on signs and symptoms reported by health care professionals vs patients. RESULTS: The best level of agreement (although moderate) was observed for number of stools last week in patients with CD (κ=0.47), and nocturnal diarrhea in patients with UC (κ=0.52). Agreement was low on level of abdominal pain (κ=0.31 for patients with CD and κ=0.37 for patients with UC) and GWB (κ=0.23 for patients with CD and κ=0.26 for patients with UC). Patients reported less severe abdominal pain and worse GWB (CD) or better GWB (UC) than that determined by health care professionals. Patient self-rated GWB correlated with IBD quality of life (r=0.68 for patients with CD and r=0.70 for patients with UC) and SF-36 physical scores (r=0.55 for patients with CD and r=0.60 for patients with UC); there was no correlation between health care professional-rated GWB and QoL. CONCLUSION: In a comparison of patient vs health care provider-reported outcomes in a Swiss IBD cohort, we found that health care professionals seem to misinterpret patients' complaints. Patients self-rated GWB correlated with QoL scores, indicating that reporting GWB in a single question is possible and relevant, but can vary based on how the data are collected

Differences in Outcomes Reported by Patients With Inflammatory Bowel Diseases vs Their Health Care Professionals

BACKGROUND & AIMS Inflammatory bowel disease (IBD) scoring systems combine patient-reported data with physicians' observations to determine patient outcomes, but these systems are believed to have limitations. We used real-world data from a large IBD cohort in Switzerland to compare results between patients and healthcare professionals from scoring systems for Crohn's disease (CD) and ulcerative colitis (UC). METHODS We collected data from the Swiss IBD cohort, beginning in 2006, using 2453 reports for 1385 patients (52% female, 58% with CD). During office visits, physicians asked patients about signs and symptoms and recorded their answers (health care professional-reported outcomes). On a later date, patients received a questionnaire at home (independently of the medical visit), complete it, and sent it back to the data center. Patients also completed the short form 36 and IBD quality of life (QoL) questionnaires. We calculated Cohen's kappa (κ) statistics to assess the level of agreement in scores between patients and health care professionals (Δt between reports collected less than 2 months apart). We used Spearman correlation coefficients (ρ) to compare general well-being (GWB) and QoL scores determined by patients vs health care professionals. Our primary aim was to investigate the overall and individual level of agreement on signs and symptoms reported by health care professionals vs patients. RESULTS The best level of agreement (although moderate) was observed for number of stools last week in patients with CD (κ = 0.47), and nocturnal diarrhea in patients with UC (κ = 0.52). Agreement was low on level of abdominal pain (κ = 0.31 for patients with CD and κ = 0.37 for patients with UC) and GWB (κ = 0.23 for patients with CD and κ = 0.26 for patients with UC). Patients reported less severe abdominal pain and worse GWB (CD) or better GWB (UC) than that determined by health care professionals. Patient self-rated GWB correlated with IBD quality of life (ρ = 0.68 for patients with CD and ρ = 0.70 for patients with UC) and SF-36 physical scores (ρ = 0.55 for patients with CD and ρ = 0.60 for patients with UC); there was no correlation between health care professional-rated GWB and QoL. CONCLUSIONS In a comparison of patient vs health care provider-reported outcomes in a Swiss IBD cohort, we found that health care professionals seem to misinterpret patients' complaints. Patients self-rated GWB correlated with QoL scores, indicating that reporting GWB in a single question is possible and relevant, but can vary based on how the data are collected

The Evolution of Health Care Utilisation and Costs for Inflammatory Bowel Disease Over Ten Years.

Inflammatory bowel disease [IBD] places an economic strain on health systems due to expensive pharmaceutical therapy, risk of hospitalisation and surgery, and long-term monitoring. The evolving treatment guidelines advocate rapid scale-up to biologic agents in order to improve health outcomes and quality of life. This study evaluated changes in health care utilisation and expenditures for IBD in Switzerland over time. We extracted clinical, patient, and resource consumption data from the Swiss IBD Cohort Study between 2006 and 2016. Average unit costs for IBD-related events were derived from Swiss claims data and pharmaceutical price lists. We used multivariate regression, controlling for patient-level characteristics, to estimate trends and determinants of direct and indirect costs and resource utilisation. We included 2365 adults diagnosed with Crohn's disease [CD; N = 1353] and ulcerative colitis [UC; N = 1012]. From 2006-16, mean health care expenditures per patient per year were 9504 euros [70% drugs, 23% inpatient, 7% outpatient] for CD and 5704 euros [68% drugs, 22% inpatient, 10% outpatient] for UC. Health care costs increased by 7% [CD] and 10% [UC] per year, largely due to rising pharmaceutical expenditures driven by increased biologic agent use. Inpatient, outpatient, and indirect costs fluctuated and did not offset increased pharmaceutical costs. Disease characteristics were important predictors of costs. Increased expenditure for IBD was marked by a shift towards greater pharmaceutical management over the past decade. This study highlights the need to identify cost-effective treatment strategies in the face of increased uptake and expenditures associated with innovative treatments

