Outcomes measurement is an important component of routine hemophilia care, clinical trials and economic evaluations. Assessing outcomes in patients living with hemophilia is challenging due to a lack of validated outcome measures. Conventional clinical outcomes, for instance, bleeding rate, structural changes of joints or functional joint scores may be less relevant for the decision-making process. Patient reported outcomes measures has been increasingly interested in routine medical care and clinical research. However, the available validated patients reported outcome measures for patients with hemophilia are not generally implemented in routine care or clinical trials. The Patent Reported Outcomes, Burdens, and Experiences (PROBE) study aims to develop a validated patient reported outcome measure for patients living with hemophilia. The PROBE questionnaire is organized in 4 sections, comprising 29 questions. Section I contains questions pertaining to demographic data. Section II contains questions pertaining to patient reported outcomes. Section III contains questions pertaining to hemophilia specific problems and treatments. Section IV contains the EuroQol five dimension 5-level instrument (EQ-5D-5L). The psychometric analysis of revealed that the PROBE questionnaire has a good internal consistency (Cronbach’s alpha coefficient=0.84). PROBE items showed moderate to strong correlations with corresponding EQ-5D-5L domains. The PROBE Score has a known group validity among known groups. The psychometric properties of the PROBE questionnaire demonstrated the validity of the instrument in both patients living with hemophilia and control population (participants without bleeding disorder). The test-retest reliability analysis demonstrated that the PROBE questionnaire has a substantial agreement when the questionnaire was repeatedly administrated. There were acceptable reliability properties between the paper-based and web-based questionnaires. The reliability properties of the PROBE questionnaire were established in both patients living with hemophilia and control population. The PROBE questionnaire was cross-cultural implemented in 21 countries. The results showed that the regions of participant contributed a trivial variability of the PROBE score, indicating that the PROBE questionnaire is valid for assessing the health status among hemophilia patients and participants without bleeding disorder across regions. Sexual health of patients living with hemophilia was evaluated using the PROBE questionnaire. The results showed that sexual difficulty was more prevalent in patients with hemophilia and associated with markers of disease severity. This finding warrants the sexual health assessment in routine hemophilia care. / Thesis / Doctor of Philosophy (PhD)

Patient-reported outcome measures (PROMs) are becoming an integral part of healthcare decision making. Clinical trials, systematic reviews, and clinical practice guidelines incorporate them to learn about the effect of medical interventions in patients’ health status, without interference or mediation from clinicians or proxies. The use of these types of measures, however, is not without challenges. In particular, the complexity of the PROMs makes it difficult for patients, clinicians, and researchers to fully grasp the extent to which a treatment effect is negligible or trivial, small but important, moderate, or large. One of the most documented ways to address this issue is the use of the minimal important difference (MID), the smallest change in a PROM, either beneficial or harmful, that patients would perceive as important. A patient-oriented way to determine this threshold is the estimation of an anchor-based MID, where PROM results are compared against an external independent criterion the anchor that is in itself understandable and relevant for patients. This dissertation is an effort to facilitate the identification, evaluation, and utilization of MID estimates for PROMs. First, this thesis describes the development and reliability assessment of a new instrument to determine the credibility of primary studies ascertaining MID estimates, Second, it describes the conduct of a systematic survey to inform the creation of an inventory of all available anchor-based MIDs in the medical literature until 2015. Third, it reports an analysis of the state of the art of current MID estimates from a reporting and credibility perspective. Finally, this work concludes with a summary of the main results, presentation of strengths and limitations, and insights related to the implications for future research. / Dissertation / Doctor of Philosophy (PhD)

