Aim: To develop an outcome measure to identify “responders” for patients who had a clinically important improvement in lupus disease activity with treatment. Methods: The outcome measure derived was based on the commonly used disease activity measure SLEDAI-2K which documents findings over the previous 10 days. Since patients in drug trials are followed at monthly intervals it was necessary to validate SLEDAI-2K 30 days against SLEDAI-2K 10 days. Two prospective studies were accomplished for this purpose. SLEDAI-2K 30 days was used to develop the new responder index, SLEDAI-2K Responder Index-50 (SRI-50). The SRI-50 data retrieval form was developed to standardize the documentation of the descriptors. The construct validity of SRI-50 was prospectively evaluated against an external construct. The reliability of SRI-50 was tested in a multi-centre study. A retrospective analysis over 10 years was used to further validate SRI-50. SRI-50 ability to enhance the SLE Responder Index (SRI) in detecting “responders” was evaluated. The performance of SRI-50 was investigated against SLEDAI-2K and SRI over 12 months. Results: SLEDAI-2K 30 days was validated to describe disease activity over the previous 30 days. The responder index to SLEDAI-2K, SRI-50 was developed including the SRI-50 Definitions and SRI-50 Data Retrieval Forms. The initial validation of SRI-50 confirmed its construct validity to identify ≥ 50 % improvement. SRI-50 is reliable and can be used by both rheumatologists and trainees. The retrospective analysis confirmed that SRI-50 is valid in identifying ≥ 50 % improvement in an efficient time. SRI-50 enhances the performance of SRI and identifies more “responders” than SLEDAI-2K and SRI at 6 and 12 months. Conclusions: SRI-50, is a valid and reliable responder index to identify patients with partial, ≥50% improvement in disease activity in an efficient time. SRI-50 can be used as an independent outcome measure of improvement in patients with SLE.

Aim: To develop an outcome measure to identify “responders” for patients who had a clinically important improvement in lupus disease activity with treatment. Methods: The outcome measure derived was based on the commonly used disease activity measure SLEDAI-2K which documents findings over the previous 10 days. Since patients in drug trials are followed at monthly intervals it was necessary to validate SLEDAI-2K 30 days against SLEDAI-2K 10 days. Two prospective studies were accomplished for this purpose. SLEDAI-2K 30 days was used to develop the new responder index, SLEDAI-2K Responder Index-50 (SRI-50). The SRI-50 data retrieval form was developed to standardize the documentation of the descriptors. The construct validity of SRI-50 was prospectively evaluated against an external construct. The reliability of SRI-50 was tested in a multi-centre study. A retrospective analysis over 10 years was used to further validate SRI-50. SRI-50 ability to enhance the SLE Responder Index (SRI) in detecting “responders” was evaluated. The performance of SRI-50 was investigated against SLEDAI-2K and SRI over 12 months. Results: SLEDAI-2K 30 days was validated to describe disease activity over the previous 30 days. The responder index to SLEDAI-2K, SRI-50 was developed including the SRI-50 Definitions and SRI-50 Data Retrieval Forms. The initial validation of SRI-50 confirmed its construct validity to identify ≥ 50 % improvement. SRI-50 is reliable and can be used by both rheumatologists and trainees. The retrospective analysis confirmed that SRI-50 is valid in identifying ≥ 50 % improvement in an efficient time. SRI-50 enhances the performance of SRI and identifies more “responders” than SLEDAI-2K and SRI at 6 and 12 months. Conclusions: SRI-50, is a valid and reliable responder index to identify patients with partial, ≥50% improvement in disease activity in an efficient time. SRI-50 can be used as an independent outcome measure of improvement in patients with SLE.

Author's name: Bc. Sára Miczová Supervisor:: Mgr. Miriama Dědková, DiS Opponent: Thesis title: Occupational therapy practise guidelines in patients with functional upper limb impairment after stroke in subacute phase To be able to provide occupational therapy intervention of high quality the occupatioal therpaists should rely on relevant standards of clinical care and clinical guidelines. (Švestková, 2018). Occupational therapists as well as other members of the interdisciplinar team provide professional care to the patients recovering from stroke. Those patients often have to deal with upper limb impairment which leads to decrese of quality of life. Relevant studies focused on this topic were searched via key words in PubMed, Web of Science and OVID Evidence Based Medicine Reviews databasis. The criterias of selection were: public access to the content, english language which were mainly focused on stroke patients rehabilitation. The suggestion of guideline was created based on methods accepted by department. The occupational therapist as well as physiotherapeutist of early rehabilitation were included in the process of creating the guideline using the GRADE programme for it. Only 48 out of 4054 studies were used, these were divided into two cathegories, one being the cathegory of assessment, the...

