POS0269-HPR CONTENT VALIDITY OF A MULTIPLE LANGUAGES QUESTIONNAIRE FOR MEASURING FLARE IN KNEE AND HIP OA: DEVELOPMENT OF THE FLARE-OA

Background:Flare in osteoarthritis (OA) of the knee and hip (KHOA) is an important outcome for patients’ daily life and clinical research. A definition of a flare was set, and a core set of domains was recently endorsed by OMERACT/OARSI. No patient reported outcome (PRO) yet focusses on flare in OA specifically.Objectives:To develop a self-reporting instrument measuring flare in 5 languages, using qualitative methods and international Delphi consensus.Methods:We generated items using a dual-language (English and French) approach involving patients with OA from Australia, France and the United States and health care professionals (HCP) from international societies (OARSI, SFR, OMERACT). Item generation relied on semi-structured individual interviews conducted with OA patients and HCP and one focus group with patients. Content analysis allowed for identifying verbatim statements that were meaningful for patients and HCP. A Delphi consensus method was used to select the most relevant items, according to core domains set (OMERACT). A cross-cultural approach using current guidelines (1) was applied to produce Spanish (in Spain), Turkish and classical Arabic (in Morocco) versions using independent translation and expert committee to preserve its content validity.Results:From semi-structured interviews with 29 patients and 16 HCPs and one focus group with 10 patients, 180 statements in French (106) and English (77) were generated. Based on similarity or redundancy, 50 items with equivalent meaning in both languages were retained by an expert committee. After two Delphi rounds involving 50 patients and 116 HCPs from 17 countries on four continents, it was reduced to 33 items (response 0=not at all, to 10=absolutely) in five domains (pain, swelling, stiffness, consequences of symptoms and psychological aspects). This questionnaire was cross-culturally adapted into Spanish, Turkish and classical Arabic. The Spanish version uncovered one inappropriate item in the original questionnaire that was amended accordingly in all 5 languages.Conclusion:Flare is more than just an exacerbation of pain. The Flare-OA questionnaire includes all OMERACT recommended core domains. High content validity was seen in 2 original and 3 cross-culturally adapted languages. The Flare-OA should be considered as fit for purpose and evaluation of responsiveness in clinical studies in 5 languages.Disclosure of Interests:None declared

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SUN-LB102 Development of a Conceptual Model to Present the Impacts of Obesity on Physical Functioning

Journal of the Endocrine Society ◽

10.1210/jendso/bvaa046.2058 ◽

2020 ◽

Vol 4 (Supplement_1) ◽

Author(s):

Jiat Ling Poon ◽

Chris Marshall ◽

Chloe Johnson ◽

Hannah Pegram ◽

Maile Hunter ◽

...

Keyword(s):

Conceptual Model ◽

Physical Functioning ◽

Content Validity ◽

Clinical Studies ◽

Negative Impact ◽

The United States ◽

Overweight And Obesity ◽

Spontaneous Reports ◽

Patient Reported ◽

The Impact

Abstract Background. Obesity is a chronic disease with a significant negative impact on health-related quality of life (HRQoL) and specifically, physical functioning, including the ability to complete activities of daily living (ADLs). Weight loss based on lifestyle management (e.g. diet, exercise), surgery, and pharmacotherapy can improve physical functioning; however, there is a need for further qualitative research to support the content validity of patient-reported outcome (PRO) measures for use in clinical studies of obesity and thus inform regulatory decision-making. Objective. To explore the impacts of obesity on physical functioning and develop a conceptual model (a visual representation of the concepts of importance and relevance to the experience of living with obesity) to ultimately support the content validity of PRO measures.Methods. Qualitative semi-structured interviews were conducted in the United States with individuals who have overweight and obesity (Body Mass Index [BMI] ≥ 27.0 kg/m2) with a history of at least one unsuccessful dietary effort to lose body weight. Recruitment quotas targeted a sample with diverse demographic and clinical characteristics, including participants with and without diabetes. Experienced qualitative interviewers used open-ended questions to elicit spontaneous reports of the impact of obesity on individuals’ daily lives, and specific probing questions to explore impacts on physical functioning. Interviews were audio-recorded, transcribed and analyzed using thematic techniques. Results. A total of 33 participants were interviewed (mean BMI of 37.6 kg/m2 [27.4 kg/m2 to 56.6 kg/m2]; mean age of 45 years [19 to 81 years]). The sample included a mix of races (Caucasian: n=12, 36%), education completed (high school: n=17, 51%) and split of gender (female: n=16, 48%). During development of the conceptual model, two separate domains were identified to group the reported impacts on physical functioning: ‘Mobility/Movement’ and ‘ADLs’. The most frequently reported impacts related to Mobility/Movement were ‘running’ (n=31/33, 94%), ‘bending’ (n=27/33, 82%), ‘walking’ (n=26/33, 79%), ‘difficulty standing for prolonged periods’ (n=22/33, 67%), and ‘lifting’ (n=19/33, 58%). All participants reported effects on some aspect of physical functioning, which were often characterized in terms of their direct impact on ADLs such as ‘household chores’ (n=21/33, 64%). Conclusions. The conceptual model will serve as a basis to identify fit-for-purpose PRO measures with strong content validity to evaluate the impact of anti-obesity therapies on physical functioning in future clinical studies. Obesity has a consistent and significant impact on physical functioning, leading to limitations in various aspects of mobility and affecting an individual’s ability to carry out specific daily activities.

