Muscle biopsies are used in clinical trials to measure target engagement of the investigational product. With many upcoming therapies for patients with facioscapulohumeral dystrophy (FSHD), the... Show moreMuscle biopsies are used in clinical trials to measure target engagement of the investigational product. With many upcoming therapies for patients with facioscapulohumeral dystrophy (FSHD), the frequency of biopsies in FSHD patients is expected to increase. Muscle biopsies were performed either in the outpatient clinic using a Bergström needle (BN-biopsy) or in a Magnetic Resonance Imaging machine (MRI-biopsy). This study assessed the FSHD patients’ experience of biopsies using a customized questionnaire. The questionnaire was sent to all FSHD patients who had undergone a needle muscle biopsy for research purposes, inquiring about biopsy characteristics and burden, and willingness to undergo a subsequent biopsy. Forty-nine of 56 invited patients (88%) completed the questionnaire, reporting on 91 biopsies. The median pain score (scale 0-10) during the procedure was 5 [2-8], reducing to 3 [1-5] and 2 [1-3] after one and 24 h, respectively. Twelve biopsies (13.2%) resulted in complications, eleven resolved within 30 days. BN-biopsies were less painful compared to MRI-biopsies (median NRS: 4 [2-6] vs. 7 [3-9], p = 0.001). The burden of needle muscle biopsies in a research setting is considerate and should not be underestimated. MRI-biopsies have a higher burden compared to BN-biopsies. Show less
Background: Rheumatic and Musculoskeletal Diseases (RMDs) substantially impact the lives of patients, with complex associations between disease severity and self-perceived health status. In this... Show moreBackground: Rheumatic and Musculoskeletal Diseases (RMDs) substantially impact the lives of patients, with complex associations between disease severity and self-perceived health status. In this regard, the Coping with Rheumatic Stressors (CORS) questionnaire was developed to measure how patients with RMDs cope with stressors such as pain, limitations or dependency. The CORS is not currently available in Spanish, and therefore the adaptation of this instrument is needed.Objective: First, to cross-culturally adapt the CORS into Spanish for Spain. Secondly, to test the conceptual equivalence of the translated version in patients with axial spondyloarthritis (axSpA). Methods: A translation of the CORS into Spanish was performed adhering to the forward-backward procedure described by Beaton. Two translators produced independent forward translations of the item content, response options, and instructions of the CORS into Spanish. Both versions were harmonized in a consensual version. Another translator back-translated the synthesized version into Dutch. A scientific committee including all the translators, one methodologist and a rheumatologist, held a meeting and reached consensus on discrepancies to develop a final draft version of the Spanish CORS. Then, a field test with cognitive debriefing was conducted, involving a sample of 10 patients with axSpA. Results: The translation process of the CORS was completed after the discussion of some discrepancies throughout the process. The first translation was done without major complications. Back-translation presented some discrepancies. These led to minor modifications in the wording in one response option and 15 questionnaire items. The scientific committee agreed upon a final version of the questionnaire. Cognitive debriefing, led to minor modifications; for example, three respondents indicated that one of the statements in the instructions was syntactically complex ("indique cuan a menudo usted ha llevado a cabo dicho comportamiento") which led to its adjustment. The process indicated that the final CORS Spanish questionnaire was clear and understandable to all patients.Conclusions: The Spanish version of the CORS showed good cross-cultural validity and good face validity according to the field test. Before the Spanish CORS is implemented, further validation is in progress to test the psychometric properties of the instrument in patients with axSpA. Show less
BackgroundRheumatic and Musculoskeletal Diseases (RMDs) substantially impact the lives of patients, with complex associations between disease severity and self-perceived health status. In this... Show moreBackgroundRheumatic and Musculoskeletal Diseases (RMDs) substantially impact the lives of patients, with complex associations between disease severity and self-perceived health status. In this regard, the Coping with Rheumatic Stressors (CORS) questionnaire was developed to measure how patients with RMDs cope with stressors such as pain, limitations or dependency. The CORS is not currently available in Spanish, and therefore the adaptation of this instrument is needed.ObjectiveFirst, to cross-culturally adapt the CORS into Spanish for Spain. Secondly, to test the conceptual equivalence of the translated version in patients with axial spondyloarthritis (axSpA).MethodsA translation of the CORS into Spanish was performed adhering to the forward-backward procedure described by Beaton. Two translators produced independent forward translations of the item content, response options, and instructions of the CORS into Spanish. Both versions