Patient Commitment to Health (PACT-Health) in the Heart Failure Population: A Focus Group Study of an Active Communication Framework for Patient-Centered Health Behavior Change.

BACKGROUND: Over 6 million Americans have heart failure, and 1 in 8 deaths included heart failure as a contributing cause in 2016. Lifestyle changes and adherence to diet and exercise regimens are important in limiting disease progression. Health coaching and public commitment are two interactive communication strategies that may improve self-management of heart failure. OBJECTIVE: This study aimed to conduct patient focus groups to gain insight into how best to implement health coaching and public commitment strategies within the heart failure population. METHODS: Focus groups were conducted in two locations. We studied 2 patients in Oakland, California, and 5 patients in Los Angeles, California. Patients were referred by local cardiologists and had to have a diagnosis of chronic heart failure. We used a semistructured interview tool to explore several patient-centered themes including medication adherence, exercise habits, dietary habits, goals, accountability, and rewards. We coded focus group data using the a priori coding criteria for these domains. RESULTS: Medication adherence barriers included regimen complexity, forgetfulness, and difficulty coping with side effects. Participants reported that they receive little instruction from care providers on appropriate exercise and dietary habits. They also reported personal and social obstacles to achieving these objectives. Participants were in favor of structured goal setting, use of online social networks, and financial rewards as a means of promoting health lifestyles. Peers were viewed as better motivating agents than family members. CONCLUSIONS: An active communication framework involving dissemination of diet- and exercise-related health information, structured goal setting, peer accountability, and financial rewards appears promising in the management of heart failure.

Quality of Care for Patients with Type 2 Diabetes Mellitus in Dubai: A HEDIS-Like Assessment.

Objective. As little data are available on the quality of type 2 diabetes mellitus (T2DM) care in the Arabian Gulf States, we estimated the proportion of patients receiving recommended monitoring at the Dubai Hospital for T2DM over one year. Methods. Charts from 150 adults with T2DM were systematically sampled and quality of care was assessed during one calendar year, using a Healthcare Effectiveness Data and Information Set- (HEDIS-) like assessment. Screening for glycosylated haemoglobin (HbA1c), low-density lipoprotein (LDL), blood pressure, retinopathy, and nephropathy was considered. Patients were classified based on their most recent test in the period, and predictors of receiving quality care were examined. Results. Mean age was 58 years (standard deviation (SD): 12.4 years) and 33% were males. Over the year, 98% underwent HbA1c screening (50% had control and 28% displayed poor control); 91% underwent LDL screening (65% had control); 55% had blood pressure control; 30% had retinopathy screening; and 22% received attention for nephropathy. No individual characteristics examined predicted receiving quality care. Conclusion. Some guideline monitoring was conducted for most patients; and rates of monitoring for selected measures were comparable to benchmarks from the United States. Greater understanding of factors leading to high adherence would be useful for other areas of preventive care and other jurisdictions.

Value-based insurance design: benefits beyond cost and utilization.

As value-based insurance design (VBID) programs proliferate, evidence is emerging on the impact of VBID. To date, studies have largely measured VBID impact on utilization, and a few studies have assessed its impact on quality, outcomes, and cost. In this commentary we discuss these domains, summarize evidence, and propose the extension of measurement of VBID impact into areas including workplace productivity and quality of life, employee and patient engagement, and talent attraction and retention. We contend that VBID evaluations should consider a broad variety of programmatic dividends on both humanistic and health-related outcomes.

Patterns of Care and Treatment Target Success among Persons with Type 2 Diabetes Mellitus in Dubai: A Retrospective Cohort Study.

