Cost-utility analysis of Dexcom G6 real-time continuous glucose monitoring versus FreeStyle Libre 1 intermittently scanned continuous glucose monitoring in adults with type 1 diabetes in Belgium.

AIMS/HYPOTHESIS: The aim of this study was to assess the long-term cost-effectiveness of Dexcom G6 real-time continuous glucose monitoring (rtCGM) with alert functionality compared with FreeStyle Libre 1 intermittently scanned continuous glucose monitoring (isCGM) without alerts in adults with type 1 diabetes in Belgium. METHODS: The IQVIA CORE Diabetes Model was used to estimate cost-effectiveness. Input data for the simulated baseline cohort were sourced from the randomised ALERTT1 trial (ClinicalTrials.gov. REGISTRATION NO: NCT03772600). The age of the participants was 42.9 ± 14.1 years (mean ± SD), and the baseline HbA1c was 57.8 ± 9.5 mmol/mol (7.4 ± 0.9%). Participants using rtCGM showed a reduction in HbA1c of 3.6 mmol/mol (0.36 percentage points) based on the 6-month mean between-group difference. In the base case, both rtCGM and isCGM were priced at €3.92/day (excluding value-added tax [VAT]) according to the Belgian reimbursement system. The analysis was performed from a Belgian healthcare payer perspective over a lifetime time horizon. Health outcomes were expressed as quality-adjusted life years. Probabilistic and one-way sensitivity analyses were used to account for parameter uncertainty. RESULTS: In the base case, rtCGM dominated isCGM, resulting in lower diabetes-related complication costs and better health outcomes. The associated main drivers favouring rtCGM were lower HbA1c, fewer severe hypoglycaemic events and reduced fear of hypoglycaemia. The results were robust under a wide range of one-way sensitivity analyses. In models where the price of rtCGM is €5.11/day (a price increase of 30.4%) or €12.34/day (a price increase of 214.8%), rtCGM was cost-neutral or reached an incremental cost-effectiveness ratio of €40,000 per quality-adjusted life year, respectively. CONCLUSIONS/INTERPRETATION: When priced similarly, Dexcom G6 rtCGM with alert functionality has both economic and clinical benefits compared with FreeStyle Libre 1 isCGM without alerts in adults with type 1 diabetes in Belgium, and appears to be a cost-effective glucose monitoring modality. Trial registration ClinicalTrials.gov NCT03772600.

Cost-utility of real-time continuous glucose monitoring versus self-monitoring of blood glucose in people with insulin-treated Type 2 diabetes in Canada.

Aim: Clinical trials and real-world data for Type 2 diabetes have shown that real-time continuous glucose monitoring (rt-CGM) lowers glycated hemoglobin (A1c) and reduces hypoglycemia relative to self-monitoring of blood glucose (SMBG). This analysis examined the long-term health and economic outcomes associated with using rt-CGM versus SMBG in people with insulin-treated Type 2 diabetes in Canada. Materials & methods: Clinical data were sourced from a real-world study, in which rt-CGM reduced A1C by 0.56% versus continued SMBG. The analysis was performed using the IQVIA Core Diabetes Model, from a Canadian payer perspective over a lifetime horizon for a cohort aged 65 years with an A1C of 8.3% at baseline. Future costs and clinical outcomes were discounted at 1.5% annually. Results: Projected total mean lifetime costs were CAD 207,466 for rt-CGM versus CAD 189,863 for SMBG (difference: CAD 17,602) and projected mean quality-adjusted life expectancy was 9.97 quality-adjusted life years (QALYs) for rt-CGM versus 9.02 QALYs for SMBG (difference: 0.95 QALYs), resulting in an incremental cost-utility ratio (ICUR) of CAD 18,523 per QALY gained for rt-CGM versus SMBG. Findings were sensitive to changes in the A1C treatment effect, annual cost and quality of life benefit associated with using rt-CGM, SMBG frequency, and baseline age, but ICURs remained below CAD 50,000 per QALY in all analyses. Conclusion: For people in Canada with insulin-treated Type 2 diabetes and poor glycemic control, use of rt-CGM is likely to be cost-effective relative to SMBG.

Cost-Effectiveness of a Real-Time Continuous Glucose Monitoring System Versus Self-Monitoring of Blood Glucose in People with Type 2 Diabetes on Insulin Therapy in the UK.