Unicompartmental knee replacement (UKR) is the principal alternative to total knee replacement (TKR) in the treatment of end-stage knee osteoarthritis. It involves less tissue resection, resulting in lower rates of morbidity and faster recoveries compared to TKR. However, UKR has a significantly higher revision rate compared to TKR. As a result, whilst over a third of patients are eligible for UKR, only around 8% receive it. A comprehensive comparison of matched patients undergoing TKR and UKR was undertaken using a large dataset from the National Joint Registry for England and Wales (NJR). Failure rates (revision, reoperation, complications and mortality), length of stay and patient-reported outcomes (PROMs) were studied. Whilst patients undergoing TKR had lower reoperation and revision rates, they had higher rates of morbidity and mortality, longer hospital stays, and inferior PROMs compared to UKR. The main reason for revision in UKR was loosening. In view of the high revision rate in UKR, NJR data was studied to identify modifiable risk factors for failure in UKR. Important patient factors were identified including age, gender and pre-operative function. Surgeons with a higher UKR caseload had significantly lower revision rates and superior patient-reported outcomes. Increasing usage (offering UKR to a greater proportion of knee replacement patients) appears to be a viable method of increasing caseload and therefore of improving results. Surgeons with optimal usage (around 50% of patients, using appropriate implants) achieved revision/reoperation rates similar to matched patients undergoing TKR. Two clinical studies were conducted to establish whether the use of cementless fixation would improve fixation and reduce the revision rate of UKR. Cementless UKR was demonstrated to be safe and reliable, with PROMs similar or superior to those demonstrated in cemented UKR. Patients with suboptimal cementless fixation were examined and pre-disposing technical factors were identified. Finally, using NJR data, the effect of the introduction of cementless UKR on overall outcomes was examined. The number of cementless cases was small, and no significant effect on implant survival was demonstrated. However, patients undergoing cementless UKR demonstrated superior PROMs. These studies demonstrate that UKR has numerous advantages over TKR in terms of morbidity, mortality and PROMs. If surgeons perform high volumes of UKR (achievable by increasing their UKR usage), these advantages can be attained without the large difference in revision rates previously demonstrated. Cementless UKR is safe and provides superior fixation and outcomes in the hands of high-volume surgeons. Further work is needed to quantify the revision rate of cementless UKR, and to assess its results in the hands of less experienced surgeons.

Introduction The use of Patient Reported Outcome Measures (PROMs) to measure the effects of care is being advocated increasingly in clinical settings. Current patient data capture involves completion of paper questionnaires which is costly and environmentally perplexing. New innovations are required to balance the challenges of introducing data capture directly from patients while considering budgets, access to Information Technology, and the capability to use technological devices. Methods Two qualitative studies were undertaken to identify the views of patients and clinicians concerning electronic PROM data capture in osteopathic practice. One qualitative study involved patient interviews to identify their views on a selection of specific PROMs. Clinician focus groups and interviews (osteopaths, chiropractors, and physiotherapists) were undertaken concerning their views and experiences of using PROMs. Scoping of PROMs in musculoskeletal practice was undertaken followed by a systematic review of one identified PROM. The review and qualitative work informed the development of content for a mobile and web app for capturing PROM data. The app was piloted to evaluate feasibility, and the clinimetric performance of the included PROMs. Feedback from the pilot informed revisions to the app prior to implementation into osteopathic practice. Results Clinicians (n=46) identified a range of barriers and facilitators to PROM use. Patients (n=22) while generally more enthusiastic than clinicians welcomed the opportunity to provide feedback and although undaunted by the use of technology highlighted the need for assurances concerning confidentiality of data, and limits on data sharing. The systematic review identified good measurement properties for the Bournemouth (BQ). Piloting of the app involved 257 participants contributing 404 data returns: it performed well requiring minimal revision prior to implementation. Conclusions The app performed well demonstrating great potential for further development to collect outcome data in a musculoskeletal clinical setting.