OBJECTIVES: Despite the high prevalence of type 2 diabetes mellitus (T2DM), few data exist describing its management in Dubai. This study characterized the treatment and estimated levels of glycemic, lipid, and blood pressure control among a sample with T2DM at a large Dubai Hospital. METHODS: This retrospective cohort study systematically sampled charts from adults seeking care for T2DM from October 2009 to March 2010 until the target (N = 250) was reached. Data on patient characteristics, pharmacotherapy, complications, and laboratory testing were abstracted until September 2011. The frequency of treatments and modifications over the period was calculated, and measures of glycosylated hemoglobin A1c, low-density lipoprotein, and blood pressure control were compared with guideline targets. Frequencies of complications were compared according to treatment type. RESULTS: One-third of the cohort comprised men, and the mean age was 58 years. At enrolment, the mean time from T2DM diagnosis was nearly 15 years and 74% had received insulin. During the study period, the most common regimens were insulin + oral combinations (55%) and oral combination therapy (39%). Overall, 67% received any insulin therapy during the study; and by study end, 78% had received insulin at any time. At the most recent assessment, guideline targets for glycosylated hemoglobin A1c, blood pressure, and low-density lipoprotein were met by 23%, 29%, and 71%, respectively. Complications were more frequent among those treated with combination or insulin therapies. CONCLUSIONS: This study provides baseline data from Dubai for future comparisons of the effectiveness of new treatments, and to better understand the humanistic and economic burden of T2DM and its complications.

Value of survival gains in chronic myeloid leukemia.

UNLABELLED: Although clinical trial data have quantified patient survival gains associated with tyrosine kinase inhibitors in chronic myeloid leukemia, the overall value of these benefits is unknown. OBJECTIVE: To estimate the total value of survival gains associated with first- and second-line TKI therapy in chronic myeloid leukemia (CML) and the fraction of tyrosine kinase inhibitor (TKI)- related survival-gain value retained by patients and drug companies. STUDY DESIGN: This retrospective study identified CML patient data from the Surveillance, Epidemiology and End Results registry, dasatinib clinical trials, and insurance claims data sets. METHODS: Multivariate Cox proportional hazard models were used to estimate improvements in CML survival associated with the introduction of first-line imatinib therapy. Survival gains associated with second-line dasatinib treatment were identified via retrospective analyses and published clinical outcomes. An economic model was developed to calculate the social value of survival gains derived from first- and second-line TKI treatment. TKI costs were used to estimate the fraction of survival gain value retained by patients and drug companies. RESULTS: The introduction of TKIs in 2001 was associated with a hazard ratio of 0.833 (P <.01). Cost analyses indicate that the TKI drug class in CML therapy has created more than $143 billion in social value. Approximately 90% of this value is retained by patients and society, while approximately 10% is recouped by drug companies. CONCLUSIONS: These estimates indicate that the introduction of TKI drugs to treat CML has generated significant social value as a result of survival gains, the vast majority of which has accrued to patients.

Coverage and use of cancer therapies in the treatment of chronic myeloid leukemia.

OBJECTIVES: This study was designed to assess the effect of tyrosine kinase inhibitor (TKI) use on nonpharmaceutical medical spending for patients with chronic myeloid leukemia (CML), and estimate the association between cost-sharing and the TKI medication possession ratio (MPR). STUDY DESIGN: The retrospective study covered the 13 years from 1997 to 2009. METHODS: Analyses were conducted using a large administrative health insurance claims database covering 45 large employers. From this database, 995 unique patients with CML were identified, with 3,765 patient-years; of these patients, 415 (or 1,689 patientyears) were TKI users. We estimated the association of TKI use with total pharmaceutical spending and total non-pharmaceutical medical spending. In addition, we characterized plan-level cost-sharing rules for TKIs and assessed whether these were associated with the MPR for TKI therapy among CML patients. RESULTS: TKI users averaged $26,406 in annual non-pharmaceutical medical spending, compared with $38,194 for non-users; this was a difference of approximately 30%, which was statistically significant at the 5% level. The median patient out-ofpocket payment was $25, which increased to $63 at the 75th percentile and to $122 at the 95th percentile. MPRs were 94.8 at the median cost-sharing level and 100.0 at the 75th percentile and higher. There was no statistically significant association between cost-sharing and MPR. CONCLUSIONS: Use of TKIs was associated with a 30% reduction in non-pharmaceutical medical spending for CML patients. This difference is approximately equal to 40% of the incremental pharmaceutical cost associated with using TKI therapy. The net annual cost of TKI therapy is roughly $15,000. An informal calculation suggests that this is well within the range of conventional cost-effectiveness thresholds. On balance, coverage of TKIs is relatively generous, with the vast majority of patients exhibiting high levels of adherence to therapy.

Proposed severity and response criteria for Routine Assessment of Patient Index Data (RAPID3): results for categories of disease activity and response criteria in abatacept clinical trials.

