Browsing by Subject "Patient-reported outcomes"
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Item Open Access A novel patient-reported outcome for paediatric localized scleroderma: A qualitative assessment of content validity.(The British journal of dermatology, 2019-09-09) Zigler, CK; Ardalan, K; Lane, S; Schollaert, KL; Torok, KSBACKGROUND:According to current standards, no existing patient-reported outcome (PRO) measures have high quality validity evidence for use with individuals diagnosed with paediatric localized scleroderma. This lack severely hinders patient-centred LS-focused research, including much needed clinical trials. OBJECTIVES:To develop a valid health-related quality of life measure for individuals with paediatric-localized scleroderma (LS) and to qualitatively evaluate its content validity using a patient-centred approach. METHODS:Previously collected qualitative data from youth with LS and their caregivers was used to develop items. The resulting item-set was administered in a clinical setting to participants aged 8-18 years old. Cognitive interviews were used to evaluate time to survey completion, readability/understanding of the items, appropriateness of the recall period, and construct representation. RESULTS:Seventeen children and adolescents with LS participated in the study. Interviews supported readability, understanding of the items, and appropriateness of the recall period in individuals >10 years old. Revisions were made to simplify the instructions and to be more inclusive of different subtypes of localized scleroderma. Three items were added to improve content representation. CONCLUSIONS:Content validity was supported by the patient-centred development process of the outcome measure and via direct feedback from individuals with LS and their families. Although an important first step, the resulting PRO, termed the "Localized Scleroderma Quality of Life Instrument", should be further evaluated in a larger sample before being implemented.Item Open Access Can PROMIS measures be used to create subgroups for patients seeking orthopaedic care?(Bone & joint open, 2021-07) George, Steven Z; Yan, Xiaofang; Luo, Sheng; Olson, Steven A; Reinke, Emily K; Bolognesi, Michael P; Horn, Maggie EAims
Patient-reported outcome measures have become an important part of routine care. The aim of this study was to determine if Patient-Reported Outcomes Measurement Information System (PROMIS) measures can be used to create patient subgroups for individuals seeking orthopaedic care.Methods
This was a cross-sectional study of patients from Duke University Department of Orthopaedic Surgery clinics (14 ambulatory and four hospital-based). There were two separate cohorts recruited by convenience sampling (i.e. patients were included in the analysis only if they completed PROMIS measures during a new patient visit). Cohort #1 (n = 12,141; December 2017 to December 2018,) included PROMIS short forms for eight domains (Physical Function, Pain Interference, Pain Intensity, Depression, Anxiety, Sleep Quality, Participation in Social Roles, and Fatigue) and Cohort #2 (n = 4,638; January 2019 to August 2019) included PROMIS Computer Adaptive Testing instruments for four domains (Physical Function, Pain Interference, Depression, and Sleep Quality). Cluster analysis (K-means method) empirically derived subgroups and subgroup differences in clinical and sociodemographic factors were identified with one-way analysis of variance.Results
Cluster analysis yielded four subgroups with similar clinical characteristics in Cohort #1 and #2. The subgroups were: 1) Normal Function: within normal limits in Physical Function, Pain Interference, Depression, and Sleep Quality; 2) Mild Impairment: mild deficits in Physical Function, Pain Interference, and Sleep Quality but with Depression within normal limits; 3) Impaired Function, Not Distressed: moderate deficits in Physical Function and Pain Interference, but within normal limits for Depression and Sleep Quality; and 4) Impaired Function, Distressed: moderate (Physical Function, Pain Interference, and Sleep Quality) and mild (Depression) deficits.Conclusion
These findings suggest orthopaedic patient subgroups differing in physical function, pain, and psychosocial distress can be created from as few as four different PROMIS measures. Longitudinal research is necessary to determine whether these subgroups have prognostic validity. Cite this article: Bone Jt Open 2021;2(7):493-502.Item Open Access Core outcomes set for research on the treatment of opioid use disorder (COS-OUD): the National Institute on Drug Abuse Clinical Trials Network protocol for an e-Delphi consensus study.(Trials, 2021-01-28) Karnik, Niranjan S; Campbell, Cynthia I; Curtis, Megan E; Fiellin, David A; Ghitza, Udi; Hefner, Kathryn; Hser, Yih-Ing; McHugh, R Kathryn; Murphy, Sean M; McPherson, Sterling M; Moran, Landhing; Mooney, Larissa J; Wu, Li-Tzy; Shmueli-Blumberg, Dikla; Shulman, Matisyahu; Schwartz, Robert P; Stephens, Kari A; Watkins, Katherine E; Marsden, JohnBackground
