Why a Complete Response Is the Treatment Aim in Chronic Spontaneous Urticaria.

This study investigated the association between urticaria activity and health-related quality of life (HRQoL). Patient evaluations from the ligelizumab Phase 2b clinical trial (N = 382) were pooled (NCT02477332). Daily patient diaries assessed urticaria activity, sleep and activity interference, the dermatology life quality index (DLQI), and work productivity and activity impairment-chronic urticaria (WPAI-CU). The number of DLQI scores, weekly sleep interference scores (SIS7), weekly activity interference scores (AIS7), and overall work impairment (OWI) evaluations with a complete response per weekly urticaria activity score (UAS7) using bands (0, 1-6, 7-15, 16-27, and 28-42) were reported. Over 50% of the patients had a mean DLQI of > 10 at baseline, indicating a significant effect of chronic spontaneous urticaria (CSU) on their HRQoL. Complete response (UAS7 = 0) evaluations corresponded with no impacts on other patient-reported outcomes. In total, 91.1% of UAS7 = 0 evaluations corresponded to DLQI scores of 0-1, 99.7% to SIS7 scores of 0, 99.7% to AIS7 scores of 0, and 85.3% to OWI scores of 0. This was significantly different compared with the UAS7 = 1-6 evaluations (61.9%, 68.5%, 67.7%, and 65.4%, respectively; p < 0.0001). Complete responses to treatment were associated with no impairments on the dermatology-QoL, no interferences with sleep and activity, and significantly improved capacities to work compared to patients who continued to have signs and symptoms, even for those with minimal disease activity.

The Benefit of Complete Response to Treatment in Patients With Chronic Spontaneous Urticaria-CURE Results.

BACKGROUND AND OBJECTIVE: Chronic spontaneous urticaria (CSU) is a distressing disease. We report real-world data from the global Chronic Urticaria Registry (CURE) about associations between various CSU states and sleep impairment, plus important health-related quality-of-life (HRQoL) outcomes and compared different methods to assess CSU states. METHODS: CURE data were collected at baseline and 6-monthly follow-ups (FU). Assessments included CSU states using the Urticaria Control Test (UCT), weekly Urticaria Activity Score (UAS7), and Physician Global Assessment (PhyGA) of treatment response. Complete response to treatment (CR, UAS7 = 0), complete control of disease (CC, UCT = 16), and PhyGA = CR were assessed, plus the Dermatology Life Quality Index and the Chronic Urticaria Quality-of-Life Questionnaire (CU-Q2oL) sleep domain. RESULTS: Overall, 2078 patients were included. At baseline, 9.8%, 17.9%, and 42.3% of patients had UCT = 16, UAS7 = 0, or PhyGA = CR, respectively, which increased at FU1 and FU2. Patients with higher UCT scores had better sleep and HRQoL. The presence of angioedema without wheals, episodic disease, omalizumab treatment, and male sex were associated with CC (P < .05). Among 469 patients who achieved CC or CR, 16.4% (n = 77) showed CC or CR with all 3 instruments. Agreement between UCT = 16 and UAS7 = 0 measurements was moderate (κ = 0.581), but poor between UCT = 16 and PhyGA = CR (κ = 0.208). CONCLUSIONS: Few patients had CR/CC of their CSU at baseline entry. Disease control strongly related to good sleep and better HRQoL; therefore, it is important to aim for CR in CSU treatment. Patient-reported UCT and UAS7 assessments demonstrated a more accurate measurement of CSU state versus physician assessments.

Double jeopardy from the COVID-19 pandemic: risk of exposure and income loss in Portugal.

BACKGROUND: Increasing evidence indicates that the first wave of the COVID-19 pandemic had immediate health and social impact, disproportionately affecting certain socioeconomic groups. Assessing inequalities in risk of exposure and in adversities faced during the pandemic is critical to inform targeted actions that effectively prevent disproportionate spread and reduce social and health inequities. This study examines i) the socioeconomic and mental health characteristics of individuals working in the workplace, thus at increased risk of COVID-19 exposure, and ii) individual income losses resulting from the pandemic across socioeconomic subgroups of a working population, during the first confinement in Portugal. METHODS: This study uses data from 'COVID-19 Barometer: Social Opinion', a community-based online survey in Portugal. The sample for analysis comprised n = 129,078 workers. Logistic regressions were performed to estimate the adjusted odds ratios (AOR) of factors associated with working in the workplace during the confinement period and with having lost income due to the pandemic. RESULTS: Over a third of the participants reported working in the workplace during the first confinement. This was more likely among those with lower income [AOR = 2.93 (2.64-3.25)], lower education [AOR = 3.17 (3.04-3.30)] and working as employee [AOR = 1.09 (1.04-1.15)]. Working in the workplace was positively associated with frequent feelings of agitation, anxiety or sadness [AOR = 1.14 (1.09-1.20)] and perception of high risk of infection [AOR = 11.06 (10.53-11.61)]. About 43% of the respondents reported having lost income due to the pandemic. The economic consequences affected greatly the groups at increased risk of COVID-19 exposure, namely those with lower education [AOR = 1.36 (1.19-1.56)] and lower income [AOR = 3.13 (2.47-3.96)]. CONCLUSIONS: The social gradient in risk of exposure and in economic impact of the pandemic can result in an accumulated vulnerability for socioeconomic deprived populations. The COVID-19 pandemic seems to have a double effect in these groups, contributing to heightened disparities and poor health outcomes, including in mental health. Protecting the most vulnerable populations is key to prevent the spread of the disease and mitigate the deepening of social and health disparities. Action is needed to develop policies and more extensive measures for reducing disproportionate experiences of adversity from the COVID-19 pandemic among most vulnerable populations.

