scholarly journals Translation and validation of two disease-specific patient-reported outcome measures (Bladder Cancer Index and FACT-Bl-Cys) in Dutch bladder cancer patients

2019 ◽  
Vol 3 (1) ◽  
Author(s):  
Charlotte T. J. Michels ◽  
Carl J. Wijburg ◽  
Inger L. Abma ◽  
J. Alfred Witjes ◽  
Janneke P. C. Grutters ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Bladder Cancer ◽  
Clinical Practice ◽  
Cancer Patients ◽  
Radical Cystectomy ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Patient Reported ◽  
Disease Specific

Abstract Background The Bladder Cancer Index (BCI) and Functional Assessment of Cancer Therapy-Bladder-Cystectomy (FACT-Bl-Cys) were developed to measure disease-specific health-related quality of life (HRQOL) in bladder cancer patients and patients treated with radical cystectomy, respectively. Both patient-reported outcome measures (PROMs) are frequently used in clinical practice, but are not yet validated according to the COSMIN criteria and not yet available in Dutch. Therefore, the aim of this study was to translate the BCI and FACT-Bl-Cys into Dutch and to evaluate their measurement properties according to the COSMIN criteria. Methods The BCI and FACT-Bl-Cys were translated into Dutch using a forward-backward method, and subsequently administered at baseline (pre-operatively) and 3 months post-operatively in bladder cancer patients who received a radical cystectomy. Validity (content and construct), reliability (internal consistency, test-retest reliability, and measurement error), floor and ceiling effects, and responsiveness were assessed according to the COSMIN criteria. Results Forward-backward translation encountered no particular linguistic problems. In total 260 patients completed the baseline measurement, while 182 patients completed the three-month measurement. Only a ceiling effect was identified for the BCI. Hypotheses testing for construct validity was satisfying, as 67% and 92% of the hypothesized correlations were confirmed. Structural validity was moderate for both measures, as confirmatory factor analyses showed limited fit. Reliability of both PROMs was good. The intraclass correlation coefficient (ICC) of the BCI domains ranged from 0.47 to 0.93, minimal value of Cronbach’s α was 0.70, smallest detectable change on group level (SDC group) ranged from 1.9 to 8.6. The ICC of the FACT-Bl-Cys domains ranged from 0.43 to 0.83, minimal value of Cronbach’s α was 0.77, SDC group was around 1. Only the FACT-Bl-Cys total score was found to be responsive to changes in generic quality of life. Conclusions The Dutch versions of the BCI and FACT-Bl-Cys were shown to be reliable and have good content validity. Structural validity was limited for both measures. Only the FACT-Bl-Cys total score was responsive to changes in generic HRQOL. Despite some limitations, both PROMs seem suitable for use in clinical practice and research.

scholarly journals Characteristics and patient-reported outcomes associated to dropout in severely affected oncological patients

2021 ◽  
Author(s):  
Pimrapat Gebert ◽  
Daniel Schindel ◽  
Johann Frick ◽  
Liane Schenk ◽  
Ulrike Grittner
Keyword(s):  
Quality Of Life ◽  
Social Support ◽  
Cancer Patients ◽  
Patient Reported Outcomes ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Poor Quality ◽  
Drop Out ◽  
Patient Reported

Abstract BackgroundPatient-reported outcome measures (PROMs) are commonly used and are surrogates for clinical outcomes in cancer research. In the research setting of very severe diseases such as cancer, it is difficult to avoid the problem of incomplete questionnaires from drop-out or missing data due to patients who deceased during observation period. We aimed to explore patient characteristics and patient-reported outcomes associated with the time-to-dropout. MethodsIn the Oncological Social Care Project (OSCAR) study the condition of participants was assessed four times within 12 months (t0: baseline, t1: 3 months, t2: 6 months, and t3: 12 months) by validated PROMs. We performed competing-risks regression based on Fine and Gray’s proportional sub-distribution hazards model for exploring factors associated with time-to-dropout. Death was considered as competing risk. ResultsThree hundred sixty-two participants were analyzed in the study. 193 (53.3%) completed follow-up at 12 months, 67 (18.5%) patients dropped out, and 102 patients (28.2%) died during the study period. Poor subjective social support was related to higher risk for drop-out (SHR=2.10; 95%CI: 1.01 – 4.35). Lower values in health-related quality of life were related to drop-out and death. The subscales global health status/QoL, role functioning, physical functioning, and fatigue symptom in the EORTC QLQ-C30 were key characteristics associated with early drop-out.ConclusionSeverely affected cancer patients with poor social support and poor quality of life seem more likely to drop out of studies compared to patients with higher levels of social support and quality of life. This should be considered when planning studies assessing cancer patients. Methods to monitor drop-outs timely and handle missing outcomes might be used. Results of such studies have to be interpreted with caution in light of the particular drop-out mechanisms.

