In deciding whether to use a particular treatment for conditions such as depression, arthritis, or heart disease, clinicians and patients must balance the benefits against the side effects and burden. To make this trade-off, they must understand the likely degree of benefit in patients’ symptoms and perceived wellbeing, best undertaken using patient-reported outcomes (PROs). PROs are measures of any aspect of a patients’ health status that are obtained from direct patient inquiry without interpretation by a clinician or anyone else. PRO measures (PROMs) are increasingly used in clinical trials and systematic reviews to evaluate health care interventions, and information obtained from PROMs can guide clinical decisions and inform shared-decision making. The use of PROMs, however, involves challenges, the most important of which is deciding if a particular treatment effect is trivial, small but important, moderate or large. One way to make this judgment is to consider the minimal important difference (MID), the smallest change in a PROM score that is important enough that patients would consider a change in treatment to achieve that benefit. The number of published studies providing anchor-based MIDs for PROMs has grown rapidly over the last three decades, and researchers have proposed several anchor-based methods to derive MID estimates, each with its own merits and limitations. This thesis begins with the development of a framework to determine the extent to which the design and conduct of studies measuring anchor-based MIDs are likely to have protected against misleading estimates. Subsequently, this thesis presents a comprehensive inventory of empirically estimated anchor-based MIDs and their associated credibility for all PROMs published in the medical literature. Further, this thesis highlights critical issues that key stakeholders should consider, and demonstrates how the use of credible MIDs may inform the development of a clinical practice guideline in which PROs were identified as critically important. Finally, this thesis concludes with insights to improve the methodological quality and transparency for researchers in the PRO and MID field. / Thesis / Doctor of Philosophy (PhD)

Background: Amyotrophic Lateral Sclerosis (ALS) is a fatal neurodegenerative disease characterized by the loss of motor neurons. Preference-based measures (PBMs) of health-related quality of life (HRQL) can be utilized for cost-effectiveness analyses of interventions in individuals with ALS. However, current measures are generic (GPBMs) and the psychometric properties of these measures have not yet been evaluated in ALS. Purpose: The purpose of this thesis was to evaluate the psychometric properties of GPBMs in ALS by 1) conducting a systematic review of the psychometric properties of GPBMs, and 2) assessing the content and convergent validity of GPBMs in ALS. Methods: Two studies were conducted. First, a systematic review was performed, and four databases were searched to identify studies that used and reported on the psychometric properties of GPBMs in ALS. Second, participants were recruited from three clinical sites across Canada and outcome measures were administered through an online or hardcopy survey. Areas of importance to the HRQL of individuals with ALS were identified using the Patient Generated Index (PGI), mapped against GPBMs to determine their coverage and scores were compared to determine convergent validity. Results: For the first study, the EQ-5D-3L was found to be the most commonly used GPBMs in ALS. It demonstrated convergent and known-groups validity however, significant floor effects were observed. For the second study, results indicated that the majority of GPBMs identified approximately half of the areas impacted by ALS. In addition, there were several domains not identified by GPBMs. Conclusion: This thesis highlights the importance of complete psychometric evaluation of measures in ALS. There is the need for the development of an ALS specific preference-based measures that reflects the health concerns of individuals with ALS; as GPBMs used in ALS were evaluated and deemed to be lacking in support for their usage in ALS. / Thesis / Master of Science Rehabilitation Science (MSc) / Amyotrophic Lateral Sclerosis (ALS) is a fatal disease that causes individuals to lose their strength and eventually the ability to speak, eat, move and breathe. Questionnaires can be used to understand the health-related quality of life (HRQL) of individuals with ALS however these measures do not always reflect the experiences of these individuals. The goal of this dissertation was to identify whether measures truly capture areas important to individuals with ALS. In our studies, we found that there is little proof in the accuracy of measures used. In addition, the measures do not fully capture the areas of life important to individuals with ALS. This is important to help researchers and health care professionals understand the effects of ALS on HRQL. These results will help them determine which treatments are worthwhile and the best to use in practice and provide recommendations for future research.