BACKGROUND: An index is needed to assess the status of patients with rheumatoid arthritis (RA), as none of the existing measures are applicable to all individual patients. The 28-joint Disease Activity Score (DAS28) is the most specific and widely used index. Routine Assessment of Patient Index Data (RAPID3) is an index containing only the 3 patient self-report core dataset measures, without a laboratory test or formal joint count, and with simple scoring. RAPID3 is correlated significantly with DAS28, but calculated in 5-10 seconds on a Multidimensional Health Assessment Questionnaire (MDHAQ), compared to 114 seconds for DAS28. METHODS: DAS28 (0-10 scale) categories for high, moderate, and low activity, and remission (≤ 2.6, 2.6-3.2, 3.21-5.1, and > 5.1, respectively) and proposed RAPID3 (0-30 scale) categories for severity (0 ≤ 3, 3.1-6, 6.1-12, and > 12) were compared in patients taking abatacept and control-treated patients at the endpoint of the Abatacept in Inadequate Response to Methotrexate (AIM) and the Abatacept Trial in Treatment of Anti-TNF INadequate Responders (ATTAIN) clinical trials, using cross-tabulations and kappa statistics. RESULTS: Overall, 92%-99% of patients classified as having high DAS28 activity had high or moderate RAPID3 severity, while 64%-83% in DAS28 remission had RAPID3 low severity or remission; 50%-82% of patients with good or poor EULAR responses had good or poor RAPID3 responses. Kappa values ranged from 0.25 to 0.48, and weighted kappas from 0.32 to 0.52, indicating fair to moderate agreement for the 2 indices. CONCLUSION: Proposed RAPID3 severity and response categories yield comparable results to DAS28 and EULAR criteria in AIM and ATTAIN. DAS28 is more specific for clinical trials. RAPID3 does not preclude also scoring DAS28, and may be informative in the infrastructure of routine care.

Development and validation of a short form of the valued life activities disability questionnaire for rheumatoid arthritis.

OBJECTIVE: To develop and validate a shortened version of the Valued Life Activities disability and accommodations scale (VLA) for individuals with rheumatoid arthritis (RA). METHODS: To shorten the existing VLA measure, item response theory analyses were conducted using data from 449 patients with RA. Next, the resulting 14-item shortened version of the VLA scale (S-VLA) was evaluated by structured interviews among 20 RA patients. Lastly, the S-VLA was administered to 150 RA patients along with other measures, including the Health Assessment Questionnaire (HAQ) and Short Form 36 (SF-36). A random sample of 50 patients completed the S-VLA 2 weeks later to assess reliability. Item statistics were calculated to evaluate correlations between individual items and the S-VLA total score. Correlations between the S-VLA and other measures were used to evaluate validity. RESULTS: Test-retest reliability was 0.91, while Cronbach's alpha for the S-VLA was 0.95. None of the 14 items was associated with improved alpha coefficients when omitted. All of the items were strongly correlated with the S-VLA total score. S-VLA scores were highly positively correlated with the HAQ (r = 0.81, P ≤ 0.001), patient-reported disease activity (r = 0.71, P ≤ 0.001), satisfaction with abilities (r = 0.82, P ≤ 0.001), and number of days with activity limitations (r = 0.65, P ≤ 0.001). In addition, as hypothesized, the S-VLA was inversely correlated with the SF-36 physical component summary score (r = -0.78, P ≤ 0.001) and the physical functioning (r = -0.80, P ≤ 0.001), role physical (r = -0.67, P ≤ 0.001), and social functioning (r = -0.72, P ≤ 0.001) subscales. CONCLUSION: The S-VLA is a short, valid, and reliable instrument that may prove useful for monitoring disability among individuals with RA.

The direct and indirect cost burden of acute coronary syndrome.

OBJECTIVE: Quantify the incremental health care costs and workplace absence and short-term disability costs, to payers and employers, of patients hospitalized for acute coronary syndrome (ACS). METHODS: Retrospective study using medical insurance claims for the years 2002 to 2007. Patients were aged 18 to 64 years and hospitalized for ACS between January 1, 2003, and December 31, 2006; comparison patients without evidence of coronary artery disease were also selected. The incremental impact of ACS was estimated using weighted regression. RESULTS: 30,200 ACS patients were selected. Incremental annual direct costs of ACS were $40,671 (P < 0.001). For the indirect cost sub-analyses, incremental short-term disability costs of ACS were $999 (P < 0.001) and incremental absence costs were insignificant (P = 0.314) but from a small sample (N = 416). CONCLUSIONS: Patients with ACS impose a substantial direct cost burden on employers and payers and a substantial indirect cost burden on employers. Acute coronary syndrome is more costly to employers and payers than other health conditions that are common among employed persons. Rehospitalizations after the initial hospitalization are common and represent a large portion of the cost.

