Reliable Patient-reported Outcome Measure Research Articles

Development, validity and reliability of the Italian version of the Copenhagen neck functional disability scale

BackgroundValid and reliable patient-reported outcome measures support health professionals in evaluating the results of clinical research and practice. The Copenhagen Neck Functional Disability Scale (CNFDS) has shown promising measurement properties to measure disability in patients with neck pain, but an Italian version of this questionnaire is not available. The objective of this study was to cross-culturally adapt the CNFDS into Italian (CNFDS-I), and to assess its validity and reliability in patients with neck pain.MethodsThe CNFDS-I was developed according to well-established guidelines for cross-cultural adaptation of patient-reported outcome measures. A cross-sectional clinimetric study was conducted to evaluate its validity and reliability. Patients with chronic neck pain (pain > 3 months) participated in this study. The following measurement properties (defined by the COSMIN initiative) were assessed: structural validity (exploratory factor analysis), internal consistency (Cronbach’s α), construct validity [by testing hypotheses on expected correlations with the Neck Disability Index (NDI), the Neck Bournemouth Questionnaire (NBQ), and pain Visual Analogue Scale (VAS)]. Test-retest reliability [Intraclass Correlation Coefficient for agreement (ICCagreement)], and measurement error [Smallest Detectable Change (SDC)] were also assessed in 50 clinically stable patients. Floor/ceiling effects and acceptability were calculated.ResultsOne-hundred and sixty-two patients (mean age = 47.9 ± 14.5 years, 70% female) were included. The CNFDS-I exhibited sufficient unidimensionality (one factor explained 83% of the variability) and internal consistency (α = 0.83). Construct validity was sufficient as all correlations with the other questionnaires were as expected (r = 0.846 with NDI, r = 0.708 with NBQ, r = 0.570 with VAS). Test-retest reliability was excellent (ICCagreement = 0.99, 95% CI from 0.995 to 0.999), while measurement error was equal to 8.31 scale points (27% scale range). No floor/ceiling effects were detected. The average time for filling the questionnaire was two minutes.ConclusionsThe CNFDS-I proved to be a valid and reliable outcome measure to assess disability in patients with chronic neck pain. Head-to-head comparison studies on the CNFDS-I measurement properties against other disability measures for neck pain (e.g. NDI and NBQ) are required to determine the relative merits of these different measures.

INFLAMMATORY MYOPATHIES: P.159Feasibility and validation of modified oculobulbar facial respiratory score (mOBFRS) in sporadic inclusion body myositis

Sporadic inclusion body myositis (sIBM) is a neurodegenerative disorder that results in progressive limb muscle weakness with bulbar and respiratory involvement. The leading cause of morbidity and mortality are secondary to bulbar dysfunction. The IBMFRS is a reliable patient reported outcome measure used in sIBM, however, we lack objective scales that can quantitate deterioration in bulbar and respiratory functions. We are conducting a prospective study to test the feasibility of using, and validate the use of the modified oculobulbar facial respiratory score (mOBFRS) in sIBM. The mOBFRS is validated in immune myasthenia gravis. We will evaluate if changes in mOBFRS in sIBM: 1) can be reliably measured on a serial basis; 2) correlate with scores of the existing subjective IBM Functional Rating Scale (IBMFRS) and objective Bulbar Rating Scale (BRS); and 3) can be used as a potential outcome measure in clinical trials. Subjects are tested at baseline, month 6 and month 12. Interim analysis was done using a linear mixed effect model to assess the BRS compared with mOBFRS as fixed effect and a random intercept to account for the with-in subject correlation. Pearson's correlation was calculated between swallow time and subjective swallow ability at each visit. Within the first 12 months 36 subjects have completed visit one, 19 completed month 6, and 4 completed month 12. The mOBFRS score is significantly associated with the BRS along all three visits (p< 0.0001). One score change of mOBFRS leads to an average of 0.39 point change in the BRS. The IBMFRS swallow ability question was compared to the mOBFRS timed swallow test which showed no correlation at any of the three visits. Conducting the mOBFRS in a clinic setting has proved to be feasible. mOBFRS is trending to be a useful measure of bulbar function but the study is ongoing.

Reliable patient-reported outcome measure and survivorship of UKA for primary spontaneous osteonecrosis.

