The scope of this dissertation focuses on issues related to the quality of life of children suffering with spinal muscular atrophy (SMA) and their carers and the associated social impact on families affected by this progressive and incurable disease. It describes ethical aspects of help to families with SMA and serious decisions in relation to the need to connect to artificial ventilation. Spinal Muscular Atrophy - SMA is a motoneuron disease i.e. disease of neurons, which are responsible for conscious movements of muscles e.g. running, head movement and swallowing. The prevalence is approximately 1 newborn for 6000 live births and approximately 1 person of 40 people is the carrier of the disease. SMA affects all the bone muscles i.e. proximal muscles are often affected the most. Everyone affected is in some point in life, depending on stage and type, reliant on mechanical or electrical wheelchair, in many cases also on artificial ventilation and permanent 24hr care. Families affected by this illness accept the fact of this progressive and incurable illness differently, this dissertation reflects upon such different perceptions on quality of life of the affected children, the carers. It forms a contribution in building a foundation for organising multi-discipline teams of experts with sole purpose of therapeutical interventions, to support the child and his/hers family. The World Health Organization (WHO) defines palliative care as "improving quality of life of patients facing life-threatening illnesses, and their families, through the prevention and relief of suffering by early identification and treatment of pain and other problems, whether physical, psychological, social or spiritual." Palliative care prepares families for these situations and should be provided along with whatever treatment options families choose. This dissertation is a comprehensive information base to support children affected by SMA and their families in early care in Czech Republic and in the process of inclusive educational integration into mainstream society.

Estudo descritivo, correlacional e longitudinal que teve como objetivos estudar a validade e a confiabilidade da Burns Specific Pain Anxiety Scale-BSPAS que avalia ansiedade frente a procedimentos dolorosos e a Impact of Event Scale-IES que avalia o estresse pós-traumático. Participaram do estudo 91 indivíduos maiores de 15 anos de idade, internados na Unidade de Queimados do Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto. Seguiramse as seguintes etapas: 1ª etapa: coleta de dados relacionados à internação e ao tratamento. 2ª etapa: seguimento de manifestações dolorosas, durante sete dias, com a aplicação da Escala Visual Analógica para Dor, em três momentos diferentes do dia: a) imediatamente antes do banho e curativo (DIABC); b) imediatamente depois do banho e curativo (DIDBC); e c) dor no período de descanso, por volta das 20 horas (DDPD). 3ª etapa: no oitavo dia, aplicação da BSPAS - Versão Português, da IES - Versão Português e do Inventário de Ansiedade-Estado de Spielberger (IDATE). 4ª etapa (composta por fases A e B): entre o 4º e o 6º meses (fase A) e entre o 9º e o 12º meses, após o acontecimento da queimadura (fase B), aplicação da IES-VP, da Escala de Autoestima de Rosenberg (EAER), do Inventário de Depressão de Beck (IDB) e do SF-36. Nas três primeiras etapas, participaram do estudo 91 pacientes (64 homens e 27 mulheres). A superfície corporal queimada (SCQ) média foi de 18% (1-60%), sendo os locais anatômicos mais atingidos membros superiores (66; 72,5%), tórax (61; 67%) e cabeça/face (43; 47,3%). Os agentes causadores de queimaduras mais comuns foram os líquidos quentes (15,4%) e os agentes inflamáveis, principalmente o álcool, (38,5%). Na 4ª etapa, participaram 77 pacientes, na fase A, e 76, na fase B. Na avaliação da validade de construto convergente, a BSPAS-VP apresentou correlações fortes e positivas com a IES-VP (0,52; p<0,01), fortes e moderadas com as subescalas que avaliam pensamentos intrusivos (0,54; p<0,01) e reações de evitação (0,37; p<0,01) e correlações moderadas com as avaliações de dor: DIABC (0,32; p<0,01), DIDBC (0,31; p<0,01) e DDPD (0,31; <0,01). A IES-VP total apresentou correlações moderadas e positivas com o IDB (0,63; p<0,01), moderadas e negativas com a EAER (-0,58; p<0,01) e moderadas a baixas e negativas com os domínios Dor (r=-0,24; p<0,05), Aspectos Sociais (r=-0,34;p<0,01) e Saúde Mental (r=-0,27; p<0,05), entre o 4º e o 6º meses e baixa e de pouca aplicabilidade para a prática com o domínio Estado Geral de Saúde (r=-0,24; p<0,05), entre o 9º e o 12º meses, do SF-36. Na aplicação da