Indirect cost-effectiveness analyses of abatacept and rituximab in patients with moderate-to-severe rheumatoid arthritis in the United States.

OBJECTIVE: To estimate the incremental cost per quality-adjusted life-years (QALYs) for abatacept and rituximab, in combination with methotrexate, relative to methotrexate alone in patients with active rheumatoid arthritis (RA). METHODS: A patient-level simulation model was used to depict the progression of functional disability over the lifetimes of women aged 55-64 years with active RA and inadequate response to a tumor necrosis factor (TNF)-alpha antagonist therapy. Future health-state utilities and medical care costs were based on projected values of the Health Assessment Questionnaire Disability Index (HAQ-DI). Patients were assumed to receive abatacept or rituximab in combination with methotrexate until death or therapy discontinuation due to lack of efficacy or adverse events. HAQ-DI improvement at month 6, after adjustments for control drug (methotrexate) response, was derived from two clinical trials. Costs of medical care and biologic drugs, discounted at 3% annually, were from the perspective of a US third-party payer and expressed in 2007 US dollars. RESULTS: Relative to methotrexate alone, abatacept/methotrexate and rituximab/methotrexate therapies were estimated to yield an average of 1.25 and 1.10 additional QALYs per patient, at mean incremental costs of $58,989 and $60,380, respectively. The incremental cost-utility ratio relative to methotrexate was $47,191 (95% CI $44,810-49,920) per QALY gained for abatacept/methotrexate and $54,891 (95% CI $52,274-58,073) per QALY gained for rituximab/methotrexate. At an acceptability threshold of $50,000 per QALY, the probability of cost effectiveness was 90% for abatacept and 0.0% for rituximab. CONCLUSION: Abatacept was estimated to be more cost effective than rituximab for use in RA from a US third-party payer perspective. However, head-to-head clinical trials and long-term observational data are needed to confirm these findings.

Complex measures and indices for clinical research compared with simple patient questionnaires to assess function, pain, and global estimates as rheumatology "vital signs" for usual clinical care.

Indices of multiple measures have been developed to assess and monitor patients with rheumatic diseases, as no single "gold standard" measure is available for diagnosis, prognosis, and monitoring of all individual patients. Rheumatology indices generally include 4 types of measures from a standard medical evaluation: patient history, physical examination, laboratory tests, and imaging studies. Well-characterized indices are available for rheumatoid arthritis (RA), psoriatic arthritis, systemic lupus erythematosus (SLE), ankylosing spondylitis, vasculitis, osteoarthritis, fibromyalgia, and other rheumatic diseases. These indices are complex and applied widely in clinical research, but rarely are scored in usual rheumatology patient encounters, which generally are conducted without quantitative data other than laboratory tests. Information from a patient often is as prominent in clinical decisions as information from a physical examination or laboratory tests, and is easily collected as standardized "scientific" data on patient questionnaires designed for usual clinical care, which require minimal professional effort. Patient-derived data-along with physical examination, laboratory, and imaging data-are useful rheumatology "vital signs" to assess and monitor patient status, provide documentation, and improve the quality of clinical care, in addition to their possible value for clinical research. Differences between complex measures for research and simple questionnaires designed for usual clinical care might be more widely recognized, to promote quantitative measurement in the infrastructure of usual rheumatology care.

Time to psychiatric hospitalization in patients with bipolar disorder treated with a mood stabilizer and adjunctive atypical antipsychotics: a retrospective claims database analysis.