Primary Spontaneous osteonecrosis of the knee (SPONK) was a result of a subchondral insufficiency fracture based on histopathological examinations. There were few studies examining patients who underwent unicompartmental knee arthroplasty (UKA) for the treatment of primary SPONK. The aim of this study was to investigate (1) patient-reported outcome measure (PROM), (2) survivorship of revision as end point and (3) survivorship of complication as end point in patients with primary SPONK. The clinical examinations of a consecutive series of 61 medial UKAs for primary SPONK of the medial femoral condyle from 2008 to 2012 were evaluated retrospectively at our institution. There were 18 males and 43 females with a mean age of 73.7years (60-91). In all patients, preoperative radiographs were analyzed according to the stage of primary SPONK. We conducted Kaplan-Meier survival analyses using revision and complications for any reasons as the end point. Mean follow-up was 6.6years (range 6-10). UKA using Physica ZUK (LIMA Corporate. UD, Italy) for SPONK improved patients' 2011 Knee Society symptom score, patient satisfaction, patient activities, EQ-5D and postoperative ranges of motion compared with their preoperative status (P < 0.01). Revision surgery was required in one knee (1.6%) due to postoperative fracture of the medial tibial plateau after a fall that occurred 6months postoperatively. The projected rate of survivorship of UKA was 90.4% at 10years (95% confidence interval 0.80-1). The projected rate of survivorship with complication at end point was 87.7% at 10years (95% confidence interval 0.76-0.99). The present study demonstrated that primary spontaneous osteonecrosis of the knee (SPONK) can be successfully be treated with UKA at a mean follow-up of 6.6years. II.

Chronic fatigue syndrome and quality of life.

Chronic fatigue syndrome (CFS), also known as myalgic encephalomyelitis (ME), is a challenging long-term condition (LTC) with complex and fluctuating symptoms. It is heterogeneous in presentation without diagnostic indicators; therefore, in health care encounters, insight must be gained from the patient’s perspective. One indicator of impact can be gained by measuring quality of life (QoL). By applying a patient-reported outcome measure (PROM), professionals can gather insights with direct relevance to the patient questioned. Such a tool can act therapeutically tool to promote holistic and individualized professional interventions and interval measurement can inform commissioning of specialist services. Standard practice appears not fully to capture the experience of CFS, while a search of the literature turned up QoL patient-reported outcome tools, but failed to reveal a CFS/ME-specific measure. The author explores a valid and reliable PROM that can monitor change and evaluate the UK National Institute of Clinical Excellence rehabilitation program, as delivered by specialist National Health Service units. An alternative, the World Health Organization’s quality-of life instrument (WHOQoL)-Bref26, is reviewed for relevance to the condition, measuring treatment outcomes and the wider debate of measuring QoL in LTCs.

PRM87 - Psychometric Validation of the 1-Month Recall Uterine Fibroid Symptom and Health-Related Quality-of-Life Questionnaire

To evaluate psychometric characteristics of the 1-month recall Uterine Fibroid Symptom and Health-related Quality-of-Life (UFS-QOL) questionnaire, a patient-reported outcome measure of uterine fibroid (UF)-specific symptom severity and health-related quality of life (HRQoL). VENUS I and II were phase 3, randomized, placebo-controlled trials in women with abnormal uterine bleeding caused by UF. Assessments included: 1-month UFS-QOL (completed at baseline [V1] and after 12 weeks of treatment [V2]); daily diary of menstrual bleeding; and Patient Global Impression of Improvement (PGI-I) scale rating change in menstrual/vaginal bleeding at V2 (VENUS II only). Internal consistency, validity, and responsiveness of the UFS-QOL were evaluated. Confirmatory factor analyses assessed model fit for a Revised Activities subscale of the UFS-QOL. 589 women were randomized; 586 completed the UFS-QOL at V1; 483 had data at V2. Mean age was 41 years; 67.4% were black (VENUS II). All UFS-QOL scales showed strong internal consistency with high Cronbach’s coefficient alphas at V1 (VENUS I and II: range, 0.76-0.96 and 0.79-0.96, respectively) and V2 (VENUS I and II: range, 0.81-0.98 and 0.85-0.98, respectively). Validity: at V2, all scales were significantly correlated with number of heavy bleeding days and PGI-I response categories (all p<0.0001). On each scale, women who achieved absence of bleeding or controlled bleeding scored significantly (p<0.001) better than those who did not. Responsiveness: anchor-based analyses showed that women responding “very much better” on the PGI-I had significantly (p<0.05) greater improvements on almost all scales versus each of the other six response categories. Confirmatory factor analyses confirmed Revised Activities subscale model fit; comparative fit index values exceeded 0.95 in both trials. The 1-month UFS-QOL, including the Revised Activities subscale, is a valid and reliable patient-reported outcome measure for assessing UF symptoms and HRQoL.

Development and validation of the mastocytosis activity score.

