Introdução: As avaliações econômicas completas do tipo custo-utilidade, suportadas por dados de efetividade do mundo real, permitem uma perspectiva diferenciada da avaliação de tecnologia em saúde. Objetivo: Realizar uma análise de custo-utilidade do uso de estatinas para a prevenção secundária de eventos cardiovasculares em portadores de doença cardiovascular isquêmica, e avaliar a variabilidade da efetividade e da razão de custo-efetividade incremental com diferentes classificações de usuários de estatina (incidentes e prevalentes). Método: Um modelo de microssimulação de Markov com 5 estados, ciclos anuais e horizonte temporal de 20 anos, com taxas de desconto de 5% foi desenvolvido. As probabilidades de transição para mortalidade por todas as causas foram extraídas após pareamento por escore de propensão dos dados e tratamento de dados ausentes de uma base secundária de registro assistencial com linkage determinístico com a base de mortalidade do Ministério da Saúde. As probabilidades dos desfechos não-fatais foram obtidas na literatura. As medidas de efetividade (QALY) foram calculadas com dados publicados dos domínios do SF-36 de um estudo realizado com a população do mesmo hospital, no mesmo período e com as mesmas condições clínicas e transformados em medidas de utilidade por modelo validado. Somente custos diretos na perspectiva do reembolso do SUS foram considerados. Resultados: 3.150 pacientes foram pareados após o escore de propensão, 1.050 não usuários de estatina, 1.050 usuários de estatinas classificados como prevalentes e 1.050 usuários de estatinas classificados como incidentes, com diagnóstico de doença cardiovascular isquêmica prévia, com seguimento médio de 5,1 anos. A efetividade das estatinas quando considerados todos os usuários em relação aos não usuários resultou em um HR para mortalidade de 0,992 (IC 95% 0,85; 0,96) e de 0,90 (IC 95% 0,85; 0,96) para os usuários incidentes. A RCEI comparando todos os usuários de estatinas versus não usuários foi de R$5.846,10/QALY e de R$7.275,61/QALY para os usuários incidentes. Conclusão: As estatinas diminuíram a mortalidade por todas as causas, e a análise incluindo usuários prevalentes diminui o tamanho do efeito. O tratamento possui custo-efetividade favorável dentro do limiar de disponibilidade a pagar definido, sendo modificado pela forma de extração do dado de efetividade / Background: The complete economic evaluations, such as cost-utility analysis, supported by real world data of effectiveness lead to a more realistic perspective of a health technology assessment. Objective: Perform a cost-utility analysis of statins for secondary prevention in ischemic cardiovascular disease patients based on effectiveness from real world data and evaluate the variability of effectiveness and incremental cost-effectiveness ratio (ICER) considering prevalent and incident users. Methods: A Markov microssimulation model with five states, annual cycle and time horizon (TH) of 20 years, with discount rate of 5% was developed. Transition probabilities for all cause mortality was derived from a secondary database of a teaching hospital after record linkage with national registry of mortality database and an analysis of propensity score matching and multiple imputation analysis for missing data. Non-fatal endpoints were derived from a published meta-analysis. Utility measures was calculated with a validated model to derive values from published domains of SF-36 QoL questionnaire, domains was measured for a published RCT in the same teaching hospital, over the same period, with similar age and diagnostic characteristics. Only direct costs were analyzed from the Brazilian public health reimbursement perspective. Results: 3150 patients were matched, 1050 non-statins users (CG), 1050 prevalent statins users (PSU) and 1050 incident statins users (ISU) with previous cardiovascular disease, with mean follow-up of 5,1 years. Treatment effects on the treatment group considering all statins users for all cause mortality had a hazard ratio of HR:0,992 (IC 95% 0,85 - 0,96) and HR: 0,90 (IC 95% 0,85 - 0,96) only for ISU. The ICER comparing all users with non-users was R$5.846,10 per QALY and for ISU was R$7.275,61 per QALY. Conclusion: Real world evidence demonstrated that statins are an effective treatment to reduce all cause mortality in secondary prevention and are a cost-effective strategy considering the willingness to pay established, but the prevalent users resulted in less effectiveness of the drug when included in the analysis and influenced the ICER