A lack of consensus on the optimal outcome measures to assess the efficacy and effectiveness of interventions for the treatment of opioid use disorder (OUD) has hampered the pooling of research data for evidence synthesis and clinical guidelines. A core outcome set (COS) is a minimum set of outcome measures that are recommended for all studies of a particular condition. The National Drug Abuse Treatment Clinical Trials Network (CTN) Core Outcome Set for OUD (COS-OUD) is a development study to identify core constructs, meaningful outcomes, and their optimal measurement for all efficacy and effectiveness studies of OUD treatment and service delivery.Methods/design
Overseen by an expert workgroup, a modified, stepwise, e-Delphi methodology will be used to gain consensus among a panel of clinical practitioners and researchers involved in the treatment of OUD, who are members of the CTN. Sequential rounds of anonymous, online questionnaires will be used to identify, rate the importance of, and refine a core outcome set. A consensus threshold will be achieved if at least 70% of the panel rate the measure as critical for inclusion in the COS-OUD. Where consensus is not reached or there are suggestions for new measures, these will be brought forward to a further round of review prior to a consensus meeting. Products from this study will be communicated via peer-reviewed scientific journals and conferences.Discussion
This initiative will develop a COS for OUD intervention trials, treatment studies, and service delivery and will support the pooling of research and clinical practice data and efforts to develop measurement-based care within the OUD treatment cascade.Trial registration
http://www.comet-initiative.org/Studies/Details/1579.Item Open Access Facets of physical function assessed by patient-reported outcome measures in oncology research.(Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation, 2024-04) Coles, Theresa; Fillipo, Rebecca; Plyler, Kate; Hernandez, Alexy; Henke, Debra M; Arizmendi, Cara; Cantrell, Sarah; Lagoo-Deenadayalan, Sandhya; Rory Goodwin, C; LeBlanc, Thomas W; Horodniceanu, Erica G; Bhatnagar, Vishal; Reeve, Bryce B; Weinfurt, KevinPurpose
The U.S. Food & Drug Administration has identified physical functioning (PF) as a core patient-reported outcome (PRO) in cancer clinical trials. The purpose of this study was to identify PF PRO measures (PROMs) in adult cancer populations and classify the PROMs by content covered (facets of PF) in each measure.Methods
As part of the Patient Reports of Physical Functioning Study (PROPS) research program, we conducted a targeted literature review to identify PROMs that could be used in clinical trials to evaluate PF from the patient perspective. Next, we convened an advisory panel to conduct a modified, reactive, Delphi study to reach consensus on which PF facets are assessed by PROMs identified in the review. The panel engaged in a "card sort" activity to classify PROM items by PF facets. Consensus was reached when 80% of panel members agreed that at least one facet was being measured by each PROM item.Results
The literature review identified 13 PROMs that met inclusion criteria. Eight facets of PF were identified for classification in the Delphi study: ability, completion, difficulty, limitation, quality, frequency, bother, and satisfaction. Through two rounds, the panel documented and classified conceptual approaches for each PRO item presented. The most prevalent PF facets were ability, difficulty, and limitation.Conclusion
Classifying PF PROMs by PF facets will promote more consistent communication regarding the aspects of PF represented in each PROM, helping researchers prioritize measures for inclusion in cancer clinical trials.Item Open Access How do patients interpret and respond to a novel patient-reported eastern cooperative oncology group performance status (ECOG)?(Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation, 2024-06) Fillipo, Rebecca; Leblanc, Thomas W; Plyler, Katelyn E; Arizmendi, Cara; Henke, Debra M; Coles, TheresaPurpose
Performance status is an important concept in oncology, but is typically clinician-reported. Efforts are underway to include patient-reported measures in cancer care, which may improve patient symptoms, quality of life and overall survival. The purpose of this study was to gain a preliminary understanding of how patients determined their physical performance status based on a novel patient-reported version of the Eastern Cooperative Oncology Group Performance Status (ECOG) scale.Methods
We conducted qualitative interviews, including concept elicitation and cognitive interviewing as part of the Patient Reports of Physical Functioning Study (PROPS) to investigate how participants selected their answers to a novel patient-reported ECOG. Participants were administered the patient-reported ECOG and asked to describe devices and modifications used to keep up with daily activities.Results
Participants generally understood the ECOG as intended. Participants with recent changes in status had some difficulty selecting an answer. Most participants used modifications and assistive devices in their daily lives but did not incorporate these into their rational for the ECOG.Conclusion