BSPAS-VP e da IES-VP, observaram-se valores médios mais altos para o sexo feminino (55,15 e 63,96, respectivamente), entre os indivíduos com SCQ maior que 20% (54,90 e 62,98, respectivamente) e para os indivíduos que referiam as cicatrizes visíveis (52,53 e 61,40, respectivamente), porém, quando testadas as diferenças por meio do Teste t de Student para amostras independentes, essas não foram estatisticamente significantes. Na análise dos componentes principais por meio da Matrix de Rotação Varimax, a IES-VP apresentou-se como uma escala bidimensional e a BSPAS-VP, unidimensional, conforme a proposta original de cada escala. O Alfa de Cronbach de ambas as escalas foi alto, 0,90 para a BSPAS-VP e 0,87 para a IES-VP, mostrando forte consistência interna entre seus itens. Quando aplicado o Teste t para amostras dependentes, a IES-VP apresentou-se sensível para detectar mudanças no tempo. Conclui-se que a BSPAS-VP e a IES-VP são instrumentos confiáveis e válidos para a avaliação de ansiedade-estado relacionada à dor no paciente queimado e para a avaliação do impacto do evento, respectivamente. / This descriptive, correlation and longitudinal study aimed to study the validity and reliability of the Burns Specific Pain Anxiety Scale-BSPAS, which assesses anxiety about painful procedures, and the Impact of Event Scale-IES, which assesses post-traumatic stress. Study participants were 91 individuals over 15 years of age, hospitalized at the Burns Unity of the Ribeirão Preto Medical School Hospital das Clínicas. The following steps were followed: 1st step: data collection about hospitalization and treatment. 2nd step: follow-up of painful manifestations for seven days, applying the Visual Analogue Scale for Pain at three different times each day: a) immediately before bathing and wound dressing (DIABC); b) immediately after bathing and wound dressing (DIDBC); and c) pain in the rest period, around 20:00h (DDPD). 3rd step: on the eighth day, application of the BSPAS Portuguese Version, IES Portuguese Version and Spielbergers State Anxiety Inventory (SAI). 4th step (including phases A and B): between the 4th and 6th month (phase A) and between the 9th and 12th month after the burn event (phase B), application of the IES-VP, Rosenbergs Self-Esteem Scale (RSES), the Beck Depression Inventory (BDI) and the SF-36. In the first three steps, study participants were 91 patients (64 men and 27 women). The mean burned body surface (BBS) was 18% (1-60%), with upper limbs (66; 72.5%), thorax (61; 67%) and head/face (43; 47.3%) as the most affected anatomical sites. The most common causal agents of the burns were hot fluids (15.4%) and inflammable agents, mainly alcohol (38.5%). In the 4th step, 77 patients participated in phase A and 76 in phase B. In convergent construct validity, the BSPAS-VP presented strong and positive correlations with the IES-VP (0.52; p<0.01), strong and moderate with the subscales that assess intrusive thoughts (0.54; p<0.01) and reactions of avoidance (0.37; p<0.01) and moderate correlations with the pain assessments: DIABC (0.32; p<0.01), DIDBC (0.31; p<0.01) and DDPD (0.31; <0.01). Total IES-VP presented moderate and positive correlations with the BDI (0.63; p<0.01), moderate and negative with the RSES (-0.58; p<0.01) and moderate to low and negative with the Pain (r=-0.24; p<0.05), Social Aspects (r=-0.34;p<0.01) and Mental Health (r=-0.27; p<0.05) domains between the 4th and 6th month, besides low correlation and little applicability to practice with the General Health State (r=-0.24; p<0.05) domain of the SF-36 between the 9th and 12th month. When applying the BSPAS-VP and IES-VP, higher mean scores were observed for women (55.15 and 63.96, respectively), among individuals with a BBS of more than 20% (54.90 and 62.98, respectively) and for people who mentioned visible scars (52.53 and 61.40, respectively). When differences were tested through Students t-test for independent samples, however, these were not statistically significant. In the main components analysis through the Varimax Rotation Matrix, IES-VP appeared as a bidimensional scale and BSPAS-VP as a unidimensional scale, in accordance with each scales original proposal. Both scales obtained a high Cronbachs Alpha, 0.90 for BSPAS-VP and 0.87 for IES-VP, showing strong internal consistency of its items. When the t-test for independent samples was applied, the IES-VP showed sensitivity to detect changes over time. It is concluded that the BSPAS-VP and the IES-VP are reliable and valid instrument to assess state-anxiety related to pain in burned patients and to assess the impact of the event, respectively.