OBJECTIVE: This study compared the time to psychiatric hospitalization in commercially insured patients with bipolar disorder who were treated with a mood stabilizer plus adjunctive aripiprazole versus adjunctive ziprasidone, olanzapine, quetiapine, or risperidone. METHODS: This was a retrospective, propensity score-matched cohort study using the Ingenix I3/LabRx integrated insurance claims data set. Patients with bipolar disorder were included if they had >or=180 days of pre-index enrollment in the health plan without atypical antipsychotic exposure. Patients received mood stabilizers and subsequently received adjunctive atypical antipsychotic agents; they were then monitored for up to 90 days after the index antipsychotic prescription. The primary analysis was a Cox proportional hazards analysis to evaluate the time until psychiatric hospitalization comparing adjunctive aripiprazole with ziprasidone, olanzapine, quetiapine, or risperidone after adjusting for age, sex, and preindex hospitalization. RESULTS: Adjunctive aripiprazole was associated with a longer time until hospitalization than adjunctive ziprasidone, olanzapine, quetiapine, or risperidone (hazard ratios 1.7, 1.6, 1.5, and 1.5, respectively; all, P < 0.05). Mean initial and maximum doses of all drugs were below those recommended by the package insert or clinical practice guidelines. Sensitivity analyses suggested the robustness of the results in the general population of patients with bipolar disorder recently treated with atypical antipsychotics. CONCLUSIONS: This retrospective claims-data analysis suggests that in these adults with bipolar disorder treated with mood stabilizers, the addition of adjunctive aripiprazole was associated with a longer time to hospitalization than adjunctive ziprasidone, olanzapine, quetiapine, or risperidone during a 90-day follow-up period.

Cost-effectiveness modeling of abatacept versus other biologic agents in DMARDS and anti-TNF inadequate responders for the management of moderate to severe rheumatoid arthritis.

To assess the cost-effectiveness of abatacept compared to different biologic treatment strategies for moderate to severe rheumatoid arthritis based on current medical practices in Canada. A model was constructed to assess the cost-effectiveness of various biologic treatments over a 2-year time horizon, using two effectiveness endpoints: "low disease activity state" (LDAS) and "remission". Abatacept, as first biologic agent after an inadequate response to DMARDs, provides greater treatment success rate for achieving LDAS (29.4% versus 15.6%) and remission (14.8% versus 5.2%), and appears significantly more cost-effective compared to the sequential use of anti-TNF agents (p<0.001). Abatacept, as second biologic agent after an inadequate response to one anti-TNF agent, provides greater treatment success rate for achieving LDAS (17.1% versus 10.2%) and remission (7.4% versus 3.9%) and appears significantly more cost-effective compared to the sequential use of anti-TNF agents (p<0.001). Abatacept is a cost-effective strategy in patients with an inadequate response to DMARDs or to one anti-TNF agent.

The direct and indirect cost burden of Crohn's disease and ulcerative colitis.

OBJECTIVE: To estimate the direct medical and indirect (absenteeism and short-term disability) cost burden of Crohn's Disease (CD) and Ulcerative Colitis (UC). METHODS: Data were obtained from 1999 to 2005 MarketScan databases. Twelve-month expenditures for patients with CD and UC were compared to expenditures among an equal number of propensity score matched comparison group patients. Regression analysis controlled for demographics and case-mix. RESULTS: Annual medical expenditures were significantly higher for commercially insured CD and UC patients compared to matched comparison group patients ($18,963 vs $5300 for CD patients, $15,020 vs $4982 for UC patients, respectively, all P < 0.001). Indirect costs were also high for employed patients with these conditions. CONCLUSIONS: CD and UC are costly diseases with a significant cost burden related to health care utilization and productivity loss.

Cost-effectiveness of abatacept in patients with moderately to severely active rheumatoid arthritis and inadequate response to tumor necrosis factor-alpha antagonists.

