Background: Patient-reported outcome measures (PROMs) can help predict clinical outcomes and improve shared clinical decision-making discussions. There remains a paucity of research assessing how... Show moreBackground: Patient-reported outcome measures (PROMs) can help predict clinical outcomes and improve shared clinical decision-making discussions. There remains a paucity of research assessing how the use of PROMs may drive improved patient experience and patient activation. Methods: New foot and ankle patients completed PROMIS physical function (PF), pain interference (PI), and depression assessments. Patients were then randomized to viewing and discussing their PROMIS scores with their surgeon or not. Following the clinic visit, patients completed a series of Clinician & Group Survey-Consumer Assessment of Healthcare Providers and Systems (CG-CAHPS) questions and the Patient Activation Measure (PAM). Responses to the CG-CAHPS questions and PAM were compared between the 2 groups and after clustering on surgeon. Potential interaction effects by social deprivation were also explored. Results: After enrolling patients but removing those lost to follow-up or with missing data, 97 and 116 patients remained in the intervention control cohorts, respectively. No difference was found in CG-CAHPS responses nor PAM scores between the 2 groups (P > .05). All surgeons were highly rated by all patients. When clustered by surgeon, intervention subjects were less likely to indicate "top box" scores for the understanding domain of the CG-CAHPS question (OR 0.51, P < .001) and had decreased odds of high patient activation compared to control subjects (OR 0.67; P = .005). Among the most socially disadvantaged patients, there was no difference in control and intervention subjects in their likelihood of having high patient activation (P = .09). Conclusion: Highly rated foot and ankle surgeons who show and discuss PROM results may not improve patient experience or activation and may, in fact, decrease understanding or patient activation in select populations. Future work is needed to determine when PROM discussions are most beneficial and how best to present PROMs data, as we suspect that how the information was presented-and not the use of PROMs-resulted in our findings. Health literacy tools and/or communication training may better engage different patient groups regarding PROMs. Show less
BackgroundThe Pulmonary Embolism Quality of Life (PEmb-QoL) questionnaire is the first disease-specific scale for assessing the quality of life in patients with a history of pulmonary embolism (PE)... Show moreBackgroundThe Pulmonary Embolism Quality of Life (PEmb-QoL) questionnaire is the first disease-specific scale for assessing the quality of life in patients with a history of pulmonary embolism (PE).ObjectivesTo assess the cross-cultural validity and reliability of the disease-specific PEmb-QoL questionnaire.MethodsThe Persian version was prepared through the forward and backward translation of the English questionnaire. Six months after the diagnosis of acute PE, consecutive Persian-speaking patients were asked to complete the PEmb-QoL, the generic 36-item Short Form (SF-36) questionnaires and undertake a 6-minute walk test (6MWT). Acceptability was assessed via item missing rate, reproducibility by the test-retest method, and internal consistency reliability by Cronbach’s α and McDonald’s ω coefficients. Convergence validity was assessed using the Spearman rank correlation between scores of PEmb-QoL, SF-36, and 6MWT. The questionnaire structure was evaluated through exploratory factor analysis.ResultsNinety-six patients with a confirmed diagnosis of PE completed the questionnaires. The Persian version of PEmb-QoL had good internal consistency (α = 0.95, 3-factor ω = 0.96), inter-item correlation (0.3–0.62), item-total correlation (0.38–0.71), reproducibility (test-retest ICC with 25 participants = 0.92–0.99), and good discriminant validity. Convergence validity was confirmed by the moderate-to-high correlations between PEmb-QoL and SF-36 scores, and a good correlation between the “limitation in daily activities” dimension of the PEmb-QoL questionnaire and 6MWT results. Exploratory factor analysis suggested a 3-component structure with functional (items 1h, 4b-5d, 6, 8, 9i, and 9j), symptoms (1b-h, 7, and 8), and emotional (5a, 6, and 9a-h) components.ConclusionThe Persian version of the PEmb-QoL questionnaire is valid and reliable for measuring the disease-specific quality of life in patients with PE. Show less
Veltkamp, D.M.J.; Wang, Y.M.; Meuleman, Y.; Dekker, F.W.; Michels, W.M.; Boog, P.J.M. van der; Vries, A.P.J. de 2023