Measurement Based Care (MBC) is considered to be an evidence-based practice. Despite its well-documented efficacy, it is underutilized in the clinical community for various reasons, including clinician and patient buy-in. A key component to the successful implementation of MBC is the routine administration of Patient Reported Outcome Measures (PROMs). There is a lack of research describing the utilization of MBC in a telehealth setting. As technological innovations continue, a greater number of clinics are offering telemental health services. Additionally, the COVID-19 pandemic caused a majority of underprepared clinics to begin implementing telehealth. The present study sought to evaluate patient compliance with PROMs in MBC after an abrupt shift to telehealth due to the COVID-19 pandemic. Participants were collected from a clinical population at a community based psychological training clinic. The participants were separated into groups: modality 1 (in-person services, n = 17), modality 2 (telehealth services, n = 17), and modality 3 (hybrid of modalities 1 and 2, n = 10), to assess the effect of modality on mean PROM compliance. The participants were separated into groups adult (≥18 years of age, n = 23) and child (<18 years of age, n = 17), to assess the effect of maturity on mean PROM compliance. Results showed that mean PROM compliance was significantly higher in the in-person modality than the telehealth modality. Results also showed that PROM compliance was significantly higher in adults than in children. There was not a significant interaction effect of modality and maturity on PROM compliance. Additionally, results showed that PROM compliance decreased significantly after the switch from in-person services to telehealth services in the hybrid modality. These findings show that clinics may face significant barriers to the implementation of MBC after a sudden shift to telehealth caused by an unforeseen stressful event. / M.S. / Measurement-Based Care (MBC) is an effective practice for the treatment of patients in psychological practices. It is a collaborative process that involves the clinician and patient tracking treatment progress and outcomes through the use of consistently administered measures known as Patient Reported Outcome Measures (PROMs). Although MBC has been shown to be effective, there is little literature regarding its use in a telehealth setting. As a greater number of clinics begin offering telehealth services, questions regarding patient adherence to interventions have arisen. Furthermore, the global COVID-19 pandemic forced a majority of underprepared clinics to offer telehealth services. The current study sought to better understand potential barriers to the implementation of MBC in a telehealth setting. Participants were collected from patients receiving therapy at a community based psychological training clinic. The participants were separated into the groups: in-person services (n = 17), telehealth services (n = 17), and hybrid of in-person and telehealth (n = 10), to assess the effect of modality on mean PROM compliance. The participants were separated into groups adult (≥18 years of age, n = 23) and child (<18 years of age, n = 17), to assess the effect of maturity on mean PROM compliance. Results showed that patients in the in-person therapy group were more likely to complete their measures than patients in the telehealth therapy group, regardless of their maturity. Additionally, adult patients were more likely to complete their measures than child patients, regardless of the modality. Results also showed that patients who experienced both in-person and telehealth services were more likely to complete their measures before the switch to telehealth. These findings show that clinics may face significant barriers to the implementation of MBC after a sudden shift to telehealth caused by an unforeseen stressful event.

Introduction: Conversation treatment for individuals with aphasia (IwAs) aims to enhance language and communication skills within naturalistic settings. Group dynamics, including psychosocial support (PSS) and vicarious learning (VL), are important components of this treatment. However, the lack of established aphasia-friendly methods for quantifying group dynamics impedes understanding of its contribution to treatment efficacy. This study addresses three key research goals: (1) Can group dynamics be reliably quantified? (2) Do measures of group dynamics vary with group size or time? (3) Are group dynamics linked to changes in self-reported psychosocial health measures? Methods: IwAs were assigned to participate in either a large group or dyadic condition as part of a larger study. Participants completed multiple standardized assessments before and after receiving conversation treatment for one hour twice a week for ten weeks. A coding system was developed to track PSS and VL during sessions. Video recordings of sessions at the start (Session 2) and end (Session 19) of the treatment period were analyzed for five large groups and six dyads, with seven additional dyads coded for Session 19 after reliability was confirmed. Intrarater and interrater reliability were assessed by recoding 21% of the videos randomly. Results: To address the first research goal, group dynamics were defined and then a code was created to represent occurrences of PSS and VL. After establishing a reliable coding system, instances of PSS and VL were compared across size conditions to address the second research goal. The results suggested that PSS differed between conditions later in treatment, but VL did not. For research goal three, the data suggest that large groups experience larger gains in quality of life measures, possibly due to increased exposure to PSS. Conclusion: Findings indicate that group dynamics can be reliably tracked and used for quantitative analysis. PSS was more common in groups than dyads later in the 10 week treatment period, but VL did not vary across conditions or at the start or end of treatment. Further, there is some evidence that PSS in larger groups contributes to improvements in quality of life measures. / Communication Sciences