were harmonized in a consensual version. Another translator back-translated the synthesized version into Dutch. A scientific committee including all the translators, one methodologist and a rheumatologist, held a meeting and reached consensus on discrepancies to develop a final draft version of the Spanish CORS. Then, a field test with cognitive debriefing was conducted, involving a sample of 10 patients with axSpA.ResultsThe translation process of the CORS was completed after the discussion of some discrepancies throughout the process. The first translation was done without major complications. Back-translation presented some discrepancies. These led to minor modifications in the wording in one response option and 15 questionnaire items. The scientific committee agreed upon a final version of the questionnaire. Cognitive debriefing, led to minor modifications; for example, three respondents indicated that one of the statements in the instructions was syntactically complex (“indique cuán a menudo usted ha llevado a cabo dicho comportamiento”) which led to its adjustment. The process indicated that the final CORS Spanish questionnaire was clear and understandable to all patients.ConclusionsThe Spanish version of the CORS showed good cross-cultural validity and good face validity according to the field test. Before the Spanish CORS is implemented, further validation is in progress to test the psychometric properties of the instrument in patients with axSpA. Show less
Glioma, a malignant brain tumor, is a serious condition with a large impact on patients’ life expectancy and Health-Related Quality of Life (HRQoL). This thesis aimed to improve knowledge on the... Show moreGlioma, a malignant brain tumor, is a serious condition with a large impact on patients’ life expectancy and Health-Related Quality of Life (HRQoL). This thesis aimed to improve knowledge on the prediagnostic symptoms, tmeasurement of HRQoL and care in the end-of-life phase of glioma patients.Regarding prediagnostic symptoms in glioma patients, it was found that several symptoms such as fatigue or headache were relatively common before diagnosis, but not more common than in patients with other conditions. Therefore, identification of patients with glioma in general practice based on their symptomatology seems extremely difficult.For the measurement of HRQoL in glioma it was demonstrated that patients, proxies and healthcare neuro-oncology professionals were overall positive about their routine implementation of its assessment in clinical practice. Regarding the timing of the measurements, the scores were not influenced by assessment either before or after the consultation with the physician.With respect to the optimization of care in the end-of-life phase, a feasibility study on the disease-specific Advanced Care Program was conducted among patients with glioblastoma, the most malignant type of glioma and their nearest. The relatively positive results of that study in terms of satisfaction warrant the need for a larger, controlled study. Show less
In this thesis, we aimed to develop and validate a patient and a physician questionnaire to measure the shared decision making (SDM) process in oncology. We identified SDM measurement challenges:... Show moreIn this thesis, we aimed to develop and validate a patient and a physician questionnaire to measure the shared decision making (SDM) process in oncology. We identified SDM measurement challenges: there is limited evidence on measurement properties, patients’ role is not assessed while it is present in SDM models, patients and healthcare professionals have been involved only to a limited extent in the development of SDM measurement instruments, and a reflective measurement model is often assumed, while a formative might be more appropriate. The aim of this thesis was therefore to develop and validate questionnaires to assess the SDM process in oncology from both the patient and the physician viewpoints. We chose the oncology setting because it is a setting in which patients often face preference-sensitive decisions, and in which most patients prefer an active or collaborative role in treatment decision making. To inform the development and validation process of our questionnaires, we used the original Consensus-based Standards for the selection of health Measurement INstruments (COSMIN) checklist and we wrote two reviews: one on existing SDM measurement instruments and one on existing SDM models. The iSHARE questionnaires shows adequate measurement properties Show less