scholarly journals Patient-Reported Outcome Measures (PROMs) in Pediatric Non-Malignant Hematology: A Systematic Review

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 2180-2180
Author(s):  
Robert J Klaassen ◽  
Julia Y. Kinahan ◽  
Johann M. I. Graham ◽  
Yamilée V. Hébert ◽  
Katie O'Hearn
Keyword(s):  
Quality Of Life ◽  
Systematic Review ◽  
Outcome Measures ◽  
Well Being ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Clinical Environment ◽  
Patient Reported ◽  
Disease Specific

Introduction: Patient reported outcome measures (PROMs) are questionnaires completed by patients or caregivers without interpretation by healthcare professionals. As such, they allow patient concerns about a variety of healthcare issues to be identified and addressed in an efficient and actionable manner. PROMs can be generic, with questions relevant to multiple disease groups or disease-specific, with questions targeting the symptoms, limitations, and feelings common to the disease group. This systematic review identified generic and disease-specific PROMs for monitoring symptoms and health-related quality of life (HRQoL) in 4 pediatric non-malignant hematologic disease groups: thalassemia, hemophilia, immune thrombocytopenia (ITP), and sickle cell disease (SCD). Methods: Databases (MEDLINE, Embase, HaPI, CINAHL, and PsycTESTS) were searched to identify publications that either validated or used PROMs as an outcome measure in the four disease groups. Articles were excluded when <30% of the population was pediatric (<18 years), when the study setting was inpatient, when the tool had not been validated, or when the article did not report the use of a PROM for monitoring symptoms or HRQoL. Notably, hemophilia records published prior to 2016 were not screened as a systematic review by Limperg et al. (2017) identified validated PROMs in the pediatric hemophilia population and was used to include relevant articles. Results: The search identified 1176 unique records, with 902 records remaining for title and abstract screening after removal of 274 hemophilia articles published prior to the systematic review. Including hemophilia records identified from the 2017 review, 217 articles met inclusion criteria incorporating 107 generic and 20 disease-specific PROMs. Of the generic tools, the most frequent categories identified include psychological well-being (26 tools), general quality of life (19 tools), and family impact (19 tools). The most frequently used tool was the PedsQL 4.0 Generic Core Scales (66 studies), appearing 33 times in SCD, 25 times in thalassemia, 5 times in ITP, and 3 times in hemophilia. Other commonly used generic tools include the Short Form Health Survey, Child Health Questionnaire, PROMIS Health Measures, and Child Behaviour Checklist (Table). Disease-specific tools identified in the review include the PedsQL SCD Module, Kids ITP Tool, Haemo-QoL, CHO-KLAT, and TranQol (Table). In addition, 10 studies reported on pain diaries and 9 of these studies were SCD focused, the other being hemophilia focused. Conclusion: This systematic review identified several generic and disease-specific PROMs that have been used in pediatric non-malignant hematology. Although generic tools have been used more frequently, many disease-specific tools have been validated and are available for use in the clinical environment. We are currently conducting focus groups with patients, parents, and clinicians to determine the optimal choice of tools for monitoring symptoms and HRQoL in the pediatric non-malignant clinical environment. Disclosures No relevant conflicts of interest to declare.

