Search tips
Search criteria

Results 1-13 (13)

Clipboard (0)

Select a Filter Below

Year of Publication
1.  The need for patient-centred clinical research in idiopathic pulmonary fibrosis 
BMC Medicine  2015;13:240.
Patient-centredness is an accepted term and is perceived by healthcare professionals to be morally and ethically desirable. We are motivated by the belief that this approach will improve the patient-professional experience of the decision-making process and improve health outcomes. We acknowledge that patients, either as participants or as co-investigators, have positive contributions to make to research. As the idiopathic pulmonary fibrosis (IPF) community enters a new era of clinical research activity we consider that there is greater capacity for patient involvement and partnership.
Patient involvement in research can be optimised through collaborations in the research design, study conduct, and dissemination. There is increasing interest in using patient- reported outcomes (PROs), such as health-related quality of life, and symptoms measures to inform decision-making and ensure patient perspectives are taken into account. PROs are an essential component of specialist IPF services, to monitor and improve care delivery and to measure and benchmark performance. In clinical trials, PROs can additionally be used to define entry criteria, evaluate efficacy of an intervention, and evaluate adverse events. We suggest that there is a much wider scope for including patient-centred PROs in clinical research and for creative thought in developing patient co-investigator roles.
Participation in research activity requires highly refined decision-making processes, particularly in a condition such as IPF, which has an often unpredictable trajectory. The IPF research landscape has changed and the design and conduct of clinical trials in IPF requires some radical rethinking. It is accepted that involving patients in the role of co-investigators will impact the research questions we ask and result in study designs that are patient-centred. IPF clinical trials have been hindered by the lack of availability of validated, disease-specific questionnaires. A conservative approach appears to have been taken to the inclusion of generic symptom or quality of life measures as PRO endpoints. Thus, the impact of new drugs on the quality of life of research participants demonstrates only minimal benefit. It is time to refocus on a patient-centred approach with regards to the co-investigator role, PRO development, and research participants.
PMCID: PMC4581492  PMID: 26399318
Clinical research; Decision-making; Idiopathic pulmonary fibrosis; Patient-centredness; Patient-reported outcomes; Patient co-investigator
2.  Predictors and correlates of adherence to combination antiretroviral therapy (ART) for chronic HIV infection: a meta-analysis 
BMC Medicine  2014;12:142.
Adherence to combination antiretroviral therapy (ART) is a key predictor of the success of human immunodeficiency virus (HIV) treatment, and is potentially amenable to intervention. Insight into predictors or correlates of non-adherence to ART may help guide targets for the development of adherence-enhancing interventions. Our objective was to review evidence on predictors/correlates of adherence to ART, and to aggregate findings into quantitative estimates of their impact on adherence.
We searched PubMed for original English-language papers, published between 1996 and June 2014, and the reference lists of all relevant articles found. Studies reporting on predictors/correlates of adherence of adults prescribed ART for chronic HIV infection were included without restriction to adherence assessment method, study design or geographical location. Two researchers independently extracted the data from the same papers. Random effects models with inverse variance weights were used to aggregate findings into pooled effects estimates with 95% confidence intervals. The standardized mean difference (SMD) was used as the common effect size. The impact of study design features (adherence assessment method, study design, and the United Nations Human Development Index (HDI) of the country in which the study was set) was investigated using categorical mixed effects meta-regression.
In total, 207 studies were included. The following predictors/correlates were most strongly associated with adherence: adherence self-efficacy (SMD = 0.603, P = 0.001), current substance use (SMD = −0.395, P = 0.001), concerns about ART (SMD = −0.388, P = 0.001), beliefs about the necessity/utility of ART (SMD = 0.357, P = 0.001), trust/satisfaction with the HIV care provider (SMD = 0.377, P = 0.001), depressive symptoms (SMD = −0.305, P = 0.001), stigma about HIV (SMD = −0.282, P = 0.001), and social support (SMD = 0.237, P = 0.001). Smaller but significant associations were observed for the following being prescribed a protease inhibitor-containing regimen (SMD = −0.196, P = 0.001), daily dosing frequency (SMD = −0.193, P = 0.001), financial constraints (SMD −0.187, P = 0.001) and pill burden (SMD = −0.124, P = 0.001). Higher trust/satisfaction with the HIV care provider, a lower daily dosing frequency, and fewer depressive symptoms were more strongly related with higher adherence in low and medium HDI countries than in high HDI countries.