Introdução: O tratamento do câncer retal melhorou ao longo das décadas com aprimoramento e surgimento de novas terapêuticas resultando em maior sobrevida. Assim, os resultados e o impacto pós-tratamento sobre a QVRS são cada vez mais considerados e não só a ausência da doença. Objetivo: Avaliar a qualidade de vida imediata e tardia relacionada à saúde em pacientes tratados de câncer retal com intenção curativa. Método: Estudo descritivo-exploratório, com delineamento de coorte prospectivo, de caráter observacional para geração de hipóteses acerca da qualidade de vida de pacientes com câncer de reto. Conduzimos com aplicação de entrevista por questionário específico para dados demográficos; questionário estruturado EORTC QLQ-C30 e EORTC-CR38 para avaliação da QVRS aplicados no início do tratamento, três meses após a cirurgia e 12 meses após. A casuística foi composta de 58 pessoas, totalizando 29 pacientes puderam participar conforme critérios de inclusão e 12 que puderam responder os questionários após 12 meses. Os escores de cada paciente foram comparados - início, após 3 meses de intervenção e 12 meses com ou sem estoma. Os dados foram organizados em planilha Excel e análise dos dados realizada utilizando o software R (R-project) versão 3.1.2. Resultados: Após três meses houve piora da satisfação sexual, Problemas sexuais femininos e Perspectiva futura. Melhoram os Sintomas Gastrointestinais, problemas esfincterianos e perda de peso. Após 12 meses a Perspectiva futura deteriorou, porém houve melhora dos Problemas relacionados ao estoma, Problemas esfincterianos e Imagem Corporal. Conclusão: Apesar de toda complexidade do tratamento multidisciplinar do câncer de reto dentro de um serviço especializado, a qualidade de vida ficou preservada e foi satisfatória na maioria dos quesitos estudados / Introduction: The treatment of rectal cancer has improved over the decades with improvement and emergence of new therapies resulting in greater survival. Thus, the results and post-treatment impact on HRQoL are increasingly considered and not just the absence of the disease. Objective: To evaluate the immediate and late health-related quality of life in patients treated for rectal cancer with curative intent. Method: A descriptive-exploratory study, with a prospective cohort design, with an observational character to generate hypotheses about the quality of life of patients with rectal cancer. We conducted with questionnaire interview application specific to demographic data; structured questionnaire EORTC QLQ-C30 and EORTC-CR38 for the evaluation of HRQoL applied at the beginning of treatment, three months after surgery and 12 months after. The sample consisted of 58 people, totaling 29 patients who could participate according to inclusion criteria and 12 who could answer the questionnaires after 12 months. The scores of each patient were compared - beginning, after 3 months of intervention and 12 months with or without stoma. The data were organized in Excel spreadsheet and data analysis performed using software R (R-project) version 3.1.2. Results: After three months there was worsening of sexual satisfaction, Female sexual problems and Future perspective. Improve Gastrointestinal Symptoms, Sphincter Problems and Weight Loss. After 12 months, the future Perspective deteriorated, but there was improvement of the problems related to the stoma, Sphincter problems and Body Image. Conclusion: Despite the complexity of the multidisciplinary treatment of rectal cancer within a specialized service, the quality of life was preserved and was satisfactory in most of the studied questions

Introdução: O envelhecimento populacional, decorrente do aumento da expectativa de vida, foi, sem dúvida, uma importante conquista em nível global. Contudo, discute-se atualmente a necessidade premente de agregar qualidade aos anos de vida ganhos. Objetivo: Identificar fatores associados à Qualidade de Vida Relacionada à Saúde (QVRS) de idosos não institucionalizados do Município de São Paulo, em 2006. Método: Este estudo faz parte do Estudo Longitudinal SABE Saúde, Bem-Estar e Envelhecimento. Os dados foram coletados em 2006, de uma amostra representativa composta por 1.160 idosos (idade 60 anos) que responderam ao Short-Form 12 (SF-12), questionário genérico que avalia QVRS. As variáveis dependentes foram os Componentes Físico (PCS) e Mental (MCS) do SF-12 e as análises foram conduzidas separadamente segundo sexo. Buscou-se a associação com variáveis demográficas, socioeconômicas, estado de saúde, incapacidade funcional, estilo de vida e relacionamento social, utilizando o método de regressão logística múltipla. Resultados: Entre as idosas, estiveram associados a baixos escores no PCS: idade 80 anos, multimorbidade, internação, ter incontinência urinária, ter depressão, ter dificuldades para executar atividades instrumentais e básicas da vida diária (AIVD e ABVD) e não praticar atividades físicas. Baixos escores no PCS dos homens associaram-se a idade 80 anos, renda insuficiente, multimorbidade, tomar dois ou mais medicamentos, ter dificuldades para ABVD e não praticar