Psoriasis is a chronic, immunological and systemic disease with an estimated prevalence of about 2-3 percent. Psoriasis is associated with the joint disease psoriasis arthropathy. There are several treatments options available for psoriasis and patients with moderate to severe psoriasis generally need systemic agents. In 2004 biologics were introduced for patients with moderate to severe psoriasis in Sweden. The overall objective of this thesis was to assess the relationship between Health Related Quality of Life (HRQOL) and clinical outcome measures in psoriasis patients, to analyse the effectiveness of biologics in psoriasis in everyday clinical practice and to explore how costs of the psoriasis population changed after the introduction of biologics in Sweden. Methods: Research was based on national administrative registers and PsoReg, the Swedish registry for systemic treatment of psoriasis. In a cross-sectional study (paper I) the three outcome measures: the generic HRQOL measure EQ-5D, the dermatology specific HRQOL measure the Dermatology Life Quality Index (DLQI) and the clinical measure of skin involvement, Psoriasis Area and Severity Index (PASI), were analysed by demographic characteristics. The generic EQ-5D among psoriasis patients was compared to previously published values for the general population in Sweden. Relationships between measures were examined with correlation tests and regression analysis. A longitudinal study included patients registered in PsoReg who switched to a biologic agent for the first time during registration (paper II). The three outcomes EQ-5D, DLQI, and PASI were analysed before and after switch in the overall patient group and in subgroups. The relative effectiveness of continuing with the standard care of conventional treatment compared to switching from standard care to biologics was analysed in patients with moderate to severe psoriasis (paper III). Patients in PsoReg were matched with propensity scores and average treatment effects were estimated. The estimated outcomes were the change of EQ-5D, DLQI, and PASI. Patients were identified in national registers at the National Board of Health and Welfare when analysing costs; either by a registration of a psoriasis diagnosis in the national patients register and/or by a registration in the prescribed drugs register of a topical treatment with calcipotriol, a substance which has the indication psoriasis only (paper IV). Direct costs included patients’ visits in specialist health care and prescribed drugs used for psoriasis treatment, retrieved from the national patients register and the prescribed drugs register, respectively. Indirect costs included productivity loss in terms of sick leave and disability pension, which estimated as excess costs compared to controls. Controls were selected from the normal population and matched on sex, age and municipality. Productivity loss was estimated based on data from the Longitudinal integration database for health insurance and labour market studies at Statistics Sweden. Results: Patients with moderate to severe psoriasis had significantly lower HRQOL in EQ-5D than the general population (paper I). Women rated their HRQOL lower than men, even though men had more severe clinical skin involvement than women. (paper I). The generic measure EQ-5D and the dermatology-specific DLQI had moderate correlations whereas EQ-5D had low correlation with the clinical measure PASI (paper I). Patients who switched to a biologic agent during registration in PsoReg had significant improvements in all outcomes (paper II). Patients who fulfilled the criteria for moderate to severe psoriasis had the highest benefits of the biologic agents (paper II). The matched conventionally and biologically treated patients with moderate to severe psoriasis were essentially equal in important observable variables (paper III). The subgroup of patients not responding to conventional treatment had high potential benefits of biologic agents (paper III). Individuals with psoriasis had sick leave and disability pension to a larger extent than their matched controls (paper IV). Direct costs increased, whereas the indirect costs of productivity loss decreased between 2006 and 2009 (paper IV). Conclusion: Psoriasis is associated both with direct costs and indirect costs, and it has a negative impact on patients’ HRQOL. When evaluating psoriasis treatments and making decisions about treatment guidelines, both generic, dermatology-specific HRQOL measures, and clinical measures are necessary; as they answer to different needs. Although dependent on data quality, generalisability, and current pricing, results suggest that conventional treatments are suitable as first line and biologic agents as second line treatment. Results indicate that the different types of systemic treatments are not allocated optimally among patients with psoriasis in Swedish clinical practice.