Mastocytosis is a heterogeneous disease characterized by a clonal expansion of mast cells in various organs. The vast majority of patients suffer from signs and symptoms caused by mediator release from mast cells. Although the disease burden is high, there is currently no specific and validated instrument to measure and monitor signs and symptoms in patients with mastocytosis. To develop and validate a disease-specific tool to measure and monitor the activity of signs and symptoms in patients with mastocytosis, the Mastocytosis Activity Score (MAS). Nineteen potential MAS items were developed in a combined approach consisting of semi-structured patient interviews, expert input and literature research. Item selection was performed by impact analysis with 76 patients followed by a review for face validity. The resulting MAS was tested for validity, reliability and influence factors. In parallel, a US American English version of the MAS was developed. A total of 68 mastocytosis patients took part in the MAS validation study. The final 9-item MAS was found to have a three-domain structure ("skin," "gastrointestinal tract" and "other"), a valid total score and an excellent test-retest reliability. Multiple regression analysis revealed that disease duration, age or gender is not a significant determinant of the MAS results. The MAS is a disease-specific, valid and reliable patient-reported outcome measure for adult patients with cutaneous and indolent systemic mastocytosis. It may serve as a valuable tool to measure and monitor mastocytosis activity, both, in clinical trials and in routine care.

Psychometric properties of the PROMIS short form measures in a U.S. cohort of 961 patients with chronic hepatitis C prescribed direct acting antiviral therapy.

To better understand symptoms experienced by patients infected with chronic hepatitis C virus (HCV), valid and reliable patient-reported outcome (PRO) measures are needed. To assess the reliability and validity of 10 patient-reported outcomes measurement information system (PROMIS) measures and the Headache Impact Test-6 (HIT-6) in a large national sample of patients with HCV. Pre-treatment data from 961 patients with HCV starting direct acting antiviral therapy at 11 U.S. liver centers were analyzed. Internal reliability was evaluated using Cronbach's alpha coefficient; frequency distributions were examined for floor and ceiling effects; structural validity was investigated via item-response-theory models; convergent validity was evaluated using correlations with theoretically-similar items from the HCV-PRO and memorial symptom assessment scale (MSAS); and known-groups validity was investigated by observing PRO differences by liver disease status and number of comorbidities. The HIT-6 and the majority of the PROMIS measures yielded excellent reliability (alphas≥0.87). Ceiling effects were infrequent (<4%), while 30%-59% of patients reported no symptoms (floor effects). The data supported structural validity of the HIT-6 and most PROMIS measures. The PROMIS measures showed moderate to strong correlations with theoretically-similar items from the HCV-PRO and MSAS (0.39-0.77). Trends were observed between worse PRO scores and advanced cirrhosis and greater number of comorbidities, lending support for known-groups validity. The psychometric properties of the HIT-6 and PROMIS measures performed satisfactorily in this large cohort of patients with HCV starting direct acting antiviral therapy. Opportunities exist for further refinement of these PROs. Evaluation of performance over time and in under-represented subgroups is needed.

Are the patient-rated wrist evaluation (PRWE) and the disabilities of the arm, shoulder and hand (DASH) questionnaire used in distal radial fractures truly valid and reliable?

ObjectivesThe patient-rated wrist evaluation (PRWE) and the Disabilities of the Arm, Shoulder and Hand (DASH) questionnaire are patient-reported outcome measures (PROMs) used for clinical and research purposes. Methodological high-quality clinimetric studies that determine the measurement properties of these PROMs when used in patients with a distal radial fracture are lacking. This study aimed to validate the PRWE and DASH in Dutch patients with a displaced distal radial fracture (DRF).MethodsThe intraclass correlation coefficient (ICC) was used for test-retest reliability, between PROMs completed twice with a two-week interval at six to eight months after DRF. Internal consistency was determined using Cronbach’s α for the dimensions found in the factor analysis. The measurement error was expressed by the smallest detectable change (SDC). A semi-structured interview was conducted between eight and 12 weeks after DRF to assess the content validity.ResultsA total of 119 patients (mean age 58 years (sd 15)), 74% female, completed PROMs at a mean time of six months (sd 1) post-fracture. One overall meaningful dimension was found for the PRWE and the DASH. Internal consistency was excellent for both PROMs (Cronbach’s α 0.96 (PRWE) and 0.97 (DASH)). Test-retest reliability was good for the PRWE (ICC 0.87) and excellent for the DASH (ICC 0.91). The SDC was 20 for the PRWE and 14 for the DASH. No floor or ceiling effects were found. The content validity was good for both questionnaires.ConclusionThe PRWE and DASH are valid and reliable PROMs in assessing function and disability in Dutch patients with a displaced DRF. However, due to the high SDC, the PRWE and DASH are less useful for individual patients with a distal radial fracture in clinical practice.Cite this article: Y. V. Kleinlugtenbelt, R. G. Krol, M. Bhandari, J. C. Goslings, R. W. Poolman, V. A. B. Scholtes. Are the patient-rated wrist evaluation (PRWE) and the disabilities of the arm, shoulder and hand (DASH) questionnaire used in distal radial fractures truly valid and reliable? Bone Joint Res 2018;7:36–45. DOI: 10.1302/2046-3758.71.BJR-2017-0081.R1.