atividades físicas. Baixos escores no MCS das mulheres estiveram associados a ser fumante, auto-percepção negativa de saúde geral e saúde bucal, ocorrência de queda no último ano e ter depressão. Baixos escores no MCS entre os homens idosos associaram-se à auto-percepção negativa de saúde, ter incontinência urinária e ter depressão. Homens mais velhos (idade 70 anos) apresentaram melhores escores no MCS em relação aos mais jovens (60-69 anos). Discussão: No PCS, as únicas variáveis coincidentes entre homens e mulheres foram idade, prática de atividades físicas, multimorbidade e dificuldade para ABVD; enquanto no MCS foram auto-percepção de saúde e depressão. A análise separada por sexo possibilitou a identificação de modelos distintos de determinantes da QVRS de idosos. Conclusão: Os fatores que se associaram significativamente ao PCS-SF12 de idosos de ambos os sexos foram: auto-percepção de saúde, multimorbidade, dificuldades para desempenhar ABVD e prática de atividades físicas. Enquanto suficiência de renda e número de medicamentos associaram-se apenas para o sexo masculino e internação, incontinência urinária, depressão e dificuldades para desempenhar AIVD, apenas para o sexo feminino. Ao MCS-SF12 de ambos os sexos associaram-se apenas auto-percepção de saúde e depressão; enquanto para o sexo masculino associaram-se também faixa etária e incontinência urinária. Já para as mulheres também apresentaram associação significativa o tabagismo, a auto-percepção de saúde bucal e quedas / Background: The world had experienced a high increased in life expectancy during the last decades, which has been undoubtedly considered as a major achievement. Because the increase of elderly population, that presents highest prevalence of chronic conditions, besides other single characteristics, several researches have been developed due to determine ways to add quality to the gained years of life. The aim of this study was to identify factors associated with elderly health-related Quality of Life (HRQoL), in São Paulo. Methods: This study is part of the Longitudinal Study SABE - Health, Welfare and Ageing (from Portuguese: Saúde, Bem-Estar e Envelhecimento). Data were collected in 2006. The sample consisted of 1,160 elderly (age 60 years) who answered the Short-Form 12 (SF-12), a generic HRQoL questionnaire. Dependent variables were Physical (PCS) and Mental Components (MCS) of SF-12. All analysis were separated by sex and the independent variables approached demographic, socioeconomic, health status, functional disability, lifestyle and social networking conditions, using the multiple logistic regression. Results: Among old women, lower PCS scores were associated with age 80 years, multimorbidity, hospitalization, urinary incontinence, depression, difficulty to perform basic and instrumental activities of daily living (BADL and IADL) and lack of physical activities. Among old men, lower PCS scores were associated with age 80 years, insufficient income, multimorbidity, taking two or more medications, difficulty to perform BADL and the lack of physical activities. Among the women, lower MCS scores were associated with being a smoker, negative self-perception of general health and oral health, occurrence of falls in the last year and depression. Among the men, lower MCS scores were associated with negative self-perception of health, urinary incontinence and depression. Older men ( 70 years) had better MCS scores than younger (60-69 years). Conclusion: Significantly associated factors with the PCS-SF12 for both male and female elderly were: self-perceived health status, multimorbidity, incapacity for BADL and physical activity; while income and number of drugs were associated only for males. Hospitalization, urinary incontinence, depression and incapacity for IADL were associated only for females. To the MCS-SF12 of both sexes were associated only self-perceived health and depression, while age and urinary incontinence were associated for males. Females MCS-SF12 also had significant association with tobacco, self-perception of oral health and falls

Submitted by Renata Lopes (renatasil82@gmail.com) on 2017-10-02T18:24:18Z No. of bitstreams: 1 andrealemoscabalzar.pdf: 1656685 bytes, checksum: e9910962b3bc92892b53761e1aa94f40 (MD5) / Approved for entry into archive by Adriana Oliveira (adriana.oliveira@ufjf.edu.br) on 2017-10-09T19:56:16Z (GMT) No. of bitstreams: 1 andrealemoscabalzar.pdf: 1656685 bytes, checksum: e9910962b3bc92892b53761e1aa94f40 (MD5) / Made available in DSpace on 2017-10-09T19:56:16Z (GMT). No. of bitstreams: 1 andrealemoscabalzar.pdf: 1656685 bytes, checksum: e9910962b3bc92892b53761e1aa94f40 (MD5) Previous issue date: 2017-08-11 / Introdução: Atividade física de vida diária e capacidade de exercício nunca foram avaliadas em pacientes com Doença de Crohn (DC) em remissão induzida por infliximabe. Objetivos: Nosso objetivo foi avaliar a atividade física de vida diária, capacidade de exercício, qualidade de vida (QV) e desordens do humor em pacientes com DC moderada a grave em remissão induzida por infliximabe, e investigar variáveis