Rheuma SPACE - Standard Practice Aiming Clinical Excellence: the first Portuguese Rheumatology Department evaluation.

The Portuguese Rheumatology Society (SPR) embraced quality as a major goal and launched, in early 2015, a program to aim for excellence in global clinical care: Rheuma SPACE - Standard Practice Aiming Clinical Excellence. Evaluating daily reality is the first step in a quality development timeline, ultimately contributing for health gains. Herein we describe the results of the evaluation of the quality indicators defined for this project and the improvement strategies identified. The Rheuma SPACE project included three phases: 1) establishing a set of quality indicators and an excellence quality model; 2) assessment of the current care at Rheumatology departments concerning the defined quality indicators in the scope of the excellence model; and 3) elaboration of global and customized reports for each participating Rheumatology department, resulting in the identification of improvement opportunities. Ten Rheumatology departments, countrywide, including larger and smaller institutions, were asked to participate in Rheuma SPACE. This resulted in an individual report for each department along with global benchmarking practices analysis. Furthermore, a list of improvement initiatives was developed. We concluded that departments lack physicians and need exclusively dedicated nurses. Time dedicated to research and audit activities should be specifically allocated. Internal contracting is well established, and professionals are committed to targets. Processes are still suboptimal, needing standardization of triage criteria, more frequent follow-up, as well as better medical records and multidisciplinary coverage. Regarding outcomes, patients are satisfied with the provided care and professionals with the working environment. However, department facilities for the former, and career related aspects, for the latter should improve. With this innovative study conducted in Portugal we expect to have enlightened tailored opportunities for improvement, ensure patient-focused practices and be able to define the indispensable quality requirements for excellence.

Population-based Estimates for High Risk of Severe COVID-19 Disease due to Age and Underlying Health Conditions.

INTRODUCTION: COVID-19 is a viral respiratory disease, which became a global threat to public health. Specific subsets of the population are more vulnerable, namely those with chronic diseases. We aimed to estimate the share of the Portuguese population at the highest risk for complications following COVID-19 infection due to both old age and specific comorbidities. MATERIAL AND METHODS: Our sample included all people aged 65 years and above (2215 men and 3486 women) who participated in the fifth Portuguese National Health Interview Survey, conducted in 2014. In order to project the potential population at highest risk for COVID-19, we used the latest available official demographic estimates from the National Institute of Statistics - INE 2018. We used a more restrictive definition of risk combining old age criteria and the following chronic conditions as potential risk factors for COVID-19 according to the available literature: hypertension, diabetes, chronic obstructive pulmonary disease, cardio- and cerebrovascular disease. RESULTS: We estimated that 15.5% (n = 1 560 667) of the Portuguese population might be at increased risk for complications from COVID-19 because of old age and existing chronic conditions. Such estimates vary across the country (from 1.7% in Azores to 33.7% in Northern Portugal). Northern Portugal not only has the highest prevalence of selected morbidity (72.8%) within mainland Portugal, but also has the largest population at risk for COVID-19 (n = 526 607). This was followed by the Lisbon and Tagus Valley region (n = 408 564) and Central Portugal (n = 388 867). DISCUSSION: Our results should encourage authorities to continue protecting those more vulnerable to the pandemic threat, particularly on those areas of the country which are more likely to be further affected. CONCLUSION: We projected a considerable number of Portuguese people at the highest risk for severe COVID-19 disease due to both old age and pre-existing chronic conditions. Such estimates vary across the country.

Hospitalization Costs Due to Hypoglycemia in Patients with Diabetes: A Microcosting Approach.