INTRODUCTION: Real-time continuous glucose monitoring (rt-CGM) involves the measurement and display of glucose concentrations, potentially improving glucose control among insulin-treated patients with type 2 diabetes (T2D). The present analysis aimed to conduct a cost-effectiveness analysis of rt-CGM versus self-monitoring of blood glucose (SMBG) based on a USA retrospective cohort study in insulin-treated people with T2D adapted to the UK. METHODS: Long-term costs and clinical outcomes were estimated using the CORE Diabetes Model, with clinical input data sourced from a retrospective cohort study. Patients were assumed to have a baseline glycated hemoglobin (HbA1c) of 8.3%. Patients using rt-CGM were assumed to have a 0.56% reduction in HbA1c based on the mean difference between groups after 12 months of follow-up. Reduced fingerstick testing when using rt-CGM was associated with a quality of life (QoL) benefit. The analysis was performed over a lifetime time horizon from a National Health Service (NHS) perspective, including only direct costs from published data. Future costs and clinical outcomes were discounted at 3.5% per annum. Extensive sensitivity analyses were performed. RESULTS: Projections showed that rt-CGM was associated with increased quality-adjusted life expectancy of 0.731 quality-adjusted life years (QALYs) and increased mean total lifetime costs of Great British pounds (GBP) 2694, and an incremental cost-effectiveness ratio of GBP 3684 per QALY compared with SMBG. Key drivers of outcomes included HbA1c reduction and reduced fingerstick testing QoL benefit. CONCLUSIONS: Over patient lifetimes, rt-CGM was associated with improved clinical outcomes and is highly likely to be cost effective versus SMBG in people with T2D on insulin therapy in the UK.

Evaluation of the Long-Term Cost-Effectiveness of the Dexcom G6 Continuous Glucose Monitor versus Self-Monitoring of Blood Glucose in People with Type 1 Diabetes in Canada.

BACKGROUND: The Dexcom G6 real-time continuous glucose monitoring (RT-CGM) system is one of the most sophisticated RT-CGM systems developed to date and became available in Canada in 2019. A health economic analysis was performed to determine the long-term cost-effectiveness of the Dexcom G6 RT-CGM system versus SMBG in adults with type 1 diabetes (T1D) in Canada. METHODS: The analysis was performed using the IQVIA Core Diabetes Model. Based on clinical trial data, patients with mean baseline HbA1c of 8.6% were assumed to have a HbA1c reduction of 1.0% with RT-CGM versus 0.4% reduction with SMBG. RT-CGM was also associated with a quality of life (QoL) benefit owing to reduced incidence of hypoglycemia, reduced fear of hypoglycemia (FoH) and elimination of fingerstick testing. Direct medical costs were sourced from published literature, and inflated to 2019 Canadian dollars (CAD). RESULTS: Dexcom G6 RT-CGM was projected to improve mean quality-adjusted life expectancy by 2.09 quality-adjusted life years (QALYs) relative to SMBG (15.52 versus 13.43 QALYs) but mean total lifetime cots were CAD 35,353 higher with RT-CGM (CAD 227,357 versus CAD 192,004) resulting in an incremental cost-effectiveness ratio (ICER) of CAD 16,931 per QALY gained. Sensitivity analyses revealed that assumptions relating to the QoL benefit associated with reduced FoH and the elimination of fingerstick testing with RT-CGM as well as SMBG usage and change in HbA1c were the key drivers of cost-effectiveness. CONCLUSION: For adults with T1D in Canada, RT-CGM is associated with improved glycemic control and QoL benefits owing to a reduced FoH and elimination of the requirement for fingerstick testing and over a lifetime time horizon is cost-effective relative to SMBG.

Long-Term Cost-Effectiveness the Dexcom G6 Real-Time Continuous Glucose Monitoring System Compared with Self-Monitoring of Blood Glucose in People with Type 1 Diabetes in France.

INTRODUCTION: The aim was to determine the long-term cost-effectiveness of the Dexcom G6 real-time continuous glucose monitoring (RT-CGM) system versus self-monitoring of blood glucose (SMBG) in adults with type 1 diabetes (T1D) in France. METHODS: The analysis was performed using the IQVIA Core Diabetes Model and utilized clinical input data from the DIAMOND clinical trial in adults with T1D. Simulated patients were assumed to have a mean baseline HbA1c of 8.6%, and those in the RT-CGM arm were assumed to have a HbA1c reduction of 1.0% compared with 0.4% in the SMBG arm. A quality of life (QoL) benefit associated with a reduced fear of hypoglycemia (FoH) and elimination of the requirement for fingerstick testing in the RT-CGM arm was also applied. RESULTS: The G6 RT-CGM system was associated with an incremental gain in quality-adjusted life expectancy of 1.38 quality-adjusted life years (QALYs) compared with SMBG (10.64 QALYs versus 9.23 QALYs). Total mean lifetime costs were 21,087 euros higher with RT-CGM (148,077 euros versus 126,990 euros), resulting in an incremental cost-effectiveness ratio of 15,285 euros per QALY gained. CONCLUSIONS: In France, based on a willingness-to-pay threshold of 50,000 euros per QALY gained, the use of the G6 RT-CGM system is cost-effective relative to SMBG for adults with long-standing T1D, driven primarily by improved glycemic control and the QoL benefit associated with reduced FoH and elimination of the requirement for fingerstick testing.