Bakgrund: Bröstcancer-relaterat lymfödem (BCRL) är en vanlig komplikation efter bröstcancerbehandling. Tidigare forskning visar varierande förekomst av BCRL, tillståndet tros vara underdiagnostiserat. Tidiga tecken kan beskrivas som en vag tyngdkänsla och/eller spänningskänsla. Detta kan leda till stora negativa konsekvenser vilket motiverar vikten att detektera BCRL i ett tidigt stadie då tidig behandling förbättrar prognosen.  Syfte: Undersöka förekomst av tidiga besvär/symtom samt undersöka sambandet mellan patientrapporterade tidiga besvär/symtom och läkarens bedömning av besvär vid samma tillfälle.  Metod: Kvinnor som genomgått kurativ behandling mot bröstcancer deltog i studien. Datainsamlingen skedde vid återbesöket på onkologmottagningen 3 månader efter avslutad behandling. Data samlades in med patientenkät (avseende besvär/symtom från armen) och läkarbedömning, inom ramen för ett kliniskt utvecklingsprojekt.   Resultat: 61 enkäter ingick i studien. 22 deltagare upplevde besvär från armen på den opererade sidan. 70% av de 43 deltagarna som skattade symtom angav förekomst av ≥1 symtom. I 76% av de 55 läkarbedömningarna hade bedömningen inga besvär gjorts. Samband kunde påvisas gällande frekvensen av patientrapporterade besvär och läkarens bedömning av armsvullnad (p=0,005) och handödem (p=0,003) samt mellan patientrapporterade symtom och läkarens bedömning av armsvullnad (p=0,008). I de fall patienten skattat besvär/symtom fanns en signifikant andel där läkaren bedömt att det inte förekom ödem/svullnad. I de fall patienten inte rapporterade besvär hade läkaren också bedömt frånvaro av ödem/svullnad. Konklusion: Det är vanligt att patienten upplever besvär/symtom från armen på den opererade sidan, och att patienter upplever symtom i större utsträckning än att läkaren gjort bedömningen att handödem, subcutant ödem eller armsvullnad förekommer. / Background: Breast cancer-related lymphedema (BCRL) is a complication following breast cancer treatment, it is probably underdiagnosed and the prevalence varies in previous studies. Early signs can be subtle sensations of heaviness and/or tightness in limbs. BCRL can have several negative impact, this motivates the importance of early detection because early intervention improves the prognosis.   Aim: Investigate the prevalence of early signs/symtoms of BCRL and investigate the association between patient reported outcomes and the physician assessment of BCRL.  Method: Women who underwent curative treatment of breast cancer was included. Data was collected at the follow-up 3 months after finished treatment in purpose for a clinical project. A questionnaire was used for the patient (regarding signs/symtoms from the arm) and physician rescpectively. Results: 61 participants were included. 22 participants reported signs from the arm on the affected side. 70% of those who reported symptoms, reported a presence of ≥1 symtoms. The physicians estimated no signs in 76% of 55 participants. There was an association between the frequency of patient reported signs from the arm and the physicians assessment of armswelling (p=0,005), hand edema (p=0,003) and between patient reported symtoms and the physicians assessment of armswelling (p=0,008). When the patient reported signs/symptoms, there was a significant proportion where the physician estimated no edema/swelling. In cases when the patient reported no signs/symptoms, the physician also assessed absence of edema/swelling. Conclusion: Patients often experience signs/symptoms from the arm on the affected side, and patients reports symptoms more frequent than physicians assess presence of early signs of BCRL.

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.

In recent years, there has been an ever increasing emphasis placed on the collection and use of patient reported outcome measures (PROM) in mental health services. This emphasis stems from a culture of evidence based practice, wherein PROM are shown to improve therapeutic outcomes at the clinical level, as well as provide information for the appropriate development of services and commissioning at a national level. This study uses an online survey to explore the use of PROM by mental health staff (n=112) in various Child and Adolescent Mental Health Services across England. Attitudes toward routine outcome monitoring practices and perceived competency around PROM use were also measured. Results found that although significant numbers of staff were using PROM, the amount of data being collected falls short of policy targets. Staff’s attitudes towards the practice are shown to be ambivalent, whereas overall perceived levels of competency were reasonably good. The relationships between attitudes, competence and PROM usage are discussed and a prediction model for PROM usage is developed in light of existing psychological theory. Results showed that training played an important role in the uptake of PROM and implications for the dissemination of training programs are emphasised.