INTRODUCTION: Hypoglycemia leading to hospitalization is associated with adverse economic outcomes, although the real burden is unknown. The HIPOS-WARD (Hypoglycemia In Portugal Observational Study-Ward) aimed to characterize ward admissions due to hypoglycemia episodes in treated patients with diabetes and assess their economic impact to the National Health System. METHODS: Observational, cross-sectional study, conducted in 16 Portuguese centers for 22 months. The applied microcosting approach was based on healthcare resource data, collected from patients' charts upon ward admission until discharge, and unitary costs from official/public data sources. Absenteeism was also estimated for active workers on the basis of the human capital approach. RESULTS: Of the 176 patients with diabetes mellitus enrolled, 86% had type 2 diabetes. Half of the patients (50.0%) were on insulin-based therapy, followed by 30.1% on a secretagogue-based regimen, 9.7% on non-secretagogue therapy, and 10.2% on a combination of insulin and secretagogue. Overall mean costs per patient were medication, 45.45 €; laboratory analysis, 218.14 €; examinations, 64.91 €; physician and nurse time, 268.55 € and 673.39 €, respectively. Bed occupancy was the main cost driver (772.09 €) and indirect cost averaged 140.44 €. Overall, the cost per hypoglycemia episode leading to hospitalization averaged 2042.52 € (range 194.76-16,762.87 €). Patients treated with insulin-based regimens (2267.76 €) and type 2 diabetes (2051.29 €) had the highest mean costs. The mean cost increased with repeated hypoglycemic events (2191.67 €), correlated complications (2109.26 €), and death (5253.38 €). CONCLUSION: HIPOS-WARD's findings confirm and support both the substantial clinical and economic impact of hospitalization due to hypoglycemia in Portugal.

Cost-effectiveness of sacubitril/valsartan for the treatment of patients with heart failure with reduced ejection fraction in Portugal.

Objectives: This study assesses the cost-effectiveness of sacubitril/valsartan versus enalapril in patients with symptomatic heart failure with reduced ejection fraction (HFrEF).Methods: We used a previously developed Markov model calibrated with patient-level data from the PARADIGM-HF trial, adapted to the Portuguese setting. The model considers two health states (alive or dead) and uses regression analyzes to estimate hospitalizations and deaths over time. A panel of experts estimated resource consumption in the outpatient setting. To estimate resource consumption with hospitalizations, the National Health Service Diagnosis Related Groups database was used. Unit costs were based on national legislation, and on the Infomed database. The model considers a societal perspective, a time horizon of 30-years, and a 5% annual discount rate. Sensitivity analyses assessed the robustness of results.Results: Sacubitril/valsartan increases life expectancy by 0.5 life-years, corresponding to 0.4 incremental quality adjusted life-years (QALY) versus enalapril. The estimated incremental cost-effectiveness ratio (ICER) is 22,702€/QALY. Sensitivity analysis shows that results are robust, but sensitive to the parameter estimates of the cardiovascular survival curve.Conclusion: Sacubitril/valsartan is a cost-effective therapeutic option in the treatment of Portuguese patients with HFrEF and translate into significant health gains and increased life expectancy versus the current standard of care.

Multimorbidity and intention to retire: a cross-sectional study on 14 European countries.

OBJECTIVES: To describe the association between multimorbidity and intention of retirement in Europe and to understand whether this relationship is modified by the working environment and disability integration policies. METHODS: Participants were 11,790 employees aged 50-65 years old who responded to the sixth wave of SHARE project (2015). We modelled intention of retirement as a function of multimorbidity, adjusting for age, gender, education level, and household income by means of logistic models with country fixed effects. We then included the working conditions and an integration policy indicator as potential effect modifiers. RESULTS: Overall, 36.6% of participants reported multimorbidity and 56.1% were willing to retire earlier. Multimorbidity was significantly associated with intention of retirement (OR = 1.58, 95% CI 1.37-1.84). Unfavourable working conditions were positively related to the intention to retire (OR = 1.99, 95% CI 1.53-2.58), while the integration policy was unrelated (OR = 1.84, 95% CI 0.80-4.23). Both did not modify the studied association (interaction terms: OR = 1.14, 95% CI 0.77-1.67, and OR = 0.85, 95% CI 0.58-1.24, respectively). CONCLUSIONS: Multimorbidity is associated with intention of retirement in Europe. This association was unaltered by working conditions and integration policies.

The impact of osteoarthritis on early exit from work: results from a population-based study.