Long-term Cost-Effectiveness of Dexcom G6 Real-time Continuous Glucose Monitoring Versus Self-Monitoring of Blood Glucose in Patients With Type 1 Diabetes in the U.K.

OBJECTIVE: A long-term health economic analysis was performed to establish the cost-effectiveness of real-time continuous glucose monitoring (RT-CGM) (Dexcom G6) versus self-monitoring of blood glucose (SMBG) alone in U.K.-based patients with type 1 diabetes (T1D). RESEARCH DESIGN AND METHODS: The analysis used the IQVIA CORE Diabetes Model. Clinical input data were sourced from the DIAMOND trial in adults with T1D. Simulations were performed separately in the overall population of patients with baseline HbA1c ≥7.5% (58 mmol/mol), and a secondary analysis was performed in patients with baseline HbA1c ≥8.5% (69 mmol/mol). The analysis was performed from the National Health Service health care payer perspective over a lifetime time horizon. RESULTS: In the overall population, G6 RT-CGM was associated with a mean incremental gain in quality-adjusted life expectancy of 1.49 quality-adjusted life years (QALYs) versus SMBG (mean [SD] 11.47 [2.04] QALYs versus 9.99 [1.84] QALYs). Total mean (SD) lifetime costs were also pounds sterling (GBP) 14,234 higher with RT-CGM (GBP 102,468 [35,681] versus GBP 88,234 [39,027]) resulting in an incremental cost-effectiveness ratio of GBP 9,558 per QALY gained. Sensitivity analyses revealed that the findings were sensitive to changes in the quality-of-life benefit associated with reduced fear of hypoglycemia and avoidance of fingerstick testing as well as the HbA1c benefit associated with RT-CGM use. CONCLUSIONS: For U.K.-based T1D patients, the G6 RT-CGM device is associated with significant improvements in clinical outcomes and, over patient lifetimes, is a cost-effective disease management option relative to SMBG on the basis of a willingness-to-pay threshold of GBP 20,000 per QALY gained.

Use of Two Self-referral Reminders and a Theory-Based Leaflet to Increase the Uptake of Flexible Sigmoidoscopy in the English Bowel Scope Screening Program: Results From a Randomized Controlled Trial in London.

Background: We previously initiated a randomized controlled trial to test the effectiveness of two self-referral reminders and a theory-based leaflet (sent 12 and 24 months after the initial invitation) to increase participation within the English Bowel Scope Screening program. Purpose: This study reports the results following the second reminder. Methods: Men and women included in the initial sample (n = 1,383) were re-assessed for eligibility 24 months after their invitation (12 months after the first reminder) and excluded if they had attended screening, moved away, or died. Eligible adults received the same treatment they were allocated 12 months previous, that is, no reminder ("control"), or a self-referral reminder with either the standard information booklet ("Reminder and Standard Information Booklet") or theory-based leaflet designed using the Behavior Change Wheel ("Reminder and Theory-Based Leaflet"). The primary outcome was the proportion screened within each group 12 weeks after the second reminder. Results: In total, 1,218 (88.1%) individuals were eligible. Additional uptake following the second reminder was 0.4% (2/460), 4.8% (19/399), and 7.9% (29/366) in the control, Reminder and Standard Information Booklet, and Reminder and Theory-Based Leaflet groups, respectively. When combined with the first reminder, the overall uptake for each group was 0.7% (3/461), 14.5% (67/461), and 21.5% (99/461). Overall uptake was significantly higher in the Reminder and Standard Information Booklet and Reminder and Theory-Based Leaflet groups than in the control (odds ratio [OR] = 26.1, 95% confidence interval [CI] = 8.1-84.0, p < .001 and OR = 46.9, 95% CI = 14.7-149.9, p < .001, respectively), and significantly higher in the Reminder and Theory-Based Leaflet group than in the Reminder and Standard Information Booklet group (OR = 1.8, 95% CI = 1.3-2.6, p < .001). Conclusion: A second reminder increased uptake among former nonparticipants. The added value of the theory-based leaflet highlights a potential benefit to reviewing the current information booklet. Trials Registry Number: ISRCTN44293755.