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Severe perineal trauma with injury to the anal sphincter at childbirth can have a profound effect on the physical and psychological wellbeing of women. This thesis describes literature examining resulting outcomes including effects on body image. It describes patient-based outcome measures used to capture this information, and evaluation of their psychometric properties. Body Image can be defined as an individual's perceptions and feelings about their own body. There is a growing interest in how this concept can influence quality of life and psychosocial dysfunction in medical disorders. This thesis aimed to examine relationships between severity of perineal trauma, general and genital specific body image and potentially influences such as symptoms of pelvic floor dysfunction. No patient-reported outcome measure validated for use in women after anal sphincter injury exists and this thesis also aimed to psychometrically evaluate an existing electronic questionnaire, ePAQ (electronic personal assessment questionnaire), for this application. In the thesis, a retrospective review of body image and physical outcomes attending a perineal clinic shows over half of women report perceived changes in body image after anal sphincter injury, with negative effects on self-esteem. A prospective observational cohort study explored genital and general body image in primiparous women grouped according to degree of perineal trauma or caesarean delivery. Women completed the Female Genital Self Image Score, the modified Body Image Score, ePAQ and the Edinburgh Postnatal Depression Scale a mean of 15.5 weeks (standard deviation 1.6) after delivery. There were significant differences in genital body image scores between the groups, but not in general body image, with regression analysis showing the greatest influence on genital body image to be the anatomical extent of the trauma. Embedded into this study was the evaluation of reliability (internal consistency and test-retest) and validity (face, content and construct) of ePAQ in the group of women with anal sphincter tears. This thesis presents the first research to quantify issues surrounding severe perineal trauma and body image and demonstrates that more severe trauma leads to a poorer genital body image. It also reports psychometric evaluation of ePAQ in women after anal sphincter injury providing the first single instrument with validity and reliability for use in this context.

This thesis uses the methods of sychometrics and clinimetrics to develop and evaluate a Pediatric Ulcerative Colitis Activity Index (PUCAI). The initial phases of item generation and reduction were performed previously. This thesis comprises five main studies. Study one: the weighting and formatting of an initial draft PUCAI using a cohort of 157 children with ulcerative colitis, enrolled prospectively in five pediatric IBD centers. Study two: the validation of the final draft on a separate prospective cohort of 48 children undergoing complete colonoscopy. The PUCAI was highly correlated with physician global assessment (PGA) (r=0.91), Mayo score (r=0.95) and colonoscopic appearance (r=0.77). The PUCAI was able to differentiate the different categories of disease activity, and cutoff points were defined. Study three: Assessment of the responsiveness of the PUCAI. The index demonstrated excellent responsiveness on 75 children seen twice during the study period (effect size=1.9, standardized response mean=2.2, responsiveness statistics=2.6, correlation with PGA of change=0.84, and area under the ROC curve=0.97 95%CI 0.93- 0.99). Study four was aimed at evaluating the predictive validity of the PUCAI, on a retrospective cohort of 99 children with severe ulcerative colitis admitted for intravenous corticosteroid therapy. The PUCAI, calculated on the third and fifth day of therapy was highly predictive of therapy failure at discharge and one year post discharge (area under the ROC curve 0.84 (95%CI 0.76-0.92). Study five: a methodological study evaluating the preferred way to determine the minimal clinically important difference (MCID) of health-related outcome measures. This study was conducted using the PUCAI and three other well established instruments. It was concluded that the MCID should be determined primarily by the anchor-based approach using the ROC curve method on the entire cohort, supplemented by calculating the minimal detectable difference beyond statistical error using the standard error of measurement. Small, moderate and large MCID values could be presented based on the degree of expected relevant change. Together, these studies have contributed to the rigorous development and thorough evaluation of a novel, non-invasive tool for assessing disease activity in pediatric ulcerative colitis clinical studies and practice.