Hirsutism is excessive hair growth in women. The prevalence is estimated at 5%. The aim of this thesis was to describe different aspects of how life is affected for women suffering from hirsutism. Both qualitative and quantitative methods were used. Study I showed that hirsutism deeply affects women’s experiences of their bodies in a negative way and was experienced as a life sorrow. In Study II the patient-physician relationship was described. The patient-physician relationship from the patient’s perspective was suboptimal, as most meetings included feelings of being rejected and even humiliation. In Study III the aim was to translate and psychometrically evaluate an instrument that measures perceived social support, “The Multidimensional Scale of Perceived Social Support” (MSPSS). The translation was performed according to WHO:s official process, and validation was performed in a sample that consisted of 281 participants, 127 women with hirsutism (main sample) and 154 nursing students. MSPSS had good psychometric properties with regard to factor structure, construct validity, internal consistency and reproducibility. Study IV described different aspects of HRQoL in the main sample, the correlation of anxiety, depression, level of hairiness, age and BMI. The F-G scores were dichotomized into minor (F-G ≤14) and major (F-G≥15) hair growth. Higher levels of hair growth were significantly correlated to a lower level of QoL measured by DLQI, EQ-5D and symptoms of both anxiety and depression measured by HADS. Study V investigated if social support was associated with quality of life and outcome of HRQoL compared to a reference group of women (n=1115). SF-36, the MSPSS and the F-G scale were used. Compared to the reference group, women with hirsutism reported lower quality of life in all dimensions of SF-36 (p<0.01) The dimension most affected was vitality (VT=41.2), which had a lower value than has been reported for patients with MS and myasthenia gravis. A Multiple Regression Analysis showed a significant relation between quality of life and social support, indicating its importance for the ability to adapt, in spite of low quality of life.

Bakgrund: Höftfrakturer håller på att bli ett stort hälso- och vårdproblem på grund av den snabbt växande åldrande befolkningen. Det är därför viktigt att undersöka hur höftfrakturer påverkar de äldres hälsorelaterade livskvalitet. Syfte: Syftet med denna studie var att undersöka hur höftfraktur hos äldre personer, över 60 år, påverkar deras uppfattade hälsorelaterade livskvalitet. Metod: Studien är en litteraturöversikt och består av 12 vetenskapliga originalartiklar med kvantitativ ansats. Resultat: Höftfrakturer har en negativ påverkan av individens hälsorelaterade livskvalitet. Framförallt försämras individens rörlighet och självständighet. Ålder utgör den främsta bidragande faktorn och störst återhämtning sker under det första halvåret. Efter ett år är den fortsatta återhämtningen måttlig eller obefintlig.  Slutsats: Höftfraktur har en direkt negativ påverkan hos den enskilda individens funktionella rörlighet och psykosociala omständigheter vilket negativt påverkar individens hälsorelaterade livskvalitet. För att möta kommande vårdbehov skulle investeringar i mer resurser behövas, för att aktivt arbeta med att utveckla rehabiliteringsprogram för individer som råkat ut för höftfraktur, för att minska bestående besvär och tidigt avvärja vidare komplikationer. Samt att sjukvården aktivt informerar om höftfrakturer, så att patienter har realistiska förväntningar på sin återhämtning. För fortsatt forskning bör fler mätinstrument än EQ-5D inkluderas för att skapa en bättre helhetssyn av individens hälsosituation.

Electronic data capture technologies, such as interactive voice response (IVR) systems, are emerging as important alternatives for collecting self-reported data. The purpose of this research was to assess the measurement equivalence between the original paper-based versions and the adapted interactive voice response (IVR) versions of the EQ-5D and the QLQ-C30. Furthermore, we examined the test-retest reliability of two consecutive administrations of the IVR versions of the EQ-5D and the QLQ-C30. The comparison of the paper and IVR versions of the EQ-5D was conducted utilizing a crossover design with subjects randomly assigned to one of two assessment orders: 1) paper then IVR or 2) IVR then paper. A convenience sample of in-treatment outpatient cancer clinic patients (n=139) were asked to complete each assessment two days apart. For the test-retest component, outpatient cancer clinic patients (n=127) were asked to complete the IVR-based EQ-5D twice, two days apart. The analyses tested for mean differences (paired t-test) and test-retest reliability (ICC).In the crossover analysis, ten of the fifteen mean differences analyzed for the scales and items of the QLQ-C30 were within the equivalence interval set a priori. The ICCs for the scales and items of the QLQ-C30 ranged from 0.698 to 0.899. Two of the items, insomnia and appetite loss, did not meet our threshold of being statistically different from an ICC of 0.70. The EQ-5D index score means were equivalent between paper and IVR, however the EQ VAS score differences were not wholly contained in the equivalence interval. The ICCs were above 0.890 for the index and the EQ VAS. In the test-retest analysis, the ICCs for the nine multi-item scales for the QLQ-C30 were all above 0.69, ranging from 0.698 to 0.891. Ten of the fifteen mean differences analyzed were within the equivalence interval set a priori. For the EQ-5D, the mean differences were wholly contained within the equivalence intervals for both the index and the EQ VAS and the ICCs were significantly different from 0.70. Overall, the IVR version of the questionnaires provided psychometrically equivalent results to those obtained on the original paper version and showed good stability over time.