associadas com atividade física de vida diária nestes pacientes. Métodos: Um estudo transversal foi conduzido, com 26 pacientes com DC foram selecionados. Controles foram indivíduos assintomáticos com dispepsia funcional. Os pacientes foram avaliados quanto à atividade física de vida diária pelo acelerômetro triaxial, à capacidade de exercício (shuttle walk test – SWT), força de preensão manual, QV e desordens de humor. As associações entre a atividade física de vida diária e a QV ou desordens do humor foram testadas. Resultados: O número de passos dado (7446 ± 3081 vs. 7898 ± 2487), o tempo ativo (80,56 ± 41,97 min vs. 89,65 ± 24,30 min), a distância percorrida no SWT (662,69 ± 252,86 m vs. 728,82 ± 346,55 m) e a força de preensão manual (34,3 ± 11,1 kgf vs. 32,9 ± 11,9 kgf) não mostraram significância estatística entre pacientes com DC e controles. O tempo gasto deitado (116,31 ± 107,38 min vs. 63,71 ± 55,79 min) e alguns domínios da QV foram superiores em pacientes com DC (p< 0,05). Nenhuma correlação foi observada entre o nível de atividade física de vida diária e QV ou desordens de humor em pacientes com DC (p>0,05). Conclusão: Nós encontramos que pacientes com DC em remissão induzida por infliximabe tem o mesmo nível de atividade física de vida diária e capacidade de exercício quando comparados com controles. / Background: Physical activity in daily life and exercise capacity have never been assessed in Crohn’s disease (CD) patients on infliximab-induced remission. Objective: We aimed to evaluate the physical activity in daily life, exercise capacity, quality of life (QoL) and mood disorders in patients with moderate-to-severe CD on infliximab-induced remission, and to investigate variables associated with physical activity in daily life in these patients. Methods: A cross-sectional study was conducted, in which 26 patients with CD were selected. Controls were currently asymptomatic individuals with functional dyspepsia. Patients underwent to evaluation of physical activity in daily life by a triaxial accelerometer, exercise capacity (shuttle walk test-SWT), handgrip strength, QoL and mood disorders. The associations between the physical activity in daily life and QoL or mood disorders were tested. Results: The number of steps taken (7446 ± 3081 vs. 7898 ± 2487), the active time (80.56 ± 41.97 min vs. 89.65 ± 24.30 min), the SWT distance (662.69 ± 252.86 m vs. 728.82 ± 346.55 m) and the handgrip (34.3 ± 11.1 kgf vs. 32.9 ± 11.9 kgf) didn’t show significant difference between CD patients and controls, respectively. The time spending lying down (116.31 ± 107.38 min vs. 63.71 ± 55.79 min) and some domains of quality of life were superior in CD patients (p< 0.05). No correlation was observed between the level of physical activity in daily life and QoL or mood disorders in CD patients (p>0.05). Conclusions: We found that patients with CD on infliximab-induced remission have the same level of physical activity in daily life and exercise capacity when compared with controls.

Abstract Morbid obesity is the most rapidly increasing health threat of developed countries, and the costs caused by it are already higher than those of smoking. In an increasing number of developing countries both starvation and morbid obesity are increasing simultaneously. Obesity in children and adolescents is also increasing rapidly. Conservative treatment almost invariably fails when treating morbid obesity. Results of pharmacotherapy have been disappointing after great expectations. Laparoscopic gastric banding has been used in the treatment of morbid obesity since 1993. The method was first used mostly in Europe. In the USA either an open or laparoscopic gastric bypass have been the most common methods of surgery. The aim of this study was to investigate the operation results of 280 patients operated in Vaasa Central Hospital during the 11 years after March 1996. Of these patients, 123 have been followed at least 5 years. The results have been analyzed with BAROS that measures the quality of life. Quality of life was measured prospectively 1 year after surgery with the 15D questionnaire that is validated in the Finnish population. The effect of gastric banding in esophageal motility and reflux was studied prospectively in 31 patients. Late results were analyzed in 123 patients 11 years after the first operation. Mean excess weight loss (EWL) was 56% in patients who had their band in place 7 years after surgery, and 46% in all patients. There was no mortality related to the operation, and there was only one serious complication. Disease-specific quality of life improved in 78.8% of the patients in 28 months of follow-up. Health-related quality of life was significantly improved 12 months after surgery, but improvement was not connected to the amount of weight loss. The band inhibited reflux 19 months after surgery. Complications, failures, and reoperations increase with longer follow-up. Weight loss is moderate 9 years after a gastric banding operation, and in carefully selected patients this operation is still a good option in the treatment of morbid obesity.