Introdução: Câncer de mama é o mais comum em mulheres. Embora sua incidência ainda esteja em ascensão, as taxas de recorrência e mortalidade têm diminuído, em especial nos países desenvolvidos. Assim o câncer pode ser considerado um choque transitório que não impede que os sobreviventes retomem a normalidade em suas vidas, incluindo atividades laborais. Na América do Norte e Europa, as taxas de RT entre as pacientes com câncer de mama variam de 24-66% após 6 meses e 53-82% após 36 meses de diagnóstico. Os fatores mais associados ao RT são: idade, quimioterapia, sequelas da terapia do câncer e apoio do empregador e colegas de trabalho. Esses achados, no entanto, variam sugerindo que outros fatores e até aspectos de diferentes legislações podem interferir no RT. Na América Latina há escassez de dados sobre RT após o diagnóstico de câncer de mama. Objetivos: Avaliar as taxas de retorno ao trabalho nos meses 12 e 24 após o diagnóstico de câncer de mama e verificar a correlação de fatores à retomada ao trabalho aos 24 meses. Métodos: Estudo prospectivo observacional avaliando taxas de RT em mulheres com câncer de mama tratadas no Instituto do Câncer do estado de São Paulo, com idade > 18 e < 57 anos e que trabalhavam de forma remunerada por pelo menos 03 meses ao diagnóstico. Pacientes com doença inoperável ou metastática foram excluídas. Nos meses 06, 12 e 24 do seguimento responderam à questionários do estudo e de qualidade de vida (FACT-B), por telefone. Resultados: Entre julho/2012 e setembro/2014, 125 pacientes assinaram o TCLE. Quatro foram excluídas da análise (02 óbitos e 02 sem contato por telefone). A idade média foi de 45.1 anos (± 8,1). A maioria (94%) gostava do trabalho, 73% receberam apoio do empregador, mas apenas 29% relataram ter recebido oferta de ajuste no trabalho. Metade apresentava doença no estádio II e 93% fizeram quimioterapia como parte de seu tratamento. As taxas de RT foram 21,5%, 30,3% e 60,4% aos 06, 12 e 24 meses, após o diagnóstico de câncer de mama. Na análise multivariada os fatores que afetaram de forma positiva as taxas de RT foram: renda familiar mensal >= 02 salários mínimos (OR 17,76, IC95% 3,33-94,75, p 0,001), cirurgia conservadora da mama (OR 9,77, IC 95% 2,03-47,05, p 0,004) e oferta de ajuste no trabalho pelo empregador (OR 37,62, IC95% 2,03-47,05, p 0,004). Fatores que se associaram de forma negativa ao RT foram: terapia endócrina (OR 0,11, IC95%0,02-0,74, p 0,023) e diagnóstico de depressão após o câncer (OR 0,07, IC95% 0,01-0,63, p 0,017). Conclusões: As taxas de RT aos 12 e 24 meses após diagnóstico de câncer de mama são inferiores a maioria dos estudos conduzidos na América do Norte e Europa. Oferta de ajuste no trabalho, maior renda familiar, cirurgia conservadora da mama, terapia endócrina adjuvante e diagnóstico de depressão após o câncer de mama desempenharam importante papel no RT / Background: Breast cancer is the most common cancer in women. While its incidence has been increasing, recurrence and mortality rates have been decreasing, mainly because of better treatment options. Because of that cancer can be regarded as a transient shock that does not prevent survivors resume normality in their lives including return to their workplace. In North America and Europe return to work (RTW) rates vary among breast cancer patients from 24- 66% after 06 months and 53-82% after 36 months of diagnosis. Factors most associated with the decision to return to work are: age, chemotherapy, sequelae related to cancer therapy and support from the employer and coworkers. However, these findings vary among the different populations evaluated, suggesting that other factors and even variations in countries laws may interfere with the decision to return to work. So far there is a lack of data on RTW after breast cancer diagnosis in Latin America. Endpoints: To evaluate return to work rates on months 12 and 24 after breast cancer diagnosis, and check the correlation of some factors with the decision to return to work at 24 months. Methods: A prospective, observational study evaluating RTW rates in patients with breast cancer diagnosis, > 18 and < 57 years old and a paid work for at least 03 months at the time of dianosis. Patients with inoperable or metastatic disease were excluded. On months 6, 12 and 24 they answered a telephone interview and the quality of life questionnaire (FACT-B). Results: Between july/2012 and september/2014, 125 patients were enrolled. Two of them died and two other could not be reached by telephone, and were excluded from the analysis. Mean age was 45,1 years (± 8,1). Most of them reported that they liked their job (94%) and received support from employer (73%), but only 29,1% reported having been offered work adjustment. Half of patients had stage II disease and 93% received chemotherapy as part of their treatment. Overall, 21,5%, 30,3% and 60,4% of patients returned to work 06, 12 and 24 months after breast cancer diagnosis, respectively. In the multivariate analysis, factors associated with positive RTW outcomes included higher income (OR: 17,76, CI95% 3,33-94,75; p = 0,001), breast conserving surgery (OR: 9,77, CI95% 2,03-47,05; p = 0,004) and work adjustment (OR: 37,62, CI95% 2,03-47,05; p= 0,004). Factors associated with negative RTW outcomes included adjuvant endocrine therapy (OR: 0,11, IC95% 0,02-0,74; p = 0,023) and depression diagnosis after breast cancer diagnosis (OR: 0,07, IC95% 0,01-0,63; p = 0,017). Conclusion: RTW rates after 12 and 24 months of breast cancer diagnosis are lower than reported in North America (with exception for low income americans) and Europe. Workplace adjustments, higher income, breast conserving surgery, endocrine therapy and depression after breast cancer played an important role in the RTW decision