BACKGROUND: Osteoarthritis (OA) is a leading cause of pain and disability, which may be a source of productivity losses. The objectives of this study were to describe the impact of OA, namely through pain and physical disability, on early exit from work and to calculate its economic burden. METHODS: We analysed data from the national, cross-sectional, population-based EpiReumaPt study (Sep2011-Dec2013) in which 10,661 individuals were randomly surveyed in order to capture all cases of rheumatic diseases. We used all participants aged 50-64, near the official retirement age, who were clinically validated by experienced rheumatologists (n = 1286), including OA cases. A national database was used to calculate productivity values by gender, age and region, using the human capital approach. The impact of OA on the likelihood of early exit from work and the population attributable fractions used to calculate due economic burden (indirect costs) were obtained at the individual level by logistic regression. All results were based on weighted data. RESULTS: Almost one third of the Portuguese population aged 50-64 had OA (29.7%; men: 16.2% and women: 43.5%) and more than half were out of paid work (51.8%). Only knee OA is associated with early exit from work (OR: 2.25; 95%CI: 1.42-3.59; p = 0.001), whereas other OA locations did not reach any statistical difference. Furthermore, we observed an association between self-reported longstanding musculoskeletal pain (OR: 1.55; 95%CI: 1.07-2.23; p = 0.02) and pain interference (OR: 1.35; 95%CI: 1.13-1.62; p = 0.001) with early exit from work. We also detected a clear relationship between levels of disability, measured by the Health Assessment Questionnaire (HAQ), and the probability of work withdrawal. The estimated annual cost of early exit from work attributable to OA was €656 million (€384 per capita; €1294 per OA patient and €2095 per OA patient out-of-work). CONCLUSIONS: In this study, we observed an association between OA and early exit from work, largely dependent on pain and disability. This relationship translates into a meaningful economic burden amounting to approximately 0.4% of the national Gross Domestic Product (GDP). The high prevalence and the impact of this disabling chronic disease highlight the need to prioritize policies targeting early exit from work in OA.

Effectiveness of a Referral Program for rheumatoid arthritis and axial spondyloarthritis Diagnosis at Primary Care Centers in Portugal - SIARA STUDY.

OBJECTIVES: Early diagnosis and treatment of Rheumatoid Arthritis (RA) and axial Spondylarthritis (axial SpA) can limit the impact of disease outcomes. This study evaluated the effectiveness of a referral program on the identification of patients with RA and axial SpA. METHODS: This was an observational, prospective, randomized (by clusters) study conducted in Portugal to evaluate the impact of the implementation of a set of referral support actions (RSA). The study was divided in two sub-studies, the RA sub-study and the axial SpA sub-study. 28 participating primary care units were randomly (by clusters) assigned to RSA or control group (with no intervention). Both RSA and control groups identified and referred patients with suspected RA or axial SpA to the rheumatology unit of the reference hospital. The primary objective was to evaluate the correct diagnosis of RA or axial SpA cases confirmed by the rheumatologist of the reference hospital. RESULTS: RA-Substudy: A total of 340 patients were recruited (144 in the RSA-exposed group; 196 in the control). RA diagnosis confirmation was 7.3% (95%CI, 2.1-12.5%) in RSA group versus 2.7% (95%CI, 0.0-5.7%) in control group RSA effect was positive but moderate (4.6%) and not statistically significant (95% CI, 0.0%-11.8%; p=0.222, adjusted for clustering effect). Rate of confirmed arthritis of any type was 16.9% (n=14/83) in the RSA group and 6.0% (n=5/83) in the control group. This difference was statistically significant and favorable to RSA group (OR=3.2; 95% CI 1.1-9.2; p=0.028). Axial SpA-Substudy: A total of 231 patients were recruited (108 in the RSA-exposed group; 123 in the control). Axial SpA diagnosis confirmation was 8.7% (95% CI, 2.1-15.4%) in RSA group versus 5.6% (95% CI, 0.0-11.73%) in control group. RSA effect was positive (3.1%) but not statistically significant (95% CI, -7.5- 12.9%; p=0.568, adjusted for clustering effect). CONCLUSIONS: This study showed a positive tendency for the RSA program, most relevantly on the diagnosis of patients with any type of arthritis in the RA sub-study. It is possible that a referral program more comprehensive than the one herein tested might improve early diagnosis of RA and SpA.

Severe hypoglycaemia among patients with type 2 diabetes requiring emergency hospital admission: The Hypoglycaemia In Portugal Observational Study-Emergency Room (HIPOS-ER).

