Patient-reported outcomes in congenital cardiac disease: are they as good as you think they are?

2010 ◽  
Vol 20 (S3) ◽  
pp. 143-148 ◽  
Author(s):  
Philip Moons

AbstractPatient-reported outcomes are “any outcome based on data provided by patients or patient proxy as opposed to data provided from other sources”. Examples of patient-reported outcomes are quality of life, well-being, functional status, symptoms, adherence to treatment, satisfaction with treatment, and utility or preference-based measures. The main question of this manuscript is whether patient-reported outcomes in patients with congenital cardiac disease are as good as we think they are. In general, we could say yes, because numerous studies show that patients with congenital cardiac disease have an excellent quality of life. By contrast, we could say no, because patients generally overestimate their functioning, and up to two out of three patients are not compliant with the prescribed therapy or recommendations for follow-up. However, most importantly, we have to say that we do not know whether the patient-reported outcomes are good, because research with patient-reported outcomes in congenital cardiac disease is limited. Hence, patient-reported outcomes should be a priority on the agenda for research in the domain of congenital cardiac disease.

2020 ◽  
Vol 41 (Supplement_2) ◽  
Author(s):  
A Salzwedel ◽  
I Koran ◽  
E Langheim ◽  
A Schlitt ◽  
J Nothroff ◽  
...  

Abstract Introduction Comprehensive cardiac rehabilitation (CR) programs based on the bio-psycho-social approach of the international classification of functioning and disease are carried out to achieve improved prognosis, superior health-related quality of life (HRQL) and social integration. We aimed to identify predictors of returning to work (RTW) and HRQL among cardiovascular risk factors and physical performance as well as patient-reported outcome measures (PROMs) modifiable during CR. Methods We designed a prospective observational multi-center study and enrolled 1,586 patients (2017/18) in 12 German rehabilitation centers regardless of their primary allocation diagnoses (e.g. acute myocardial infarction (AMI), coronary artery bypass grafting (CABG), coronary artery disease (CAD), valvular disease). Besides general data (e.g. age, gender, diagnoses), parameters of risk factor management (e.g. smoking, lipid profile, hypertension, lifestyle change motivation), physical performance (e.g. maximum exercise capacity, endurance training load, 6-min walking distance), and PROMs (e.g. depression, heart-focused anxiety, HRQL, subjective well-being, somatic and mental health, pain, general self-efficacy, pension desire as well as self-assessment of occupational prognosis using several questionnaires) were documented at CR admission and discharge. 6 months after discharge, status of RTW and HRQL (SF-12) were captured by a follow-up (FU) survey and analyzed in multivariable regression models with multiple imputation of missing values. Results Out of the study participants, 1,262 patients (54±7 years, 77% men) responded to the follow-up survey and could be analyzed regarding the outcome parameters. Most of them were assigned to CR primarily due to AMI (40%) or CAD without myocardial infarction (18%), followed by heart valve diseases in 12% of patients and CABG (8%). 864 patients (69%) returned to work within the follow-up period. Pension desire, negative self-assessed occupational prognosis, heart-focussed anxiety, major life events, smoking and heart failure were negatively associated with RTW, while higher endurance training load, HRQL and work stress were positively associated (Figure 1). HRQL after 6 months was determined more by PROMs (e.g. pension desire, heart-focused anxiety, physical/mental HRQL in SF-12, physical/mental health in indicators of rehab-status questionnaire (IRES-24), stress, well-being in the World Health Organization well-being index and self-efficacy expectations) than by clinical parameters or physical performance. Conclusions Patient-reported outcome measures predominantly influenced RTW and HRQL in heart-disease patients, whereas patients' pension desire and heart-focussed anxiety had a dominant impact on all investigated endpoints. Therefore, the multi-component CR approach focussing on psychosocial support is crucial for subjective health prognosis and occupational resumption. Figure 1. Predictors of returning to work Funding Acknowledgement Type of funding source: Public grant(s) – National budget only. Main funding source(s): German pension insurance


Author(s):  
Ilona Stolpner ◽  
Jörg Heil ◽  
Fabian Riedel ◽  
Markus Wallwiener ◽  
Benedikt Schäfgen ◽  
...  