Background: Community acquired pneumonia (CAP) is a high incidence disease resulting in about 260,000 hospital admissions per year in Germany, more than myocardial infarction or stroke. Worldwide, CAP is the most frequent infectious disease with high lethality ranging from 1.2 % in those 20–29 years old to over 10 % in patients older than 70 years, even in industrial nations. CAP poses numerous medical challenges, which the PROGRESS (Pneumonia Research Network on Genetic Resistance and Susceptibility for the Evolution of Severe Sepsis) network aims to tackle: Operationalization of disease severity throughout the course of disease, outcome prediction for hospitalized patients and prediction of transitions from uncomplicated CAP to severe CAP, and finally, to CAP with sepsis and organ failure as a life-threatening condition. It is a major aim of PROGRESS to understand and predict patient heterogeneity regarding outcome in the hospital and to develop novel treatment concepts. Methods: PROGRESS was designed as a clinical, observational, multi-center study of patients with CAP requiring hospitalization. More than 1600 patients selected for low burden of co-morbidities have been enrolled, aiming at a total of 3000. Course of disease, along with therapy, was closely monitored by daily assessments and long-term follow-up. Daily blood samples allow in depth molecular-genetic characterization of patients. We established a well-organized workflow for sample logistics and a comprehensive data management system to collect and manage data from more than 50 study centers in Germany and Austria. Samples are stored in a central biobank and clinical data are stored in a central data base which also integrates all data from molecular assessments. Discussion: With the PROGRESS study, we established a comprehensive data base of high quality clinical and molecular data allowing investigation of pressing research questions regarding CAP. In-depth molecular characterization will contribute to the discovery of disease mechanisms and establishment of diagnostic and predictive biomarkers. A strength of PROGRESS is the focus on younger patients with low burden of co-morbidities, allowing a more direct look at host biology with less confounding. As a resulting limitation, insights from PROGRESS will require validation in representative patient cohorts to assess clinical utility. Trial registration: The PROGRESS study was retrospectively registered on May 24th, 2016 with ClinicalTrials.gov: NCT02782013

Les troubles anxieux apparaissent très tôt chez les jeunes. Les études corrélationnelles suggèrent que plusieurs facteurs, tels que le stress prénatal maternel (SPM) et la surimplication maternelle contribueraient à leur développement et à leur maintien. L’objectif de l’étude est de mieux comprendre si la surimplication maternelle est une cause ou une conséquence de l’anxiété chez l’enfant, le tout dans un contexte de stress prénatal maternel causé par un désastre naturel. L’échantillon fait partie d’un projet plus grand dans lequel les stress objectif et subjectif ont été mesurés chez les femmes enceintes lors de l’inondation en 2011 à Queensland en Australie. L’échantillon de l’étude inclut 72 dyades mère-enfant, ayant complété une tâche difficile de casse-tête, d’une durée de 5 minutes, lorsque les enfants étaient âgés de 4 ans. À partir des vidéos, les observateurs ont évalué le comportement maternel (la surimplication) et le comportement de l’enfant (l’anxiété) en continu. Des techniques d’analyse séquentielle ont été utilisées pour déterminer la probabilité qu’un comportement maternel de surimplication se produise avant ou après un comportement anxieux et vice versa. Les corrélations obtenues entre le SPM (objectif et subjectif), le comportement anxieux de l’enfant et le comportement de surimplication maternelle n’ont pas été significatives. Le comportement de surimplication n’est pas un médiateur entre le SPM (objectif et subjectif) et le comportement anxieux de l’enfant. Toutefois, les chances qu’un comportement maternel de surimplication suive un comportement anxieux chez l’enfant étaient plus grandes que les chances qu’un comportement anxieux suive un comportement maternel de surimplication. Nos résultats suggèrent que le stress prénatal maternel n’est pas associé avec le comportement de surimplication maternelle, ni avec le comportement anxieux de l’enfant. La surimplication maternelle est plutôt une réaction au comportement anxieux de l’enfant. Il est essentiel, pour de futures recherches, de se rappeler que l’enfant est un participant actif dans la relation mère-enfant et qu’il est important d’étudier la bidirectionnalité de la relation pour mieux comprendre et pour mieux intervenir. / Anxiety disorders start very early in young people and several factors, such as prenatal