The EQ-VAS, a measure of self-reported health status, has been operationalized in ways that depart from the original format. The primary purpose of the study was to examine the equivalence of the original paper-based vertical format with a touch screenbased horizontal format. Non-probability sampling was used to recruit 314 subjects intended to reflect the primary socio-demographic characteristics of the general adult population. A two-part questionnaire completed roughly 10 minutes apart was administered in a randomized crossover design. One part was the original paper-based 20cm vertical EQ-VAS; the other part was touch screen computer-based (designed by Assist Technologies) and included, among other items/scales, a horizontal EQ-VAS, the SF-36, and socio-demographic items. A mean difference of ± eight points between the two versions was specified as the minimally important difference (MID). Almost a third (30.1%) of the respondents reported identical scores on both formats and 80.1% of the respondents had difference scores within ± 8 points. The 95% confidence intervals for both samples indicated that the difference in scores was relatively small and below our equivalence threshold. In addition, data collected via touch screen may be more reliable since 22% of subjects did not complete the EQ-VAS paper format as instructed. These results provided evidence for the measurement equivalence of the touch screen EQ-VAS with the original paper format. A secondary purpose was to examine the psychometric properties of an electronic version of the SF-36. Floor and ceiling effects were comparable to that observed in other studies using the paper SF-36 in the general population. All reliability coefficients exceeded 0.70; the range was from 0.75 to 0.93. There was support for the construct validity of the touch screen SF-36, as the direction and strength of the correlations between the SF-36 scales and the EQ-5D domains were as hypothesized. Overall, there was a high level of correspondence between the touch screen SF-36 scores and previously reported paper based SF-36 scores in the general population. The comparable psychometric properties and low level of missing data make touch screen questionnaires a very viable alternative to their paper-based formats.

<p><strong>Background </strong></p> <p>The EQ-5D is one of the most widely used instruments to measure health status. It consists of a descriptive profile with a corresponding scoring algorithm. Multiple scoring algorithms have since been developed from EQ-5D preference elicitation studies.</p> <p><strong>Objectives </strong></p> <p>To identify key methodological issues in the construction of EQ-5D preference elicitation studies and to assess the validity of using a standard methodology in the construction of EQ-5D scoring algorithms.</p> <p><strong>Search methods </strong></p> <p>We searched the MEDLINE, EMBASE, Cochrane Library, NHS Economic Evaluation Database, and Health Economic Evaluation Database, (1990 to 2012). The EuroQol Group website was also searched.</p> <p><strong>Selection criteria </strong></p> <p>EQ-5D preference elicitation studies that reported the directly estimated health state scores and estimated scoring algorithm.</p> <p><strong>Data collection and analysis </strong></p> <p>Two reviewers independently assessed articles for inclusion. The observed and estimated EQ-5D preference scores were compared across studies. A standard scoring algorithm with fixed variables was estimated. The model performance of the standard algorithm and the study reported algorithm were assessed and compared.</p> <p><strong>Results </strong></p> <p>A total of 38 preference elicitation studies were included in this review. Key differences identified include: method of valuation, selection of health states, transformation of health state values, and method of estimation of the scoring algorithm. The observed health state values were found to be significantly different. The predicted health state values showed high levels of rank correlation. In general, a standard scoring algorithm was found to be no different in model performance than study specific scoring algorithms, with only three studies reporting a significant better model performance using the study specified scoring algorithm.</p> <p><strong>Conclusion</strong></p> <p>Methodological differences were identified across EQ-5D valuation studies. A standard scoring algorithm may yield similar model performance to study specific scoring algorithms, however further research is needed to identify when the use of a standard algorithm is appropriate.</p> / Master of Science (MSc)