OBJECTIVE: To assess cost-effectiveness of abatacept in patients with rheumatoid arthritis (RA) with inadequate response to tumor necrosis factor-alpha antagonists (anti-TNF). METHODS: We developed a simulation model to depict progression of disability [in terms of Health Assessment Questionnaire Disability Index (HAQ-DI)] in women aged 55-64 years with moderately to severely active RA and inadequate response to anti-TNF. At model entry, patients were assumed to receive either oral disease modifying antirheumatic drugs (DMARD) only or oral DMARD plus abatacept. Patients were then tracked from model entry until death. Future health-state utilities and medical-care costs (except study therapy) were estimated based on predicted values of the HAQ-DI. The model was estimated using data from a Phase III clinical trial of abatacept plus secondary sources. Cost-effectiveness was expressed in terms of incremental cost (2006 US$) per quality-adjusted life-year (QALY) gained alternatively over 10 years and a lifetime. Future costs and health effects were discounted at 3% annually. RESULTS: Over 10 years, abatacept would yield 1.0 additional QALY (undiscounted) per patient (4.0 vs 3.0 for oral DMARD) at an incremental (discounted) cost of $45,497 (100,648 vs $55,151) respectively; over a lifetime, corresponding figures were 1.6 QALY (5.8 vs 4.2) and $64,978 ($140,714 vs $82,489). Cost-effectiveness was [mean (95% CI)] $50,576 ($47,056, $54,944) per QALY gained over 10 years, and $45,979 ($42,678, $49,932) per QALY gained over a lifetime. Findings were robust in sensitivity analyses. CONCLUSION: Abatacept is cost-effective by current standards of medical practice in patients with moderately to severely active RA and inadequate response to an anti-TNF.

Productivity cost model of the treatment of rheumatoid arthritis with abatacept.

BACKGROUND: The cost of the biological drug abatacept may be partly offset by reductions in the cost of productivity losses due to employee absences and reduced effectiveness at work because of rheumatoid arthritis (RA). METHODS: This was a 1-year productivity cost model based on epidemiologic and economic data. The setting was private industry in the US and the primary outcome measure was the difference in the costs of lost productivity and drug treatment with and without abatacept ('cost difference'). RESULTS: The lost productivity cost of RA for a firm of 10,000 was $1.69 million, largely due to the cost of RA-related absenteeism ($1.55 million) rather than to worker displacement ($0.12 million) or care-giving for spouses with RA ($0.02 million). In the base case analysis (excluding presenteeism), 37% of the acquisition cost of abatacept was offset by reductions in the cost of RA-related productivity losses. In some industry groups (Utilities and Finance), and in models that included presenteeism, reductions in lost productivity costs exceeded the abatacept cost. CONCLUSIONS: Much of the acquisition cost of abatacept may be offset by reductions in the cost of productivity losses due to RA. Abatacept treatment could be cost saving in some industry groups.

A proposed continuous quality improvement approach to assessment and management of patients with rheumatoid arthritis without formal joint counts, based on quantitative routine assessment of patient index data (RAPID) scores on a multidimensional health assessment questionnaire (MDHAQ).

A continuous quality improvement approach is proposed for the assessment and management of patients with rheumatoid arthritis (RA) based on scores on a one-page patient self-report multidimensional health assessment questionnaire (MDHAQ), without formal joint counts. The approach includes five simple steps before the patient is seen by the physician: (1) an MDHAQ is completed by every patient at every visit; (2) scores are calculated for patient function, pain, and global estimate, with options for a self-report joint count and other scales; (3) scores are entered on flow sheets with data from prior visits, which might also include laboratory and medication information; (4) scores are compiled into an index termed Routine Assessment of Patient Index Data (RAPID), analogous to a Disease Activity Score (DAS); (5) RAPID scores are classified to guide treatment decisions. RAPID 3 includes the three patient-reported outcome (PRO) measures in the RA Core Data Set - physical function, pain, and global estimate. RAPID 4 adds a self-report joint count, and RAPID 5, a physician global estimate. RAPID 3 can be calculated in about 10 seconds, RAPID 4 in about 19 seconds, and RAPID 5 in about 20 seconds. RAPID 3, RAPID 4, and RAPID 5 give similar results to distinguish active from control treatments in RA clinical trials, at levels similar to American College of Rheumatology or DAS improvement criteria, and are all correlated significantly with DAS28 (rho=0.62-0.64, P<0.001). A proposed classification of RAPID scores, analogous to four DAS28 categories, includes: 'near remission' (0-1), 'low severity' (1.01-2), 'moderate severity' (2.01-4), and 'high severity' (>4). RAPID scoring is feasible in standard clinical care to support continuous quality improvement.

Item response theory methods can improve the measurement of physical function by combining the modified health assessment questionnaire and the SF-36 physical function scale.