maternal stress (PNMS) and maternal overinvolvement are believed to contribute to their development and maintenance. The objective of this study is to better understand if the overinvolvement of the mother is a cause or a consequence of the child’s anxiety, all in a context of prenatal maternal stress due to a natural disaster. The sample is part of a larger project that assessed the objective hardship and the subjective distress in women who were pregnant during the 2011 Queensland flood in Australia. The current sample included 72 mother-child dyads, completing a 5-minute puzzle task when the children were 4 years old. Observers rated maternal (overinvolved) and child (anxious) behaviors from videos continuously. Sequential analysis techniques were used to determine the likelihood of a maternal overinvolved behavior occurring before or after a child anxious behavior and vice versa. The correlations between PNMS (objective and subjective) and the child's anxiety and maternal over-involvement were not significant. Maternal overinvolvement did not mediate the relationship between PNMS (objective and subjective) and anxiety in children. However, the odds of maternal overinvolvement following child anxiety, was stronger than the odds of child anxiety following maternal overinvolvement. Our results suggest that prenatal maternal stress is not associated with maternal overinvolvement nor with child anxiety. It also suggests that maternal overinvolvement is a reaction to the child’s anxious behavior rather than a cause of it. Future research needs to keep in mind that the child is an active participant in the mother-child relationship and that it is important to study the bidirectionality of the relationship to better understand it and intervene.

lt has been shown that engagement in secondary tasks may contribute to cyclists crash risk [1 ], meditated by cycling errors or risky behaviors. For influences on secondary task: engagement, it is generally found that phone use is negatively correlated with age. In most studies, males are more found engaged in phone tasks than females. lt was also found that users of a bicycle-sharing program more often to wear headphones and engage in more unsafe behavior. The use of safety gear (e.g., wearing a helmet, using reflectors) is often negatively correlated with distracted cycling. Also, cyclists engaged in a secondary task exhibit other risky behaviors more often [2]. The present study's first aim was to get (an updated) estimate of the observable frequency of different secondary tasks, use of additional safety equipment, and rule violations while riding bicycles and e-scooters in Germany. The second aim was to examine possible differences in secondary task: engagement, use of additional safety equipment, and rule violations between different types of users of the cycling infrastructure, i.e., riders of conventional bikes, e-bikes, scooters, and e-scooters. A third aim was to explore whether riders' secondary task engagement is related to active safety precautions (e.g., wearing a helmet), traffic rule violations, and at-fault conflicts and if there are rider profiles regarding safety-related behaviors. As the study is explorative, no hypotheses were formulated. [From: Introduction]

Background and aims Heart failure (HF) is a major cause of serious morbidity and death in the population and one of the leading medical causes of hospitalization among people older than 60 years. The aim of this thesis was to describe how to create and how to analyze a Heart Failure Registry with emphasis on Anemia and Quality of Life. (Paper I) We described the creation of the Swedish Heart Failure Registry (SwedeHF) as an instrument, which may help to optimize the handling of HF patients and show how the registry can be used to improve the management of patients with HF. (Paper II) In order to show how to analyze a HF registry we investigated the prevalence of anemia, its predictors, and its association with mortality and morbidity in a large cohort of unselected patients with HFrEF included in the SwedeHF, and to explore if there are subgroups of HF patients identifying high--‐risk patients in need of treatment. (Paper III) In order to show another way of analyzing a HF registry we assessed the prevalence of, associations with, and prognostic impact of anemia in patients with HFmrEF and HFpEF. (Paper IV) Finally we examined the usefulness of EQ--‐ 5D as a measure of patient--‐reported outcomes among HF patients using different analytical models and data from the SwedeHF, and comparing results about HRQoL for patients with HFpEF and HFrEF. Methods An observational study based on the SwedeHF database, consisting of about 70 variables, was undertaken to describe how a registry is created and can be used (Paper I). One comorbidity (anemia) was applied to different types of HF patients, HFrEF (EF &lt;40%) (II) and HFmrEF (EF 40--‐49% ) or HFpEF (&gt; 50%) (III) analyzing the data with different statistical methods. The usefulness of EQ--‐5D as measure of patient--‐ reported outcomes was studied and the results about HRQoL were compared for patients with HFpEF and HFrEF (IV). Results In the first paper (Paper I) we showed how to create a HF registry and presented some characteristics of the patients included, however not adjusted since this was not the purpose of the study. In the second paper (Paper II) we studied anemia in patients with HFrEF and found that the prevalence of anemia in HFrEF were 34 % and the most important independent predictors were higher age, male gender and renal dysfunction. One--‐year survival was 75 % with anemia vs. 81 % without (p&lt;0,001). In the matched cohort after propensity score the hazard ratio associated with anemia was for all--‐cause death 1.34. Anemia was associated with greater risk with lower age, male gender, EF 30--‐39%, and NYHA--‐class I--‐II. In the third paper (Paper III) we studied anemia in other types of HF patients and found that the prevalence in the overall cohort in patients with EF &gt; 40% was 42 %, in HFmrEF 38 % and in HFpEF (45%). Independent associations with anemia were HFpEF, male sex, higher age, worse New York Heart Association class and renal function, systolic blood pressure &lt;100 mmHg, heart rate ≥70 bpm, diabetes, and absence of atrial fibrillation. One--‐year survival with vs. without anemia was 74% vs. 89% in HFmrEF and 71% vs. 84% in HFpEF (p&lt;0.001 for all). Thus very similar results in paper II and III but in different types of HF patients. In the fourth paper (Paper IV) we studied the usefulness of EQ--‐5D in two groups of patients with HF (HFpEF and HFrEF)) and found that the mean EQ--‐5D index showed small reductions in both groups at follow--‐up. The patients in the HFpEF group reported worsening in all five dimensions, while those in the HFrEF group reported worsening in only three. The Paretian classification showed that 24% of the patients in the HFpEF group and 34% of those in the HFrEF group reported overall improvement while 43% and 39% reported overall worsening. Multiple logistic regressions showed that treatment in a cardiology clinic affected outcome in the HFrEF group but not in the HFpEF group (Paper IV). Conclusions The SwedeHF is a valuable tool for improving the management of patients with HF, since it enables participating centers to focus on their own potential for improving diagnoses and medical treatment, through the online reports (Paper I). Anemia is associated with higher age, male gender and renal dysfunction and increased risk of mortality and morbidity (II, III). The influence of anemia on mortality was significantly greater in younger patients in men and in those with more stable HF (Paper II, III). The usefulness of EQ--‐5D is dependent on the analytical method used. While the index showed minor differences between groups, analyses of specific dimensions showed different patterns of change in the two groups of patients (HFpEF and HFrEF). The Paretian classification identified subgroups that improved or worsened, and can therefore help to identify needs for improvement in health services (Paper IV).

L’hypoglycémie est une barrière au traitement du diabète de type 1 (DbT1). La collation au coucher est recommandée pour prévenir l’hypoglycémie nocturne (HN), mais son efficacité n’est pas démontrée. Objectif : Déterminer si une prise alimentaire en soirée est associée à la survenue d’HN. Étude observationnelle : 100 DbT1 ont porté un lecteur de glucose en continu et complété un journal alimentaire pendant 72 heures. L’HN est survenue durant 28 % des nuits. Une prise alimentaire en soirée n’était pas associée à l’HN. Toutefois, dans un modèle ajusté, l’apport en glucides en soirée était positivement associé aux HN (avec injection d’insuline rapide) et l’apport en protéines inversement associé aux HN (sans injection d’insuline rapide). Manger en soirée ne semble pas associé à moins d’HN. Des études contrôlées sont nécessaires pour comprendre l’effet de la collation au coucher sur le contrôle glycémique et le rôle de l’insuline rapide injectée en soirée. / Hypoglycemia remains a limiting factor of type 1 diabetes (T1D) treatment. Bedtime snack is often suggested to reduce nocturnal hypoglycemia (NH), but its effectiveness is not supported by evidence-based data. Objective: To determine the association between post-dinner dietary intake and NH occurrence. This is an observational study during which 100 T1D wore a blinded continuous glucose monitoring system and completed a food diary for 72 hours. NH occurred on 28 % of the 282 nights studied. Post-dinner dietary intake was not associated with NH. However, in multivariate models, carbohydrate intake was positively associated with NH (when rapid insulin was injected) and protein intake was inversely associated with NH (without rapid insulin injected). Post-dinner dietary intake does not seem to be associated with a reduce occurrence of NH. Further studies are needed to better understand the impact of bedtime snack on glycemic control and the role of the injection of rapid insulin in the evening.