AIMS: To analyse the prevalence of severe hypoglycaemia in patients with type 2 diabetes (T2DM) treated with antihyperglycaemic agents (AHA) and requiring emergency room (ER) assistance, and to analyse the prevalence according to type of AHA therapy. METHODS: The present study, the Hypoglycaemia In Portugal Observational Study-Emergency Room (HIPOS-ER), was a cross-sectional, observational, multicentre, nationwide study, with specific hypoglycaemia source data collection. RESULTS: Within the study period, a total of 425 706 admissions were recorded in the ERs of participating hospitals. The prevalence of severe hypoglycaemic episodes in patients with T2DM was 0.074%. In all, 238 patients were included, more than half of whom were on insulin-based therapy (55.0%) and a third of whom (31.5%) were on oral secretagogue-based therapy. In 61.2% of patients primary care was the main diabetes care setting. The median patient age was 77.5 years and the mean duration of diabetes was 19 years. Missing a meal or low carbohydrate meal content was the most frequent cause of hypoglycaemia (55.9%) and the most frequent triggers for seeking emergency assistance were pre-syncope (19.2%) and transient loss of consciousness (17.4%). A total of 44.1% of patients were hospitalized for a median of 5.1 days. Patients in the secretagogue group were admitted to hospital more often than patients in the insulin group (70.7% vs 29.0%; P < .001). Nine patients died. CONCLUSIONS: These findings confirm that severe hypoglycaemia in patients with T2DM requiring ER assistance occurs mainly in those on insulin- and secretagogue-based therapies and is associated with a significant medical burden. Antidiabetic therapy should be individualized to minimize the risk of severe iatrogenic hypoglycaemia, and any intervention to this end should always involve primary care stakeholders.

Inadequate pain relief among patients with primary knee osteoarthritis.

BACKGROUND: Despite the widespread treatments for osteoarthritis (OA), data on treatment patterns, adequacy of pain relief, and quality of life are limited. The prospective multinational Survey of Osteoarthritis Real World Therapies (SORT) was designed to investigate these aspects. OBJECTIVES: To analyze the characteristics and the patient reported outcomes of the Portuguese dataset of SORT at the start of observation. METHODS: Patients ≥50 years with primary knee OA who were receiving oral or topical analgesics were eligible. Patients were enrolled from seven healthcare centers in Portugal between January and December 2011. Pain and function were evaluated using the Brief Pain Inventory (BPI) and WOMAC. Quality of life was assessed using the 12-Item Short Form Health Survey (SF-12). Inadequate pain relief (IPR) was defined as a score >4/10 on item 5 of the BPI. RESULTS: Overall, 197 patients were analyzed. The median age was 67.0 years and 78.2% were female. Mean duration of knee OA was 6.2 years. IPR was reported by 51.3% of patients. Female gender (adjusted odds ratio - OR 2.15 [95%CI 1.1, 4.5]), diabetes (OR 3.1 [95%CI 1.3, 7.7]) and depression (OR 2.24 [95%CI 1.2, 4.3]) were associated with higher risk of IPR. Patients with IPR reported worst outcomes in all dimensions of WOMAC (p<0.001) and in all eight domains and summary components of SF-12 (p<0.001). CONCLUSIONS: Our findings indicate that improvements are needed in the management of pain in knee OA in order to achieve better outcomes in terms of pain relief, function and quality of life.

Inadequate pain relief among patients with primary knee osteoarthritis / Alívio inadequado da dor em pacientes com osteoartrite de joelho primária

ABSTRACT Background: Despite the widespread treatments for osteoarthritis (OA), data on treatment patterns, adequacy of pain relief, and quality of life are limited. The prospective multinational Survey of Osteoarthritis Real World Therapies (SORT) was designed to investigate these aspects. Objectives: To analyze the characteristics and the patient reported outcomes of the Portuguese dataset of SORT at the start of observation. Methods: Patients ≥50 years with primary knee OA who were receiving oral or topical analgesics were eligible. Patients were enrolled from seven healthcare centers in Portugal between January and December 2011. Pain and function were evaluated using the Brief Pain Inventory (BPI) and WOMAC. Quality of life was assessed using the 12-Item Short Form Health Survey (SF-12). Inadequate pain relief (IPR) was defined as a score >4/10 on item 5 of the BPI. Results: Overall, 197 patients were analyzed. The median age was 67.0 years and 78.2% were female. Mean duration of knee OA was 6.2 years. IPR was reported by 51.3% of patients. Female gender (adjusted odds ratio - OR 2.15 [95%CI 1.1, 4.5]), diabetes (OR 3.1 [95%CI 1.3, 7.7]) and depression (OR 2.24 [95%CI 1.2, 4.3]) were associated with higher risk of IPR. Patients with IPR reported worst outcomes in all dimensions of WOMAC (p < 0.001) and in all eight domains and summary components of SF-12 (p < 0.001). Conclusions: Our findings indicate that improvements are needed in the management of pain in knee OA in order to achieve better outcomes in terms of pain relief, function and quality of life.