OBJECTIVE: To compare the measurement properties of the Modified Health Assessment Questionnaire [MHAQ], the SF-36((R)) Health Survey 10 item Physical Functioning scale [PF10], and scores from an item response theory (IRT) based scale combining the two measures. STUDY DESIGN: Rheumatoid arthritis (RA) patients (n = 339) enrolled in a multi-center, randomized, double-blind, placebo-controlled trial completed the MHAQ and the SF-36 pre- and post-treatment. Psychometric analyses used confirmatory factor analysis and IRT models. Analyses of variance were used to assess sensitivity to changes in disease severity (defined by the American College of Rheumatism (ACR)) using change scores in MHAQ, PF10, and IRT scales. Analyses of covariance were used to assess treatment responsiveness. RESULTS: For the entire score range, the 95% confidence interval around individual patient scores was smaller for the combined (total) IRT based scale than for other measures. The MHAQ and PF10 were about 70% and 50% as efficient as the total IRT score of physical functioning in discriminating among ACR groups, respectively. The MHAQ and PF10 were also less efficient than the total IRT score in discriminating among treatment groups. CONCLUSIONS: Combining scales from the two short forms yields a more powerful tool with greater sensitivity to treatment response.

Determining the minimal clinically important differences in activity, fatigue, and sleep quality in patients with rheumatoid arthritis.

OBJECTIVE: To determine the minimal clinically important differences (MCID) in the patient-reported outcomes of activity (0-30, number of days of limitation), fatigue (0 = none, 100 = complete), and sleep quality (0 = no problems, 100 = worst case) for patients with rheumatoid arthritis (RA). METHODS: Two randomized controlled trials comparing abatacept to placebo in RA patients were considered: ATTAIN (n = 391) and AIM (n = 652). An internal anchor-based approach was used to derive the MCID using the Health Assessment Questionnaire, patient global assessment, and pain as anchors. Minimal important change in activity, fatigue, and sleep were determined by estimating mean changes in these outcomes in patients showing change in a narrow range about the MCID of the internal anchor. Correlation analysis was used to determine the consistency of the changes in the outcomes and anchors, and a Delphi process was used to determine the final MCID values. RESULTS: For the 2 trials, consistent patterns of change for activity, fatigue, and sleep and the internal anchors were found with correlations in the range of 0.5, 0.7, and 0.4, respectively. The mean changes for activity, fatigue, and sleep in a narrow range about the MCID of the 3 internal anchors corresponding to the 2 trials were: 3.4 to 4.3 for activity; 6.7 to 17.0 for fatigue; and 4.1 to 7.3 for sleep. Following the Delphi process the MCID determined were 4 for activity, 10 for fatigue, and 6 for sleep. CONCLUSION: These MCID for activity limitation, fatigue, and sleep problems can be used in designing clinical trials and providing benchmarks in assessing patient improvement.

Peripheral arterial disease and renal transplantation.

It was hypothesized that peripheral arterial disease (PAD) is less frequent after kidney transplantation than among comparable patients who are on the deceased-donor waiting list. The cumulative incidences and risk factors for PAD were compared among 43,427 adult transplant recipients and 53,309 adults who were placed on the renal transplant waiting list between 1995 and 2003. All patients had Medicare primary insurance coverage, and Medicare claims were used to identify PAD. For patients with diabetes, the 3-yr cumulative incidence of de novo PAD was 24% on the waiting list versus 20% after transplantation. For patients without diabetes, the 3-yr cumulative incidence was 9% on the waiting list versus 5% after transplantation. The adjusted relative risk (RR) for PAD among patients without diabetes was 0.73 (95% confidence interval [CI] 0.66 to 0.80; P < 0.0001) in the transplant population versus the waiting list population, whereas among patients with diabetes, it was 0.88 (95% CI 0.82 to 0.96; P = 0.0024). A diagnosis of PAD on the waiting list was associated with an almost three-fold increase in the RR for death for patients without diabetes (2.98; 95% CI 2.71 to 3.27; P < 0.0001) and with diabetes (2.92; 95% CI 2.71 to 3.15; P < 0.0001). After transplantation, de novo PAD increased the RR for death almost two-fold in patients without diabetes (1.92; 95% CI 1.63 to 2.26; P < 0.0001) and with diabetes (1.83; 95% CI 1.58 to 2.12; P < 0.0001). The incidence of PAD is higher on the waiting list than after transplantation and is associated with an increased risk for death among patients with and without diabetes.