Au cours des dernières années, le développement des connaissances au niveau de l’étiologie de la maladie ainsi que l’arrivée de nouveaux médicaments et de lignes directrices guidant la pratique clinique sont susceptibles d’avoir entraîné une meilleure gestion de la polyarthrite rhumatoïde (PAR) et de l’ostéoporose, une comorbidité fréquente chez ces patients. Dans cette thèse, trois questions de recherche sont étudiées à l’aide des banques de données administratives québécoises (RAMQ, MED-ÉCHO). Une première étude documente l’utilisation des médicaments pour la PAR au Québec. À ce jour, il s’agit de la seule étude canadienne à rapporter les tendances d’utilisation des DMARD (disease-modifying antirheumatic drug) biologiques depuis leur introduction dans la pratique clinique. Au cours de la période à l’étude (2002-2008), l’utilisation de DMARD (synthétiques et biologiques) a augmenté légèrement dans la population atteinte de PAR (1,9%, 95% CI : 1,1 - 2,8). Cependant, malgré la présence de recommandations cliniques soulignant l’importance de commencer un traitement rapidement, et la couverture de ces traitements par le régime général d’assurance médicaments, les résultats démontrent une initiation sous-optimale des DMARD chez les patients nouvellement diagnostiqués (probabilité d’initiation à 12 mois : 38,5%). L’initiation de DMARD était beaucoup plus fréquente lorsqu’un rhumatologue était impliqué dans la provision des soins (OR : 4,31, 95% CI : 3,73 - 4,97). Concernant les DMARD biologiques, le facteur le plus fortement associé avec leur initiation était l’année calendrier. Chez les sujets diagnostiqués en 2002, 1,2 sur 1 000 ont initié un DMARD biologique moins d’un an après leur diagnostic. Pour ceux qui ont été diagnostiqués en 2007, le taux était de 13 sur 1 000. Les résultats démontrent que si la gestion pharmacologique de la PAR s’est améliorée au cours de la période à l’étude, elle demeure tout de même sous-optimale. Assurer un meilleur accès aux rhumatologues pourrait, semble-t-il, être une stratégie efficace pour améliorer la qualité des soins chez les patients atteints de PAR. Dans une deuxième étude, l’association entre l’utilisation des DMARD biologiques et le risque de fractures ostéoporotiques non vertébrales chez des patients PAR âgés de 50 ans et plus a été rapportée. Puisque l’inflammation chronique résultant de la PAR interfère avec le remodelage osseux et que les DMARD biologiques, en plus de leur effet anti-inflammatoire et immunosuppresseur, sont des modulateurs de l’activité cellulaire des ostéoclastes et des ostéoblastes pouvant possiblement mener à la prévention des pertes de densité minérale osseuse (DMO), il était attendu que leur utilisation réduirait le risque de fracture. Une étude de cas-témoin intra-cohorte a été conduite. Bien qu’aucune réduction du risque de fracture suivant l’utilisation de DMARD biologiques n’ait pu être démontrée (OR : 1,03, 95% CI : 0,42 - 2,53), l’étude établit le taux d’incidence de fractures ostéoporotiques non vertébrales dans une population canadienne atteinte de PAR (11/1 000 personnes - années) et souligne le rôle d’importants facteurs de risque. La prévalence élevée de l’ostéoporose dans la population atteinte de PAR justifie que l’on accorde plus d’attention à la prévention des fractures. Finalement, une troisième étude explore l’impact de la dissémination massive, en 2002, des lignes directrices du traitement de l’ostéoporose au Canada sur la gestion pharmacologique de l’ostéoporose et sur les taux d’incidence de fractures ostéoporotiques non vertébrales chez une population de patients PAR âgés de 50 ans et plus entre 1998 et 2008. Étant donné la disponibilité des traitements efficaces pour l’ostéoporose depuis le milieu des années 1990 et l’évolution des lignes directrices de traitement, une réduction du taux de fractures était attendue. Quelques études canadiennes ont démontré une réduction des fractures suivant une utilisation étendue des médicaments contre l’ostéoporose et de l’ostéodensitométrie dans une population générale, mais aucune ne s’est attardée plus particulièrement sur une population adulte atteinte de PAR. Dans cette étude observationnelle utilisant une approche de série chronologique, aucune réduction du taux de fracture après 2002 (période suivant la dissémination des lignes directrices) n’a pu être démontrée. Cependant, l’utilisation des médicaments pour l’ostéoporose, le passage d’ostéodensitométrie, ainsi que la provision de soins pour l’ostéoporose en post-fracture ont augmenté. Cette étude démontre que malgré des années de disponibilité de traitements efficaces et d’investissement dans le développement et la promotion de lignes directrices de traitement, l’effet bénéfique au niveau de la réduction des fractures ne s’est toujours pas concrétisé dans la population atteinte de PAR, au cours de la période à l’étude. Ces travaux sont les premiers à examiner, à l’aide d’une banque de données administratives, des sujets atteints