Objectives: We have developed two Dutch questionnaires to assess the shared decision-making (SDM) process in oncology; the iSHAREpatient and iSHAREphysician. In this study, we aimed to determine:... Show moreObjectives: We have developed two Dutch questionnaires to assess the shared decision-making (SDM) process in oncology; the iSHAREpatient and iSHAREphysician. In this study, we aimed to determine: scores, construct validity, test-retest agreement (iSHAREpatient), and inter-rater (iSHAREpatient-iSHAREphysician) agreement. Methods: Physicians from seven Dutch hospitals recruited cancer patients, and completed the iSHAREphysician and SDM-Questionnaire-physician version. Their patients completed the: iSHAREpatient, nine-item SDM-Questionnaire, Decisional Conflict Scale, Combined Outcome Measure for Risk communication And treatment Decision-making Effectiveness, and five-item Perceived Efficacy in Patient-Physician Interactions. We formulated, respectively, one (iSHAREphysician) and 10 (iSHAREpatient) a priori hypotheses regarding correlations between the iSHARE questionnaires and questionnaires assessing related constructs. To assess test-retest agreement patients completed the iSHAREpatient again 1-2 weeks later. Results: In total, 151 treatment decision-making processes with unique patients were rated. Dimension and total iSHARE scores were high both in patients and physicians. The hypothesis on the iSHAREphysician and 9/10 hypotheses on the iSHAREpatient were confirmed. Test-retest and inter-rater agreement were >.60 for most items. Conclusions: The iSHARE questionnaires show high scores, have good construct validity, substantial test-retest agreement, and moderate inter-rater agreement. Practice implications: Results from the iSHARE questionnaires can inform both physician- and patient-directed efforts to improve SDM in clinical practice. Show less
The management of dysphagia may differ by country and clinical setting. The purpose of this study was to describe the management and care pathways for elderly people with dysphagia in nursing homes... Show moreThe management of dysphagia may differ by country and clinical setting. The purpose of this study was to describe the management and care pathways for elderly people with dysphagia in nursing homes across Norway using an online survey. A national survey was developed that consisted of 23 questions covering various areas related to dysphagia care in nursing homes: background information of respondents, nursing homes, and residents and staff; screening and assessment of dysphagia including use of specialist consultation; management, practice patterns, and interventions targeting residents with dysphagia; training of staff; and perceived quality of current clinical practices in their nursing home. A total of 121 respondents completed the online survey, resulting in an overall response rate of 23.2%. Substantial discrepancies in dysphagia management were identified between nursing homes. In approximately 75% of nursing homes, residents were not routinely screened or assessed for swallowing problems. Although nursing homes used a broad range of strategies and routines for people with eating and swallowing difficulties, bolus modification seemed standard practice. Oral hygiene strategies were lacking in over 80% of nursing homes, and almost 50% did not have access to external experts, including speech therapists. Although nursing home staff rated the overall quality of care for people with eating and swallowing problems as high, their rating seemed mainly based on care for malnutrition and not directly aimed at dysphagia. The survey identified an evident need for training and upskilling staff in Norwegian nursing homes and raising awareness of the serious consequences and comorbidities that can result from dysphagia. Show less
Rationale The impact of prophylactic implantable cardioverter-defibrillator (ICD) implantation on the psychological well-being of patients on dialysis is unknown.Objective We aimed to identify the... Show moreRationale The impact of prophylactic implantable cardioverter-defibrillator (ICD) implantation on the psychological well-being of patients on dialysis is unknown.Objective We aimed to identify the effect of primary ICD implantation on quality of life (QoL), mood and dispositional optimism in patients undergoing dialysis.Methods and results We performed a prespecified subanalysis of the randomized controlled ICD2 trial. In total, 177 patients on chronic dialysis, with an age of 55-81 years, and a left ventricular ejection fraction of >= 35%, were included in the per-protocol analysis. Eighty patients received an ICD for primary prevention, and 91 patients received standard care. The Short Form-36 (SF-36), Geriatric Depression Scale-15 (GDS-15), Revised Life Orientation Test (LOT-R) questionnaires were administered prior to ICD implantation (T0), and at 1-year follow-up (T1) to assess QoL, depression and optimism, respectively. The patients were predominantly male (76.0%), with a median age of 67 years. Hemodialysis was the predominant mode of dialysis (70.2%). The GDS-15 score difference (T1 - T0) was 0.5 (2.1) in the ICD group compared with 0.3 (2.2) in the control group (mean difference - 0.3; 95% CI - 1.1 to 0.6; P = 0.58). The LOT-R score difference was - 0.2 (4.1) in the ICD group compared with - 1.5 (4.0) in the control group (mean difference - 1.1 (0.8); 95% CI - 2.6 to 0.4; P = 0.17). The mean difference scores of all subscales of the SF-36 were not significantly different between randomization groups.Conclusions In our population of patients on dialysis, ICD implantation did not affect QoL, mood or dispositional optimism significantly during 1-year follow-up. Show less