Assessing patient-reported outcomes in asthma and COPD patients: which can be recommended in clinical practice?

There is a clear need for simple and reliable patient-reported outcome measures for chronic obstructive pulmonary disease (COPD) and asthma in daily practice. The purpose of this review is to facilitate the choice for clinicians of patient-reported outcomes which they can use in their daily practice. More than 50 patient-reported outcome measures for asthma and COPD exist and clinicians are often left confused on which to use. Four tools (two for asthma and two for COPD) can be suggested based on validity/reliability, responsiveness, practicality and are particularly convenient in terms of time to measure. On the basis of ample evidence, the COPD assessment test and the clinical COPD questionnaire for COPD and asthma control questionnaire and the asthma control test for asthma can be recommended for use in both primary care and other clinical settings. A simple guide figured as smiley faces has been designed to assist physicians to easily select the appropriate measure. With the current direction of thinking into treatable traits, targeted measures that evaluate the upper airways like the control of allergic rhinitis and asthma test may also be more used in the future.

Relation between the Disability of the Arm, Shoulder and Hand Score and Muscle Strength in Post-Cardiac Surgery Patients.

Background: The Disabilities of the Arm, Shoulder, and Hand (DASH) questionnaire is a valid and reliable patient-reported outcome measure. DASH can be assessed by self-reported upper extremity disability and symptoms. We aimed to examine the relationship between the physiological outcome of muscle strength and the DASH score after cardiac surgery. Methods: This cross-sectional study assessed 50 consecutive cardiac patients that were undergoing cardiac surgery. Physiological outcomes of handgrip strength and knee extensor muscle strength and the DASH score were measured at one month after cardiac surgery and were assessed. Results were analyzed using Spearman correlation coefficients. Results: The final analysis comprised 43 patients (men: 32, women: 11; age: 62.1 ± 9.1 years; body mass index: 22.1 ± 4.7 kg/m2; left ventricular ejection fraction: 53.5 ± 13.7%). Respective handgrip strength, knee extensor muscle strength, and DASH score were 27.4 ± 8.3 kgf, 1.6 ± 0.4 Nm/kg, and 13.3 ± 12.3, respectively. The DASH score correlated negatively with handgrip strength (r = −0.38, p = 0.01) and with knee extensor muscle strength (r = −0.32, p = 0.04). Conclusion: Physiological outcomes of both handgrip strength and knee extensor muscle strength correlated negatively with the DASH score. The DASH score appears to be a valuable tool with which to assess cardiac patients with poor physiological outcomes, particularly handgrip strength as a measure of upper extremity function, which is probably easier to follow over time than lower extremity function after patients complete cardiac rehabilitation.

Responsiveness and minimal important change of the Norwegian version of the Disabilities of the Arm, Shoulder and Hand questionnaire (DASH) in patients with subacromial pain syndrome

BackgroundThe Disabilities of the Arm, Shoulder, and Hand questionnaire (DASH) is a valid and reliable patient-reported outcome measure (PROM). It was designed to measure physical disability and symptoms in patients with musculoskeletal disorders of the upper extremity, and is one of the most commonly used PROMs for patients with shoulder pain. The aim of this study was to examine responsiveness, the smallest detectable change (SDC) and the minimal important change (MIC) of the DASH, in line with international (COSMIN) recommendations.MethodsThe study sample consisted of 50 patients with subacromial pain syndrome, undergoing physical therapy for 3–4 months. Responsiveness to change was examined by calculating area under the receiver operating characteristic curves (AUC) and testing a priori-formulated hypothesis regarding correlations with changes in other instruments that measuring the same construct. The SDC was calculated using a test re-test protocol, and the MIC was calculated by the anchor-based MIC distribution. MIC values for patients with low and high baseline scores were also calculated.ResultsDASH appeared to be responsive, as it was able to distinguish patients who reported to be improved from those unchanged (AUC 0.77). All of the hypotheses were accepted. The SDC was 11.8, and the MIC was 4.4.ConclusionThis study shows that the Norwegian version of the DASH has good responsiveness to change and may thus be recommended to measure outcome in patients with shoulder pain in Norway.

Surgical Management of Gout in the Foot and Ankle A Systematic Review.