Abstract Background Poor patient-reported satisfaction after breast-conserving therapy (BCT) has been associated with impaired health-related quality of life (HRQOL) and subsequent depression in retrospective analysis. This prospective cohort study aimed to assess the HRQOL of patients who have undergone BCT using the BREAST-Q, and to identify clinical risk factors for lower patient satisfaction. Methods Patients with primary breast cancer undergoing BCT were asked to complete the BREAST-Q preoperatively (T1) for baseline evaluation, then 3 to 4 weeks postoperatively (T2), and finally 1 year after surgery (T3). Clinicopathologic data were extracted from the patients’ charts. Repeated measures analysis of variance (ANOVA) was used to determine significant differences in mean satisfaction and well-being levels among the test intervals. Multiple linear regression was used to evaluate risk factors for lower satisfaction. Results The study enrolled 250 patients. The lowest baseline BREAST-Q score was reported for “satisfaction with breast” (mean, 61 ± 19), but this increased postoperatively (mean, 66 ± 18) and was maintained at the 1 year follow-up evaluation (mean, 67 ± 21). “Physical well-being” decreased from T1 (mean, 82 ± 17) to T2 (mean, 28 ± 13) and did not recover much by T3 (mean, 33 ± 13), being the lowest BREAST-Q score postoperatively and in the 1-year follow-up evaluation. In multiple regression, baseline psychosocial well-being, body mass index (BMI), and type of incision were risk factors for lower “satisfaction with breasts.” Conclusion Both the aesthetic/surgery-related and psychological aspects are equally important with regard to “satisfaction with breasts” after BCT. The data could serve as the benchmark for future studies.


2021 ◽  
Vol 43 (1) ◽  
Author(s):  
Robert M. Kaplan ◽  
Ron D. Hays

Patient-reported outcomes are recognized as essential for the evaluation of medical and public health interventions. Over the last 50 years, health-related quality of life (HRQoL) research has grown exponentially from 0 to more than 17,000 papers published annually. We provide an overview of generic HRQoL measures used widely in epidemiological studies, health services research, population studies, and randomized clinical trials [e.g., Medical Outcomes Study SF-36 and the Patient-Reported Outcomes Measurement Information System (PROMIS®)-29]. In addition, we review methods used for economic analysis and calculation of the quality-adjusted life year (QALY). These include the EQ-5D, the Health Utilities Index (HUI), the self-administered Quality of Well-being Scale (QWB-SA), and the Health and Activities Limitation Index (HALex). Furthermore, we consider hybrid measures such as the SF-6D and the PROMIS-Preference (PROPr). The plethora of HRQoL measures has impeded cumulative science because incomparable measures have been used in different studies. Linking among different measures and consensus on standard HRQoL measurement should now be prioritized. In addition, enabling widespread access to common measures is necessary to accelerate future progress. Expected final online publication date for the Annual Review of Public Health, Volume 43 is April 2022. Please see http://www.annualreviews.org/page/journal/pubdates for revised estimates.


2020 ◽  
Vol 30 (5) ◽  
pp. 596-601
Author(s):  
Dana Chase ◽  
Helen Q Huang ◽  
Bradley J Monk ◽  
Lois Michelle Ramondetta ◽  
Richard T Penson ◽  
...  

IntroductionTo describe patient-reported outcomes and toxicities at time of treatment discontinuation secondary to progression or toxicities in advanced/recurrent cervical cancer patients receiving chemotherapy with bevacizumab.MethodsSummarize toxicity, grade, and health-related quality of life within 1 month of treatment discontinuation for women receiving chemotherapy with bevacizumab in GOG240.ResultsOf the 227 patients who received chemotherapy with bevacizumab, 148 discontinued study protocol treatment (90 for disease progression and 58 for toxicity). The median survival time from treatment discontinuation to death was 7.9 months (95% CI 5.0 to 9.0) for those who progressed versus 12.1 months (95% CI 8.9 to 23.2) for those who discontinued therapy due to toxicities. The most common grade 3 or higher toxicities included hematologic, gastrointestinal, and pain. Some 57% (84/148) of patients completed quality of life assessment within 1 month of treatment discontinuation. Those patients who discontinued treatment due to progression had a mean decline in the FACT-Cx TOI of 3.2 points versus 2.2 in patients who discontinued therapy due to toxicity. This was a 9.9 point greater decline in the FACT-Cx TOI scores than those who discontinued treatment due to progression (95% CI 2.8 to 17.0, p=0.007). The decline in quality of life was due to worsening physical and functional well-being. Those who discontinued treatment due to toxicities had worse neurotoxicity and pain.DiscussionPatients who discontinued chemotherapy with bevacizumab for toxicity experienced longer post-protocol survival but significantly greater declination in quality of life than those with progression. Future trial design should include supportive care interventions that optimize physiologic function and performance status for salvage therapies.