Bakgrund: Integrated Palliative care Outcome Scale (IPOS) syftar till att ge en multidimensionell bild av patienters situation. IPOS används som ett sätt att bedöma patienters upplevelse av sina symtom, sin funktionsförmåga och sitt välbefinnande i relation till svår sjukdom. Få studier har dock utgått från patienternas perspektiv för att undersöka upplevelser av att använda IPOS. Syfte: Att undersöka patienters erfarenheter av att använda Integrated Palliative care Outcome Scale under pågående specialiserad palliativ hemsjukvård. Metod: Studien är genomförd med en kvalitativ ansats och en tolkande beskrivande design. Rekrytering till studien gjordes från tre specialiserade palliativa hemsjukvårdsenheter. Intervjuer genomfördes med 10 patienter, sju män och tre kvinnor med medelåldern 72 år. En majoritet var diagnostiserade med obotlig cancer. Data analyserades genom tolkande beskrivning så som den beskrivs av Sally Thorne. Resultat: Patienterna upplevde att använda IPOS medförde säker vård eftersom att det underlättade för sjuksköterskorna att göra korrekta bedömningar av patienternas behov. Således var IPOS en hjälp att planera vården utifrån patienternas specifika behov. Detta gav patienterna en känsla av att vården de fick var anpassad för dem, vilket gav en känsla av trygghet. De upplevde även att använda IPOS försäkrade att information fördes vidare på ett korrekt sätt från patienterna till vårdpersonalen genom att det minskade risken för missförstånd. De uppgav att IPOS underlättade diskussioner mellan dem och sjuksköterskorna om sådant de upplevde som viktigt. Patienterna upplevde även att använda IPOS möjliggjorde för dem att reflektera över sitt välbefinnande och sin livssituation. Att göra detta tillsammans med en sjuksköterska var berikande eftersom att det kunde ge nya perspektiv. Slutsats: Resultatet indikerar att patienterna i denna studie upplever att använda IPOS var givande för dem. Det kan konstateras att IPOS med fördel kan användas i specialiserad palliativ hemsjukvård. / Background: The Integrated Palliative care Outcome Scale (IPOS) is intended to provide a multidimensional view of patients’ concerns. IPOS can be used with the purpose of assessing patients’ perceptions of their functional status and wellbeing. Few studies have undertaken the patients’ perspective when exploring experiences of using IPOS. Aim: To explore patients’ experiences of using the Integrated Palliative care Outcome Scale during specialized palliative home care. Method: The study adopted a qualitative approach with an interpretive descriptive design. The patients were recruited from three different specialized palliative home care settings. Interviews were performed with 10 patients, seven men and three women, with a mean age of 72 years. A majority of the patients were diagnosed with incurable cancer. Data were analysed using interpretive description, as described by Sally Thorne. Results: Patients experienced that the use of IPOS entailed secure care as it facilitated nurses in making accurate assessments of patient care needs. IPOS helped to plan the care according to patients’ specific needs, making them feel confident that the care provided was tailored to them, giving a sense of security. Patients expressed that IPOS facilitated discussions between them and the nurse about care needs. They believed that using IPOS enabled opportunities for reflection on their wellbeing and life situation. Doing so with a nurse present was enriching, providing new perspectives. Conclusions: Patients experienced that using IPOS was beneficial. It can be concluded that IPOS provide an effective way to enable person-centred care and with advantage could be used in specialized palliative home care.

The World Health Organization Disability Assessment Schedule 2.0 (WHODAS 2.0) is a leading patient-reported outcome measure of disability. However, patients' perspective of the WHODAS 2.0 self-administered Swedish version have not been sufficiently described. Similarly, knowledge of its construct validity in the general population is missing. This creates a gap because updated norm data to use as reference is also missing. The overall aim of this study was to establish evidence of the measurement properties of the self-administered Swedish version of the WHODAS 2.0. Both qualitative and quantitative methods were adopted. In Study I, interviews with patients (n = 12) with orthopaedic or psychiatric conditions were performed and data were analysed by deductive content analysis. In Study II, a cross-sectional general population survey (n = 3 482) was conducted, and statistical methods based on classical test theory were used in the data analysis. The results show that the items were well understood, acceptable and easy to answer by outpatients, with the exception of six items (Study I).The internal consistency reliability was good or excellent and the construct validity was overall acceptable, with partial support for the factor structure in the general populations (Study II). The self-administered Swedish 36-item version of the WHODAS 2.0 is comparable to other language versions of the instrument. Some of the previous known weaknesses of its construct validity in relation to the item content and insufficient instructions were confirmed. The evidence of overall good content and construct validity together with available norm data supports its use in clinical settings and research.