The surgical management of tophaceous gout has been well documented in the literature, including its effect on foot pain and disability. To date, there have been no systematic reviews assessing the quality of the literature relating to the operative management of tophaceous gout and the outcomes in the foot and ankle. The following electronic databases were searched (1980-2014): Allied and Complementary Medicine, Cumulative Index to Nursing and Allied Health Literature, MEDLINE, Scopus, and the Cochrane Library. The articles identified were published in English and included adult participants (age ≥18 years) with diagnosed gout and surgical intervention to the foot and ankle. The Downs and Black Quality Index was modified to assess the quality of the articles being reviewed. Six articles were reviewed and were of moderate quality (mean quality score of 71%). Surgical management was conducted on men (88%) with a mean age of 52 years. There was wide variation in the types of surgical procedures performed, with 28% of studies reporting surgery to the first metatarsophalangeal joint. Most studies were retrospective. A wide range of outcome measures were reported: foot pain, function, preoperative and postoperative activity levels, monitoring of uric acid levels, and patient satisfaction after surgery. The review demonstrated a limited number of good-quality studies. Several surgical procedures for the foot and ankle in people with chronic tophaceous gout were reported. Future studies should include prospective observational studies using validated and reliable patient-reported outcome measures.

Validity and reliability of the Chronic Oral Mucosal Diseases Questionnaire in a UK population.

The objective of this study was to investigate the validity and reliability of a the Chronic Oral Mucosal Diseases Questionnaire in a UK population Two hundred patients with chronic oral mucosal disease (oral lichen planus, recurrent aphthous stomatitis, mucous membrane pemphigoid, pemphigus vulgaris) were enrolled in this study from the Oral Medicine Department of University College London Hospitals Trust (UCLHT) Eastman Dental Hospital. Individuals were interviewed using Oral Health Impact Profile (OHIP-14), Visual Analogue Scale (VAS) and Chronic Oral Mucosal Diseases Questionnaire (COMDQ), and the construct validity and internal reliability were examined. Of the 200 study participants, 100 respondents had oral lichen planus, 42 had recurrent aphthous stomatitis and 58 had vesiculobullous conditions (mucous membrane pemphigoid or pemphigus vulgaris). With regard to construct validity, a moderate to good degree of convergent validity was found between OHIP-14 and VAS and most subscales and the total COMDQ score except the patient support subscale of COMDQ (0.21-0.37). COMDQ is a valid and reliable patient-reported outcome measure for patients with chronic oral mucosal diseases in a UK population. It can be considered a valuable instrument in both clinical practice and in oral medicine research.

A Patient-Reported Outcome Measure for Symptoms and Symptom Burden of Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS)