La Survie globale (SG) est le critère de jugement de référence dans les essais cliniques en oncologie mais les critères de jugement dit intennédiaires centrés sur la tumeur qui sont évalués plus précocement sont de plus en plus utilisés comme critères substitutifs de la survie globale La qualité de vie (QdV) constitue un critère de jugement pour évaluer un bénéfice clinique direct pour le patient. Nos résultats ont montré que 32% des critères de jugement dans les essais de phase III de CBNPC avancés étaient pas du tout définis et seulement 43% était clairement défini et qu'aucun critère intermédiaire n'a démontré sa substitutivité sur la SG. Nous avons montré que la mesure, l'analyse et le report des données de QdV étaient hétérogènes entre les essais de phase III de CBNPC avancés d'où la nécessité de recommandations. Nous avons comparé longitudinalement la QdV chez 451 patients âgés de 70-89 ans atteints de CBNPC avancés randomisés entre une bichimiothérapie et une monochimiothérapie selon la technique du temps jusqu'à détérioration définitive (TJD) d'un score de QdV. Cette étude a montré : sur le plan clinique, le bénéfice du doublet de chimiothérapie pour les patients est renforcé par les données de QdV; sur le plan méthodologique: le TJD est une technique d'analyse faisable dans les essais de phase III de CBNPC et fournit des résultats faciles d'interprétation. Nous avons proposé l'utilisation de co-critères de jugement principaux associant QdV et critère centré sur la tumeur. Enfin, nous avons démontré la valeur pronostique de la dimension santé globale de QdV à baseline du questionnaire sur la SG chez les patients âgés atteints de CBNPC avancés. / Overall survival (OS) is the gold standard endpoint in oncology clinical trials. Nevertheless, intermediate tumor-centered endpoints which are assessed earlier are more and more used as surrogate of OS. Health-related quality of life is an endpoint which assesses a direct benefit for the patient. We demonstrated that 32% of endpoints in advanced non-small-cell lung phase III clinical trials were not defined at all, 43% were not clearly defined and none ofthem has demonstrated its surrogacy on OS. We demonstrated the weakness and the heterogeneity of the measurement, analysis, and reporting of HRQoL in phase III advanced NSCLC trials. Precise and uniform recommendations are needed. We longitudinally compared HRQoL using tüne until definitive deterioration (TUDD) method in 451 patients aged 70-89 years with advanced NSCLC randomly assigned to receive a doublet of chemotherapy or a monochemotherapy. We demonstrated the benefit of the doublet chemotherapy in terms ofHRQoL. On a methodological point ofview TUDD method is feasible and provide clinically meaningful results. We proposed another approach which would be to combine intermediate endpoint• with HRQoL as co-primary endpoints. Finally, we demonstrated the additional prognostic value of HRQoL data at baseline to identify vulnerable subpopulations in elderly NSCLC patients.

Abstract The aim of this thesis was to measure patient reported outcomes, such as health related quality of life and general diabetes knowledge of elderly diabetes mellitus type 2 (DMT2) patients in Slovenia. Patient reported outcomes demonstrate patient perspectives when evaluating the delivery of care. In Slovenia, a new, multidisciplinary model of chronic care was introduced in 2011, which yet needs to demonstrate the efficiency, care improvement and cost reduction. Thus, another aim of the study was to research if multidisciplinary teams in diabetes care are economically viable. To achieve the above, the study was divided into 4 subprojects. Firstly, the general level of diabetes knowledge of elderly DMT2 patients (n=179) was measured. Secondly, the reliability and validity of generic, European Quality of Life- 5 Dimensions (EQ-5D), and disease specific, Audit on Diabetes Dependent Quality of Life (ADDQoL ) instruments were examined. Thirdly, health related quality of life of elderly diabetic patients (n=285) was assessed. Fourthly, the systematic literature review on the cost-effectiveness of multidisciplinary teams was conducted. The data were collected during the period 2011–2012. The main contributions of the current thesis can be summarised as follows: This was the first study to measure general diabetes knowledge of elderly DMT2 patients in Slovenia. The results showed that the place of living does not have an impact neither on diabetes knowledge nor the health related quality of life of these patients. Secondly, a pioneering example of measuring health related quality of life (HRQoL) in elderly diabetic patients in Slovenia, using a validated and reliable instrument (ADDQoL) was provided. A study to evaluate the relationships between diabetic and other co-existing chronic medical conditions on health related quality of life was performed. As part of that study, the reliability and validity of the instruments (EQ-5D and ADDQoL) were measured, and the analysis showed that both instruments are reliable. Thirdly, a systematic way of finding evidence for understanding the cost-effectiveness of multidisciplinary teams was applied. The results of the literature review show weak improvements in