Improving uptake of flexible sigmoidoscopy screening: a randomized trial of nonparticipant reminders in the English Screening Programme.

Background and study aims Uptake of flexible sigmoidoscopy screening in the English Bowel Scope Screening (BSS) Programme is low. The aim of this study was to test the impact of a nonparticipant reminder and theory-based leaflet to promote uptake among former nonresponders (previously did not confirm their appointment) and nonattenders (previously confirmed their appointment but did not attend). Patients and methods Eligible adults were men and women in London who had not attended a BSS appointment within 12 months of their invitation. Individuals were randomized (1:1:1) to receive no reminder (control), a 12-month reminder plus standard information booklet (TMR-SIB), or a 12-month reminder plus bespoke theory-based leaflet (TMR-TBL) designed to address barriers to screening. The primary outcome of the study was the proportion of individuals screened within each group 12 weeks after the delivery of the reminder. Results A total of 1383 men and women were randomized and analyzed as allocated (n = 461 per trial arm). Uptake was 0.2 % (n = 1), 10.4 % (n = 48), and 15.2 % (n = 70) in the control, TMR-SIB, and TMR-TBL groups, respectively. Individuals in the TMR-SIB and TMR-TBL groups were significantly more likely to attend screening than individuals in the control group (adjusted odds ratio [OR] 53.7, 95 % confidence interval [CI] 7.4 - 391.4, P < 0.001 and OR 89.0, 95 %CIs 12.3 - 645.4, P < 0.01, respectively). Individuals in the TMR-TBL group were also significantly more likely to attend screening than individuals in the TMR-SIB group (OR 1.7, 95 %CIs 1.1 - 2.5, P = 0.01). Across all groups, former nonattenders were more likely to participate in screening than former nonresponders (uptake was 14.2 % and 8.0 %, respectively; OR 2.5, 95 %CIs 1.4 - 4.4, P < 0.01). The adenoma detection rate among screened adults was 7.6 %, which is comparable to the rate in initial attenders. Conclusions Reminders targeting former nonparticipants can improve uptake and are effective for both former nonresponders and nonattenders. Theory-based information designed to target barriers to screening added significantly to this strategy.

Health-related quality of life in acute schizophrenia patients treated with RBP-7000 once monthly risperidone: An 8-week, randomized, double-blind, placebo-controlled, multicenter phase 3 study.

BACKGROUND: There is increased interest in the impact of new long-acting treatments on health-related quality of life (HRQoL) in patients with schizophrenia. The aim of this study was to evaluate the impact of treatment with subcutaneous injections of RBP-7000, a new sustained-release formulation of risperidone, compared with placebo on health status, subjective well-being, treatment satisfaction, and preference of medicine in subjects with acute schizophrenia. METHODS: HRQoL data were derived from an 8-week double-blind, randomized, placebo-controlled, phase 3 study that assessed efficacy, safety, and tolerability of once monthly RBP-7000 (90mg and 120mg) compared with placebo in subjects with acute schizophrenia (n=337). HRQoL was measured with the EuroQol EQ-5D-5L, well-being using the Neuroleptic Treatment-Short Version (SWN-S), satisfaction using the Medication Satisfaction Questionnaire (MSQ), and preference using the Preference of Medicine Questionnaire (POM). RESULTS: The EQ-5D-5L VAS increased significantly in the RBP-7000 120mg group compared to Placebo (p=0.0212). In RBP-7000 120mg, subjects reported significant improvements in SWN-S physical functioning (p=0.0093), social integration (p=0.0368), and total score (p=0.0395). Subjects were significantly more satisfied with RBP-7000 versus placebo (90mg p=0.0009, 120mg p=0.0006) and preferred RBP-7000 over their previous medication (90mg p<0.0001, 120mg p=0.0619). CONCLUSIONS: Significantly greater improvements in HRQoL and overall well-being were demonstrated in patients randomized to RBP-7000 compared to placebo. The effect was more pronounced in the RBP-7000 120mg group. Patient satisfaction improved significantly and patient preference for their medicine favored RBP-7000 90mg and 120mg versus Placebo.