Background: Patient report of disease- and treatment-related symptom burden in acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) is scarce. Symptom burden is the combined impact of disease and treatment symptoms on daily functioning. Lack of recognition and monitoring of symptoms and symptom burden can lead to inadequate management and possible treatment non-adherence.Aims: Our aim is to develop a short, valid, reliable patient-reported outcome measure of symptoms and symptom burden experienced by AML and MDS patients and to determine the validity of a single measure for research and practice.Methods: After obtaining IRB approval, patients with AML (N=152) and MDS (N=97) were recruited to this cross-sectional study. Patients rated the 13 core symptom items (pain, fatigue, nausea, disturbed sleep, distress, shortness of breath, trouble remembering, lack of appetite, drowsiness, dry mouth, sadness, vomiting, and numbness and tingling), 6 proposed AML/MDS symptom items (muscle weakness, malaise, fever, headache, diarrhea, skin problems), and 6 interference items (general activities, mood, work, relations with others, walking, and enjoyment of life) of the MD Anderson Symptom Inventory (MDASI) on 0-to-10 scales (0 = not present or no interference; 10 = as bad as can be imagined or complete interference) twice 1-2 days apart. Clinical and demographic information was collected from medical records and analyzed using descriptive statistics. Means of the symptom and interference ratings for the AML and MDS patients were compared using T-tests. Standard psychometric techniques were used to determine the reliability, stability, and validity of the instrument in patients with AML and MDS.Results: All MDS patients were outpatients while 75 of the AML patients were inpatients and 77 were outpatients. The AML and MDS patients had been diagnosed a mean of 13.8 months (standard deviation [SD]=23.9) and 30.5 months (SD=32.4) respectively. The mean (Mn) symptom and interference ratings respectively for the AML inpatients (Mn=2.8, SD=1.6; Mn=4.0, SD=2.4) were significantly higher than for the AML outpatients (Mn=1.8, SD=1.4, p<0.01; Mn=2.7, SD=2.3, p<0.01) or MDS patients (Mn=1.9, SD=1.5, p<0.01; Mn=2.7, SD=2.5, p<0.01). The mean ratings for the 5 most severe symptom means for AML and MDS patients respectively were: fatigue (Mn=4.0, SD=2.8; Mn=4.0, SD=2.5; p =0.97), disturbed sleep (Mn=3.3, SD=3.2; Mn=2.7, SD=3.3; P =0.19), drowsiness (Mn=3.0, SD=2.8; Mn=2.8, SD=3.1; P =0.70), muscle weakness (Mn=2.9, SD=2.8; Mn=2.9, SD=3.0; P =0.91), dry mouth for AML patients (Mn=3.4, SD=3.2; Mn=2.2,SD=2.8; p<0.01), and shortness of breath for MDS patients (Mn=2.7, SD=2.8; 1.9, SD=2.3; P =0.02). Two of the 6 AML/MDS symptom items (fever and headache) were dropped because so few patients said they experienced the symptoms at more than a mild (0-4 rating) level (12% and 11% respectively). Both groups of patients endorsed similar symptoms, and none of the means of the 4 final AML/MDS symptoms were significantly different between the groups. Cronbach's reliability for all symptom items for AML and MDS respectively were 0.88 and 0.91 and for all interference items were 0.86 and 0.92. The test-retest reliability intra-class correlations were 0.85 for the core symptoms, 0.77 for AML/MDS symptoms, and 0.84 for the interference items. The MDASI-AML/MDS can be completed by patients in less than 5 minutes.Conclusion/Summary: Lack of recognition of symptoms experienced by patients with AML and MDS can lead to inadequate management of symptoms, interfere with the ability of patients to function and enjoy life, and impact the tolerability of and adherence to treatment regimens. While the symptoms experienced by the two groups had some variation in severity, a similar group of symptoms were the most common and relevant for both groups of patients, and the same measure was appropriate for both groups. The MDASI-AML/MDS is a brief, easily-completed, and validated measure of symptom burden for patients with AML and MDS that can be used for accurate and consistent monitoring of symptoms by clinicians and researchers. DisclosuresWilliams:Amgen: Consultancy; Novartis: Research Funding. Cortes:Pfizer: Consultancy, Research Funding; BMS: Consultancy, Research Funding; Novartis: Consultancy, Research Funding; BerGenBio AS: Research Funding; Teva: Research Funding; Ariad: Consultancy, Research Funding; Astellas: Consultancy, Research Funding; Ambit: Consultancy, Research Funding; Arog: Research Funding; Celator: Research Funding; Jenssen: Consultancy. Mendoza:Amgen Inc.: Consultancy. Shi:Amgen Inc.: Consultancy. Cleeland:Amgen Inc.: Consultancy.

A novel method for expediting the development of patient-reported outcome measures and an evaluation of its performance via simulation.

BackgroundDeveloping valid and reliable patient-reported outcome measures (PROMs) is a critical step in promoting patient-centered health care, a national priority in the U.S. Small populations or rare diseases often pose difficulties in developing PROMs using traditional methods due to small samples.MethodsTo overcome the small sample size challenge while maintaining psychometric soundness, we propose an innovative Ordinal Bayesian Instrument Development (OBID) method that seamlessly integrates expert and participant data in a Bayesian item response theory (IRT) with a probit link model framework. Prior distributions obtained from expert data are imposed on the IRT model parameters and are updated with participants’ data. The efficiency of OBID is evaluated by comparing its performance to classical instrument development performance using actual and simulation data.Results and Discussion The overall performance of OBID (i.e., more reliable parameter estimates, smaller mean squared errors (MSEs) and higher predictive validity) is superior to that of classical approaches when the sample size is small (e.g. less than 100 subjects). Although OBID may exhibit larger bias, it reduces the MSEs by decreasing variances. Results also closely align with recommendations in the current literature that six subject experts will be sufficient for establishing content validity evidence. However, in the presence of highly biased experts, three experts will be adequate.ConclusionsThis study successfully demonstrated that the OBID approach is more efficient than the classical approach when the sample size is small. OBID promises an efficient and reliable method for researchers and clinicians in future PROMs development for small populations or rare diseases.Electronic supplementary materialThe online version of this article (doi:10.1186/s12874-015-0071-5) contains supplementary material, which is available to authorized users.

Patient Perspectives in OMERACT Provide an Anchor for Future Metric Development and Improved Approaches to Healthcare Delivery in Connective Tissue Disease Related Interstitial Lung Disease (CTD-ILD).

