Introduction/Aims: As life expectancy improves for patients with Duchenne muscular dystrophy (DMD), new symptoms are likely to arise. This aims of this study are: (1) to explore the prevalence of a... Show moreIntroduction/Aims: As life expectancy improves for patients with Duchenne muscular dystrophy (DMD), new symptoms are likely to arise. This aims of this study are: (1) to explore the prevalence of a broad variety of symptoms in the various stages of DMD (with and without steroid use); (2) to explore the prevalence of common secondary diagnoses; and (3) to evaluate the social participation level of patients with DMD older than 16 y of age; and to explore correlations between social participation and symptoms.Methods A cross-sectional self-report questionnaire, including questions on functional level and health status, as well as a standardized participation scale was distributed among Dutch patients with DMD.Results Eighty-four male patients with a mean age of 22.0 (SD = 10.0) y were enrolled. The most prevalent and limiting symptoms were difficulty coughing (58%), coldness of hands (57%), contractures (51%), stiffness (49%), fatigue (40%), myalgia (38%), and low speech volume (33%). Prevalent secondary diagnoses included cardiac disease (14%), neurobehavioral diagnosis (13%), low blood pressure (13%), and arthrosis (5%). Social participation correlated negatively with coldness of hands (r = - .29; P < .03), decreased intelligibility (r = - .40; P < .003), and chewing problems (r = - .33; P < .02).Discussion The prevalence of a broad spectrum of symptoms and secondary diagnoses is high in patients with DMD, and some of these symptoms are correlated with social participation. Growing awareness of new symptoms and secondary diagnoses among patients, caregivers, and professionals can enhance their recognition, possibly facilitating prevention and early treatment. Show less
Verberne, W.R.; Wittenboer, I.D. van den; Voorend, C.G.N.; Abrahams, A.C.; Buren, M. van; Dekker, F.W.; ... ; Bos, W.J.W. 2021
Background. Non-dialytic conservative care (CC) has been proposed as a viable alternative to maintenance dialysis for selected older patients to treat end-stage kidney disease (ESKD). This... Show moreBackground. Non-dialytic conservative care (CC) has been proposed as a viable alternative to maintenance dialysis for selected older patients to treat end-stage kidney disease (ESKD). This systematic review compares both treatment pathways on health-related quality of life (HRQoL) and symptoms, which are major outcomes for patients and clinicians when deciding on preferred treatment.Methods. We searched PubMed, Embase, Cochrane Library, Cumulative Index to Nursing and Allied Health Literature (CINAHL) Plus and PsycINFO from inception to 1 October 2019 for studies comparing patient-reported HRQoL outcomes or symptoms between patients who chose either CC or dialysis for ESKD.Results. Eleven observational cohort studies were identified comprising 1718 patients overall. There were no randomized controlled trials. Studies were susceptible to selection bias and confounding. In most studies, patients who chose CC were older and had more comorbidities and worse functional status than patients who chose dialysis. Results were broadly consistent across studies, despite considerable clinical and methodological heterogeneity. Patient-reported physical health outcomes and symptoms appeared to be worse in patients who chose CC compared with patients who chose dialysis but had not yet started, but similar compared with patients on dialysis. Mental health outcomes were similar between patients who chose CC or dialysis, including before and after dialysis start. In patients who chose dialysis, the burden of kidney disease and impact on daily life increased after dialysis start.Conclusions. The available data, while heterogeneous, suggest that in selected older patients, CC has the potential to achieve similar HRQoL and symptoms compared with a dialysis pathway. High-quality prospective studies are needed to confirm these provisional findings. Show less
Background. The Response Assessment in Neuro-Oncology Patient-Reported Outcome (RANO-PRO) working group aims to provide guidance on the use of PROs in brain tumor patients. PRO measures should be... Show moreBackground. The Response Assessment in Neuro-Oncology Patient-Reported Outcome (RANO-PRO) working group aims to provide guidance on the use of PROs in brain tumor patients. PRO measures should be of high quality, both in terms of relevance and other measurement properties. This systematic review aimed to identify PRO measures that have been used in brain tumor studies to date.Methods. A systematic literature search for articles published up to June 25, 2020 was conducted in several electronic databases. Pre-specified inclusion criteria were used to identify studies using PRO measures assessing symptoms, (instrumental) activities of daily living [(I)ADL] or health-related quality of life (HRQoL) in adult patients with glioma, meningioma, primary central nervous system lymphoma, or brain metastasis.Results. A total of 215 different PRO measures were identified in 571 published and 194 unpublished studies. The identified PRO measures include brain tumor-specific, cancer-specific, and generic instruments, as well as instruments designed for other indications or multi- or single-item study-specific questionnaires. The most frequently used instruments were the EORT QLQ-C30 and QLQ-BN20 (n = 286 and n = 247),and the FACT-Br (n = 167), however, the majority of the instruments were used only once or twice (150/215).Conclusion. Many different PRO measures assessing symptoms, (I)ADL or HROoL have been used in brain tumor studies to date. Future research should clarify whether these instruments or their wales/items exhibit good content validity and other measurement properties for use in brain tumor patients. Show less