de PAR sur une période s’étalant sur 11 ans, permettant non seulement l’étude des changements de pratique clinique suivant l’apparition de nouveaux traitements ou bien de nouvelles lignes directrices, mais également de leur impact sur la santé. De plus, via l’étude des déterminants de traitement, les résultats offrent des pistes de solution afin de combler l’écart entre la pratique observée et les recommandations cliniques. Enfin, les résultats de ces études bonifient la littérature concernant la qualité des soins pharmacologiques chez les patients PAR et de la prévention des fractures. / Over the past two decades, progresses in the understanding of disease etiology, the arrival of new drugs, and the development of clinical practice guidelines may have led to a better pharmacological management of rheumatoid arthritis (RA) and osteoporosis, a common comorbidity. In this thesis, three research questions were investigated using well characterized administrative databases (RAMQ and MED-ECHO). A first study documented RA drug use in the province of Quebec. To this date, this is the only Canadian study to report on patterns of biologic DMARD use since their introduction in clinical practice. Over our study time horizon (2002-2008), the use of any DMARDs (synthetic and biologic) slightly increased in the overall RA population (1.9%, 95% CI: 1.1-2.8). However, despite clinical practice guidelines stressing the importance of early treatment, and the reimbursement of treatments by the Quebec drug plan, the results demonstrated suboptimal DMARD initiation in newly diagnosed RA (probability at 12 months: 38.5%), though DMARD initiation increased when rheumatologists were overseeing care (OR: 4.31, 95%CI: 3.73-4.97). For biologic DMARDs, the strongest predictor of initiation was the calendar year of study entry. Of subjects newly diagnosed in 2002, 1.2 in 1000 had a biologic initiated within one year, while for those newly diagnosed in 2007, it was 13.0 in 1000. The results showed that the pharmacological management of RA is improving over time, but remains below expectations. Ensuring better access to rheumatologists should be an area of focus in order to enhance the quality of RA care. A second study reported on biologic DMARD use and the risk of non-vertebral osteoporotic fractures in RA patients aged ≥50 years. Because chronic inflammation in RA interferes with bone remodeling and biologic DMARDs, in addition to their anti-inflammatory and immunosuppressive effects, are modulators of the cellular activity of osteoblasts and osteoclasts possibly leading to the preservation of bone mineral density (BMD), it was believed that their use may reduce the risk of fractures. A nested-case control study was conducted. Although a reduction in the risk of fractures subsequent to biologic DMARD use could not be demonstrated (OR: 1.03, 95% CI: 0.42-2.53), the study established the incidence rate of non-vertebral osteoporotic fractures in a Canadian RA population (11/1000 person-years) and highlighted some important risk factors. The high prevalence of osteoporosis in the RA population justifies that more attention be paid to preventing fractures. Finally, a third study investigated the impact of the 2002 Canadian osteoporosis guidelines on the pharmacological management of osteoporosis and on the rates of non-vertebral osteoporotic fractures in a RA population aged ≥50 years between 1998 and 2008. With the availability of effective osteoporosis treatments since the mid 90s, and the evolving clinical practice guidelines, a reduction in the rate of fractures was expected. Some Canadian studies have shown reductions in the rate of fractures following broader use of osteoporosis drugs and BMD testing in a general adult population, but none have specifically investigated the impact in RA. In this observational study using a time series approach, no reduction in the rate of fractures after 2002 (post guidelines dissemination) could be demonstrated. However, the use of osteoporosis drugs, BMD testing, and provision of post fracture osteoporosis care improved. This study demonstrated that years of availability of effective preventive measures and investments in the development and promotion of clinical practice guidelines have not yet translated into further reduction in the rate of fractures in our RA population over our study time horizon. This body of work is the first to examine, using healthcare administrative data, subjects with RA over a period of 11 years, allowing not only to study the changes in clinical practice following the introduction of new treatments and guidelines, but also to capture the impact on health. In addition, by studying predictors of treatment, the results provide good insights in terms of solutions to fill the gap between the observed clinical practice and guideline recommendations. Finally, the results of these studies substantiate the literature regarding the quality of RA care and the prevention of fractures.