2016 ◽  
Vol 34 (2_suppl) ◽  
pp. 157-157 ◽  
Author(s):  
Daniel Xiao Yang ◽  
Jackson Thea ◽  
Yi An ◽  
James B. Yu

157 Background: The use of digital health technology, including mobile applications, in the clinical setting is becoming increasingly more prevalent. Such technology is currently being explored as clinical research tools. While the side effects of prostate radiotherapy are well documented after treatment, there remains a paucity of data on patient-reported outcomes and changes in quality of life (QOL) during the treatment period. Therefore, mobile applications represent a practical platform to enable patient reporting in real-time during prostate radiotherapy. Methods: Using an existing open source code framework (Apple ResearchKit), we developed a novel mobile application that enables prostate cancer patients to report, either during or immediately following daily radiation treatment, changes in urinary, bowel, sexual, and hormonal QOL domains. The mobile application utilizes validated questions from the Expanded Prostate Index Composite for Clinical Practice (EPIC-CP) Survey, and allows for survey responses to be tracked over time throughout the treatment period and at routine follow up. Results: For the initial phase of our study, we are currently piloting the mobile application at a single institution with a goal of accruing 50 patients. Study results will be compared to data from traditional surveys, which are available at follow-up but impracticable for real-time symptom reporting. By ASCO 2016 Genitourinary Cancers Symposium, we plan to begin the second phase of our study where any patient can enroll online through a mobile software distribution platform (Apple App Store). Conclusions: We demonstrate the feasibility of using a mobile application to enable patients to report quality of life changes in real-time during prostate radiotherapy. Moreover, our application facilitates clinical trials where patient data collection can be automated and completed at scale. Future prospective studies are planned to evaluate validity of clinical trial data gathered through such methodology.


2016 ◽  
Vol 20 (64) ◽  
pp. 1-86 ◽  
Author(s):  
Rebecca K Simmons ◽  
Knut Borch-Johnsen ◽  
Torsten Lauritzen ◽  
Guy EHM Rutten ◽  
Annelli Sandbæk ◽  
...  

BackgroundIntensive treatment (IT) of cardiovascular risk factors can halve mortality among people with established type 2 diabetes but the effects of treatment earlier in the disease trajectory are uncertain.ObjectiveTo quantify the cost-effectiveness of intensive multifactorial treatment of screen-detected diabetes.DesignPragmatic, multicentre, cluster-randomised, parallel-group trial.SettingThree hundred and forty-three general practices in Denmark, the Netherlands, and Cambridge and Leicester, UK.ParticipantsIndividuals aged 40–69 years with screen-detected diabetes.InterventionsScreening plus routine care (RC) according to national guidelines or IT comprising screening and promotion of target-driven intensive management (medication and promotion of healthy lifestyles) of hyperglycaemia, blood pressure and cholesterol.Main outcome measuresThe primary end point was a composite of first cardiovascular event (cardiovascular mortality/morbidity, revascularisation and non-traumatic amputation) during a mean [standard deviation (SD)] follow-up of 5.3 (1.6) years. Secondary end points were (1) all-cause mortality; (2) microvascular outcomes (kidney function, retinopathy and peripheral neuropathy); and (3) patient-reported outcomes (health status, well-being, quality of life, treatment satisfaction). Economic analyses estimated mean costs (UK 2009/10 prices) and quality-adjusted life-years from an NHS perspective. We extrapolated data to 30 years using the UK Prospective Diabetes Study outcomes model [version 1.3;©Isis Innovation Ltd 2010; seewww.dtu.ox.ac.uk/outcomesmodel(accessed 27 January 2016)].ResultsWe included 3055 (RC,n = 1377; IT,n = 1678) of the 3057 recruited patients [mean (SD) age 60.3 (6.9) years] in intention-to-treat analyses. Prescription of glucose-lowering, antihypertensive and lipid-lowering medication increased in both groups, more so in the IT group than in the RC group. There were clinically important improvements in cardiovascular risk factors in both study groups. Modest but statistically significant differences between groups in reduction in glycated haemoglobin (HbA1c) levels, blood pressure and cholesterol favoured the IT group. The incidence of first cardiovascular event [IT 7.2%, 13.5 per 1000 person-years; RC 8.5%, 15.9 per 1000 person-years; hazard ratio 0.83, 95% confidence interval (CI) 0.65 to 1.05] and all-cause mortality (IT 6.2%, 11.6 per 1000 person-years; RC 6.7%, 12.5 per 1000 person-years; hazard ratio 0.91, 95% CI 0.69 to 1.21) did not differ between groups. At 5 years, albuminuria was present in 22.7% and 24.4% of participants in the IT and RC groups, respectively [odds ratio (OR) 0.87, 95% CI 0.72 to 1.07), retinopathy in 10.2% and 12.1%, respectively (OR 0.84, 95% CI 0.64 to 1.10), and neuropathy in 4.9% and 5.9% (OR 0.95, 95% CI 0.68 to 1.34), respectively. The estimated glomerular filtration rate increased between baseline and follow-up in both groups (IT 4.31 ml/minute; RC 6.44 ml/minute). Health status, well-being, diabetes-specific quality of life and treatment satisfaction did not differ between the groups. The intervention cost £981 per patient and was not cost-effective at costs ≥ £631 per patient.ConclusionsCompared with RC, IT was associated with modest increases in prescribed treatment, reduced levels of risk factors and non-significant reductions in cardiovascular events, microvascular complications and death over 5 years. IT did not adversely affect patient-reported outcomes. IT was not cost-effective but might be if delivered at a reduced cost. The lower than expected event rate, heterogeneity of intervention delivery between centres and improvements in general practice diabetes care limited the achievable differences in treatment between groups. Further follow-up to assess the legacy effects of early IT is warranted.Trial registrationClinicalTrials.gov NCT00237549.Funding detailsThis project was funded by the NIHR Health Technology Assessment programme and will be published in full inHealth Technology Assessment; Vol. 20, No. 64. See the NIHR Journals Library website for further project information.