the economic outcomes. In general, the thesis contributes to the improved understanding of patient reported outcomes in elderly diabetic patients, which can be a measure in assessing diabetes care program in Slovenia, and offers a basis for a national evaluation of the Model Practices. Furthermore, patient reported outcomes of elderly diabetic patients is important to Slovenian decision makers to identify and implement appropriate interventions for achieving better management of diabetes and ultimately improving the quality of life of diabetes patients. / Tiivistelmä Tutkimuksen tarkoitus oli mitata iäkkäiden tyypin 2 diabetespotilaiden itse ilmoittamia tuloksia, kuten terveyteen liittyvä elämänlaatu ja yleinen diabetekseen liittyvä tietämys Sloveniassa. Tulokset valottavat potilaiden näkökulmaa hoidon arvioinnissa. Sloveniassa otettiin 2011 käyttöön monitieteellinen hoitomalli, jonka tehoa sekä kykyä parantaa hoitoa ja vähentää hoitokustannuksia ei ole osoitettu. Toisena tavoitteena oli selvittää, onko moniammatillisten tiimien käyttö diabeteshoidossa taloudellisesti järkevää. Tutkimus jaettiin neljään osaprojektiin. Ensin mitattiin iäkkäiden tyypin 2 diabetespotilaiden (n=179) yleistä diabetestietämystä. Toiseksi selvitettiin geneerisen European Quality of Life- 5 Dimensions- (EQ-5D) ja diabeteskohtaisen ADDQoL -mittarin luotettavuus ja validiteetti. Kolmanneksi arvioitiin iäkkäiden diabetespotilaiden (n=285) terveyteen liittyvää elämänlaatua. Neljänneksi tehtiin moniammatillisten tiimien kustannus¬tehokkuutta koskeva systemaattinen kirjallisuuskatsaus. Tiedot kerättiin vuosina 2011–2012. Tulokset voidaan tiivistää seuraavasti: Kyseessä oli ensimmäinen tutkimus, jossa mitattiin iäkkäiden tyypin 2 diabetespotilaiden yleistä diabetestietämystä Sloveniassa. Tulosten mukaan asuinpaikka ei vaikuta potilaiden diabetestietämykseen tai terveyteen liittyvään elämänlaatuun. Toiseksi, Sloveniassa toteutettiin pioneerihanke, jossa mitattiin iäkkäiden potilaiden terveyteen liittyvää elämänlaatua (HRQoL) validoidun ja luotettavan instrumentin (ADDQoL) avulla. Tutkimuksessa selvitettiin diabeteksen ja muiden pitkäaikaissairauksien yhteisvaikutusta terveyteen liittyvään elämänlaatuun. Osana tutkimusta selvitettiin instrumenttien (EQ-5D ja ADDQoL) luotettavuus ja validiteetti. Molemmat osoittautuivat luotettaviksi. Kolmanneksi, tutkimuksessa etsittiin systemaattisesti näyttöä moniammatillisten tiimien kustannustehokkuuden arvioimiseksi. Kirjallisuuskatsauksen mukaan taloudellinen tulos paranee vain vähän. Tutkimus lisää tietoa potilaiden ilmoittamista tuloksista iäkkäiden diabetespotilaiden kohdalla, mitä voidaan käyttää diabeteshoito-ohjelman arvioinnissa Sloveniassa sekä mallikäytäntöjen kansallisen arvioinnin perustana. Diabetespotilaiden itse ilmoittava terveystieto on Slovenian päätöksentekijöille tärkeää sopivien interventioiden löytämisessä ja toteuttamisessa, kun halutaan parantaa diabeteshoitoa ja potilaiden elämänlaatua.

Abstract Leukemia treatment has been implicated to be responsible for a diversity of long-term adverse effects (Pui 2008), which can occur even years after diagnosis and can seriously impair survivors’ performance status and quality of life (Campbell et al. 2007, Zeltzer et al. 2008). The aim of the present work was to assess health-related quality of life in long-term survivors of acute lymphoblastic leukemia (ALL), and to find out whether vocational rehabilitation can promote the coping at work of young people who take part in a training intervention or help them find employment. Neuropsychological testing was performed on 64 survivors. Cranial irradiation had been administered to 44 of the survivors, while 20 survivors had been treated solely with chemotherapy. A control group consisted of 45 healthy young adults. We found that young adult survivors of childhood ALL treated with cranial irradiation had clear progressive deficits in their neurocognitive functioning at a mean of 20 years after diagnosis compared with healthy controls. Non-irradiated ALL survivors performed significantly better, but even they had statistically significant impairments in some of the neuropsychological test scores compared with the controls. Rand-36-Item health Survey (RAND-36) was used to assess subjective HRQoL, depressive symptoms were assessed with Beck Depression Inventory (BDI-21), and mental distress with General Health Questionnaire (GHQ-12) in 74 survivors of ALL. The control group consisted of 146 healthy young adults selected from local population registry. ALL survivors showed good HRQoL scores in comparison to the control group. Patients who had been treated for an ALL relapse and had received the most intensive chemo- and radiotherapy had significantly higher scores on mental health and vitality than the controls. Survivors of ALL report fewer depressive symptoms and equal mental well-being compared to healthy controls. Nine out of 63 survivors were selected for rehabilitation based on their occupation and psychosocial difficulties. The occupational rehabilitation course improved physical fitness and decreased overweight in long-term survivors of ALL, but impaired their HRQoL. Our findings support the idea that ALL survivors’ subjective experience of well-being is