Les patients atteints de diabète de type 1 (DbT1) présentent un risque accru de maladies cardiovasculaires. De plus, sous l’influence sociétale et iatrogénique, leur profil de risque cardiométabolique a défavorablement évolué au cours des dernières décennies. Cette population aurait aussi davantage de barrières à l’adoption d’un mode de vie sain. L’objectif de ce mémoire visait à explorer le mode de vie des adultes DbT1 et sa relation avec les facteurs de risque cardiométabolique. Une étude transversale observationnelle menée auprès de 124 adultes DbT1 a permis d’obtenir une caractérisation de leur profil cardiométabolique et de leurs habitudes de vie. Les résultats démontrent que la majorité des adultes atteints de DbT1 n'adoptent pas les saines habitudes de vie recommandées (bonne qualité alimentaire, pratique régulière d’activité physique, non fumeur) et que ceux-ci présentent un profil cardiométabolique altéré comparativement aux adultes DbT1 qui les adoptent. De plus, il y aurait une relation proportionnelle entre l’adoption d’un plus grand nombre de saines habitudes de vie et l’amélioration du profil cardiométabolique. Des études d’interventions ciblant les patients DbT1 et visant à améliorer ces trois grandes composantes des habitudes de vie sont nécessaires. / Cardiovascular disease is a major cause of morbidity and mortality in patients with type 1 diabetes (T1D). In addition, over the past decades, there was a major upward shift in the prevalence of cardiometabolic risk factors (central obesity, insulin resistance, hypertension and dyslipidemia) among this population which could have either an additive or synergistic effect on cardiovascular disease risk. Those patients also have more barriers to the adoption of a healthy lifestyle. The objective of this master thesis was to explore the lifestyle habits of adults with T1D and its relationship with cardiometabolic risk factors. An observational cross-sectional study among 124 adults with T1D has resulted in a characterization of their cardiometabolic profile and their lifestyle habits. The results show that the majority of T1D adults do not adopt the recommended healthy lifestyles habits (good diet quality, regular physical activity, none-smoking status) and present altered cardiometabolic profile compared to those who adopt them. In addition, there would be a proportional relationship between the adoption of a healthier lifestyle and the improvement of the cardiometabolic profile. Prospective studies and clinical trials interventions among patients with T1D are although needed to confirm these findings.

Os efeitos dos dados defeituosos sobre os resultados dos processos analíticos são notórios. Aprimorar a qualidade dos dados exige estabelecer alternativas a partir de vários métodos, técnicas e procedimentos disponíveis. O processo de Avaliação da Qualidade dos Dados - pAQD - provê relevantes insumos na definição da alternativa mais adequada por meio do mapeamento dos defeitos nos dados. Relevantes abordagens computacionais apoiam esse processo. Tais abordagens utilizam métodos quantitativos ou baseados em asserções que usualmente restringem o papel humano a interpretação dos seus resultados. Porém, o pAQD depende do conhecimento do contexto dos dados visto que é impossível confirmar ou refutar a presença de defeitos baseado exclusivamente nos dados. Logo, a supervisão humana é essencial para esse processo. Sistemas de visualização pertencem a uma classe de abordagens supervisionadas que podem tornar visíveis as estruturas dos defeitos nos dados. Apesar do considerável conhecimento sobre o projeto desses sistemas, pouco existe para o domínio da avaliação visual da qualidade dos dados. Isto posto, este trabalho apresenta duas contribuições. A primeira reporta uma taxonomia que descreve os defeitos relacionados aos critérios de qualidade da acuracidade, completude e consistência para dados estruturados e atemporais. Essa taxonomia seguiu uma metodologia que proporcionou a cobertura sistemática e a descrição aprimorada dos defeitos em relação ao estado-da-arte das taxonomias. A segunda contribuição reporta relacionamentos entre propriedades-defeitos que estabelecem que certas propriedades visuais-interativas são mais adequadas para a avaliação visual de certos defeitos em dadas resoluções de dados. Revelados por um estudo de caso múltiplo e exploratório, esses relacionamentos oferecem indicações que reduzem a subjetividade durante o projeto de sistemas de visualização de apoio a avaliação visual da qualidade dos dados. / The effects of poor data quality on the reliability of the outcomes of analytical processes are notorious. Improving data quality requires alternatives that combine procedures, methods, techniques and technologies. The Data Quality Assessment process - DQAp - provides relevant and practical inputs for choosing the most suitable alternative through a data defects mapping. Relevant computational approaches support this process. Such approaches apply quantitative or assertions-based methods that usually limit the human interpretation of their outcomes. However, the DQAp process strongly depends on data context knowledge since it is impossible to confirm or refute a defect based only on data. Hence, human supervision is essential throughout this process. Visualization systems belong to a class of supervised approaches that can make visible data defect structures. Despite their considerable design knowledge encodings, there is little support design to data quality visual assessment. Therefore, this work reports two contributions. The first reports a taxonomy that organizes a detailed description of defects on structured and timeless data related to the quality criteria of accuracy, completeness and consistency. This taxonomy followed a methodology which enabled a systematic coverage of data defects and an improved description of data defects in regard to state-of-art literature. The second contribution reports a set of property-defect relationships that establishes that certain visual and interactive properties are more suitable for visual assessment of certain data defects in a given data resolution. Revealed by an exploratory and multiple study case, these relationships provides implications that reduce the subjectivity in the visualization systems design for data quality visual assessment.