Patient-Reported Outcome Measures in Symptomatic, Non–Limb-Threatening Peripheral Artery Disease: A State-of-the-Art Review

2021 ◽  
Author(s):  
Jennifer A. Rymer ◽  
Dennis Narcisse ◽  
Michael Cosiano ◽  
John Tanaka ◽  
Mary M. McDermott ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Outcome Measures ◽  
Peripheral Artery Disease ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Peripheral Artery ◽  
Patient Reported ◽  
Artery Disease ◽  
Disease Specific

Patient-reported outcome measures (PROMs) are health outcomes directly reported by the patient that can be used to measure the effect of disease and treatments on patient perceived well-being. This review summarizes current evidence regarding the validation of PROMs in people with symptomatic, nonlimb-threatening peripheral artery disease. A literature search was conducted to identify studies of symptomatic peripheral artery disease without limb-threatening ischemia that included PROMs and had sample sizes ≥25. PROMs were summarized along a continuum of validation using classical test theory framework and according to whether they fulfilled defined criteria for (1) content validity; (2) psychometric validation; and (3) further validation evidence base expansion. Of 2198 articles identified, 157 (7.1%) met inclusion criteria. Twenty-four PROMs in patients with symptomatic peripheral artery disease were reviewed. Among disease-specific PROMs, 8 of 15 had excellent reliability as measured by a Cronbach alpha ≥0.80. Based on established criteria for PROM responsiveness, 6 of 15 disease-specific PROMs demonstrated excellent sensitivity to change. Of these, the disease-specific peripheral artery questionnaire, vascular quality of life questionnaire, and walking impairment questionnaire met criteria for validation at each stage of the continuum. For generic (nondisease specific) PROMs, the European Quality of Life 5-Dimension and SF-36 had the most extensive evidence of validation. Evidence from this review can inform selection of PROMs aligned with scientific and clinical goals, given the variable degree of validation and potential complementary nature of the measures.

scholarly journals Non-disease specific patient-reported outcome measures of health-related quality of life in juvenile idiopathic arthritis: a systematic review of current research and practice

2021 ◽  
Author(s):  
Justyna Młyńczyk ◽  
Paweł Abramowicz ◽  
Maciej K. Stawicki ◽  
Jerzy Konstantynowicz
Keyword(s):  
Quality Of Life ◽  
Juvenile Idiopathic Arthritis ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Health Related Quality ◽  
Patient Reported ◽  
Related Quality ◽  
Health Related ◽  
Disease Specific

AbstractJuvenile idiopathic arthritis (JIA), as a chronic condition, is associated with symptoms negatively impacting health-related quality of life (HRQL). Regarding growing interest in the implementation of the patient-reported outcome measures (PROMs), we aimed to review the non-disease specific PROMs addressing HRQL assessment, potentially useful in the clinical care of JIA and daily practice. A systematic literature search was conducted using MEDLINE/PubMed, Google Scholar, Scopus and Embase databases (1990 to 2021), with a focus on the recent 5-years period. Entry keywords included the terms: “children”, “adolescents”, “JIA”, “chronic diseases”, “HRQL”, “PROMs” and wordings for the specific tools. Several available PROMs intended to measure HRQL, non-specific to JIA, were identified. The presented outcomes differed in psychometric properties, yet all were feasible in assessing HRQL in healthy children and those with chronic diseases. Both EQ-5D-Y and PedsQL have already been tested in JIA, showing relevant reliability, validity, and similar efficiency as disease-specific measurements. For PROMIS® PGH-7 and PGH-7 + 2, such validation and cross-cultural adaptation need to be performed. Considering the future directions in pediatric rheumatology, the large-scale implementation of PROMIS® PGH-7 and PGH-7 + 2 in JIA offers a particularly valuable opportunity. The PROMs reflect the patient perception of the chronic disease and allow to understand child’s opinions. The PROMs may provide an important element of the holistic medical care of patients with JIA and a standardized tool for clinical outcomes, monitoring disease severity and response to treatment.

scholarly journals Systematic Review of the Use of Patient Reported Outcome Measures in Studies of Electively-Managed Hand Conditions