Background Health-related quality of life (HRQOL) is an increasingly important patient-reported outcome in kidney transplant recipients (KTRs). This study explored relationships between symptom... Show moreBackground Health-related quality of life (HRQOL) is an increasingly important patient-reported outcome in kidney transplant recipients (KTRs). This study explored relationships between symptom prevalence and burden with HRQOL, and age and gender differences in symptom experience. Methods Eligible Dutch KTRs transplanted in Leiden University Medical Center were invited for this cross-sectional study. HRQOL, and occurrence and burden of 62 symptoms were measured using validated questionnaires. Univariate and multivariate regression analysis were used for investigating the associations of symptom experience with mental and physical HRQOL, and differences in symptom experience between genders and KTRs of diverse age groups. Results A total of 631 KTRs were analyzed; the mean (standard deviation) age was 61.3 (11.3) years, and 62% were male. The median (interquartile range) number of symptoms was 14 (7-22), with a burden of 20 (8-37; range 0-244). Per extra symptom, physical and mental HRQOL decreased [-0.41 (-0.50; -0.31) and -0.51 (-0.59; -0.42), respectively, P < .001]. Most occurring symptoms were bruises, tiredness, lack of energy, urge to urinate at night and dry skin. Sexual problems were considered most burdensome. Female KTRs reported more symptoms than men. Amongst others, younger KTRs experienced more (18-50 > 50-65 >= 65 years) feelings of depression and both female and younger KTRs reported higher symptom prevalence concerning changes in physical appearance. Conclusion KRTs' symptom experience differed depending on gender and age, highlighting the need to develop tailored treatment strategies to reduce symptom experience and subsequently improve HRQOL. Show less
Survivors of acute pulmonary embolism (PE) are at risk of developing persistent, sometimes disabling symptoms of dyspnea and/or functional limitations despite adequate anticoagulant treatment,... Show moreSurvivors of acute pulmonary embolism (PE) are at risk of developing persistent, sometimes disabling symptoms of dyspnea and/or functional limitations despite adequate anticoagulant treatment, fulfilling the criteria of the post-PE syndrome (PPES). PPES includes chronic thromboembolic pulmonary hypertension (CTEPH), chronic thromboembolic pulmonary disease, post-PE cardiac impairment (characterized as persistent right ventricle impairment after PE), and post-PE functional impairment. To improve the overall health outcomes of patients with acute PE, adequate measures to diagnose PPES and strategies to prevent and treat PPES are essential. Patient-reported outcome measures are very helpful to identify patients with persistent symptoms and functional impairment. The primary concern is to identify and adequately treat patients with CTEPH as early as possible. After CTEPH is ruled out, additional diagnostic tests including cardiopulmonary exercise tests, echocardiography, and imaging of the pulmonary vasculature may be helpful to rule out non-PE-related comorbidities and confirm the ultimate diagnosis. Most PPES patients will show signs of physical deconditioning as main explanation for their clinical presentation. Therefore, cardiopulmonary rehabilitation provides a good potential treatment option for this patient category, which warrants testing in adequately designed and executed randomized trials. In this review, we describe the definition and characteristics of PPES and its diagnosis and management. Show less
Survivors of acute pulmonary embolism (PE) are at risk of developing persistent, sometimes disabling symptoms of dyspnea and/or functional limitations despite adequate anticoagulant treatment,... Show moreSurvivors of acute pulmonary embolism (PE) are at risk of developing persistent, sometimes disabling symptoms of dyspnea and/or functional limitations despite adequate anticoagulant treatment, fulfilling the criteria of the post-PE syndrome (PPES). PPES includes chronic thromboembolic pulmonary hypertension (CTEPH), chronic thromboembolic pulmonary disease, post-PE cardiac impairment (characterized as persistent right ventricle impairment after PE), and post-PE functional impairment. To improve the overall health outcomes of patients with acute PE, adequate measures to diagnose PPES and strategies to prevent and treat PPES are essential. Patient-reported outcome measures are very helpful to identify patients with persistent symptoms and functional impairment. The primary concern is to identify and adequately treat patients with CTEPH as early as possible. After CTEPH is ruled out, additional diagnostic tests including cardiopulmonary exercise tests, echocardiography, and imaging of the pulmonary vasculature may be helpful to rule out non-PE-related comorbidities and confirm the ultimate diagnosis. Most PPES patients will show signs of physical deconditioning as main explanation for their clinical presentation. Therefore, cardiopulmonary rehabilitation provides a good potential treatment option for this patient category, which warrants testing in adequately designed and executed randomized trials. In this review, we describe the definition and characteristics of PPES and its diagnosis and management. Show less