RESUMO Antecedentes: Apesar dos tratamentos muito difundidos para a osteoartrite (OA), dados sobre os padrões de tratamento, a adequação do alívio da dor e a qualidade de vida são limitados. O estudo multinacional prospectivo Survey of Osteoarthritis Real World Therapies (SORT) foi projetado para investigar esses aspectos. Objetivos: Analisar as características e os desfechos relatados pelo paciente do conjunto de dados português do Sort no início da observação. Métodos: Consideraram-se elegíveis os pacientes com 50 anos ou mais com OA de joelho primária que recebiam analgésicos orais ou tópicos. Os pacientes foram recrutados de sete centros de saúde de Portugal entre janeiro e dezembro de 2011. A dor e a função foram avaliadas pelo Brief Pain Inventory (BPI) e pelo WOMAC. A qualidade de vida foi avaliada com o 12-item Short Form Health Survey (SF-12). O alívio inadequado da dor (AID) foi definido como uma pontuação > 4/10 no item 5 do BPI. Resultados: Foram analisados 197 pacientes. A idade média foi de 67 anos e 78,2% eram do sexo feminino. A duração média da OA de joelho foi de 6,2 anos. O AID foi relatado por 51,3% dos pacientes. O sexo feminino (odds ratio ajustado - OR 2,15 [IC 95% 1,1-4,5]), o diabetes (OR = 3,1 [IC 95% 1,3-7,7]) e a depressão (OR 2,24 [IC 95% 1,2-4,3]) estiveram associados a um maior risco de AID. Os pacientes com AID relataram piores desfechos em todas as dimensões do Womac (p < 0,001) e em todos os oito domínios e nos dois componentes sumários do SF-12 (p < 0,001). Conclusões: Os resultados do presente estudo indicam que é necessário melhorar o manejo da dor na OA de joelho a fim de alcançar melhores desfechos em termos de alívio da dor, função e qualidade de vida.

Impact of hypoglycemic events and HbA1c level on sulfonylurea discontinuation and down-titration.

BACKGROUND: A retrospective cohort study using GE Centricity electronic medical records assessed the association between post-index hypoglycemia and HbA1c with discontinuation and down-titration of sulfonylureas among patients with Type 2 diabetes mellitus. METHODS: Adult patients with an index prescription for a sulfonylurea and ≥12 months' continuous records pre- and post-index were eligible. Sulfonylurea discontinuation and down-titration was assessed 1-year post-index. Discontinuation occurred if the date of a prescription was >90 days from the preceding prescription plus days of supply. Down-titration occurred when a subsequent prescription was lower than the index dose. Cox regression assessed the association between post-index hypoglycemia and HbA1c with time to sulfonylurea discontinuation and down-titration, as well as other factors. RESULTS: 28,371 participants were included in the study; 13,459 (47.4%) were discontinuers, 717 (2.5%) were down-titraters, and 14,195 (50.0%) were continuers. 0.6% of continuers experienced hypoglycemia 1-year post-index, compared with 3.1% of down-titraters and 0.8% of discontinuers (p < 0.0001). Patients with post-index hypoglycemia had a significantly higher rate of discontinuation (hazard ratio [HR] = 1.82, 95% CI: 1.47-2.23) and down-titration (HR = 4.25, 95% CI: 1.92-8.03). Patients with higher post-index HbA1c and use of 2nd generation sulfonylureas had an increased rate of discontinuation (HR = 1.05, 95% CI: 1.04-1.06; HR = 1.19, 95% CI: 1.14-1.24, respectively). CONCLUSION: Approximately half of participants who initiated sulfonylureas discontinued or down-titrated therapy within one year. Both post-index hypoglycemia and higher HbA1c were significant risk factors for sulfonylurea treatment change.

Inadequate pain relief among patients with primary knee osteoarthritis. / Alívio inadequado da dor em pacientes com osteoartrite de joelho primária.

BACKGROUND: Despite the widespread treatments for Osteoarthritis (OA), data on treatment patterns, adequacy of pain relief, and quality of life are limited. The prospective multinational Survey of Osteoarthritis Real World Therapies (SORT) was designed to investigate these aspects. OBJECTIVES: To analyze the characteristics and the patient reported outcomes of the Portuguese dataset of SORT at the start of observation. METHODS: Patients ≥ 50 years with primary knee OA who were receiving oral or topical analgesics were eligible. Patients were enrolled from seven healthcare centers in Portugal between January and December 2011. Pain and function were evaluated using the Brief Pain Inventory (BPI) and WOMAC. Quality of life was assessed using the 12-Item Short Form Health Survey (SF-12). Inadequate Pain Relief (IPR) was defined as a score>4/10 on item 5 of the BPI. RESULTS: Overall, 197 patients were analyzed. The median age was 67.0 years and 78.2% were female. Mean duration of knee OA was 6.2 years. IPR was reported by 51.3% of patients. Female gender (adjusted odds ratio - OR 2.15 [95%CI 1.1, 4.5]), diabetes (OR 3.1 [95%CI 1.3, 7.7]) and depression (OR 2.24 [95%CI 1.2, 4.3]) were associated with higher risk of IPR. Patients with IPR reported worst outcomes in all dimensions of WOMAC (p<0.001) and in all eight domains and summary components of SF-12 (p<0.001). CONCLUSIONS: Our findings indicate that improvements are needed in the management of pain in knee OA in order to achieve better outcomes in terms of pain relief, function and quality of life.