Background. Initiation of renal replacement therapy often results from a combination of kidney function deterioration and symptoms related to chronic kidney disease (CKD) progression. We... Show moreBackground. Initiation of renal replacement therapy often results from a combination of kidney function deterioration and symptoms related to chronic kidney disease (CKD) progression. We investigated the association between kidney function decline and symptom development in patients with advanced CKD.Methods. In the European Quality study on treatment in advanced CKD (EQUAL study), a European prospective cohort study, patients with advanced CKD aged >= 65 years and a kidney function that dropped <20 mL/min/1.73 m(2) were followed for 1 year. Linear mixed-effects models were used to assess the association between kidney function decline and symptom development. The sum score for symptom number ranged from 0 to 33 and for overall symptom severity from 0 to 165, using the Dialysis Symptom Index.Results. At least one kidney function estimate with symptom number or overall symptom severity was available for 1109 and 1019 patients, respectively. The mean (95% confidence interval) annual kidney function decline was 1.70 (1.32; 2.08) mL/min/1.73 m(2). The mean overall increase in symptom number and severity was 0.73 (0.28; 1.19) and 2.93 (1.34; 4.52) per year, respectively. A cross-sectional association between the level of kidney function and symptoms was lacking. Furthermore, kidney function at cohort entry was not associated with symptom development. However, each mL/min/1.73 m(2) of annual kidney function decline was associated with an extra annual increase of 0.23 (0.07; 0.39) in the number of symptoms and 0.87 (0.35; 1.40) in overall symptom severity.Conclusions. A faster kidney function decline was associated with a steeper increase in both symptom number and severity. Considering the modest association, our results seem to suggest that repeated thorough assessment of symptom development during outpatient clinic visits, in addition to the monitoring of kidney function decline, is important for clinical decision-making. Show less
Background: Whereas biochemical response is often used as a primary study outcome, improvement in symptoms and health-related quality of life (HRQoL) is the relevant goal for patients to consider... Show moreBackground: Whereas biochemical response is often used as a primary study outcome, improvement in symptoms and health-related quality of life (HRQoL) is the relevant goal for patients to consider treatment successful. We performed a systematic review and meta-analysis to assess the effect of treatment on symptoms and HRQoL in acromegaly.Methods: Seven electronic databases were searched for longitudinal studies assessing patient-reported symptoms or HRQoL in acromegaly. Meta-analyses were performed to assess differences during treatment for the Acromegaly Quality of Life Questionnaire (AcroQoL) and Patient-Assessed Acromegaly Symptom Questionnaire (PASQ), and standardized mean difference (SMD) for individual symptoms (interpretation: 0.2 small, 0.5 moderate, and 0.8 large effect). Treatment-naive and previously treated patients were assessed separately.Results: Forty-six studies with 3301 patients were included; 24 contributed to quantitative analyses. Thirty-six studies used medication as main treatment, 1 transsphenoidal adenomectomy, and 9 various treatments. Symptoms and HRQoL both improved: AcroQoL increased 2.9 points (95% CI, 0.5 to 5.3 points), PASQ decreased -2.3 points (95% CI, -1.3 to -3.3 points), and individual symptom scores decreased for paresthesia -0.9 (95% CI, -0.6 to -1.2), hyperhidrosis -0.4 (95% CI, -0.1 to -0.6), fatigue -0.3 (95% CI, -0.1 to -0.6), arthralgia -0.3 (95% CI, -0.1 to -0.5), headache -0.3 (95% CI, 0.0 to -0.6), and soft-tissue swelling -0.2 (95% CI, 0.0 to -0.4).Conclusion: Symptoms and HRQoL improved during acromegaly treatment. Consensus is needed on which symptoms should be included in a potential core outcome set, taking into account symptom frequency, severity, and sensitivity to change, which can be used in clinical practice and as outcome in trials. Show less
Introduction: This prospective cohort study determined which questions in patient history are most likely to identify symptoms that are independently associated with a diagnosis of benign... Show moreIntroduction: This prospective cohort study determined which questions in patient history are most likely to identify symptoms that are independently associated with a diagnosis of benign paroxysmal positional dizziness (BPPV) in patients presenting with dizziness, and to evaluate whether the patient's age and type of BPPV are of influence.Methods: We included adult patients with dizziness referred to our dizziness center, Apeldoorn, the Netherlands, from December 2018 to November 2019. All patients completed a questionnaire, underwent vestibular testing and received a diagnosis. Symptoms strongly suggesting BPPV were tested with multivariable analysis to determine their independent associations with BPPV. Subgroup analysis was performed for patient age, and the type of BPPV.Results: We