Background and objective: To develop targeted and efficient follow-up programmes for patients hospitalized with coronavirus disease 2019 (COVID-19), structured and detailed insights in recovery... Show moreBackground and objective: To develop targeted and efficient follow-up programmes for patients hospitalized with coronavirus disease 2019 (COVID-19), structured and detailed insights in recovery trajectory are required. We aimed to gain detailed insights in long-term recovery after COVID-19 infection, using an online home monitoring programme including home spirometry. Moreover, we evaluated patient experiences with the home monitoring programme. Methods: In this prospective multicentre study, we included adults hospitalized due to COVID-19 with radiological abnormalities. For 6 months after discharge, patients collected weekly home spirometry and pulse oximetry measurements, and reported visual analogue scales on cough, dyspnoea and fatigue. Patients completed the fatigue assessment scale (FAS), global rating of change (GRC), EuroQol-5D-5L (EQ-5D-5L) and online tool for the assessment of burden of COVID-19 (ABCoV tool). Mixed models were used to analyse the results. Results: A total of 133 patients were included in this study (70.1% male, mean age 60 years [SD 10.54]). Patients had a mean baseline forced vital capacity of 3.25 L (95% CI: 2.99-3.44 L), which increased linearly in 6 months with 19.1% (Delta 0.62 L, p < 0.005). Patients reported substantial fatigue with no improvement over time. Nevertheless, health status improved significantly. After 6 months, patients scored their general well-being almost similar as before COVID-19. Overall, patients considered home spirometry useful and not burdensome. Conclusion: Six months after hospital admission for COVID-19, patients' lung function and quality of life were still improving, although fatigue persisted. Home monitoring enables detailed follow-up for patients with COVID-19 at low burden for patients and for the healthcare system. Show less
Simple Summary Radiotherapy is widely used as treatment for localized prostate cancer. Due to a high incidence and a good survival after treatment, a large number of prostate cancer survivors are... Show moreSimple Summary Radiotherapy is widely used as treatment for localized prostate cancer. Due to a high incidence and a good survival after treatment, a large number of prostate cancer survivors are at risk of developing late radiation toxicity. Symptoms may significantly affect quality of life; therefore, the monitoring of toxicities and evaluating their impact are increasingly important matters. Toxicities have always been assessed by physicians, but there is a growing interest in the use of questionnaires to be completed by patients themselves, so-called patient-reported outcome measures. The aim of this study was to compare both outcomes in long-term prostate cancer survivors, and to determine which outcome correlates best with a biological predictor of late radiation toxicity. In symptomatic patients, we found a low agreement; patients assigned greater severity to symptoms than the trial physician assistant did. Neither outcome correlated with the biological predictor. Consideration of both perspectives seems warranted to provide the best care. Patient-reported outcome measures (PROMs) are advocated for the monitoring of toxicity after radiotherapy. However, studies comparing physician- and patient-reported toxicity show low concordance. In this study, we compared physician- and patient-reported toxicity in long-term prostate cancer survivors after radiotherapy, and we determined the correlation with a presumable risk factor for late toxicity: gamma-H2AX foci decay ratio (FDR). Patients formerly included in a prospective study were invited to participate in this new study, comprising one questionnaire and one call with a trial physician assistant. Concordance was calculated for seven symptoms. Gamma-H2AX FDRs were determined in ex vivo irradiated lymphocytes in a previous analysis. Associations between FDR and long-term prevalence of toxicity were assessed using univariable logistic regression analyses. The 101 participants had a median follow-up period of 9 years. Outcomes were discordant in 71% of symptomatic patients; in 21%, the physician-assessed toxicity (using CTCAE) was higher, and, in 50%, the patients reported higher toxicity. We did not find a correlation between presence of toxicity at long-term follow-up and FDR. In conclusion, patients assigned greater severity to symptoms than the trial physician assistant did. Consideration of both perspectives may be warranted to provide the best care. Show less