Cost-effectiveness of adding ezetimibe to atorvastatin vs switching to rosuvastatin therapy in Portugal.

BACKGROUND: Statin monotherapy is the mainstay of low-density lipoprotein cholesterol (LDL-C) management for high cardiovascular risk patients in Portugal; however, several therapeutic options are available and predicted to have different clinical and economic impacts. The aim of this study was to evaluate the cost-effectiveness of adding ezetimibe 10 mg (EZ10) to atorvastatin 10 or 20 mg (A10/20) vs switching to rosuvastatin 10 or 20 mg (R10/20) in Portuguese patients with coronary heart disease (CHD) and/or diabetes who are currently above the LDL-C goal. METHODS: A Markov model was used to describe CHD disease progression and the lifetime costs and utilities associated with each disease state were used to estimate the gains in life-years and quality-adjusted life-years (QALYs), as well as the incremental cost-effectiveness ratio (ICER), of the two treatment regimens. Model inputs, such as age, gender, and prevalence of cardiovascular risk factors of the dyslipidemic Portuguese patients were obtained from the Portuguese cohort of the Dyslipidemia International Study (DYSIS). The efficacy of each treatment regimen, the cost of drugs and of treating CHD events, and the utilities for each disease state were derived from published sources. RESULTS: The estimated lifetime discounted number of QALYs gained by patients treated with A10/20 was 8.70, while in those switching to R10/20 it was 8.81 and in those adding EZ10 it was 8.93. Discounted total health costs were estimated to be €11,131 for A10/20, but €14,511 and €16,571 for R10/20 and A10/20 + EZ10, respectively. The ICER of adding ezetimibe vs switching to rosuvastatin was €16,465/QALY. Based on the Portuguese cost-effectiveness willingness-to-pay threshold of €30,000/QALY, adding ezetimibe vs switching to rosuvastatin would be a cost-effective use of resources in Portugal. Sensitivity analyses in patients with differing clinical histories (CHD or diabetes or both) yielded similar values, with no ICER over €30,000/QALY. CONCLUSIONS: From the perspective of the National Health Service, prescribing ezetimibe to high cardiovascular risk patients being treated with atorvastatin vs switching them to rosuvastatin is projected to be a cost-effective use of resources in Portugal.

Indirect costs associated with early exit from work attributable to rheumatic diseases.

BACKGROUND: Rheumatic diseases (RD) cause physical disability that may lead to early exit from work, generating indirect costs to society. We aimed to measure these costs in a population approaching the statutory retirement age. METHODS: The analysis was based on the prevalence of self-reported RD using a bottom-up approach. Health and sociodemographic data were retrieved from the fourth National Health Survey (INS), for all people between 50 and 64 years of age (3762 men and 4241 women), whereas an official national database was used to estimate productivity values by gender, age group and region, using the human capital approach. The effects of RD on the likelihood of early exit from paid employment and the attributable fractions estimates were obtained at the individual level by logistic regression. RESULTS: At the time of the survey, 37.2% of the population aged 50-64 years self-reported at least one RD. Among these, 52.6% were not employed, compared with 40.7% of those without RD (P < 0.001). The annual indirect costs following premature exit from work attributable to RD were €650 million (€892 per RD patient). Early retirement amounted to €367 million, whereas early retirement and unemployment totalized €385 million (€504 and €528 per RD patient, respectively). Females are responsible for about 60% of these costs; however, males contribute with higher individual productivity losses. CONCLUSION: Early exit from work attributable to RD amounts to approximately 0.4% of the national GDP. The public health concern and the economic impact highlight the need to prioritize investments in health and social protection policies targeting patients with rheumatic conditions.

Association of rheumatic diseases with early exit from paid employment in Portugal.

To examine the association between rheumatic diseases (RD) and other chronic morbidity with early exit from paid employment in the Portuguese population. The study population consisted of all people between 50 and 64 years of age (3,762 men and 4,241 women) who participated in the Portuguese National Health Survey, conducted in 2005/2006. Data were collected on demographics, ill-health, lifestyle, and socioeconomic factors. Logistic regression was used to estimate the isolated effect of rheumatic diseases and other chronic diseases on the likelihood of exit from paid employment. At the time of the survey, 45.1 % of the Portuguese population with ages between 50 and 64 years old were not employed. In the nonemployed population, 31.6 % self-reported "poor" to "very poor" health, whereas 16.4 % did so in the employed population. A larger average number of major chronic diseases per capita were also found in those not employed (1.9 vs. 1.4, p < 0.001). In the multivariate models, chronic diseases were associated with early exit from paid employment. In particular, rheumatic diseases were more prevalent (43.4 vs. 32.1 %) and associated with early exit from work (OR 1.31; CI 1.12-1.52, p = 0.001). This study suggests an association between RD and other major chronic diseases with early exit from paid employment in Portugal. Thus, health and social protection policies should target these chronic disorders in order to better address sustainability issues and social protection effectiveness.