Evaluating the cost-effectiveness of early compared to late or no biologic treatment to manage Crohn's disease using real world data

We evaluated the cost-effectiveness of early (≤2 years after diagnosis) compared to late or no biologic initiation (starting biologics >2 years after diagnosis or no biologic use) for adults with Crohn's disease in Switzerland.; We developed a Markov cohort model over the patient's lifetime from the health system and societal perspectives. Transition probabilities, quality of life, and costs were estimated using real world data. Propensity score matching was used to ensure comparability between patients in the early (intervention) and late/no (comparator) biologic initiation strategies. The incremental cost-effectiveness ratio (ICER) per quality-adjusted life year (QALY) gained is reported in Swiss francs (CHF). Sensitivity and scenario analyses were performed.; Total costs and QALYs were higher for the intervention (CHF384,607; 16.84 QALYs) compared to comparator (CHF340,800; 16.75 QALYs) strategy, resulting in high ICERs (health system: CHF887,450 per QALY; societal: CHF449,130 per QALY). Assuming a threshold of CHF100,000 per QALY, in probabilistic sensitivity analysis the intervention strategy had a 0.1 and 0.25 probability of being cost-effective from the health system and societal perspectives, respectively. In addition, ICERs improved when we assumed a 30% reduction in biologic prices (health system: CHF134,502 per QALY; societal: intervention dominant).; Early biologic use was not cost-effective considering a threshold of CHF100,000 per QALY compared to late/no biologic use. However, early identification of patients likely to need biologics and future drug price reductions through increased availability of biosimilars may improve the cost-effectiveness of an early treatment approach

The cost of inflammatory bowel disease in high-income settings: a Lancet Gastroenterology & Hepatology Commission

The cost of caring for patients with inflammatory bowel disease (IBD) continues to increase worldwide. The cause is not only a steady increase in the prevalence of Crohn's disease and ulcerative colitis in both developed and newly industrialised countries, but also the chronic nature of the diseases, the need for long-term, often expensive treatments, the use of more intensive disease monitoring strategies, and the effect of the diseases on economic productivity. This Commission draws together a wide range of expertise to discuss the current costs of IBD care, the drivers of increasing costs, and how to deliver affordable care for IBD in the future. The key conclusions are that (1) increases in health-care costs must be evaluated against improved disease management and reductions in indirect costs, and (2) that overarching systems for data interoperability, registries, and big data approaches must be established for continuous assessment of effectiveness, costs, and the cost-effectiveness of care. International collaborations should be sought out to evaluate novel models of care (eg, value-based health care, including integrated health care, and participatory health-care models), as well as to improve the education and training of clinicians, patients, and policy makers

Somatic mosaicism and common genetic variation contribute to the risk of very-early-onset inflammatory bowel disease

Very-early-onset inflammatory bowel disease (VEO-IBD) is a heterogeneous phenotype associated with a spectrum of rare Mendelian disorders. Here, we perform whole-exome-sequencing and genome-wide genotyping in 145 patients (median age-at-diagnosis of 3.5 years), in whom no Mendelian disorders were clinically suspected. In five patients we detect a primary immunodeficiency or enteropathy, with clinical consequences (XIAP, CYBA, SH2D1A, PCSK1). We also present a case study of a VEO-IBD patient with a mosaic de novo, pathogenic allele in CYBB. The mutation is present in ~70% of phagocytes and sufficient to result in defective bacterial handling but not life-threatening infections. Finally, we show that VEO-IBD patients have, on average, higher IBD polygenic risk scores than population controls (99 patients and 18,780 controls; P < 4 × 10-10), and replicate this finding in an independent cohort of VEO-IBD cases and controls (117 patients and 2,603 controls; P < 5 × 10-10). This discovery indicates that a polygenic component operates in VEO-IBD pathogenesis