included a total of 885 patients, 113 of whom (13%) were diagnosed with BPPV. The duration of dizziness spells <1 min (Q2) and dizziness provoked by rolling over in bed (Q4) were independently associated with the diagnosis BPPV. Q2 showed a sensitivity of 43%, and a specificity of 75%; Q4 scored 81% and 68%, respectively. Overall, the way patients perceived their dizziness (vertigo, light-headedness or instability) was not independently associated with the diagnosis BPPV. In younger patients, light-headedness and instability decreased the likelihood of BPPV compared to vertigo.Conclusion: The most reliable predictors for BPPV in patient history are a short duration of the dizziness spell and provocation of dizziness by rolling over in bed. Unlike younger patients, elderly patients with BPPV do not only perceive the dizziness as vertigo, but also as a feeling of instability. Show less
Voskamp, P.W.M.; Diepen, M. van; Evans, M.; Caskey, F.J.; Torino, C.; Postorino, M.; ... ; EQUAL Study Investigators 2019
Background. Quality of life (QoL) is an important outcome in chronic kidney disease (CKD). Patients feel that symptoms are an important determinant of QoL. However, this relation is unknown. The... Show moreBackground. Quality of life (QoL) is an important outcome in chronic kidney disease (CKD). Patients feel that symptoms are an important determinant of QoL. However, this relation is unknown. The aims of this study were to investigate the impact of the number and severity of symptoms on QoL in elderly pre-dialysis patients, assessed by both the effect of symptoms and their importance relative to kidney function, and other clinical variables on QoL.Methods. The European Quality study (EQUAL study) is an ongoing European prospective follow-up study in late Stage 4/5 CKD patients aged >= 65 years. We used patients included between March 2012 and December 2015. Patients scored their symptoms with the Dialysis Symptom Index, and QoL with the research and development-36 (RAND-36) item Health Survey (RAND-36). The RAND-36 results in a physical component summary (PCS) and a mental component summary (MCS). We used linear regression to estimate the relation between symptoms and QoL at baseline and after 6 months, and to calculate the variance in QoL explained by symptoms.Results. The baseline questionnaire was filled in by 1079 (73%) patients (median age 75 years, 66% male, 98% Caucasian), and the follow up questionnaire by 627 (42%) patients. At baseline, every additional symptom changed MCS with -0.81 [95% confidence interval (CI): -0.91 to -0.71] and PCS with -0.50 (95% CI: -0.62 to -0.39). In univariable analyses, number of symptoms explained 22% of MCS variance and 11% of PCS variance, whereas estimated glomerular filtration rate only explained 1%.Conclusions. In elderly CKD Stage 4/5 patients, symptoms have a substantial impact on QoL. This indicates symptoms should have a more prominent role in clinical decision-making. Show less
Background: Informing patients with cancer about the possible implications of prospective treatment is a crucial yet challenging task. Unfortunately, patients' recall of medical information is... Show moreBackground: Informing patients with cancer about the possible implications of prospective treatment is a crucial yet challenging task. Unfortunately, patients' recall of medical information is generally poor and their information needs are not met. Effective information giving entails that oncologists help patients understand and recall the implications of their treatment, meanwhile fostering a trusting physician-patient relationship. Communication strategies that are often suggested to be effective are structuring and tailoring (cognition-oriented) but also are oncologists' expressions of caring or empathy (affect-oriented).Objective: The aim of this study is to provide evidence concerning the pathways linking physician communication to (improved) consultation outcomes for patients. More specifically, the aim is to determine the effects of information structuring and information tailoring, combined with physician caring, on information recall, satisfaction with information, and trust in the physician (primary objective) and on symptom distress (secondary objective).Methods: A randomized controlled trial, systematically testing the effects of information structuring and information tailoring, each combined with caring, in 2 video-vignette experiments (2 x2 and 2 x2 x2 design). Using an online survey platform, participants will be randomly allocated (blinded) to 1 of 12 conditions in which they are asked to view a video vignette (intervention) in which an oncologist discusses a treatment plan for malignant lymphoma with a patient. The independent variables of interest are systematically varied across conditions. The outcome measures are assessed in a survey, using validated instruments. Study participants are (former) patients with cancer and their relatives recruited via online panels and patient organizations. This protocol discusses the trial design, including the video-vignette design, intervention pretesting, and a pilot study.Results: Data collection has now been completed, and preliminary analyses will be available in Spring 2019. A total of 470 participants completed the first part of the survey and were randomized to receive the intervention.Conclusions: The results of the proposed trial will provide evidence concerning the pathways linking physician information, giving skills to (improved) consultation outcomes for patients. Show less