These findings suggest that adherence-enhancing interventions should particularly target psychological factors such as self-efficacy and concerns/beliefs about the efficacy and safety of ART. Moreover, these findings suggest that simplification of regimens might have smaller but significant effects.
Electronic supplementary material
The online version of this article (doi:10.1186/s12916-014-0142-1) contains supplementary material, which is available to authorized users.
PMCID: PMC4148019  PMID: 25145556
Adherence; Compliance; HIV infection; Antiretroviral therapy; Meta-analysis
3.  Developing core outcomes sets: methods for identifying and including patient-reported outcomes (PROs) 
Trials  2014;15:49.
Synthesis of patient-reported outcome (PRO) data is hindered by the range of available PRO measures (PROMs) composed of multiple scales and single items with differing terminology and content. The use of core outcome sets, an agreed minimum set of outcomes to be measured and reported in all trials of a specific condition, may improve this issue but methods to select core PRO domains from the many available PROMs are lacking. This study examines existing PROMs and describes methods to identify health domains to inform the development of a core outcome set, illustrated with an example.
Systematic literature searches identified validated PROMs from studies evaluating radical treatment for oesophageal cancer. PROM scale/single item names were recorded verbatim and the frequency of similar names/scales documented. PROM contents (scale components/single items) were examined for conceptual meaning by an expert clinician and methodologist and categorised into health domains. A patient advocate independently checked this categorisation.
Searches identified 21 generic and disease-specific PROMs containing 116 scales and 32 single items with 94 different verbatim names. Identical names for scales were repeatedly used (for example, ‘physical function’ in six different measures) and others were similar (overlapping face validity) although component items were not always comparable. Based on methodological, clinical and patient expertise, 606 individual items were categorised into 32 health domains.
This study outlines a methodology for identifying candidate PRO domains from existing PROMs to inform a core outcome set to use in clinical trials.
PMCID: PMC3916696  PMID: 24495582
Core outcome set; Patient reported outcome (PRO); Patient reported outcome measure (PROM); Randomised controlled trial (RCT); Trial methodology; Health domains; Quality of life; Systematic review
4.  Patients with unexplained physical symptoms have poorer quality of life and higher costs than other patient groups: a cross-sectional study on burden 
To determine whether healthcare resources are allocated fairly, it is helpful to have information on the quality of life (QoL) of patients with Unexplained Physical Symptoms (UPS) and on the costs associated with them, and on how these relate to corresponding data in other patient groups. As studies to date have been limited to specific patient populations with UPS, the objective of this study was to assess QoL and costs in a general sample of patients with UPS using generic measures.
In a cross-sectional study, 162 patients with UPS reported on their QoL, use of healthcare resources and lost productivity in paid and unpaid work. To assess QoL, the generic SF-36 questionnaire was used, from which multidimensional quality-of-life scores and a one-dimensional score (utility) using the SF-6D scorings algorithm were derived. To assess costs, the TiC-P questionnaire was used.
Patients with UPS reported a poor QoL. Their QoL was mostly decreased by limitations in functioning due to physical health, and the least by limitations in functioning due to emotional problems. The median of utilities was 0.57, and the mean was 0.58 (SD = .09).
The cost for the use of healthcare services was estimated to be €3,123 (SD = €2,952) per patient per year. This cost was enlarged by work-related costs: absence from work (absenteeism), lower on-the-job productivity (presenteeism), and paid substitution of domestic tasks. The resulting mean total cost was estimated to be €6,815 per patient per year.
These findings suggest that patients with UPS have a high burden of disease and use a considerable amount of healthcare resources. In comparison with other patient groups, the QoL values of patients with UPS were among the poorest and their costs were among the highest of all patient groups. The burden for both patients and society helps to justify the allocation of sufficient resources to effective treatment for patients with UPS.
Trial registration
Nederlands Trial Register, NTR1609
PMCID: PMC3878564  PMID: 24344899
Absenteeism; Burden; Chronic disease; Costs; Healthcare utilization; Presenteeism; Production loss; Quality of life; Somatic symptom disorder; Somatoform disorder; Unexplained physical symptoms
5.  The life in sight application study (LISA): design of a randomized controlled trial to assess the role of an assisted structured reflection on life events and ultimate life goals to improve quality of life of cancer patients 
BMC Cancer  2013;13:360.