The Lung Screen Uptake Trial (LSUT): protocol for a randomised controlled demonstration lung cancer screening pilot testing a targeted invitation strategy for high risk and 'hard-to-reach' patients.

BACKGROUND: Participation in low-dose CT (LDCT) lung cancer screening offered in the trial context has been poor, especially among smokers from socioeconomically deprived backgrounds; a group for whom the risk-benefit ratio is improved due to their high risk of lung cancer. Attracting high risk participants is essential to the success and equity of any future screening programme. This study will investigate whether the observed low and biased uptake of screening can be improved using a targeted invitation strategy. METHODS/DESIGN: A randomised controlled trial design will be used to test whether targeted invitation materials are effective at improving engagement with an offer of lung cancer screening for high risk candidates. Two thousand patients aged 60-75 and recorded as a smoker within the last five years by their GP, will be identified from primary care records and individually randomised to receive either intervention invitation materials (which take a targeted, stepped and low burden approach to information provision prior to the appointment) or control invitation materials. The primary outcome is uptake of a nurse-led 'lung health check' hospital appointment, during which patients will be offered a spirometry test, an exhaled carbon monoxide (CO) reading, and an LDCT if eligible. Initial data on demographics (i.e. age, sex, ethnicity, deprivation score) and smoking status will be collected in primary care and analysed to explore differences between attenders and non-attenders with respect to invitation group. Those who attend the lung health check will have further data on smoking collected during their appointment (including pack-year history, nicotine dependence and confidence to quit). Secondary outcomes will include willingness to be screened, uptake of LDCT and measures of informed decision-making to ensure the latter is not compromised by either invitation strategy. DISCUSSION: If effective at improving informed uptake of screening and reducing bias in participation, this invitation strategy could be adopted by local screening pilots or a national programme. TRIAL REGISTRATION: This study was registered with the ISRCTN (International Standard Registered Clinical/soCial sTudy Number: ISRCTN21774741) on the 23rd September 2015 and the NIH ClinicalTrials.gov database (NCT0255810) on the 22nd September 2015.

Use of a 12 months' self-referral reminder to facilitate uptake of bowel scope (flexible sigmoidoscopy) screening in previous non-responders: a London-based feasibility study.

BACKGROUND: In March 2013, NHS England extended its national Bowel Cancer Screening Programme to include 'one-off' Flexible Sigmoidoscopy screening (NHS Bowel Scope Screening, BSS) for men and women aged 55. With less than one in two people currently taking up the screening test offer, there is a strong public health mandate to develop system-friendly interventions to increase uptake while the programme is rolling out. This study aimed to assess the feasibility of sending a reminder to previous BSS non-responders, 12 months after the initial invitation, with consideration for its potential impact on uptake. METHOD: This study was conducted in the ethnically diverse London Boroughs of Brent and Harrow, where uptake is below the national average. Between September and November 2014, 160 previous non-responders were randomly selected to receive a reminder of the opportunity to self-refer 12 months after their initial invitation. The reminder included instructions on how to book an appointment, and provided options for the time and day of the appointment and the gender of the endoscopist performing the test. To address barriers to screening, the reminder was sent with a brief locally tailored information leaflet designed specifically for this study. Participants not responding within 4 weeks were sent a follow-up reminder, after which there was no further intervention. Self-referral rates were measured 8 weeks after the delivery of the follow-up reminder and accepted as final. RESULTS: Of the 155 participants who received the 12 months' reminder (returned to sender, n=5), 30 (19.4%) self-referred for an appointment, of which 24 (15.5%) attended and were successfully screened. Attendance rates differed by gender, with significantly more women attending an appointment than men (20.7% vs 8.8%, respectively; OR=2.73, 95% CI=1.02-7.35, P=0.05), but not by area (Brent vs Harrow) or area-level deprivation. Of the 30 people who self-referred for an appointment, 27 (90%) indicated a preference for a same-sex practitioner, whereas three (10%) gave no preference. Preference for a same-sex practitioner was higher among women than men (χ(2)=7.78, P<0.05), with only 67% of men (six of nine) requesting a same-sex practitioner, compared with 100% of women (n=21). CONCLUSIONS: Sending previous non-responders a 12 months' reminder letter with a brief information leaflet is a feasible and efficacious intervention, which merits further investigation in a randomised controlled trial.

Hemoglobin, exercise training, and health status in patients with chronic heart failure (from the HF-ACTION randomized controlled trial).