2020 ◽  
Vol 20 (1) ◽  
Author(s):  
Marjolein M. Iversen ◽  
Jannicke Igland ◽  
Hilde Smith-Strøm ◽  
Truls Østbye ◽  
Grethe S. Tell ◽  
...  

Abstract Background Follow-up care provided via telemedicine (TM) is intended to be a more integrated care pathway to manage diabetes-related foot ulcers (DFU) than traditionally-delivered healthcare. However, knowledge of the effect of TM follow-up on PROMs including self-reported health, well-being and QOL in patients with DFUs is lacking and often neglected in RCT reports in general. Therefore, in this study of secondary outcomes from the DiaFOTo trial, the aim was to compare changes in self-reported health, well-being and QOL between patients with DFUs receiving telemedicine follow-up care in primary healthcare in collaboration with specialist healthcare, and patients receiving standard outpatient care. Methods The current study reports secondary endpoints from a cluster randomized controlled trial whose primary endpoint was ulcer healing time. The trial included 182 adults with diabetes-related foot ulcers (94/88 in the telemedicine/standard care groups) in 42 municipalities/districts, recruited from three clinical sites in Western Norway. Mean (SD) diabetes duration for the study population was 20.8 (15.0). The intervention group received care in the community in collaboration with specialist healthcare using an asynchronous telemedicine intervention. The intervention included an interactive web-based ulcer record and a mobile phone enabling counseling and communication between the community nurses and specialist healthcare; the control group received standard outpatient care. In total 156 participants (78/78) reported on secondary endpoints: self-reported health, well-being and quality of life evaluated by generic and disease-specific patient-reported outcome measures (e.g. Euro-QOL, the Hospital Anxiety and Depression Scale (HADS), Problem Areas in Diabetes (PAID), Neuropathy and Foot Ulcer–Specific Quality of Life Instrument (NeuroQOL)). Linear mixed-effects regression was used to investigate possible differences in changes in the scores between the intervention and control group at the end of follow-up. Results In intention to treat analyses, differences between treatment groups were small and non-significant for the health and well-being scale scores, as well as for diabetes-related distress and foot ulcer-specific quality of life. Conclusions There were no significant differences in changes in scores for the patient reported outcomes between the intervention and control group, indicating that the intervention did not affect the participants’ health, well-being and quality of life. Trial registration Clinicaltrials.gov, NCT01710774. Registered October 19th, 2012.


BMJ Open ◽  
2018 ◽  
Vol 8 (7) ◽  
pp. e021859
Author(s):  
Ilana N Ackerman ◽  
Richard S Page ◽  
Kathy Fotis ◽  
Peter Schoch ◽  
Nigel Broughton ◽  
...  