Background: There is increased demand for valid, reliable, and responsive patient-reported outcome measures (PROMs) to evaluate treatment for Achilles tendon rupture, but not all PROMs currently in... Show moreBackground: There is increased demand for valid, reliable, and responsive patient-reported outcome measures (PROMs) to evaluate treatment for Achilles tendon rupture, but not all PROMs currently in use are reliable and responsive for this condition. Purpose: To evaluate the measurement properties of the Patient-Reported Outcomes Measurement Information System Physical Function (PROMIS PF) compared with other PROMs used after treatment for acute Achilles tendon rupture. Study Design: Cohort study (diagnosis); Level of evidence, 2. Methods: A retrospective cohort study with a follow-up questionnaire was performed. All adult patients with an acute Achilles tendon rupture between June 2016 and June 2018 with a minimum 12-month follow-up were eligible for inclusion. Functional outcome was assessed using the PROMIS PF computerized adaptive test (CAT), Foot and Ankle Ability Measure (FAAM) Activities of Daily Living (ADL), FAAM-Sports, and Achilles Tendon Total Rupture Score (ATRS). Pearson correlation (r) was used to assess the correlations between PROMs. Absolute and relative floor and ceiling effects were calculated. Results: In total, 103 patients were included (mean age, 44.7 years; 74% male); 82 patients (79.6%) underwent operative repair, while 21 patients (20.4%) underwent nonoperative management. The mean time between treatment and collection of PROMs was 25.3 months (range, 15-36 months). The mean scores were 55.4 +/- 9.2 (PROMIS PF), 92.9 +/- 12.2 (FAAM-ADL), 77.7 +/- 22.9 (FAAM-Sports), and 83.0 +/- 19.4 (ATRS). The ATRS was correlated with FAAM-ADL (r = 0.80; 95% CI, 0.72-0.86; P < .001) and FAAM-Sports (r = 0.86; 95% CI, 0.80-0.90; P < .001). The PROMIS PF was correlated with the FAAM-ADL (r = 0.66; 95% CI, 0.53-0.75; P < .001), FAAM-Sports (r = 0.65; 95% CI, 0.53-0.75; P < .001), and ATRS (r = 0.69; 95% CI, 0.58-0.78; P < .001). The PROMIS PF did not show absolute floor or ceiling effects (0%). The FAAM-ADL (35.9%), FAAM-Sports (15.8%), and ATRS (20.4%) had substantial absolute ceiling effects. Conclusion: The PROMIS PF, FAAM-ADL, and FAAM-Sports all showed a moderate to high mutual correlation with the ATRS. Only the PROMIS PF avoided substantial floor and ceiling effects. The results suggest that the PROMIS PF CAT is a valid, reliable, and perhaps the most responsive tool to evaluate patient outcomes after treatment for an Achilles tendon rupture. Show less
Loef, M.; Gademan, M.G.J.; Latijnhouwers, D.A.J.M.; Kroon, H.M.; Kaptijn, H.H.; Marijnissen, W.J.C.M.; ... ; LOAS Study Grp 2021
Background: We aimed to investigate the application of the Knee Injury and Osteoarthritis Outcome Score (KOOS) percentile curves, using preoperative and postoperative data of patients with knee... Show moreBackground: We aimed to investigate the application of the Knee Injury and Osteoarthritis Outcome Score (KOOS) percentile curves, using preoperative and postoperative data of patients with knee osteoarthritis undergoing total knee arthroplasty (TKA).Methods: We used Longitudinal Leiden Orthopedics Outcomes of Osteo-Arthritis study data of patients between 45 and 65 years and undergoing primary TKA. KOOS scores (0-100) were obtained preoperatively and 6, 12, and 24 months after TKA. Preoperative knee radiographs were assessed according to Kellgren-Lawrence (KL) in a subset (37%) of patients. Comorbidities were self-reported using a standardized questionnaire. The median (interquartile range) population-level KOOS scores were plotted on previously developed population-based KOOS percentile curves. In addition, we assessed the application of the curves on patient level and investigated differences in scores between patients with preoperative KL scores <= 2 and >= 3 and presence (vs absence) of comorbidities.Results: The study population consisted of 853 patients (62% women, mean age 59 years, body mass index 30 kg/m(2)) with knee osteoarthritis undergoing primary TKA. Preoperatively, median KOOS scores of all subscales were at or below the 2.5th percentile. Scores increased to approximately the 25th percentile 12 months postoperatively. Greater improvements were observed in pain and less improvements in sport and recreational function and quality of life. Patients with higher preoperative KL scores and without comorbidities showed greater improvements.Conclusion: The KOOS percentile curves provided visual insights in knee complaints of patients relative to the general population. Furthermore, the KOOS percentile curves give insight in how preoperative patient characteristics are correlated with postoperative results. (C) 2021 The Author(s). Published by Elsevier Inc. Show less