It is widely recognized that spiritual care plays an important role in physical and psychosocial well-being of cancer patients, but there is little evidence based research on the effects of spiritual care. We will conduct a randomized controlled trial on spiritual care using a brief structured interview scheme supported by an e-application. The aim is to examine whether an assisted reflection on life events and ultimate life goals can improve quality of life of cancer patients.
Based on the findings of our previous research, we have developed a brief interview model that allows spiritual counsellors to explore, explicate and discuss life events and ultimate life goals with cancer patients. To support the interview, we created an e-application for a PC or tablet. To examine whether this assisted reflection improves quality of life we will conduct a randomized trial. Patients with advanced cancer not amenable to curative treatment options will be randomized to either the intervention or the control group. The intervention group will have two consultations with a spiritual counsellor using the interview scheme supported by the e-application. The control group will receive care as usual. At baseline and one and three months after randomization all patients fill out questionnaires regarding quality of life, spiritual wellbeing, empowerment, satisfaction with life, anxiety and depression and health care consumption.
Having insight into one’s ultimate life goals may help integrating a life event such as cancer into one’s life story. This is the first randomized controlled trial to evaluate the role of an assisted structured reflection on ultimate life goals to improve patients’ quality of life and spiritual well being. The intervention is brief and based on concepts and skills that spiritual counsellors are familiar with, it can be easily implemented in routine patient care and incorporated in guidelines on spiritual care.
Trial registration
The study is registered at NCT01830075
PMCID: PMC3727984  PMID: 23889978
Spiritual care; Quality of life; Meaning; Ultimate life goals; Palliative care; Contingency; Cancer patients; Spiritual wellbeing; Empowerment
6.  The effectiveness of cognitive behavioral therapy on the quality of life of patients with inflammatory bowel disease: multi-center design and study protocol (KL!C- study) 
BMC Psychiatry  2012;12:227.
Inflammatory Bowel Disease (IBD) patients report poorer quality of life (QoL) and more anxiety and depressive symptoms than controls from the general population. Cognitive behavioral therapy (CBT) is effective for anxiety and depression, but questionable in case of co-morbidity with IBD. Therefore, an adapted new CBT specifically designed for IBD patients was developed. The objective of this study is to evaluate the effectiveness of adapted CBT on QoL.
IBD patients with a poor level of mental QoL (score less than or equal to 23 on the mental health scale of SF-36) will be randomly assigned to the experimental (n = 40) or waiting-list control condition (n = 40). The experimental condition will then immediately start CBT. The waiting-list control condition will wait 3,5 months before CBT begins with pre- and post assessments. Both conditions will complete a baseline and follow-up assessment following CBT and a mid-treatment assessment. The primary outcome is IBD-specific QoL (IBDQ). Secondary outcomes are generic QoL (SF-36) and anxiety and depression complaints (HADS, CES-D). Additionally, we will examine the working mechanism of the psychological intervention by investigating the impact of the intervention on illness-related cognitions, attitudes, coping styles and their associations with outcome. Data will be analysed on an intention to treat (ITT) as well as treatment completer basis (greater than or equal to five sessions followed).
If found effective, this IBD-specific CBT is a first step to enhance poor QoL in IBD patients and possibly, other gastroenterological diseases. By enhancing IBD patients’ QoL, we may also improve their mental and physical health, and lower unnecessary health care consumption.
Trial registration number
NTR (TC = 1869)
PMCID: PMC3570397  PMID: 23237076
Inflammatory Bowel Disease; Cognitive behavioral therapy; Quality of life; Anxiety; Depression
7.  The efficacy of a health-related quality-of-life intervention during 48 weeks of biologic treatment of patients with moderate to severe psoriasis: study protocol for a multicenter randomized controlled trial 
Trials  2012;13:236.