Anemia is common in patients with chronic heart failure (HF), with a prevalence ranging from 10% to 56%, and may be a risk factor for poor outcomes. Anemia in HF remains poorly understood, with significant gaps in its impact on health-related quality of life (HRQoL), with most studies in HF being retrospective or from registries. The purpose of this study was to explore the relation of hemoglobin (Hgb) with HRQoL and training-induced changes in HRQoL in a cohort of patients in Heart Failure: A Controlled Trial Investigating Outcomes of Exercise Training (HF-ACTION). Using data from HF-ACTION, a randomized controlled trial of exercise training in patients with HF and low left ventricular ejection fractions, HRQoL was measured using the Kansas City Cardiomyopathy Questionnaire (KCCQ) at baseline, 3 and 12 months, and annually up to 4 years. Treatment group effects on HRQoL were estimated using linear mixed models according to the intention-to-treat principle. It was hypothesized that baseline Hgb would be correlated with baseline KCCQ scales and that Hgb would moderate the beneficial effect of exercise training on HRQoL. Hgb level was not significantly correlated with baseline HRQoL. Baseline Hgb did not moderate the beneficial effect of exercise training on KCCQ overall or subscales relative to usual care. In conclusion, in the HF-ACTION cohort, there was no correlation with baseline Hgb and baseline HRQoL as measured by the KCCQ. In addition, the beneficial effects of HRQoL from exercise training were not modulated by baseline Hgb.

Low hemoglobin levels and recurrent falls in U.S. men and women: prospective findings from the REasons for Geographic And Racial Differences in Stroke (REGARDS) cohort.

BACKGROUND: There are few data available on low hemoglobin and incident falls in the general U.S. population. METHODS: Of 30,239 black and white U.S. adults ≥45 years in the population-based REasons for Geographic And Racial Differences in Stroke study, 16,782 had hemoglobin measured at baseline and follow-up data on falls. Hemoglobin was categorized by 1.0 g/dL increments relative to the World Health Organization anemia threshold (<13.0 g/dL for men, <12.0 g/dL for women). Recurrent falls (≥2 falls in the 6 months after baseline) were assessed during a telephone interview. RESULTS: Recurrent falls occurred in 3.9% of men and 4.8% of women. Compared with those with a hemoglobin level 1 to 2 g/dL above the anemia cut-off, multivariable adjusted odds ratios (95% confidence intervals) for recurrent falls associated with hemoglobin levels ≥3, 2 to <3 and 0 to 1 g/dL above the cut-off point, and 0 to <1 and ≥1 g/dL below the cut-off point were 0.73 (0.45-1.19), 0.84 (0.57-1.24), 1.29 (0.88-1.90), 1.32 (0.0.80-1.2.18) and 2.12 (1.23-3.63), respectively, among men (linear trend P < 0.001), and 1.59 (1.10-2.3), 1.24 (0.95-1.62), 1.42(1.11-1.81), 1.28 (0.91-1.80) and 1.76 (1.13-2.74), respectively, among women (linear trend P = 0.45; quadratic trend P = 0.016). CONCLUSIONS: Among men, lower hemoglobin levels were associated with an increased risk for recurrent falls. Although our findings suggest an increased risk for recurrent falls at both lower and higher hemoglobin levels among women, these findings should be confirmed in subsequent studies.

Associations between hemoglobin level, resource use, and medical costs in patients with heart failure: findings from HF-ACTION.

BACKGROUND: Reports that patients with heart failure and anemia incur greater costs and medical resource use have relied largely on data with limited clinical detail. METHODS: HF-ACTION, a large trial of exercise training in heart failure, recorded hemoglobin at baseline. Medical resource use and hospital bills for inpatient and emergency department visits were collected throughout the study. We analyzed hemoglobin as a continuous variable to evaluate relationships with medical resource use and costs over 1 year. RESULTS: Among 1,763 patients with baseline hemoglobin levels, those with lower hemoglobin levels tended to be older, African American, and women and to have more severe heart failure. Lower hemoglobin was significantly associated with more hospital admissions, inpatient days, outpatient visits, and urgent care or emergency department visits (all P < .005, unadjusted). Although cost outliers influenced estimates, these observations were distributed across hemoglobin levels. Mean 1-year costs across hemoglobin levels defined as ≤ 11, >11-12, >12-13, >13-14, >14-15, and >15 g/dL were $21,106, $20,189, $16,249, $17,989, $13,216, and $12,492, respectively (P < .001, unadjusted). Significant associations remained after multivariable adjustment. CONCLUSIONS: Patients with lower baseline hemoglobin levels experienced progressively greater resource use and higher costs.