To improve the quality and accuracy of the patient-reported outcome measures that assess health-related quality of life (HRQoL), guidelines have been developed to standardize the development and... Show moreTo improve the quality and accuracy of the patient-reported outcome measures that assess health-related quality of life (HRQoL), guidelines have been developed to standardize the development and validation process. Considering the increasing importance of HRQoL questionnaires in research, we set out to review the literature and evaluate whether existing questionnaires developed for deep vein thrombosis (DVT) and pulmonary embolism (PE) fulfill state-of-the-art requirements. The literature search was conducted in March 2019 and updated in September 2020. Seven databases were searched. No time limit was set for the search to include all available questionnaires. The inclusion criteria were original publications describing the development of disease-specific HRQoL questionnaires specific to DVT or PE in adults and available in English. The questionnaires were assessed to determine whether they fulfill the requirements in the latest guidelines. A total of 3826 references were identified. After the exclusion process, 15 papers were reviewed in full, of which 7 were included. Four questionnaires were developed for chronic venous disease, two were specific to DVT, and one was specific to PE. Most questionnaires we found in this review, fulfilled some but none fulfilled all recommendations in existing guidelines. Because the development of current available HRQoL questionnaires specific to DVT or PE do not fulfil all recommendations of existing guidelines, there is room for improvements within this field. Such improvements could likely enhance the quality associated with the use of these end points in clinical trials and practice. Show less
Traumatic brain injury (TBI) may lead to impairments in various outcome domains. Since most instruments assessing these are only available in a limited number of languages, psychometrically... Show moreTraumatic brain injury (TBI) may lead to impairments in various outcome domains. Since most instruments assessing these are only available in a limited number of languages, psychometrically validated translations are important for research and clinical practice. Thus, our aim was to investigate the psychometric properties of the patient-reported outcome measures (PROM) applied in the CENTER-TBI study. The study sample comprised individuals who filled in the six-months assessments (GAD-7, PHQ-9, PCL-5, RPQ, QOLIBRI/-OS, SF-36v2/-12v2). Classical psychometric characteristics were investigated and compared with those of the original English versions. The reliability was satisfactory to excellent; the instruments were comparable to each other and to the original versions. Validity analyses demonstrated medium to high correlations with well-established measures. The original factor structure was replicated by all the translations, except for the RPQ, SF-36v2/-12v2 and some language samples for the PCL-5, most probably due to the factor structure of the original instruments. The translation of one to two items of the PHQ-9, RPQ, PCL-5, and QOLIBRI in three languages could be improved in the future to enhance scoring and application at the individual level. Researchers and clinicians now have access to reliable and valid instruments to improve outcome assessment after TBI in national and international health care. Show less
Willik, E.M. van der; Hemmelder, M.H.; Bart, H.A.J.; Ittersum, F.J. van; Hoogendijk-van den Akker, J.M.; Bos, W.J.W.; ... ; Meuleman, Y. 2021
Background. The use of patient-reported outcome measures (PROMS) is becoming increasingly important in healthcare. However, incorporation of PROMS into routine nephrological care is challenging.... Show moreBackground. The use of patient-reported outcome measures (PROMS) is becoming increasingly important in healthcare. However, incorporation of PROMS into routine nephrological care is challenging. This study describes the first experience with PROMS in Dutch routine dialysis care.Methods. A pilot study was conducted in dialysis patients in 16 centres. Patients were invited to complete PROMS at baseline and 3 and 6 months. PROMS consisted of the 12-item short-form and Dialysis Symptom Index to assess health-related quality of life (HRQoL) and symptom burden. Response rates, HRQoL and symptom burden scores were analysed. Qualitative research methods were used to gain insight into patients' views on using PROMS in clinical practice.Results. In total, 512 patients (36%) completed 908 PROMS (24%) across three time points. Response rates varied from 6 to 70% among centres. Mean scores for physical and mental HRQoL were 35.6 [standard deviation (SD) 10.2] and 47.7 (SD 10.6), respectively. Patients experienced on average 10.8 (SD 6.1) symptoms with a symptom burden score of 30.7 (SD 22.0). Only 1-3% of the variation in PROM scores can be explained by differences between centres. Patients perceived discussing their HRQoL and symptom scores as insightful and valuable. Individual feedback on results was considered crucial.Conclusions. The first results show low average response rates with high variability among centres. Dialysis patients experienced a high symptom burden and poor HRQoL. Using PROMs at the individual patient level is suitable and may improve patient-professional communication and shared decision making. Further research is needed to investigate how the collection and the use of PROMs can be successfully integrated into routine care to improve healthcare quality and outcomes. Show less