IntroductionPersistent musculoskeletal conditions can impact profoundly on younger people’s quality of life, psychological distress and capacity to work, as shown by previous research involving younger people with osteoarthritis. The personal impacts, in particular, work and parenting impacts, of other musculoskeletal conditions (such as persistent shoulder pain) on younger patient groups remain poorly understood. Furthermore, the personal financial burden associated with managing musculoskeletal conditions is rarely documented. This study aims to investigate well-being, work participation and productivity, shoulder-related parenting disability and out-of-pocket healthcare expenditure among younger people with shoulder pain and evaluate changes over 12 months.Methods and analysisOne hundred and fifty people aged 20–55 years with shoulder pain of more than 6 weeks’ duration (excluding those with recent history of fracture or dislocation) will be recruited for this cohort study. Participants will be recruited from three major public hospitals in Victoria, Australia, following screening of orthopaedic outpatient clinics lists and referrals. Participants will be asked to complete a baseline questionnaire and 2-week healthcare costs diary, with follow-up data collected at 12 months. Patient-reported outcomes will be collected, including health-related quality of life (HRQoL), shoulder pain and function, psychological distress, shoulder-related parenting disability and work productivity. Information on sociodemographics, employment, health services utilisation and shoulder-related healthcare expenditure will also be collected. Descriptive analysis of baseline data will provide a comprehensive snapshot of the personal burden of shoulder pain. Baseline HRQoL and psychological distress data will be compared with Australian population norms to provide context around well-being. Associations between sociodemographic factors and patient-reported outcomes will be evaluated using univariate and multivariate analyses. Changes in patient-reported outcomes from baseline to 12 months will be analysed using paired t-tests.Ethics and disseminationEthics approval has been obtained. The study findings will be submitted to peer-reviewed journals and presented at relevant scientific meetings.


BMJ Open ◽  
2018 ◽  
Vol 8 (3) ◽  
pp. e017571 ◽  
Author(s):  
Irmela Gnass ◽  
Michaela Ritschel ◽  
Silke Andrich ◽  
Silke Kuske ◽  
Kai Moschinski ◽  
...  

IntroductionSurvivors of polytrauma experience long-term and short-term burden that influences their lives. The patients’ view of relevant short-term and long-term outcomes should be captured in instruments that measure quality of life and other patient-reported outcomes (PROs) after a polytrauma. The aim of this systematic review is to (1) collect instruments that assess PROs (quality of life, social participation and activities of daily living) during follow-up after polytrauma, (2) describe the instruments’ application (eg, duration of period of follow-up) and (3) investigate other relevant PROs that are also assessed in the included studies (pain, depression, anxiety and cognitive function).Methods and analysisThe systematic review protocol is developed in line with the Preferred Reporting Items for Systematic Review and Meta-Analysis Protocols statement. MEDLINE, EMBASE, Cumulative Index to Nursing and Allied Health Literature, PsycINFO, Cochrane Central Register of Controlled Trials and the trials registers ClinicalTrials.gov and WHO International Clinical Trials Registry Platform will be searched. Keywords, for example, ‘polytrauma’, ‘multiple trauma’, ‘quality of life’, ‘activities of daily living’ or ‘pain’ will be used. Publications published between January 2005 and the most recent date (currently: August 2016) will be included. In order to present the latest possible results, an update of the search is conducted before publication. The data extraction and a content analysis will be carried out systematically. A critical appraisal will be performed.Ethics and disseminationFormal ethical approval is not required as primary data will not be collected. The results will be published in a peer-reviewed publication.PROSPERO registration numberCRD42017060825.


2013 ◽  
Vol 2013 ◽  
pp. 1-13 ◽  
Author(s):  
Michael J. Mackenzie ◽  
Linda E. Carlson ◽  
Panteleimon Ekkekakis ◽  
David M. Paskevich ◽  
S. Nicole Culos-Reed

Little attention has been paid to the psychological determinants by which benefits are accrued via yoga practice in cancer-related clinical settings. Using a longitudinal multilevel modeling approach, associations between affect, mindfulness, and patient-reported mental health outcomes, including mood disturbance, stress symptoms, and health-related quality of life (HRQL), were examined in an existing seven-week yoga program for cancer survivors. Participants(N=66)were assessed before and after the yoga program and at three- and six-month follow-ups. Decreases in mood disturbance and stress symptoms and improvements in HRQL were observed upon program completion. Improvements in mood disturbance and stress symptoms were maintained at the three- and six-month follow-ups. HRQL exhibited further improvement at the three-month follow-up, which was maintained at the six-month follow-up. Improvements in measures of well-being were predicted by initial positive yoga beliefs and concurrently assessed affective and mindfulness predictor variables. Previous yoga experience, affect, mindfulness, and HRQL were related to yoga practice maintenance over the course of the study.


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