The impact and natural history of connective tissue disease related interstitial lung disease (CTD-ILD) are poorly understood; and have not been previously described from the patient's perspective. This investigation sought insight into CTD-ILD from the patients' perspective to add to our knowledge of CTD-ILD, identify disease-specific areas of unmet need and gather potentially meaningful information towards development of disease-specific patient-reported outcome measures (PROMs). A mixed methods design incorporating patient focus groups (FGs) querying disease progression and life impact followed by questionnaires with items of importance generated by >250 ILD specialists were implemented among CTD-ILD patients with rheumatoid arthritis, idiopathic inflammatory myopathies, systemic sclerosis, and other CTD subtypes. FG data were analyzed through inductive analysis with five independent analysts, including a patient research partner. Questionnaires were analyzed through Fisher's Exact tests and hierarchal cluster analysis. Six multicenter FGs included 45 patients. Biophysiologic themes were cough and dyspnea, both pervasively impacting health related quality of life (HRQoL). Language indicating dyspnea was unexpected, unique and contextual. Psycho-social themes were Living with Uncertainty, Struggle over Self-Identity, and Self-Efficacy - with education and clinician communication strongly emphasised. All questionnaire items were rated 'moderately' to 'extremely' important with 10 items of highest importance identified by cluster analysis. Patients with CTD-ILD informed our understanding of symptoms and impact on HRQoL. Cough and dyspnea are central to the CTD-ILD experience. Initial FGs have provided disease-specific content, context and language essential for reliable PROM development with questionnaires adding value in recognition of patients' concerns.

PD14-07 PHASE I DEVELOPMENT OF A PATIENT REPORTED OUTCOME MEASURE FOR MALE URETHRAL STRICTURE DISEASE

You have accessJournal of UrologyTrauma/Reconstruction: Urethral Reconstruction (including Stricture) II1 Apr 2015PD14-07 PHASE I DEVELOPMENT OF A PATIENT REPORTED OUTCOME MEASURE FOR MALE URETHRAL STRICTURE DISEASE Benjamin Breyer, Todd Edwards, Donald Patrick, and Bryan Voelzke Benjamin BreyerBenjamin Breyer More articles by this author , Todd EdwardsTodd Edwards More articles by this author , Donald PatrickDonald Patrick More articles by this author , and Bryan VoelzkeBryan Voelzke More articles by this author View All Author Informationhttps://doi.org/10.1016/j.juro.2015.02.1336AboutPDF ToolsAdd to favoritesDownload CitationsTrack CitationsPermissionsReprints ShareFacebookTwitterLinked InEmail INTRODUCTION AND OBJECTIVES Patient reported outcome measures (PROMs) are important for assessing aspects of urethral stricture disease that are known only to the patient. PROM-based results are tied increasingly to health care funding and management. The availability of comprehensive, valid and reliable PROMs for urethral stricture is important. Our objective is to develop a urethral stricture-specific PROM to be used for assessing treatments for urethral stricture. Here we describe a Phase I study to elicit vital content related to men with a urethral stricture. METHODS We performed concept elicitation interviews with men with urethral strictures in the pre- and post-op setting. Qualitative interviews were stopped after concept saturation was reliably noted (n=16). Interviewees included 12 men with bulbar and 4 men with penile strictures. Median age was 51 (range 29-70). The cohort included Caucasian (13), Hispanic (1), African-American (1), and Asian (1) men. The interviews were audio-recorded and transcribed. Specific content regarding impaired urinary/sexual function and quality of life were excerpted and coded by investigators using a qualitative analysis program. We organized the content into domains by consensus. Existing PROMs with content relevant for urinary and sexual function were reviewed to incorporate legacy items. RESULTS We identified 35 potential items (13 legacy and 22 new) for possible PROM inclusion. Most content pertained to urinary impact (n=15), function (n=7) and symptoms (n=6). Seven items were related to sexual impact (n=4), symptoms (n=2) and function (n=1). Urinary content included emotional impact of urethral stricture including worry (n=4), embarrassment (n=2), and depression (n=1), as well as those related to changing daily activities to manage stricture symptoms (n=4) including “I quit doing things I like to do” and “I had to plan everything ahead.” Urinary function content included obstruction (n=4) and trouble aiming the stream (n=1). Urinary symptom content related to pain (n=3), nocturia (n=1) and urgency (n=1). Of the items related to sexual activity (n=7), most related to ejaculation (n=5), both weak (n=2) and unsatisfying (n=1), while pain was also elicited (n=2). CONCLUSIONS Using qualitative inductive methodology, we have obtained patient-centered data suitable for a comprehensive urethral stricture-specific PROM. Future studies will include cognitive interviews of the draft instrument and multi-center field testing to assess the instrument's measurement properties, including its sensitivity to detect change following treatment. © 2015 by American Urological Association Education and Research, Inc.FiguresReferencesRelatedDetails Volume 193Issue 4SApril 2015Page: e321 Advertisement Copyright & Permissions© 2015 by American Urological Association Education and Research, Inc.MetricsAuthor Information Benjamin Breyer More articles by this author Todd Edwards More articles by this author Donald Patrick More articles by this author Bryan Voelzke More articles by this author Expand All Advertisement Advertisement PDF downloadLoading ...