The potential benefits of a patient-reported ECOG are numerous and this study demonstrates that participants were able to understand and answer the patient-reported ECOG as intended. We recommend future evaluation for the most-appropriate recall period, whether to include modifications in the ECOG instructions, and if increasing the number of response options to the patient-reported ECOG may improve confidence when providing an answer.Item Open Access Obese Patients Benefit, but do not Fare as Well as Nonobese Patients, Following Lumbar Spondylolisthesis Surgery: An Analysis of the Quality Outcomes Database.(Neurosurgery, 2020-01) Chan, Andrew K; Bisson, Erica F; Bydon, Mohamad; Glassman, Steven D; Foley, Kevin T; Potts, Eric A; Shaffrey, Christopher I; Shaffrey, Mark E; Coric, Domagoj; Knightly, John J; Park, Paul; Wang, Michael Y; Fu, Kai-Ming; Slotkin, Jonathan R; Asher, Anthony L; Virk, Michael S; Kerezoudis, Panagiotis; DiGiorgio, Anthony M; Haid, Regis W; Mummaneni, Praveen VBACKGROUND:Given recent differing findings following 2 randomized clinical trials on degenerative lumbar spondylolisthesis (DLS) surgery, there is a need to better define how subsets of patients fare following surgery. OBJECTIVE:To investigate the impact of obesity on patient-reported outcomes (PROs) following DLS surgery. METHODS:A total of 12 high-enrolling sites were queried, and we found 797 patients undergoing surgery for grade 1 DLS. For univariate comparisons, patients were stratified by BMI ≥ 30 kg/m2 (obese) and < 30 kg/m2 (nonobese). Baseline, 3-mo, and 12-mo follow-up parameters were collected. PROs included the North American Spine Society satisfaction questionnaire, numeric rating scale (NRS) back pain, NRS leg pain, Oswestry Disability Index (ODI), and EuroQoL-5D (EQ-5D) Questionnaire. RESULTS:We identified 382 obese (47.9%) and 415 nonobese patients (52.1%). At baseline, obese patients had worse NRS back pain, NRS leg pain, ODI, and EQ-5D scores (P < .001, P = .01, P < .001, and P = .02, respectively). Both cohorts improved significantly for back and leg pain, ODI, and EQ-5D at 12 mo (P < .001). At 12 mo, similar proportions of obese and nonobese patients responded that surgery met their expectations (62.6% vs 67.4%, P = .24). In multivariate analyses, BMI was independently associated with worse NRS leg pain and EQ-5D at 12 mo (P = .01 and P < .01, respectively) despite adjusting for baseline differences. CONCLUSION:Obesity is associated with inferior leg pain and quality of life-but similar back pain, disability, and satisfaction-12 mo postoperatively. However, obese patients achieve significant improvements in all PRO metrics at 12 mo.Item Open Access Responsiveness of the PROMIS® measures to changes in disease status among pediatric nephrotic syndrome patients: a Midwest pediatric nephrology consortium study.(Health Qual Life Outcomes, 2017-08-23) Selewski, David T; Troost, Jonathan P; Cummings, Danyelle; Massengill, Susan F; Gbadegesin, Rasheed A; Greenbaum, Larry A; Shatat, Ibrahim F; Cai, Yi; Kapur, Gaurav; Hebert, Diane; Somers, Michael J; Trachtman, Howard; Pais, Priya; Seifert, Michael E; Goebel, Jens; Sethna, Christine B; Mahan, John D; Gross, Heather E; Herreshoff, Emily; Liu, Yang; Carlozzi, Noelle E; Reeve, Bryce B; DeWalt, Darren A; Gipson, Debbie SBACKGROUND: Nephrotic syndrome represents a condition in pediatric nephrology typified by a relapsing and remitting course, proteinuria and the presence of edema. The PROMIS measures have previously been studied and validated in cross-sectional studies of children with nephrotic syndrome. This study was designed to longitudinally validate the PROMIS measures in pediatric nephrotic syndrome. METHODS: One hundred twenty seven children with nephrotic syndrome between the ages of 8 and 17 years participated in this prospective cohort study. Patients completed a baseline assessment while their nephrotic syndrome was active, a follow-up assessment at the time of their first complete proteinuria remission or study month 3 if no remission occurred, and a final assessment at study month 12. Participants completed six PROMIS measures (Mobility, Fatigue, Pain Interference, Depressive Symptoms, Anxiety, and Peer Relationships), the PedsQL version 4.0, and two global assessment of change items. RESULTS: Disease status was classified at each assessment: nephrotic syndrome active in 100% at baseline, 33% at month 3, and 46% at month 12. The PROMIS domains of Mobility, Fatigue, Pain Interference, Depressive Symptoms, and Anxiety each showed a significant overall improvement over time (p < 0.001). When the PROMIS measures were compared to the patients' global assessment of change, the domains of Mobility, Fatigue, Pain Interference, and Anxiety consistently changed in an expected fashion. With the exception of Pain Interference, change in PROMIS domain scores did not correlate with changes in disease activity. PROMIS domain scores were moderately correlated with analogous PedsQL domain scores. CONCLUSION: This study demonstrates that the PROMIS Mobility, Fatigue, Pain Interference, and Anxiety domains are sensitive to self-reported changes in disease and overall health status over time in children with nephrotic syndrome. The lack of significant anchoring to clinically defined nephrotic syndrome disease active and remission status may highlight an opportunity to improve the measurement of HRQOL in children with nephrotic syndrome through the development of a nephrotic syndrome disease-specific HRQOL measure.