Study Design. Retrospective, observational study. Objective. To determine the association of patient socioeconomic disadvantage, insurance type, and other characteristics on presenting symptom... Show moreStudy Design. Retrospective, observational study. Objective. To determine the association of patient socioeconomic disadvantage, insurance type, and other characteristics on presenting symptom severity in patients with isolated lumbar disc herniation. Summary of Background Data. Little is known of the impact of socioeconomic disadvantage and other patient characteristics on the level of self-reported symptom severity when patients first seek care for lumbar disc herniation. Methods. Between April 2015 and December 2018, 734 patients newly presenting for isolated lumbar disc herniation who completed the Patient-Reported Outcomes Measurement Information System Physical Function (PF), Pain Interference (PI), and Depression Computer Adaptive Tests (CATs) were identified. Socioeconomic disadvantage was determined using the Area Deprivation Index, a validated measure of socioeconomic disadvantage at the census block group level (0-100, 100 = highest socioeconomic disadvantage). Bivariate analyses were used. Multivariable linear regression was used to determine if there was an association between socioeconomic disadvantage, insurance type, and other patient factors and presenting patient-reported health status. Results. Significant differences in age, insurance type, self-reported race, marital status, and county of residence were appreciated when comparing patient characteristics by socioeconomic disadvantage levels (all comparisons, P < 0.01). In addition, significant differences in age, insurance type, marital status, and county of residence were appreciated when comparing patient characteristics by self-reported race (all comparisons, P < 0.01). Being in the most socioeconomically disadvantaged cohort was associated with worse presenting Patient-Reported Outcomes Measurement Information System scores (Physical Function: beta = -3.27 (95% confidence interval [CI]: -4.89 to -1.45), P < 0.001; Pain Interference: beta = 3.20 (95% CI: 1.58-4.83), P < 0.001; Depression: beta = 3.31 (95% CI: 1.08-5.55), P = 0.004. Conclusion. The most socioeconomically disadvantaged patients with symptomatic lumbar disc herniations present with worse functional limitations, pain levels, and depressive symptoms as compared to patients from the least socioeconomically disadvantaged cohort when accounting for other key patient factors. Show less
Background: Patient-relevant health outcomes for persons with hemophilia should be identified and prioritized to optimize and individualize care for persons with hemophilia. Therefore, an... Show moreBackground: Patient-relevant health outcomes for persons with hemophilia should be identified and prioritized to optimize and individualize care for persons with hemophilia. Therefore, an international group of persons with hemophilia and multidisciplinary health care providers set out to identify a globally applicable standard set of health outcomes relevant to all individuals with hemophilia.Methods: A systematic literature search was performed to identify possible health outcomes and risk adjustment variables. Persons with hemophilia and multidisciplinary health care providers were involved in an iterative nominal consensus process to select the most important health outcomes and risk adjustment variables for persons with hemophilia. Recommendations were made for outcome measurement instruments.Results: Persons with hemophilia were defined as all men and women with an X-linked inherited bleeding disorder caused by a deficiency of coagulation factor VIII or IX with plasma activity levels <40 IU/dL. We recommend collecting the following 10 health outcomes at least annually, if applicable: (i) cure, (ii) impact of disease on life expectancy, (iii) ability to engage in normal daily activities, (iv) severe bleeding episodes, (v) number of days lost from school or work, (vi) chronic pain, (vii) disease and treatment complications, (viii) sustainability of physical functioning, (ix) social functioning, and (x) mental health. Validated clinical as well as patient-reported outcome measurement instruments were endorsed. Demographic factors, baseline clinical factors, and treatment factors were identified as risk-adjustment variables.Conclusion: A consensus-based international set of health outcomes relevant to all persons with hemophilia, and corresponding measurement instruments, was identified for use in clinical care to facilitate harmonized longitudinal monitoring and comparison of outcomes. Show less