Yes / Purpose: To develop and validate the first patient-reported outcome measure (PROM) to quantify vision-related dizziness. Dizziness is a common, multifactorial syndrome that causes reductions in quality of life and is a major risk factor for falls, but the role of vision is not well understood. Methods: Potential domains and items were identified by literature review and discussions with experts and patients to form a pilot PROM, which was completed by 335 patients with dizziness. Rasch analysis was used to determine the items with good psychometric properties to include in a final PROM, to check undimensionality, differential item functioning, and to convert ordinal questionnaire data into continuous interval data. Validation of the final 25-item instrument was determined by its convergent validity, patient, and item-separation reliability and unidimensionality using data from 223 patients plus test–retest repeatability from 79 patients. results: 120 items were originally identified, then subsequently reduced to 46 to form a pilot PROM. Rasch analysis was used to reduce the number of items to 25 to produce the vision-related dizziness or VRD-25. Two subscales of VRD-12-frequency and VRD-13-severity were shown to be unidimensional, with good psychometric properties. Convergent validity was shown by moderately good correlations with the Dizziness Handicap Inventory (r = 0.75) and good test–retest repeatability with intra-class correlation coefficients of 0.88. conclusion: VRD-25 is the only PROM developed to date to assess vision-related dizziness. It has been developed using Rasch analysis and provides a PROM for this under-researched area and for clinical trials of interventions to reduce vision-related dizziness. / College of Optometrists (UK) research studentship.

No / Improving cognition is service users' top research priority for life after stroke, and future research should include outcomes that they deem important. Patient perspectives on outcomes are collected using patient-reported outcome measures (PROMs). There is currently no patient-centred PROM specific for cognitive rehabilitation trials. Objective Inform PROM development by exploring stroke survivor perspectives on the important, measurable impacts of persisting post-stroke cognitive problems. Design Qualitative semi-structured interviews in participants' homes. Participants Purposive sample of 16 cognitively impaired stroke survivors at least six months post-stroke. Methods Interviews used a schedule and communication aids developed through patient consultation. Interviews were transcribed verbatim with non-verbal communication recorded using field notes. Data were analysed using a framework approach to find commonalities to shape the focus and content of an outcome measure. Results Participants identified important impacts of their ‘invisible’ cognitive problems, outside of other stroke-related impairments. Cognitive problems exacerbated emotional issues and vice versa. Changes in self-identity and social participation were prominent. Impact was not spoken about in terms of frequency but rather in terms of the negative affect associated with problems; terms like ‘bothered’ and ‘frustration’ were often used. Conclusions The results support the development of a PROM specifically designed to address the impact of cognitive problems. It should: include items addressing a comprehensive range of cognitive skills; ask questions about mood, self-identity and social participation; use accessible wording that respondents understand and endorse; measure impact rather than frequency; and explore perceived impact on carers.

京都大学 / 新制・課程博士 / 博士(人間健康科学) / 甲第25503号 / 人健博第123号 / 新制||人健||8(附属図書館) / 京都大学大学院医学研究科人間健康科学系専攻 / (主査)教授 宮下 美香, 教授 千葉 理恵, 特定教授 古川 壽亮 / 学位規則第4条第1項該当 / Doctor of Human Health Sciences / Kyoto University / DFAM