Interest in health-related quality of life (HRQoL) outcome research in dermatology is increasing, especially in the systemic treatment of psoriasis with biologic agents. In other specialties, such as oncology, the application of a HRQoL intervention is considered to be an aid for monitoring disease and treatment over time, for the communication with the patient, and for improving treatment outcome. However, in dermatology practice, the application of this intervention is relatively new. Moreover, evidence on the effectiveness of a HRQoL intervention in dermatology is missing. It is hypothesized that the application of a HRQoL intervention in dermatology practice will have a positive impact on patients’ HRQoL as well as on doctor-patient communication.
In a prospective multicenter cluster randomized controlled trial, patients diagnosed with moderate to severe psoriasis who receive biologic treatment, will be followed for 48 weeks. The study sites, and not the patients, will be randomly allocated via a computer-based randomization system to either the intervention (treatment with etanercept and standardized HRQoL assessment and communication) or the control group (treatment with etanercept alone). The HRQoL intervention will include 1) the electronic assessment of the Skindex-29, a well-studied dermatology-specific HRQoL questionnaire, and 2) the communication of the resulting Skindex-29 data with the patient. Prior to study start, dermatologists in the intervention group will be educated and trained in standardized HRQoL assessment and communication using the Skindex-29. At six consecutive visits, patients at study sites in the intervention group will be asked to complete the Skindex-29 on a desk-top pc at the clinic, just before their consultation with the dermatologist. A print-out of the completed questionnaire will be made and, guided by this print-out, feedback on the HRQoL scores will be given during the consultation. Primary outcome parameters are the impact of the HRQoL intervention on patients’ HRQoL, and the effect of the HRQoL intervention on doctor-patient communication. Secondary outcomes include health status and disease severity.
Trial registration
The Netherlands National Trial Register (NTR): NTR1364.
PMCID: PMC3560082  PMID: 23216943
Dermatology; Randomized controlled trial; Patient-reported outcomes; Health-related quality of life; Intervention; Skindex-29; Psoriasis; Biologic; Etanercept
8.  Health care providers underestimate symptom intensities of cancer patients: A multicenter European study 
Many patients with advanced cancer depend upon health care providers for symptom assessment. The extent of agreement between patient and provider symptom assessments and the association of agreement with demographic- and disease-related factors was examined.
This cross-sectional study included 1933 patient-health care provider dyads, from 11 European countries. Patients reported symptoms by using the four-point scales of the European Organization of Research and Treatment of Cancer Core Quality of Life Questionnaire (EORTC QLQ-C30) version 3, and providers used corresponding four-point categorical scales. Level of agreement was addressed at the group level (Wilcoxon Signed-Rank test), by difference scores (provider score minus patient score), at the individual level (Intraclass Correlation Coefficients, ICCs) and visually by Bland-Altman plots. Absolute numbers and chi-square tests were used to investigate the relationship between agreement and demographic-, as well as disease-related factors.
The prevalence of symptoms assessed as moderate or severe by patients and providers, respectively, were for pain (67 vs.47%), fatigue (71 vs. 54%), generalized weakness (65 vs. 47%), anorexia (47 vs. 25%), depression (31 vs. 17%), constipation (45 vs. 30%), poor sleep (32 vs. 21%), dyspnea (30 vs. 16%), nausea (27 vs. 14%), vomiting (14 vs. 6%) and diarrhea (14 vs. 6%). Symptom scores were identical or differed by only one response category in the majority of patient-provider assessment pairs (79-93%). Providers underestimated the symptom in approximately one of ten patients and overestimated in 1% of patients. Agreement at the individual level was moderate (ICC 0.38 to 0.59). Patients with low Karnofsky Performance Status, high Mini Mental State-score, hospitalized, recently diagnosed or undergoing opioid titration were at increased risk of symptom underestimation by providers (all p < 0.001). Also, the agreement was significantly associated with drug abuse (p = 0.024), provider profession (p < 0.001), cancer diagnosis (p < 0.001) and country (p < 0.001).
Considerable numbers of health care providers underestimated symptom intensities. Clinicians in cancer care should be aware of the factors characterizing patients at risk of symptom underestimation.
PMCID: PMC2949821  PMID: 20858248
9.  Differential item functioning (DIF) analyses of health-related quality of life instruments using logistic regression 
Differential item functioning (DIF) methods can be used to determine whether different subgroups respond differently to particular items within a health-related quality of life (HRQoL) subscale, after allowing for overall subgroup differences in that scale. This article reviews issues that arise when testing for DIF in HRQoL instruments. We focus on logistic regression methods, which are often used because of their efficiency, simplicity and ease of application.