Objective: To compare the effect on disability and quality of life, of conventional rehabilitation (control group) with individualized, tailored eRehabilitation intervention alongside conventional... Show moreObjective: To compare the effect on disability and quality of life, of conventional rehabilitation (control group) with individualized, tailored eRehabilitation intervention alongside conventional rehabilitation (Fast@home; intervention group), for people with stroke.Methods: Pre-post design. The intervention comprised cognitive (Braingymmer (R)) and physical (Telere-validatie (R)/Physitrack (R)) exercises, activity-tracking (Activ8 (R)) and psycho-education. Assessments were made at admission (T0) and after 3 (T3) and 6 months (T6). The primary outcome concerned disability (Stroke Impact Scale; SIS). Secondary outcomes were: health-related qual-ity of life, fatigue, self-management, participation and physical activity. Changes in scores be-tween T0-T3, T3-T6, and T0-T6 were compared by analysis of variance and linear mixed models.Results: The study included 153 and 165 people with stroke in the control and intervention groups, respectively. In the intervention group, 82 (50%) people received the intervention, of whom 54 (66%) used it. Between T3 and T6, the change in scores for the SIS subscales Communication (control group/ intervention group -1.7/-0.3) and Physical strength (-5.7/3.3) were significantly greater in the total intervention group (all mean differences< minimally clinically important differences). No significant differences were found for other SIS subscales or secondary outcomes, or between T0-T3 and T0-T6.Conclusion: eRehabilitation alongside conventional stroke rehabilitation had a small positive effect on communication and physical strength on the longer term, compared to conventional rehabilitation only. Show less
Introduction Although patient-reported outcome measures (PROMs) are increasingly used in clinical practice and research, it is unclear whether these instruments cover the perspective of young... Show moreIntroduction Although patient-reported outcome measures (PROMs) are increasingly used in clinical practice and research, it is unclear whether these instruments cover the perspective of young people with inflammatory arthritis (IA). The aims of this study were to explore whether PROMs commonly used in IA adequately cover the perspective of young people from different European countries.Methods A multinational qualitative study was conducted in Austria, Croatia, Italy and the Netherlands. Young people with either rheumatoid arthritis (RA), juvenile idiopathic arthritis (JIA), Still's disease, psoriatic arthritis (PsA) or spondyloarthritis (SpA), aged 18-35 years, participated in semistructured focus group interviews. Thematic analysis was used and data saturation was defined as no new emergent concepts in at least three subsequent focus groups.Results Fifty-three patients (21 with RA/JIA/Still's, 17 with PsA, 15 with SpA; 72% women) participated in 12 focus groups. Participants expressed a general positive attitude towards PROMs and emphasised their importance in clinical practice. In addition, 48 lower level concepts were extracted and summarised into 6 higher level concepts describing potential issues for improvement. These included: need for lay-term information regarding the purpose of using PROMs; updates of certain outdated items and using digital technology for data acquisition. Some participants admitted their tendency to rate pain, fatigue or disease activity differently from what they actually felt for various reasons.Conclusions Despite their general positive attitude, young people with IA suggested areas for PROM development to ensure that important concepts are included, making PROMs relevant over the entire course of a chronic disease. Show less
Strand, V.; Heijde, D. van der; Tanaka, Y.; Keystone, E.; Kremer, J.; Zerbini, C.A.F.; ... ; ORAL Scan Investigators 2020
ObjectiveTofacitinib is an oral Janus kinase inhibitor for the treatment of rheumatoid arthritis (RA). Here we present data from the completed Phase 3 randomised controlled trial (RCT) ORAL Scan ... Show moreObjectiveTofacitinib is an oral Janus kinase inhibitor for the treatment of rheumatoid arthritis (RA). Here we present data from the completed Phase 3 randomised controlled trial (RCT) ORAL Scan (NCT00847613), which evaluated the impact of tofacitinib on patient-reported outcomes (PROs) through 24 months in patients with active RA and inadequate responses to methotrexate (MTX-IR).MethodsPatients were randomised 4:4:1:1 to receive tofacitinib 5 or 10 mg twice daily (BID), or placebo advanced to tofacitinib 5 or 10 mg, plus background MTX. Patients receiving placebo advanced to tofacitinib at month 3 (non-responders) or month 6 (remaining patients). Mean changes from baseline in PROs, assessed