possibly affected by a repressive adaptive style. / Tiivistelmä Leukemiahoidoilla on todettu olevan pitkäaikaisvaikutuksia (Pui 2008), jotka voivat ilmetä jopa vuosia diagnoosin jälkeen heikentäen sairaudesta selvinneiden toimintakykyä ja elämänlaatua (Campbell et al. 2007, Zeltzer et al. 2008). Tämän tutkimuksen tavoitteena oli arvioida akuutista lymfaattisesta (ALL) leukemiasta selvinneiden terveyteen liittyvää elämänlaatua pitkällä aikavälillä sekä selvittää, voidaanko ammatillisen kuntoutuksen avulla edistää interventioon osallistuneiden työssä selviytymistä tai työllistymistä. Neuropsykologiseen tutkimukseen osallistui 64 ALL pitkäaikaisselviytyjää. Heistä 44 oli saanut pään alueen sädehoitoa ja 20 pelkästään solunsalpaajahoitoa. Verrokkiryhmässä oli 45 tervettä nuorta aikuista. Havaitsimme, että lapsuudessa pään alueen sädehoitoa saaneilla ALL-selviytyjillä oli verrokkeihin verrattuna selkeitä progressiivisia muutoksia neurokognitiivisessa toimintakyvyssä keskimäärin 20 vuotta diagnoosista. Ne, jotka eivät olleet saaneet sädehoitoa suoriutuivat huomattavasti paremmin, mutta heilläkin havaittiin tilastollisesti merkittäviä puutoksia joillakin neuropsykologisten testien osa-alueilla verrokkeihin verrattuna. 74 entisen ALL-potilaan terveyteen liittyvää elämänlaatua (HRQoL) mitattiin Rand-36 -mittarilla, masennusoireita Beckin masennustestillä (BDI-21) ja henkistä hyvinvointia General Health Questionnaire -mittarilla (GHQ-12). Verrokkiryhmäksi valittiin väestörekisteristä 146 tervettä nuorta aikuista. ALL-ryhmässä tutkittavien terveyteen liittyvää elämänlaatua mittaavat pisteet olivat hyvät verrokkiryhmään verrattuna. Ne potilaat, joita oli hoidettu leukemian uusiutumisen takia ja jotka olivat saaneet intensiivisintä solunsalpaaja- ja sädehoitoa saivat huomattavasti paremmat pisteet psyykkisen hyvinvoinnin ja vitaalisuuden osa-alueilla kuin verrokit. ALL -selviytyjät raportoivat vähemmän masennusoireita, ja he arvioivat psyykkinen hyvinvointinsa yhtä hyväksi kuin verrokit. Yhdeksän ALL pitkäaikaisselviytyjää osallistui heille suunnattuun ammatilliseen kuntoutusinterventioon. Ammatillinen kuntoutuskurssi paransi ALL -selviytyjien fyysistä suorituskykyä ja vähensi ylipainoa, mutta heikensi terveyteen liittyvää elämänlaatua. Tutkimustulokset tukevat ajatusta, että repressiivinen adaptaatio voi vaikuttaa lapsuusiän leukemiasta selvinneiden subjektiiviseen hyvinvoinnin kokemukseen.

L'évaluation de la qualité de vie des patients a une place centrale en santé, en particulier dans les recherches et les politiques de santé publique. De nombreux instruments de mesure sont développés et les discours sur la qualité de vie en santé se multiplient. Diverses approches du concept cohabitent. Mais ce développement et cette diversité s'accompagnent de doutes persistants sur la validité des concepts de qualité de vie en santé. Pour comprendre et peut-être réduire cette confusion, il faut d'abord s'accorder sur un niveau d'analyse commun du concept de qualité de vie en santé. Or, à notre connaissance, un tel niveau d'analyse n'existe pas. Ce niveau d'analyse est pourtant nécessaire pour expliciter, comparer et examiner les choix théoriques qui déterminent la conception de la qualité de vie, et surtout de la bonne qualité de vie, sous-jacente aux instruments de mesure. C'est la conceptualisation de la valeur, de ce qu'il y a de bon dans une vie qui est d'abord en jeu. Or ceci est occulté. En effet, l'approche de la qualité de vie en santé est conçue généralement, mais à tort, comme neutre par rapport à la valeur. Cette occultation de la valeur explique probablement pourquoi un cadre d'analyse commun des concepts en usage de qualité de vie liée à la santé fait défaut. A partir d'une compréhension de la logique de la valeur spécifique à la qualité de vie, nous tentons de développer ici ce cadre d'analyse conceptuelle. / The evaluation of quality of the patients' life is central in health, especially in Public Health research programs and policies. Many instruments are developed and discourses on health related quality of life grow in number and diversity. Several approaches and accounts of the concept coexist. However, this growth and diversity is synonymous with enduring doubts about the validity of the existing concepts of health related quality of life. In order to understand and maybe dissolve the confusion, we have first to agree on a common framework of analysis for the concept of quality of life. But, to our knowledge, this framework of analysis is not available. And yet this framework is necessary in order to explicit, compare and test the theoretical choices that determine the concept of quality of life, and most of all of the good quality of life, underlying the measuring instruments. It is indeed the conceptualization of value, of what is good in life, which is at stake. But this aspect is concealed: the approach of quality of life in health is generally but mistakenly conceived as value free or neutral. This concealment probably explains why a common framework of analysis of the existing concepts of health related quality of life is not available. From an understanding of the logic of value specific to quality of life, we try to develop such a framework of conceptual analysis.