A review of logistic regression DIF analyses in HRQoL was undertaken. Methodological articles from other fields and using other DIF methods were also included if considered relevant.
There are many competing approaches for the conduct of DIF analyses and many criteria for determining what constitutes significant DIF. DIF in short scales, as commonly found in HRQL instruments, may be more difficult to interpret. Qualitative methods may aid interpretation of such DIF analyses.
A number of methodological choices must be made when applying logistic regression for DIF analyses, and many of these affect the results. We provide recommendations based on reviewing the current evidence. Although the focus is on logistic regression, many of our results should be applicable to DIF analyses in general. There is a need for more empirical and theoretical work in this area.
PMCID: PMC2924271  PMID: 20684767
10.  A 'short walk' is longer before radiotherapy than afterwards: a qualitative study questioning the baseline and follow-up design 
Numerous studies have indirectly demonstrated changes in the content of respondents' QoL appraisal process over time by revealing response-shift effects. This is the first known study to qualitatively examine the assumption of consistency in the content of the cognitive processes underlying QoL appraisal over time. Specific objectives are to examine whether the content of each distinct cognitive process underlying QoL appraisal is (dis)similar over time and whether patterns of (dis)similarity can be discerned across and within patients and/or items.
We conducted cognitive think-aloud interviews with 50 cancer patients prior to and following radiotherapy to elicit cognitive processes underlying the assessment of 7 EORTC QLQ-C30 items. Qualitative analysis of patients' responses at baseline and follow-up was independently carried out by 2 researchers by means of an analysis scheme based on the cognitive process models of Tourangeau et al. and Rapkin & Schwartz.
The interviews yielded 342 comparisons of baseline and follow-up responses, which were analyzed according to the five cognitive processes underlying QoL appraisal. The content of comprehension/frame of reference changed in 188 comparisons; retrieval/sampling strategy in 246; standards of comparison in 152; judgment/combinatory algorithm in 113; and reporting and response selection in 141 comparisons. Overall, in 322 comparisons of responses (94%) the content of at least one cognitive component changed over time. We could not discern patterns of (dis)similarity since the content of each of the cognitive processes differed across and within patients and/or items. Additionally, differences found in the content of a cognitive process for one item was not found to influence dissimilarity in the content of that same cognitive process for the subsequent item.
The assumption of consistency in the content of the cognitive processes underlying QoL appraisal over time was not found to be in line with the cognitive processes described by the respondents. Additionally, we could not discern patterns of (dis)similarity across and within patients and/or items. In building on cognitive process models and the response shift literature, this study contributes to a better understanding of patient-reported QoL appraisal over time.
PMCID: PMC2915972  PMID: 20637086
11.  Reflections on changeability versus stability of health-related quality of life: distinguishing between its environmental and genetic components 
The field of health-related quality of life (HRQOL) could benefit from a broadening of perspectives to include recent advancements in research on adaptation, positive psychology, and genetics. These advances shed new light on the extent to which HRQOL is changeable or fixed. The objective of this paper is to integrate these insights and to discuss their implications for HRQOL research. We describe the Hedonic Treadmill theory, which asserts that positive events only temporarily affect happiness since people quickly return to hedonic neutrality. New empirical evidence suggests important revisions of this theory, providing a more optimistic picture of the possibility for change. Advances in positive psychology show that relatively simple interventions have the power to induce a sustainable increase in levels of happiness. Finally, a small but growing number of studies have found independent genetic influences in well-being, life satisfaction, perceived health, and even HRQOL. Given the increasing empirical evidence that HRQOL can be sustainably enhanced and is in part genetically determined, it may be useful to consider HRQOL as a concept that has state (environmental) and trait (genetic) components. This distinction will allow us to explore new pathways of improving theory, methods, and clinical practice. The overarching novel questions concern the extent to which HRQOL components are environmentally or genetically determined, and which factors lead to lasting improvement. This distinction begs for new research approaches, such as time-sampling techniques and interdisciplinary research investigating the genetic variants of HRQOL. Distinguishing between those aspects that are amenable to change from those that are relatively fixed and stable will help better target specific support interventions.