at months 1-24, included Health Assessment Questionnaire-Disability Index, Patient Global Assessment of disease activity (visual analogue scale [VAS]), Patient Assessment of Arthritis Pain (VAS), health-related quality of life (Short Form-36 version 2), Functional Assessment of Chronic Illness Therapy-Fatigue and Medical Outcomes Study-Sleep.ResultsOverall, 539/797 ( 67.6%) patients completed 24 months' treatment. At month 3, tofacitinib-treated patients reported significant (p<0.05) mean changes from baseline versus placebo across all PROs, and significantly more patients reported improvements = minimum clinically important differences versus placebo. Improvements in PROs with tofacitinib were sustained to month 24. Following advancement to tofacitinib, placebo-treated patients generally reported changes of similar magnitude to tofacitinib-treated patients.ConclusionPatients with RA and MTX-IR receiving tofacitinib 5 or 10 mg BID plus MTX reported significant and clinically meaningful improvements in PROs versus placebo at month 3, which were sustained through 24 months. Show less
Introduction:Patient-reported outcome measures (PROMs) are used increasingly, but for young patients with hip complaints, traditional scoring systems have a ceiling effect that limits their... Show moreIntroduction:Patient-reported outcome measures (PROMs) are used increasingly, but for young patients with hip complaints, traditional scoring systems have a ceiling effect that limits their usability. We developed and validated the Super Simple Hip (SUSHI) score, a PROM specifically targeted at younger patients with hip complaints. Although the SUSHI measured hip problems adequately, the responsiveness of its activity rating component was considered inadequate. Consequently, we replaced the activity rating component by the University of California, Los Angeles (UCLA) activity scale. The aim of this study was to validate the resulting new 10-item SUSHI-UCLA score.Methods:A prospective multicentre observational cohort study was performed. Patients between 18 and 59 years, who visited the Orthopaedic Department with hip complaints, completed the SUSHI-UCLA and hip osteoarthritis outcome score (HOOS) twice before their 1st appointment, and once 16 months after.Results:142 patients were included (mean age 49 years; SD 8.8). The SUSHI-UCLA score correlated well with the HOOS, both before and after treatment (Spearman's rho = 0.739 and 0.847, respectively, both p < 0.001). The responsiveness of both the SUSHI-UCLA score and the UCLA activity scale was high (standardised response mean = 2.51 and 1.35 respectively). The reproducibility was good (interclass correlation coefficient for agreement = 0.962). The minimal important change was 21.2. No significant floor or ceiling effect was observed.Conclusion:The SUSHI-UCLA score is an adequate PROM to measure hip complaints in younger patients and includes a validated activity rating. Show less
Background: Cultural differences between continents may also affect the outcome on interventions. This study compared an Asian and North American cohort of total knee replacement (TKR) patients.... Show moreBackground: Cultural differences between continents may also affect the outcome on interventions. This study compared an Asian and North American cohort of total knee replacement (TKR) patients. Questions/purposes: This study aims to compare the patient-reported outcome measures as well as a functional outcome after TKR between these two different patient populations with a different cultural societal background in two different countries. Patients and Methods: A retrospective study on two cohorts of 76 Asian TKR patients and 64 North American TKR patients were compared. Demographics, patient-reported outcome measures (Knee Society Score (KSS), Patient-Administered Questionnaire (PAQ), and Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC)), knee range of motion (RoM), and radiographic component position were compared. Results: The Asian cohort had more females compared to the North American and significantly worse preoperative RoM, and worse KSS function score and PAQ pain scores. The preoperative KSS knee score and WOMAC scores were comparable between the two groups. Postoperatively, the differences in WOMAC and KSS knee scores were significant, while KSS function and PAQ were comparable between groups. Conclusions: Even though Asian TKR patients had significantly worse preoperative scores, their postoperative outcomes were comparable to North Americans. The higher preoperative functional deficit and the higher pain levels in the Asian population might be due to cultural differences and/or socioeconomic reasons, which made Asian patients present with more severe conditions in the preoperative consultation for a possible surgical treatment compared to North Americans. More research is needed to investigate the difference between these cultural impacts on TKR outcomes. Show less