Analysis of EQ-5D scores from two phase 3 clinical trials of romiplostim in the treatment of immune thrombocytopenia (ITP).

OBJECTIVE: Utility-based measures of quality of life are recommended by some decision makers in health care. No studies have addressed changes in utility in patients with chronic idiopathic thrombocytopenia (ITP). This study sought to assess the health utilities of patients with chronic ITP and to evaluate the association of change in health utility with changes in other variables, including platelet response status and bleeding events. METHODS: We analyzed EQ-5D data from two international, randomized, placebo-controlled, double-blind, 24-week trials that evaluated the efficacy and safety of romiplostim in adult patients with chronic ITP. RESULTS: A total of 125 subjects participated. Mean change, adjusted for age, gender, splenectomy status, and baseline score using multiple linear regression models was greater for romiplostim versus placebo for the EQ-5D index score (0.05 vs. -0.03, P = 0.015) and the VAS score (6.42 vs. 0.48, P = 0.066); similar for durable platelet responders versus nonresponders; and greater for EQ-5D index scores for subjects who did not have a bleeding event during the study (n = 29) compared to subjects who did have a bleeding event (n = 47) (0.06 vs. 0.005; P = 0.066). CONCLUSIONS: Using romiplostim in cases of chronic ITP was associated with improvement in health-related quality of life as measured by the EQ-5D index score. Health utility scores derived using the EQ-5D in this study may be useful for determining quality-adjusted life years (QALYs) in economic evaluations of romiplostim for treatment of chronic ITP.

Measurement of utility values in the UK for health states related to immune thrombocytopenic purpura.

OBJECTIVE: To measure utility values associated with immune (idiopathic) thrombocytopenic purpura (ITP), as perceived by the United Kingdom (UK) general public. RESEARCH DESIGN AND METHODS: A multi-step process, including clinical trial data, literature review, and patient focus group, was used to develop ITP health states valued in a web survey. Six ITP health states were defined based on platelet levels, risk of bleeding and key adverse events/disease complications. Clinical trial data on bleeding and ITP-specific quality of life data were key sources for developing health-state descriptions. 359 respondents, randomly selected from a managed web panel in the UK, completed the web-based Time Trade-Off survey. Wilcoxon signed-rank test was used to compare differences between each pair of health states. RESULTS: Sample characteristics (mean age: 47.9 +/- 16.9 years; 54% female) were comparable to the UK general population. ITP health states were valued as significantly worse than perfect health. Experiencing bleeding episodes was a more important driver than low platelet levels in valuing a health state to be worse. Substantial disutilities were associated with surviving an intracranial haemorrhage. Mean (SD) utility values for each ITP health state are: HS1: platelets >or=50 x 10(9)/L, no outpatient bleed: 0.863 +/- 0.15; HS2: platelets >or=50 x 10(9)/L, outpatient bleed: 0.734 +/- 0.19; HS3: platelets <50 x 10(9)/L, no outpatient bleed: 0.841 +/- 0.19; HS4: platelets <50 x 10(9)/L, outpatient bleed: 0.732 +/- 0.19; HS5: intracranial haemorrhage (2-6 months): 0.038 +/- 0.46; HS6: steroid treatment adverse events: 0.758 +/- 0.20. Potential limitations relate to web user population characteristics and lack of comparative testing of web-based TTO methods. CONCLUSIONS: Results provide evidence that the UK general population associate substantial loss of value living with ITP, suggesting an important role for new ITP treatments. Utility values based on these health states may be useful in future cost-effectiveness studies of existing and/or new ITP treatments.

Impact of ITP on physician visits and workplace productivity.