Bakgrund: Hypertoni är en global folksjukdom och den största orsaken till kardiovaskulära sjukdomar och dödlighet. Sjukdomen benämns “the silent killer” eftersom den ofta inte medför några symtom, vilket i sin tur kan innebära att individerna inte upplever sig som sjuka och därför avstår medicinering. Studier visar att många individer med hypertoni lever med nedsatt hälsa och stort lidande. Vid skattning av livskvalitet inom hälso- och sjukvården används begreppet hälsorelaterad livskvalitet (HRQoL) som i denna allmänna litteraturöversikt har valts som teoretisk referensram. Syfte: Att undersöka hälsorelaterad livskvalitet hos individer med hypertoni samt vilka faktorer som påverkar deras hälsorelaterade livskvalitet. Metod: En allmän litteraturöversikt med 19 kvantitativa studier. Huvudresultat: Individer med hypertoni hade sämre HRQoL än individer utan hypertoni. Därtill framkom att medvetenhet om sin hypertonidiagnos även bidrog till sämre HRQoL. Kvinnor hade sämre HRQoL än män. Hög ålder var en faktor som påverkar HRQoL till det sämre, liksom samsjuklighet och okontrollerat högt blodtryck. Anställning, högre inkomst och högre utbildningsnivå bidrog till bättre HRQoL. Levnadsvanor som gav positiva effekter på HRQoL var en viss alkoholkonsumtion och fysisk aktivitet. Faktorer som undersöktes och visade sig påverka HRQoL positivt var individens förmåga att hantera sin sjukdom, individens psykologiska motståndskraft, hälsolitteracitet, vårdkontinuitet, att få regelbunden medicinsk undersökning samt följsamhet till farmakologisk behandling. Slutsats: Den allmänna litteraturöversiktens resultat kan vara värdefullt för all hälso- och sjukvårdspersonal, liksom i arbetet som distriktssköterska, för att kunna stötta individer med hypertoni till en bättre hälsorelaterad livskvalitet och anpassa vården utifrån individernas enskilda förutsättningar. / Background: Hypertension is a global public disease and the leading cause of cardiovascular disease and mortality. The disease is often called the “silent killer” because it often does not cause any symptoms, which can mean that individuals do not feel sick and therefore refrain from medication. Studies show that many individuals with hypertension live with impaired health and great suffering. When estimating quality of life in health care, the term health-related quality of life (HRQoL) is used, which in this general literature review has been chosen as theoretical framework. Aim: To investigate health-related quality of life in individuals with hypertension and the factors that affect their health-related quality of life. Method: A general literature review with 19 quantitative studies.                                  Main results: Individuals with hypertension had poorer HRQoL than individuals without hypertension, in addition, a study showed that awareness of their hypertension diagnosis also contributed to poorer HRQoL. Women had lower HRQoL than men. Old age impaired HRQoL as well as comorbidity and uncontrolled high blood pressure. Employment, higher income and a higher level of education contributed to better HRQoL. Living habits which had positive effects on HRQoL were a certain alcohol consumption and physical activity. Factors examined that were found to have a positive effect on HRQoL where the individual´s’ ability to manage their illness, psychological resilience, health literacy, continuity of care, regular medical examination and adherence to pharmacological treatment. Conclusion: The results of the general literature review can be valuable for all health care and medical staff, as well as in the work as a district nurse, to be able to support individuals with hypertension to a better health-related quality of life and adapt care based on the individuals' conditions.

La notion d’instauration rapide d’un traitement efficace et agressif dans la polyarthrite débutante a renforcé la nécessité de disposer de mesures d’utilité validées afin d’optimiser l’évaluation médico-économique de ces traitements. Actuellement, il n’y a pas de consensus sur le choix du questionnaire multi-attributs à utiliser pour le calcul des utilités. L’objectif de cette thèse est donc d’étudier les niveaux, les performances et les déterminants des 2 outils indirects de mesure d’utilité (EQ-5D et SF-6D) dans la polyarthrite débutante, au travers des données de la cohorte ESPOIR, cohorte multicentrique française de polyarthrites débutantes. En conclusion, l’EQ-5D et le SF-6D ne sont pas interchangeables dans la polyarthrite débutante : ils ont des échelles, des distributions, une reproductibilité et une sensibilité au changement différentes. Ils ne donnent pas les mêmes résultats surtout quand les patients ont un mauvais état de santé et plus particulièrement une incapacité fonctionnelle importante. La distribution bimodale de l’EQ-5D, sa moins bonne reproductibilité, le fait qu’il soit moins sensible à l’amélioration, sa plus grande variabilité dans l’estimation du gain d’utilité et donc des QALYs, les problèmes soulevés par la signification d’un état « pire que la mort » (qui n’est pas celle du patient), favoriseraient plutôt l’utilisation du SF-6D dans les essais sur les nouvelles biothérapies dans la polyarthrite débutante / The explosion of drug development for rheumatoid arthritis and the revolution of early aggressive therapy for the disease have fuelled the search for better approaches to establish cost-effectiveness in early arthritis (EA), but consensus is lacking on the choice of utility instrument. We aimed to compare the EQ-5D and SF-6D, 2 indirect utility measures widely used to calculate quality-adjusted life-years (QALYs), in terms of their utility values, their performance and their determinants in a large prospective cohort of patients with EA. In conclusion, the EQ-5D and SF-6D are not interchangeable: their scales, distributions, reliability and responsiveness are different. There is systematic disagreement between the EQ-5D and SF-6D in EA especially in patients with worse clinical outcomes and more specifically with high functional disability. Several elements suggest that the SF-6D may be more appropriate for clinical trials of biologics in patients with EA: the bimodal distribution of the EQ-5D, its lower reliability and responsiveness for EA improvement than the SF-6D, its high variability in the estimation of the utility gains and thus QALYs gains and the problems raised by the meaning of a state labeled " worse than the death " (which is not the one from the patient)