Made available in DSpace on 2016-12-12T17:32:58Z (GMT). No. of bitstreams: 1 Fernanda Fonseca.pdf: 58160 bytes, checksum: c9c16d7d5ac8b50498e52c991095d785 (MD5) Previous issue date: 2015-07-02 / Coordenação de Aperfeiçoamento de Pessoal de Nível Superior / Patients with chronic obstructive pulmonary disease (COPD) have reduced level and intensity of daily physical activity. The physical activity is related to the COPD prognosis and, therefore, the functional performance of these patients should be routinely assessed. The Self-Reported Functional Limitation scale (SRFL) has been used in COPD patients cohorts to assess functional performance, but neither its classification (with or without self-reported functional limitation) measurement properties have been tested nor a translation into Portuguese and cross-cultural adaptation to Brazil have been done. The aim of this study was to translate the SRFL into Portuguese and cross-culturally adapt it to Brazil, to assign scores for its response categories and to test its measurement properties in patients with COPD. Initially, the scale was translated into Portuguese, applied in six patients with COPD, then backtranslated into English and sent to one of the original version authors to have her appreciation of the Brazilian version. Then, scores (from zero to four) were assigned to their response categories. Later, the Brazilian version was applied in thirty patients with COPD (mean ± standard deviation: age = 64 ± 8 years old; post-BD FEV1 = 38.9 ± 15.3 %pred; BMI = 25.7 ± 4.71 kg/m2) for its inter-rater and test-retest reliability analysis. Nutritional status assessment, spirometry, handgrip dinamometry, physical activity in daily life (PADL) monitorization, and the application of the COPD Assessment Test (CAT), the modified Medical Research Council scale and the London Chest Activity of Daily Living scale (LCADL) were performed for the validity analysis. There was inter-rater (&#954;=1.00; ICC=0.92 and CI95%=0.85-0.96) and test-retest (&#954;=0.79; ICC=0.92 and CI95%=0.84-0.96) reliability for the SRFL classification and score (p<0.01 for all). In the validity analysis, there were associations of self-reported functional limitation classification with the COPD impact on health status, risk and symptoms, fat-free mass (FFM) depletion and body composition impairment (p<0.05 for all) and correlations between SRFL score and total (r=0.75) and %total (r=0.77) LCADL scores, handgrip strength (r=-0.36), CAT score (r=0.80) and fat-free mass index (FFMI) (r=-0.49) (p<0.05 for all). It was also observed absence floor and ceiling effects. The translated and cross-culturally adapted SRFL version was reliable, when applied by different raters or in diferente moments, and valid for assessing the perception of functional limitation in patients with COPD, considering both its classification and score. In addition, the SRFL score had a missing floor or ceiling effect. / Pacientes com doença pulmonar obstrutiva crônica (DPOC) apresentam redução do nível e da intensidade de atividade física diária. A atividade física está relacionada ao prognóstico da DPOC e, portanto, a performance funcional desses pacientes deve ser avaliada rotineiramente. A escala Self-Reported Functional Limitation (SRFL) tem sido utilizada em coortes de pacientes com DPOC para avaliação da performance funcional, mas ainda não foram testadas as propriedades de medida de sua classificação (com ou sem limitação funcional autorrelatada) e nem foi realizada sua tradução para a língua portuguesa e sua adaptação transcultural para o Brasil. Assim, o objetivo deste estudo foi traduzir a SRFL para a língua portuguesa, adaptá-la transculturalmente para o Brasil, atribuir escores para as categorias de resposta da mesma e testar suas propriedades de medida em pacientes com DPOC. Inicialmente, a escala foi traduzida para a língua portuguesa, aplicada em seis pacientes com DPOC, retrotraduzida para a língua inglesa e discutida com uma das autoras da versão original para conclusão de sua versão brasileira. Em seguida, foram atribuídos escores (de zero a quatro) as suas categorias de resposta. Posteriormente, a versão brasileira da escala foi aplicada em trinta pacientes com DPOC (média ± desvio-padrão: idade = 64 ± 8 anos; VEF1 pós-BD = 38,9 ± 15,3 %prev; IMC = 25,7 ± 4,71 kg/m2) para análise de sua confiabilidade interavaliadores e teste-reteste. Para análise de sua validade, foram realizadas a avaliação do estado nutricional, a espirometria e a dinamometria de preensão palmar, monitorizadas as atividades físicas na vida diária (AFVD) e aplicados o COPD Assessment Test (CAT), a escala Medical Research Council modificada (mMRC) e a escala London Chest Activity of Daily Living (LCADL). Observou-se confiabilidade interavaliadores (&#954;=1,00; CCI=0,92 e IC95%=0,85-0,96) e teste-reteste (&#954;=0,79; CCI=0,92 e IC95%=0,84-0,96) para a classificação da SRFL e para o escore da mesma (p<0,01 para todos). Na análise de validade, observaram-se associações da classificação de limitação funcional autorrelatada com as classificações de impacto da DPOC no estado de saúde, de risco e sintomas, de depleção de massa livre de gordura (MLG) e de prejuízo na composição corporal (p<0,05 para todos) e correlações entre o escore da SRFL e os escores total (r=0,75) e em %total (r=0,77) da LCADL, a força de preensão palmar (r=-0,36), o escore do CAT (r=0,80) e o índice de massa livre de gordura (IMLG) (r=- 0,49) (p<0,05 para todos). Foram observados também efeitos chão e teto ausentes. A versão traduzida e adaptada transculturalmente da SRFL mostrou-se confiável, quando aplicada por diferentes avaliadores e em diferentes momentos, e válida para a avaliação da percepção de limitação funcional de pacientes com DPOC, considerando tanto a sua classificação quanto o seu escore. Além disso, o escore da SRFL não apresentou efeito chão ou teto.