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Patient-Reported Disease Burden: In-Depth Interviews of Patients with CAD

Blood ◽

10.1182/blood-2020-136788 ◽

2020 ◽

Vol 136 (Supplement 1) ◽

pp. 29-30

Author(s):

Jun Su ◽

Katherine Kosa ◽

Dana DiBenedetti

Keyword(s):

Content Validity ◽

Leisure Activities ◽

Panel Member ◽

The United States ◽

Daily Activities ◽

Outdoor Activities ◽

Concept Elicitation ◽

Cold Environments ◽

Patient Reported ◽

Social Leisure

Introduction Cold agglutinin disease (CAD) is a rare disorder (a prevalence of 16 cases per 1 million people), accounting for approximately 15%-25% of all autoimmune hemolytic anemias (Berentsen. Semin Hematol. 2018). Currently, there is no approved drug for the treatment of CAD. Debilitating fatigue that greatly affects quality of life is common among patients with CAD (Roth et al. Blood. 2019). The Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) Scale has recently been used to quantify fatigue in patients with CAD (Roth et al. Blood. 2019). However, the appropriateness of FACIT-F in these patients has not been assessed. In-depth individual interviews of adults with CAD were conducted primarily to identify the symptoms and effects of CAD reported as important by patients and to better understand their experiences with CAD. The secondary objective was to obtain patient feedback regarding the relevance and importance of the items on the FACIT-F. Methods Participants were identified and recruited from a proprietary panel of patients with CAD. Of 1,250 panel members, approximately 600 members from the United States (US) were invited to participate in an in-depth interview. Systematic random sampling was used to select every nth panel member from each of 4 US regions to receive an email invitation. A semi-structured interview guide was used to ensure consistent systematic collection of data pertaining to study objectives, while maintaining a conversational tone and encouraging spontaneity of answers. Results Twenty-two members expressed interest in participating; of these, 18 could be reached for screening and 16 were eligible for interview. The sample was 100% White and non-Hispanic, 62.5% female, and had an average age of 67.8 years. Participants resided in the West (37.5%), Midwest and South (both 25%), and Northeast (12.5%). The date of CAD diagnosis averaged 6 years before the interview, and 68.8% of patients had received their diagnosis within the last 1.5 years. Seven (43.8%) patients reported that they were not taking any prescription or over-the-counter (OTC) medication for CAD, whereas 9 (56.3%) were taking prescribed or OTC folic acid for CAD (of these, 4 also took prescription strength vitamin B12). In this sample, no patient reported current use of other prescription medications for CAD. A few patients spontaneously reported previous treatments for CAD (eg, transfusions and rituximab or other injections). However, previous CAD treatment was not systematically collected for all subjects and is a limitation of the study. Concept-elicitation of symptoms The most frequently reported symptoms were fatigue/tiredness/lack of energy (93.8%), reaction to cold environments (93.8%), breathlessness (68.8%), and trouble thinking/concentrating (68.8%). When probed, 43.8% of patients reported sleep disturbances (Figure 1A). Concept-elicitation of effects Most participants (68.8%) reported lifestyle or behavioral changes to help treat CAD symptoms (eg, taking gloves/extra clothing to places that may be cold). Others reported giving up/limiting enjoyable or outdoor activities and making major changes (eg, discontinuing outdoor activities, restricting travel). Other CAD-related effects included impairment in daily activities, overall health/physical activities, and social/leisure activities/relationships (Figure 1B). For the evaluation of the FACIT-F in this CAD population, participants reported that FACIT-F instructions and items were clear and easy to understand, response options were distinct from each other, and they could recall their fatigue over the previous 7 days. They reported that FACIT-F measured one or more concepts personally important to them and important concepts were not missing. This provides support for the content validity of the FACIT-F in patients with CAD. Conclusions Patients with CAD experienced negative effects on daily activities, physical health/activities, social/ leisure activities/relationships, the ability to concentrate/recall, and mood/emotions mostly owing to fatigue/lack of energy and reactions to cold environments. Most patients required modifying their lifestyle/behavior to manage CAD symptoms. FACIT-F measured concepts important to patients with CAD, which supports its content validity and use in this patient population. Disclosures Su: Sanofi: Current Employment. Kosa:Sanofi: Other: RTI Health Solutions was contracted by Sanofi to conduct this work; RTI Health Solutions: Current Employment. DiBenedetti:Sanofi: Other: RTI Health Solutions was contracted by Sanofi to conduct this work; RTI Health Solutions: Current Employment, Other.