World Workshop on Oral Medicine VI: Patient-reported outcome measures and oral mucosal disease: current status and future direction

This systematic review aimed to (1) explore the patient-reported outcome measures (PROMs) currently used in the oral mucosal disease literature and report on the type and context of the use of these instruments and (2) provide a future direction for PROMs in Oral Medicine practice and research. A systematic review of published English-language articles relating to the use of PROMs in the oral mucosal diseases literature was performed in November2013. In total, 131 articles met the inclusion criteria; these articles addressed the following oral mucosal conditions: lichen planus (75); recurrent aphthous stomatitis (30); mucous membrane pemphigoid/pemphigus vulgaris (14); orofacial granulomatosis (1); and multiple oral mucosal diseases (11). The most commonly used instruments were visual analog scales (VAS) and the oral health impact profile (OHIP). Limited progress has been achieved with use of PROMs in Oral Medicine in the last few decades in both clinical practice and a research setting. With the engagement of allied medical disciplines in PROM usage and the promotion of PROMs by national health care bodies globally, advancement of PROMs is imperative for Oral Medicine. Exposure through the World Workshop on Oral Medicine (WWOM), along with potential involvement in the Core Outcome Measures in Effectiveness Trials (COMET) or other such initiatives, will enable worldwide collaboration to promote the development and utilization of valid and reliable PROMs in oral medicine, and improve patient care.

EDGE Task Force on Head and Neck Cancer Outcomes: A Systematic Review of Outcome Measures for Temporomandibular-related Dysfunction

Background: Patients with head and neck cancer (HNC) often experience significant postoperative limitations in temporomandibular joint (TMJ) function, facial pain, reduced nutritional intake, speech impairments, and compromised activities of daily living due to side effects of cancer treatment. Physical therapists treating these individuals must use valid and reliable patientreported outcome measures to quantify change related to physical therapy intervention for the TMJ. Purpose: As part of the activities of the Oncology Section EDGE Task Force on Head and Neck Cancer Outcomes, we report evidence-based recommendations for patient-reported outcome measures for individuals with HNC-related temporomandibular dysfunction (TMD). Methods: A systematic literature review of TMD-related patient-reported outcome measures that are clinically feasible and relevant to the HNC patient population was conducted using the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) model. Recommendations are based on the quality of psychometric properties, clinical utility, and previous use in HNC-related research. Twenty-two outcome measures were selected for review; 4 received a rating of 3, “recommended” for use in the HNC population. Conclusions: A variety of outcome measures have been reported in the literature for individuals with HNC-related TMD. Four measures, the Graded Chronic Pain Scale, 8 and 20-item Jaw Functional Limitation Scale and TMD Pain Screener, are recommended for clinical use by the researchers on this task force although it is important to note psychometric properties specific to the HNC population are lacking. Systematic Review Registration Number: PROSPERO CRD42013004898

Capturing Patient-Reported Outcome (PRO) Data Electronically: The Past, Present, and Promise of ePRO Measurement in Clinical Trials.

Patient-reported outcomes (PROs) are an important means of evaluating the treatment benefit of new medical products. It is recognized that PRO measures should be used when assessing concepts best known by the patient or best measured from the patient’s perspective. As a result, there is growing emphasis on well defined and reliable PRO measures. In addition, advances in technology have significantly increased electronic PRO (ePRO) data collection capabilities and options in clinical trials. The movement from paper-based to ePRO data capture has enhanced the integrity and accuracy of clinical trial data and is encouraged by regulators. A primary distinction in the types of ePRO platforms is between telephone-based interactive voice response systems and screen-based systems. Handheld touchscreen-based devices have become the mainstay for remote (i.e., off-site, unsupervised) PRO data collection in clinical trials. The conventional approach is to provide study subjects with a handheld device with a device-based proprietary software program. However, an emerging alternative for clinical trials is called bring your own device (BYOD). Leveraging study subjects’ own Internet-enabled mobile devices for remote PRO data collection (via a downloadable app or a Web-based data collection portal) has become possible due to the widespread use of personal smartphones and tablets. However, there are a number of scientific and operational issues that must be addressed before BYOD can be routinely considered as a practical alternative to conventional ePRO data collection methods. Nevertheless, the future for ePRO data collection is bright and the promise of BYOD opens a new chapter in its evolution.