This study investigated the relationship of out-of-class English use and proficiency gains. It also explored the relationship of gender, proficiency level and native language and the possible effect of these demographics on out-of-class English use and language gains in English. Though some studies have shown that those who spend more out-of-class time using the target language have higher language gain (Seliger, 1977), other studies have not found this to be true (Day, 1985; Freed 1990; Spada, 1986). Some reasons for the discrepancy in findings may be differences in the length of the time data is collected, samples of study participants and types of tests used to measure proficiency. Sixty-one students at an intensive English language program came from 12 different language backgrounds and 4 proficiency levels. They participated in a 31-week-long study. Participants took a proficiency pre- and posttest (Elicited Imitation Test) and responded to a questionnaire designed to elicit information about out-of-class language use (Language Contact Profile). In addition to the questionnaire, six students participated in semi-structured interviews that offered additional support for the data gathered by the questionnaire. Data obtained from the questionnaire and interviews was compared to gains in proficiency between the pre- and posttest. The results suggest that using English out-of-class helps improve oral proficiency. In addition, the study shows that gender, proficiency level and native language are not significant predictors of out-of-class English use and proficiency gains. These findings are discussed in light of what teachers and school administrators can do to help their students use the target language in and out of class for best results.

The topic of this thesis is on how text mining could be used on patient-reported symptom descriptions, and how it could be used to aid doctors in their diagnostic process. Healthcare delivery today is struggling to provide care to remote settings, and costs are increasing together with the aging population. The aid provided to doctors from text mining on patient descriptions is unknown.Investigating if text mining can aid doctors by presenting additional information, based on what patients who write similar things to what their current patient is writing about, could be relevant to many settings in healthcare. It has the potential to improve the quality of care to remote settings and increase the number of patients treated on the limited resources available. In this work, patient texts were represented using the Bag-of-Words model and clustered using the k-means algorithm. The final clustering model used 41 clusters, and the ten most important words for the cluster centroids were used as representative words for the cluster. An experiment was then performed to gauge how the doctors were aided in their diagnostic process when patient texts were paired with these additional words. The results were that the words aided doctors in cases where the patient case was difficult and that the clustering algorithm can be used to provide the current patient with specific follow-up questions. / Ämnet för detta examensarbete är hur text mining kan användas på patientrapporterade symptombeskrivningar, och hur det kan användas för att hjälpa läkare att utföra den diagnostiska processen. Sjukvården har idag svårigheter med att leverera vård till avlägsna orter, och vårdkostnader ökar i och med en åldrande population. Idag är det okänt hur text mining skulle kunna hjälpa doktorer i sitt arbete. Att undersöka om läkare blir hjälpta av att presenteras med mer information, baserat på vad patienter som skriver liknande saker som deras nuvarande patient gör, kan vara relevant för flera olika områden av sjukvården. Text mining har potential att förbättra vårdkvaliten för patienter med låg tillgänglighet till vård, till exempel på grund av avstånd. I detta arbete representerades patienttexter med en Bag-of-Words modell, och klustrades med en k-means algoritm. Den slutgiltiga klustringsmodellen använde sig av 41 kluster, och de tio viktigaste orden för klustercentroider användes för att representera respektive kluster. Därefter genomfördes ett experiment för att se om och hur läkare blev behjälpta i sin diagnostiska process, om patienters texter presenterades med de tio orden från de kluster som texterna hörde till. Resultaten från experimentet var att orden hjälpte läkarna i de mer komplicerade patientfallen, och att klustringsalgoritmen skulle kunna användas för att ställa specifika följdfrågor till patienter.