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Characterizing mobility from the prosthetic limb user’s perspective: Use of focus groups to guide development of the Prosthetic Limb Users Survey of Mobility

Prosthetics and Orthotics International ◽

10.1177/0309364615579315 ◽

2016 ◽

Vol 40 (5) ◽

pp. 582-590 ◽

Cited By ~ 20

Author(s):

Brian J Hafner ◽

Sara J Morgan ◽

Daniel C Abrahamson ◽

Dagmar Amtmann

Keyword(s):

Focus Group ◽

Focus Groups ◽

Lower Limb ◽

The United States ◽

Patient Reported Outcome Measures ◽

Individual Characteristics ◽

Prosthetic Limb ◽

Patient Reported ◽

Lower Limb Prosthesis ◽

Limb Prosthesis

Background: Input from target respondents in the development of patient-reported outcome measures is necessary to ensure that the instrument is meaningful. Objectives: To solicit perspectives of prosthetic limb users about their mobility experiences and to inform development of the Prosthetic Limb Users Survey of Mobility. Study design: Qualitative study. Methods: Four focus groups of lower limb prosthesis users were held in different regions of the United States. Focus group transcripts were coded, and themes were identified. Feedback from participants was used to develop a framework for measuring mobility with a lower limb prosthesis. Results: Focus group participants ( N = 37) described mobility as a confluence of factors that included characteristics of the individual, activity, and environment. Identified themes were defined as individual characteristics, forms of movement, and environmental situations. Prosthetic mobility was conceptualized as movement activities performed in an environmental or situational context. Conclusion: Respondent feedback used to guide development of Prosthetic Limb Users Survey of Mobility established a foundation for a new person-centered measure of mobility with a prosthetic limb. Clinical relevance Perspectives of target respondents are needed to guide development of instruments intended to measure health outcomes. Focus groups of prosthetic limb users were conducted to solicit experiences related to mobility with a lower limb prosthesis. Results were used to inform development of a clinically meaningful, person-centered instrument.

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A Population-Based Study of Peyronie's Disease: Prevalence and Treatment Patterns in the United States

Advances in Urology ◽

10.1155/2011/282503 ◽

2011 ◽

Vol 2011 ◽

pp. 1-9 ◽

Cited By ~ 111

Author(s):

Dana Britt DiBenedetti ◽

Dat Nguyen ◽

Laurie Zografos ◽

Ryan Ziemiecki ◽

Xiaolei Zhou

Keyword(s):