PMCID: PMC2607267  PMID: 18976504
12.  Laparoscopic ileocolic resection versus infliximab treatment of distal ileitis in Crohn's disease: a randomized multicenter trial (LIR!C-trial) 
BMC Surgery  2008;8:15.
With the availability of infliximab, nowadays recurrent Crohn's disease, defined as disease refractory to immunomodulatory agents that has been treated with steroids, is generally treated with infliximab. Infliximab is an effective but expensive treatment and once started it is unclear when therapy can be discontinued. Surgical resection has been the golden standard in recurrent Crohn's disease. Laparoscopic ileocolic resection proved to be safe and is characterized by a quick symptom reduction.
The objective of this study is to compare infliximab treatment with laparoscopic ileocolic resection in patients with recurrent Crohn's disease of the distal ileum with respect to quality of life and costs.
The study is designed as a multicenter randomized clinical trial including patients with Crohn's disease located in the terminal ileum that require infliximab treatment following recent consensus statements on inflammatory bowel disease treatment: moderate to severe disease activity in patients that fail to respond to steroid therapy or immunomodulatory therapy. Patients will be randomized to receive either infliximab or undergo a laparoscopic ileocolic resection. Primary outcomes are quality of life and costs. Secondary outcomes are hospital stay, early and late morbidity, sick leave and surgical recurrence. In order to detect an effect size of 0.5 on the Inflammatory Bowel Disease Questionnaire at a 5% two sided significance level with a power of 80%, a sample size of 65 patients per treatment group can be calculated. An economic evaluation will be performed by assessing the marginal direct medical, non-medical and time costs and the costs per Quality Adjusted Life Year (QALY) will be calculated. For both treatment strategies a cost-utility ratio will be calculated. Patients will be included from December 2007.
The LIR!C-trial is a randomized multicenter trial that will provide evidence whether infliximab treatment or surgery is the best treatment for recurrent distal ileitis in Crohn's disease.
Trial registration
Nederlands Trial Register NTR1150
PMCID: PMC2533646  PMID: 18721465
13.  Colonic stenting as bridge to surgery versus emergency surgery for management of acute left-sided malignant colonic obstruction: a multicenter randomized trial (Stent-in 2 study) 
BMC Surgery  2007;7:12.
Acute left-sided colonic obstruction is most often caused by malignancy and the surgical treatment is associated with a high mortality and morbidity rate. Moreover, these operated patients end up with a temporary or permanent stoma. Initial insertion of an enteral stent to decompress the obstructed colon, allowing for surgery to be performed electively, is gaining popularity. In uncontrolled studies stent placement before elective surgery has been suggested to decrease mortality, morbidity and number of colostomies. However stent perforation can lead to peritoneal tumor spill, changing a potentially curable disease in an incurable one. Therefore it is of paramount importance to compare the outcomes of colonic stenting followed by elective surgery with emergency surgery for the management of acute left-sided malignant colonic obstruction in a randomized multicenter fashion.
Patients with acute left-sided malignant colonic obstruction eligible for this study will be randomized to either emergency surgery (current standard treatment) or colonic stenting as bridge to elective surgery. Outcome measurements are effectiveness and costs of both strategies. Effectiveness will be evaluated in terms of quality of life, morbidity and mortality. Quality of life will be measured with standardized questionnaires (EORTC QLQ-C30, EORTC QLQ-CR38, EQ-5D and EQ-VAS). Morbidity is defined as every event leading to hospital admission or prolonging hospital stay. Mortality will be analyzed as total mortality as well as procedure-related mortality. The total costs of treatment will be evaluated by counting volumes and calculating unit prices. Including 120 patients on a 1:1 basis will have 80% power to detect an effect size of 0.5 on the EORTC QLQ-C30 global health scale, using a two group t-test with a 0.05 two-sided significance level. Differences in quality of life and morbidity will be analyzed using mixed-models repeated measures analysis of variance. Mortality will be compared using Kaplan-Meier curves and log-rank statistics.
The Stent-in 2 study is a randomized controlled multicenter trial that will provide evidence whether or not colonic stenting as bridge to surgery is to be performed in patients with acute left-sided colonic obstruction.
Trial registration
Current Controlled Trials ISRCTN46462267.
PMCID: PMC1925059  PMID: 17608947

Results 1-13 (13)