This thesis uses the methods of sychometrics and clinimetrics to develop and evaluate a Pediatric Ulcerative Colitis Activity Index (PUCAI). The initial phases of item generation and reduction were performed previously. This thesis comprises five main studies. Study one: the weighting and formatting of an initial draft PUCAI using a cohort of 157 children with ulcerative colitis, enrolled prospectively in five pediatric IBD centers. Study two: the validation of the final draft on a separate prospective cohort of 48 children undergoing complete colonoscopy. The PUCAI was highly correlated with physician global assessment (PGA) (r=0.91), Mayo score (r=0.95) and colonoscopic appearance (r=0.77). The PUCAI was able to differentiate the different categories of disease activity, and cutoff points were defined. Study three: Assessment of the responsiveness of the PUCAI. The index demonstrated excellent responsiveness on 75 children seen twice during the study period (effect size=1.9, standardized response mean=2.2, responsiveness statistics=2.6, correlation with PGA of change=0.84, and area under the ROC curve=0.97 95%CI 0.93- 0.99). Study four was aimed at evaluating the predictive validity of the PUCAI, on a retrospective cohort of 99 children with severe ulcerative colitis admitted for intravenous corticosteroid therapy. The PUCAI, calculated on the third and fifth day of therapy was highly predictive of therapy failure at discharge and one year post discharge (area under the ROC curve 0.84 (95%CI 0.76-0.92). Study five: a methodological study evaluating the preferred way to determine the minimal clinically important difference (MCID) of health-related outcome measures. This study was conducted using the PUCAI and three other well established instruments. It was concluded that the MCID should be determined primarily by the anchor-based approach using the ROC curve method on the entire cohort, supplemented by calculating the minimal detectable difference beyond statistical error using the standard error of measurement. Small, moderate and large MCID values could be presented based on the degree of expected relevant change. Together, these studies have contributed to the rigorous development and thorough evaluation of a novel, non-invasive tool for assessing disease activity in pediatric ulcerative colitis clinical studies and practice.

The psychometric properties of a new high-level mobility outcome measure for Service Members (SMs) with traumatic lower limb loss called the Comprehensive High-level Activity Mobility Predictor (CHAMP) was developed in order objectively evaluate functional abilities and measure change in function throughout the rehabilitation process. The CHAMP was administered to a population of SMs with traumatic lower limb loss who were representative of those who have suffered limb loss in recent conflicts. In addition, a population of non-amputee Active Duty soldiers completed the CHAMP and provided normative data for high-level mobility and threshold levels of performance for those SMs with lower limb loss. A simple grading system, absent of floor and ceiling effects, generates a composite score providing a single numeric value representing the physical performance factors of high-level mobility in different plane of motion and under different conditions. The CHAMP was found to be a safe, reliable, valid, and responsive performance-based outcome measure of high-level mobility. It was found to have excellent interrater and test-retest reliability suggesting that it is a stable and repeatable measure of high-level mobility. Convergent construct validity and known group methods were utilized to establish the CHAMP as a valid measure of high-level mobility. Predictive models of CHAMP performance were established utilizing variables representing impairments of body structure and function and contextual factors by level of lower limb amputation. The CHAMP has the potential to be used for lower limb amputees throughout the rehabilitation process and could translate to the non-amputee population for assessment of high-level mobility capabilities.

Syftet med studien var att validera den svenska översättningen av självskattningsinstrumentet Norwegian Outcome Response System for Evaluation (NORSE), ett instrument som används för att mäta psykiskt mående. Data har samlats in genom en digital enkät. Urvalet var icke-kliniskt och bestod av totalt 228 individer. Av de 228 individerna gjorde 30 personer enkäten vid två tillfällen för att testa instrumentets stabilitet över tid. För att validera NORSE användes ett redan validerat och etablerat instrument, Clinical Outcomes in Routine Evaluation Outcome Measure (CORE-OM) som standardreferens. Resultatet visade att den svenska översättningen av NORSE har hög intern konsistens och god test-retest reliabilitet, såväl som god reliabilitet och validitet. Resultaten visade även att NORSE och CORE-OM täcker liknande områden men är samtidigt långt ifrån identiska. Föreliggande studie indikerar att det kan finnas ett användningsområde för den svenska översättningen av NORSE i den svenska primärvården. / The aim of this study was to validate the Swedish translation of the self-report instrument Norwegian Outcome Response System for Evaluation (NORSE), an instrument used to evaluate psychological well-being. Data was collected using a digital questionnaire. The sample was non-clinical and consisted of a total of 228 individuals. Out of the 228 individuals, 30 participants took the questionnaire on two occasions to test the stability of the instrument over time. In order to validate NORSE, an already validated and established instrument, Clinical Outcomes in Routine Evaluation Outcome Measure (CORE-OM) was used as a reference standard. The results showed that the Swedish translation of NORSE has a high internal consistency and good test-retest reliability, as well as a good internal reliability and concurrent validity. Results also showed that NORSE and CORE-OM cover similar areas but are far from identical, thus indicating that there could be a use for NORSE in Swedish primary health care.