Purpose Being able to function independently in society is an important aspect of quality of life. This ability goes beyond self-care, requires higher order cognitive functioning, and is typically... Show morePurpose Being able to function independently in society is an important aspect of quality of life. This ability goes beyond self-care, requires higher order cognitive functioning, and is typically measured with instrumental activities of daily living (IADL) questionnaires. Cognitive deficits are frequently observed in brain tumour patients, however, IADL is almost never assessed because no valid and reliable IADL measure is available for this patient group. Therefore, this measure is currently being developed. Methods This international multicentre study followed European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Group module development guidelines. Three out of four phases are completed: phases (I) generation of items, (II) construction of the item list, and (III) pre-testing. This paper reports the item selection procedures and preliminary psychometric properties of the questionnaire. Brain tumour patients (gliomas and brain metastases), their informal caregivers, and health care professionals (HCPs) were included. Results Phase I (n = 44 patient-proxy dyads and 26 HCPs) generated 59 relevant and important activities. In phase II, the activities were converted into items. In phase III (n = 85 dyads), the 59 items were pre-tested. Item selection procedures resulted in 32 items. Exploratory factor analysis revealed a preliminary dimensional structure consisting of five scales with acceptable to excellent internal consistency (alpha = 0.73-0.94) and two single items. For three scales, patients with cognitive impairments had significantly more IADL problems than patients without impairments. Conclusion A phase IV validation study is needed to confirm the psychometric properties of the EORTC IADL-BN32 questionnaire in a larger international sample. Show less
Background Little is known about the symptoms glioma patients experience in the year before diagnosis, either or not resulting in health care usage. This study aimed to determine the incidence of... Show moreBackground Little is known about the symptoms glioma patients experience in the year before diagnosis, either or not resulting in health care usage. This study aimed to determine the incidence of symptoms glioma patients experienced in the year prior to diagnosis, and subsequent visits to a general practitioner (GP).Methods Glioma patients were asked to complete a 30-item study-specific questionnaire focusing on symptoms they experienced in the 12 months before diagnosis. For each indicated symptom, patients were asked whether they consulted the GP for this issue.Results Fifty-nine patients completed the questionnaires, 54 (93%) with input of a proxy. The median time since diagnosis was 4 months (range 1-12). The median number of symptoms experienced in the year before diagnosis was similar between gliomas with favourable and poor prognosis, i.e. 6 (range 0-24), as were the five most frequently mentioned problems: fatigue (n = 34, 58%), mental tiredness (n = 30, 51%), sleeping disorder (n = 24, 41%), headache (n = 23, 39%) and stress (n = 20, 34%). Twenty-six (44%) patients visited the GP with at least one issue. Patients who did consult their GP reported significantly more often muscle weakness (11 vs 3, p = 0.003) than patients who did not, which remained significant after correction for multiple testing, which was not the case for paralysis in hand/leg (10 vs 4), focussing (11 vs 6) or a change in awareness (9 vs 4).Conclusions Glioma patients experience a range of non-specific problems in the year prior to diagnosis, but only patients who consult the GP report more often neurological problems. Show less
Exposure to traumatic stress increases the odds of developing a broad range of psychiatric conditions. Genetic studies targeting multiple stress-related quantitative phenotypes may shed light on... Show moreExposure to traumatic stress increases the odds of developing a broad range of psychiatric conditions. Genetic studies targeting multiple stress-related quantitative phenotypes may shed light on mechanisms underlying vulnerability to psychopathology in the aftermath of stressful events. We applied a multivariate genome-wide association study (GWAS) to a unique military cohort (N = 583) in which we measured biochemical and behavioral phenotypes. The availability of pre- and post-deployment measurements allowed to capture changes in these phenotypes in response to stress. For genome-wide significant loci, we performed functional annotation, phenome-wide analysis and quasi-replication in PTSD case-control GWASs. We discovered one genetic variant reaching genome-wide significant association, surviving permutation and sensitivity analyses (rs10100651, p = 9.9 x 10(-9)). Functional annotation prioritized the genes INTS8 and TP53INP1. A phenome-wide scan revealed a significant association of these same genes with sleeping problems, hypertension and subjective well-being. Finally, a targeted lookup revealed nominally significant association of rs10100651 in a PTSD case-control GWAS in the UK Biobank (p = 0.02). We provide comprehensive evidence from multiple resources hinting at a role of the highlighted genetic variant in the human stress response, marking the power of multivariate genome-wide analysis of quantitative measures in stress research. Future genetic and functional studies can target this locus to further assess its effects on stress mediation and its possible role in psychopathology or resilience. (C) 2019 Elsevier B.V. and ECNP. All rights reserved. Show less