La qualité de vie liée à la santé (QdV) s’est imposée comme une préoccupation essentielle dans la prise en charge des patients. Elle est devenue un des objectifs majeurs des essais cliniques.Notre revue bibliographique a montré un réel manque de données sur la QdV de la femme enceinte, puisqu’entre 2005 et 2015, elle n’a identifié que 75 articles portant sur cette problématique. Nous avons donc voulu déterminer l’évolution de la QdV chez les femmes enceintes du premier trimestre au 9ème mois, à l’aide du questionnaire EQ5D-3L version française, en différenciant les grossesses physiologiques des grossesses pathologiques, et en comparant cette évolution inter et intra grossesse. A cette fin, nous avons suivi une cohorte de 500 femmes enceintes entre 2015 et 2017 au CHU de Toulouse qui ont complété mensuellement un cahier d’observation en ligne. Après avoir réalisé une régression par morceaux, entre le 4ème et le 8ème mois, la QdV était significativement plus basse pour les grossesses pathologiques et diminuait significativement pour chaque type de grossesse. Ces résultats nous ont amené à nous demander si la QdV de la femme enceinte pouvait avoir un impact sur le mode d’allaitement à la naissance (maternel ou artificiel). A partir de cette même cohorte, où nous avions également recueilli le mode d’allaitement à la naissance, nous n’avons pas montré de lien entre QdV au cours de la grossesse et mode d’allaitement à la naissance, que ce soit lors de l’analyse mensuelle ou lors de l’analyse catégorielle qui était fonction de la QdV au 3ème mois de grossesse et de son évolution au cours de la grossesse / Health Related Quality of life (HRQoL) has emerged as a major concern in the care of patients and has become a major objective of clinical trials.Our literature review showed us a real lack of data on the QoL of the pregnant woman, since between 2005 and 2015, she identified only 75 articles dealing with this problem.We therefore wanted to determine the evolution of QoL in pregnant women from the first trimester to the 9th month using the EQ5D-3L French version, by differentiating physiological pregnancies from pathological pregnancies and comparing this inter and intra pregnancy progression. . We therefore followed a cohort of 500 pregnant women between 2015 and 2017 at Toulouse University Hospital, who completed a monthly online observation form. After performing piecemeal regression, between the 4th and 8th month, QoL was significantly lower for pathological pregnancies and significantly decreased for each type of pregnancy.These results led us to question whether the QoL of the pregnant woman could have an impact on breastfeeding at birth (maternal or artificial). From this same cohort, where we also collected breastfeeding at birth, we did not show a link between QoL during pregnancy and breastfeeding at birth, whether at birth. monthly analysis or categorical analysis that was based on QoL at the 3rd month of pregnancy and its course during pregnancy

Objective: To analyze the change in health-related quality-of-life (HRQoL) in patients with Parkinson’s disease (PD) and to identify different classes of HRQoL decline. Methods: A longitudinal cohort study was performed to assess clinical parameters (unified PD rating scale, Beck Depression Inventory) and HRQoL data (EuroQol, Parkinson’s Disease Questionnaire [PDQ]-39) at baseline, 3, 6, 12, 24, and 36 months. A total of 145 patients with PD were consecutively recruited in the county of Northern Hessia, Germany, between January and June 2000. A latent growth mixture model was applied to analyse the heterogeneity in HRQoL trajectories. Results: We successfully applied latent mixture growth modeling in order to identify different classes of HRQoL trajectories in PD. Three growth models were developed and each resulted in a four-class model of distinct patterns using the generic EuroQol instruments’ outcomes (EuroQol-5 Dimensions and visual analogue scale) and the disease-specific PDQ- 39. The four classes were defined by individual trajectory characteristics. Classes one and two represented trajectories with moderate declines over 36 months, but with different initial intercepts. Class three consisted mainly of patients who passed away during the observation period and therefore had a large HRQoL decline. Class four was characterized by a low level of HRQoL at baseline and a significant subsequent decline. Conclusions: The findings provide a more elaborate understanding of the variability in HRQoL reduction in PD over time. The classification of different HRQoL subgroups may help to explain the response of PD patients to the natural history of the disease. Future research will enable the identification of HRQoL responder subgroups on different treatment regimens.