OBJECTIVE: To assess the impact of immune thrombocytopenic purpura (ITP) on primary care and specialist visits and workplace productivity. RESEARCH DESIGN AND METHODS: This was a cross-sectional, descriptive study comparing ITP patients to age- and gender-matched controls. Subjects completed a one-time web-based survey, which included questions on work loss, work productivity, and physician visits. ITP patients and controls were compared on these outcomes. For ITP patients, the relationship between work-related issues and physician visits with clinical characteristics (time since diagnosis, platelet count, number of treatments received, and an ITP-specific health-related quality of life measure, the ITP-PAQ) was explored. RESULTS: A total of 1002 ITP patients and 1031 controls completed the survey. On average, ITP patients were 46 years old, diagnosed 9 years ago, and had platelet counts of 148 x 10(9)/L; 37% had undergone splenectomy. More ITP patients had primary care (20% vs. 11%) and specialist (28% vs. 11%) visits in the past month versus controls (p < or = 0.001). Higher proportions of ITP patients have ever taken sick leave (56% vs. 30%), and missed chore hours in the past week (18% vs. 13%) (p < or = 0.003). ITP patients scored significantly worse than controls on all six work productivity items. Patients diagnosed within the past year were more likely to have specialist visits and to miss chore hours versus those diagnosed less recently. Worse ITP-PAQ scores and more ITP-related treatments were related to more physician visits and worse work-related and productivity outcomes. Platelet count is not associated with these outcomes. The study is limited by the potential for biased samples due to recruitment approaches, the inherent issues of a cross-sectional study design and recall bias in questionnaire responses. CONCLUSIONS: ITP was consistently associated with more physician visits and worse work and productivity outcomes. Future research should build on these findings by calculating a comprehensive cost-of-illness of ITP including both direct and indirect costs.

Mucositis-related morbidity and resource utilization in head and neck cancer patients receiving radiation therapy with or without chemotherapy.

The objective of this study was to estimate health care-resource utilization in head and neck cancer (HNC) patients. This was a prospective, longitudinal, multicenter, noninterventional study of mucositis in patients receiving radiation with or without chemotherapy for HNC. Mouth and throat soreness and functional impairment were measured using the Oral Mucositis Weekly Questionnaire-HNC. Resource utilization data were obtained from patient interviews and recorded from the patient's medical chart. Seventy-five patients were enrolled from six centers. Fifty (67%) patients received concurrent chemoradiation therapy; 34 (45%) received intensity-modulated radiation therapy. Over the course of treatment, 57 (76%) patients reported severe mouth and throat soreness. Pain and functional impairment because of mouth and throat soreness increased during the course of therapy despite the use of opioid analgesics in 64 (85%) of the patients. Complications of radiation therapy resulted in increased patient visits to physicians, nurses, and nutritionists. Thirty-eight (51%) patients had a feeding tube placed. Twenty-eight patients (37%) were hospitalized, five of whom were hospitalized twice; of the 33 admissions, 10 (30%) were designated as secondary to mucositis by their treating physician. Mean length of hospitalization was 4.9 days (range: 1-16). This study demonstrates that mucositis-related pain and functional impairment is associated with increased use of costly health resources. Effective treatments to reduce the pain and functional impairment of oral mucositis are needed in this patient population.

Health-related quality of life of immune thrombocytopenic purpura patients: results from a web-based survey.

OBJECTIVE: To assess the health-related quality of life (HRQOL) of immune thrombocytopenic purpura (ITP) patients. RESEARCH DESIGN AND METHODS: This was a cross-sectional, descriptive study comparing ITP patients' HRQOL to age and gender matched controls. ITP patients from the Platelet Disorder Support Association were recruited until 1000 surveys had been completed. Controls were randomly sampled from the Harris Interactive Online Panel. ITP patients and controls completed a one-time web-based survey, including a comprehensive HRQOL assessment. ITP patients completed the SF-36, the EQ-5D, and the ITP-Patient Assessment Questionnaire (ITP-PAQ). Controls completed the SF-36 and EQ-5D only. ITP patients' SF-36 and EQ-5D scores were compared to controls in unadjusted and adjusted analyses. Associations between splenectomy status, duration of illness, and platelet count with ITP patients' HRQOL scores were also examined. RESULTS: This analysis included 1002 ITP patients and 1031 controls. ITP patients scored worse on seven of eight SF-36 domains and the Physical and Mental Summary scores (all p < 0.05) and on the EQ-5D visual analog scale (65.5 vs. 82.3; p = 0.002). ITP patients who had undergone splenectomy had similar SF-36 and EQ-5D scores to non-splenectomy patients but scored significantly worse on 5 of 10 ITP-PAQ scales: Bother, Psychological, Fear, Social Activity, and Work (all p < 0.05). ITP patients diagnosed within the past 5 years had worse Bother and Overall Quality of Life scores than less recently diagnosed patients but were similar on other ITP-PAQ scales. Lower platelet count was consistently associated with worse ITP-PAQ scores and had weaker associations with SF-36 and EQ-5D scores. CONCLUSIONS: ITP was associated with consistent and statistically significant deficits on generic HRQOL measures. The ITP-PAQ demonstrated differences based on disease severity and treatments. The self-selection bias in the two samples limits the generalizability of the results to all patients with ITP. Further research is needed in more generalizable samples.