Background Patient-reported outcome measures (PROMs) are becoming increasingly important in healthcare. In nephrology, there is no agreement on which chronic kidney disease (CKD) symptom... Show moreBackground Patient-reported outcome measures (PROMs) are becoming increasingly important in healthcare. In nephrology, there is no agreement on which chronic kidney disease (CKD) symptom questionnaire to use. Therefore, the aim of this study is to select a valid symptom questionnaire for routine assessment in patients with advanced CKD. Methods A four-phase mixed methods approach, using qualitative and quantitative research methods, was applied. First, a systematic literature search was conducted to retrieve existing symptom questionnaires. Second, a symptom list was created including all symptoms in existing questionnaires and symptoms mentioned in interviews with patients with CKD, from which symptom clusters were identified. Next, questionnaires were selected based on predefined criteria regarding content validity. Last, two online feedback panels of patients with CKD (n = 151) and experts (n = 6) reviewed the most promising questionnaires. Results The literature search identified 121 questionnaires, of which 28 were potentially suitable for symptom assessment in patients with advanced CKD. 101 unique symptoms and 10 symptom clusters were distinguished. Based on predefined criteria, the Dialysis Symptom Index (DSI) and Palliative Care Outcome Scale-Renal Version (IPOS-Renal) were selected and reviewed by feedback panels. Patients needed 5.4 and 7.5 min to complete the DSI and IPOS-Renal, respectively (p < 0.001). Patients experienced the DSI as more specific, complete and straightforward compared to the IPOS-Renal. Conclusions The DSI was found to be valid and reliable, the most relevant, complete, and comprehensible symptom questionnaire available for routine assessment in patients with advanced CKD. Routine PROMs collection could be of great value to healthcare, both at individual patient and national level. Feedback on scores and involvement of healthcare providers may promote adaptation and implementation in healthcare. Show less
Hendriksma, M.; Loon, Y. van; Klop, W.M.C.; Hakkesteegt, M.M.; Heijnen, B.J.; Hasnaoui, I. el; ... ; Sjogren, E.V. 2019
As early diagnosis of swallowing and feeding difficulties in infants and children is of utmost importance, there is a need to evaluate the quality of the psychometric properties of pediatric... Show moreAs early diagnosis of swallowing and feeding difficulties in infants and children is of utmost importance, there is a need to evaluate the quality of the psychometric properties of pediatric assessments of swallowing and feeding. A systematic review was performed summarizing the psychometric properties of non-instrumental assessments for swallowing and feeding difficulties in pediatrics; no data were identified for the remaining twelve assessments. The COSMIN taxonomy and checklist were used to evaluate the methodological quality of 23 publications on psychometric properties. For each assessment, an overall quality score for each measurement property was determined. As psychometric data proved incomplete, conflicting or indeterminate for all assessments, only preliminary conclusions could be drawn; the most robust assessment based on current data is the dysphagia disorder survey (DDS). However, further research is needed to provide additional information on all psychometric properties for all assessments. Show less
Willik, D. van der; Pelzer, N.; Algra, A.; Terwindt, G.M.; Wermer, M.J.H. 2017
Post-stroke navigation complaints are frequent (about 30%) and intervention is possible, but there is no assessment instrument to identify patients with navigation complaints. We therefore studied... Show morePost-stroke navigation complaints are frequent (about 30%) and intervention is possible, but there is no assessment instrument to identify patients with navigation complaints. We therefore studied the clinical validity of the Wayfinding Questionnaire (WQ) in a cross-sectional study with 158 chronic stroke patients and 131 healthy controls. Patients with low (more navigation complaints) versus normal WQ scores were compared for demographics, stroke characteristics, emotional and cognitive complaints, and health-related quality of life (HRQoL). Actual navigation performance of 78 patients was assessed in a virtual reality setting. Effect sizes (d) were calculated. WQ responses (22 items) of stroke patients were compared with those of controls (discriminant validity). Results showed that patients with a low WQ score (n = 49, 32%) were more often women (p = 0.013) and less educated (p = 0.004), reported more cognitive complaints (d = 0.69), more emotional problems (d = 0.38 and 0.52), and lower HRQoL (d = 0.40 and 0.45) and, last but not least, performed worse on the navigation ability tasks (d = 0.23–0.80). Patients scored lower than controls on 21/22 WQ items, predominantly with small to medium effect sizes (d = 0.20–0.51). We conclude that the WQ is valid as a measure of navigation complaints in stroke patients, and thus strongly advocate its use in stroke care. Show less