Patient's access to healthcare and treatment in rheumatoid arthritis: the views of stakeholders in Portugal.

BACKGROUND: The access to healthcare and treatment by rheumatoid arthritis (RA) patients, particularly to biologics, differs significantly among European countries.We aimed to explore the views and experiences of Portuguese healthcare stakeholders on key barriers which limit the access to treatment, and ultimately to biologics, by RA patients and to find potential solutions (leverage points) to overcome the identified barriers. METHODS: This was a qualitative research consisting of semi-structured face-to-face interviews with key stakeholders in RA framework. Thirty four individuals from eight groups of stakeholders were interviewed: rural and urban general practitioners (GPs), rheumatologists, hospital managers, hospital pharmacists, budget holders, representatives from the Portuguese Rheumatology Society and the RA Patient Association. Interviews were conducted between May and June 2011. Conventional content analysis with research triangulation was used. RESULTS: The key barriers identified were related to the accessibility to primary healthcare services, difficulties in RA diagnosis among GPs, inefficient referral to secondary healthcare and controlled process of biologics prescription in public hospitals. The leverage points identified included the improvement of epidemiological and clinical knowledge about RA in Portugal, a better understanding of the disease among patients and GPs, the clarification of biologics benefits among budget holders and a raised awareness of the current treatment guidelines. In order to further address the leverage points, the following key initiatives were proposed: optimization of RA national registry; dissemination of information on rheumatic symptoms in primary care facilities and among the general public; increase interaction between rheumatologists and GPs through clinical discussions of successfully treated patients or workshops; broader utilization of disease diagnosis and monitoring tools, such as DAS28, and implementation of hospital-based research to collect real-world data. CONCLUSIONS: Most of the key barriers limiting the access to treatment, including biologics, in RA in Portugal are upstream of rheumatology practice. Our findings suggest that future actions should be focused on the primary care level to improve referral to rheumatologists. In addition, the collection of real-world data seems essential to characterise the RA population, to improve disease management and to increase compliance with current treatment guidelines.

Cost-effectiveness analysis of boceprevir for the treatment of chronic hepatitis C virus genotype 1 infection in Portugal.

BACKGROUND: The recent approval of two protease inhibitors, boceprevir and telaprevir, is likely to change the management of chronic hepatitis C virus (HCV) genotype 1 infection. OBJECTIVES: We evaluated the long-term clinical outcomes and the cost effectiveness of therapeutic strategies using boceprevir with peginterferon plus ribavirin (PR) in comparison with PR alone for treating HCV genotype 1 infection in Portugal. METHODS: A Markov model was developed to project the expected lifetime costs and quality-adjusted life-years (QALYs) associated with PR alone and the treatment strategies outlined by the European Medicines Agency in the boceprevir summary of product characteristics. The boceprevir-based therapeutic strategies differ according to whether or not the patient was previously treated and whether or not the patient had compensated cirrhosis. The model simulated the experience of a series of cohorts of chronically HCV-infected patients (each defined by age, sex, race and fibrosis score). All treatment-related inputs were obtained from boceprevir clinical trials - SPRINT-2, RESPOND-2 and PROVIDE. Estimates of the natural history parameters and health state utilities were based on published studies. Portugal-specific annual direct costs of HCV health states were estimated by convening a panel of experts to derive health state resource use and multiplying the results by national unit costs. The model was developed from a healthcare system perspective with a timeframe corresponding to the remaining duration of the patients' lifetimes. Both future costs and QALYs were discounted at 5 %. To test the robustness of the conclusions, we conducted deterministic and probabilistic sensitivity analyses. RESULTS: In comparison with the treatment with PR alone, boceprevir-based regimens were projected to reduce the lifetime incidence of advanced liver disease, liver transplantation, and liver-related death by 45-51 % and increase life expectancy by 2.3-4.3 years. Although the addition of BOC increased treatment costs by €13,300-€19,700, the reduction of disease burden resulted in a decrease of €5,400-€9,000 in discounted health state costs and an increase of 0.68-1.23 in discounted QALYs per patient. The incremental cost-effectiveness ratios of the boceprevir-based regimens compared with PR among previously untreated and previously treated patients were €11,600/QALY and €8,700/QALY, respectively. The results were most sensitive to variations in sustained virologic response rates, discount rates and age at treatment. CONCLUSIONS: Adding boceprevir to PR was projected to reduce the number of liver complications and liver-related deaths, and to be cost effective in treating both previously untreated and treated patients.