2019 ◽  
Vol 24 (03) ◽  
pp. 329-341
Author(s):  
Hawys Lloyd-Hughes ◽  
Luke Geoghegan ◽  
Jeremy Rodrigues ◽  
Michele Peters ◽  
David Beard ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Outcome Measures ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Inclusion Criteria ◽  
Site Specific ◽  
Patient Reported ◽  
Generic Quality ◽  
Disease Specific

Background: Electively-managed conditions account for over 100 000 inpatient surgeries a year in the English National Health Service alone, with further procedures in other regions of the UK, or performed on an outpatient basis. To quality assure this care and to conduct research, effective outcome measurement is critical. Traditional surgeon-centric outcome measures correlate poorly with hand function and are seldom important to patients. There has been an advent in the use of patient reported outcome measures (PROMs) in hand surgery although consensus of PROM choice appears to be lacking. This systematic review aimed to describe the use of relevant PROMs in clinical research of electively-managed hand conditions. Methods: A PRISMA-compliant methodology was used. A bespoke search strategy was developed in conjunction with a search strategist, and applied to Medline, EMBASE, CINAHL and PSYCHINFO from 1992 to June 2017. Pre-specified stepwise inclusion criteria were used to identify studies describing adult patients undergoing treatment for electively-managed hand conditions distal to the distal radius, with clinical outcomes measured using one or more PROMs. Results: Of 4554 results, 834 met inclusion criteria. PROMs identified included 9 disease-specific, 8 site-specific and 4 generic quality of life measures. Across all, the Disabilities of the Arm, Shoulder and Hand (DASH) was the most commonly used (overall frequency 41.0%). The most commonly reported disease-specific measure was the Boston Carpal Tunnel Questionnaire (overall frequency 23.0%). The most commonly reported generic quality of life of measure was the SF-36 (overall frequency 4%). Time-analysis demonstrated predominance of site-specific PROMs since the year 2000. Conclusions: Various PROMs have been used to study electively-managed hand conditions, with site-specific PROMs most popular. However, there appears to be limited consensus on choices. A future systematic evaluation of the published psychometric properties of identified PROMs may inform standardisation of measurement.

scholarly journals Characteristics and Patient-reported Outcomes Associated to Dropout in Severely Affected Oncological Patients

2020 ◽  
Author(s):  
Pimrapat Gebert ◽  
Daniel Schindel ◽  
Johann Frick ◽  
Liane Schenk ◽  
Ulrike Grittner
Keyword(s):  
Quality Of Life ◽  
Social Support ◽  
Cancer Patients ◽  
Outcome Measures ◽  
Patient Reported Outcomes ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Drop Out ◽  
Patient Reported

Abstract BackgroundPatient-reported outcome measures are commonly used and are surrogates for clinical outcomes in cancer research. In the research setting of very severe diseases such as cancer, it is difficult to avoid the problem of incomplete questionnaires from drop-out or missing data due to patients who deceased during observation period. We aimed to explore patient characteristics and patient-reported outcomes associated with the time-to-dropout. MethodsIn the Oncological Social Care Project (OSCAR) study the condition of participants was assessed four times within 12 months (t0: baseline, t1: 3 months, t2: 6 months, t3:12 months) by validated patient-reported outcome measures. We performed competing-risks regression based on Fine and Gray’s proportional sub-distribution hazards model for exploring factors associated with time-to-dropout. Death was considered a competing risk. ResultsThree hundred sixty-two participants were analyzed in the study. 193 (53.3%) completed follow-up at 12 months, 67 (18.5%) patients dropped out, and 102 patients (28.2%) died during the study period. Poor subjective social support was related to higher risk for drop-out (SHR=2.10; 95%CI: 1.01 – 4.35). Lower values in quality of life (EORTC QLQ-C30) were related to drop-out and death. The subscales global health status/QoL, role functioning, physical functioning, and fatigue symptom were key characteristics associated with drop-out.ConclusionSeverely affected cancer patients with poor social support and poor quality of life seem more likely to drop out of studies compared to patients with higher levels of social support and quality of life. This should be considered when planning studies assessing cancer patients. Methods to monitor drop-outs timely and handle missing outcomes might be used. Results of such studies have to be interpreted with caution in light of the particular drop-out mechanisms.

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