Health Care Professionals ◽

Phase 1 ◽

The United States ◽

Treatment Patterns ◽

Care Physician ◽

Peyronie’S Disease ◽

Phase 2 ◽

Patient Reported ◽

The Us ◽

Peyronie's Disease

Purpose. To estimate the US prevalence of Peyronie's disease (PD) from patient-reported data and to identify diagnosis and treatment patterns.Methods. 11,420 US males ≥18 years old completed a brief web-based survey regarding the presence of PD, past treatments, and penile symptoms (Phase 1). Phase 1 respondents with PD diagnosis, history of treatment, or PD-related symptoms then completed a disease-specific survey (Phase 2).Results. Estimated prevalence of PD ranged from 0.5% (diagnosis of PD) to 13% (diagnosis, treatment, or penile symptoms). Thirty-six percent of Phase 2 participants reported that penile symptoms interfered with sexual activities. Of participants who sought treatment for penile symptoms (), 73% initially saw a primary care physician, 74% did not receive treatment from their first doctor, and 92% were not diagnosed with PD.Conclusions. PD may be underdiagnosed/undertreated in the US. Improved awareness is needed of PD symptoms and treatment options among health care professionals.

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Practices and pitfalls in medication adherence in hemodialysis settings – a focus-group study of health care professionals

BMC Nephrology ◽

10.1186/s12882-021-02514-8 ◽

2021 ◽

Vol 22 (1) ◽

Author(s):

Trine Mechta Nielsen ◽

Nina Schjerning ◽

Gudrun Kaldan ◽

Mads Hornum ◽

Bo Feldt-Rasmussen ◽

...

Keyword(s):

Health Care ◽

Medication Adherence ◽

Focus Group ◽

Health Care Professionals ◽

Patient Adherence ◽

Patient Reported Outcome Measures ◽

Patient Reported Outcome ◽

Focus Group Study ◽

Patient Reported ◽

The Impact

Abstract Background Medication nonadherence is common among patients with hemodialysis, leading to poorer patient outcomes. Health care professionals have an important role in assessing risk of nonadherence and intervening to support adherence. The aim of this study was to explore physicians’ and nurses’ current medication adherence practices in hemodialysis settings. Method A generic qualitative design with inductive content analysis and focus group methodology. Focus groups with health care professionals were conducted in four Nephrology Centers, representing three different regions of Denmark. An interview guide was developed in collaboration with 3 patient representatives. Results Six focus group interviews involving a total of forty-two health care professionals were conducted. Five main categories were identified; Laboratory tests are the “gold standard” for assessing adherence, suggesting that abnormal results motivated investigation of adherence, Varying practices for supporting adherence, alluding to the impact of individual clinician priority and preference on choice of adherence interventions, Unclear allocation of roles and responsibility, specifically referring to uncertainty in the delegation of roles between physicians and nurses, Navigating time and resource limitations, intimating the resources needed to support medication adherence and Suggestions for future strategies. Conclusions We suggest implementing systematic use of validated patient-reported outcome measures for assessing adherence and deprescribing tools to support adherence, as these instruments might identify the patients who are in most need of support and promote patient adherence to their prescribed medications. The findings also point to a need for interdisciplinary clarification of roles and responsibilities regarding medication adherence, with the aim of building a strong collaborative partnership between professions.

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Guidelines for the definitions of time-to-event endpoints in randomized clinical trials: Results of the DATECAN Project for Breast Group.

Journal of Clinical Oncology ◽

10.1200/jco.2013.31.15_suppl.1094 ◽

2013 ◽

Vol 31 (15_suppl) ◽

pp. 1094-1094

Author(s):

Sophie Gourgou ◽

Tienhan Sandrine Dabakuyo ◽

Bernard Asselain ◽

David Azria ◽

David A. Cameron ◽

...

Keyword(s):

Breast Cancer ◽

Randomized Clinical Trials ◽

Progression Free Survival ◽

Consensus Method ◽

Expert Committee ◽

Time To Event ◽

Delphi Consensus ◽

Consensus Process ◽

Number Of Patients ◽

Independent Expert

1094 Background: With the necessity of reducing randomized clinical trial (RCT) duration, cost and number of patients, surrogate endpoints of overall survival (OS) are increasingly being used in cancer RCTs. However, most of these endpoints currently lack of standardized definition enabling a comparison of RCT results. Some recommendations have been proposed for specific cancer sites but they do not rely on a formal consensus methodology. The objective of the Definition for the Assessment of Time-to-event Endpoints in CANcer trials (DATECAN) project is to provide guidelines to standardize definitions of time-to-event endpoints in RCTs for different cancer sites. Here, we present results for BREAST cancer. Methods: We relied on the modified Delphi consensus method, a validated formalized consensus process for the development of practice guidelines. International experts with various backgrounds and expertises were involved. First, the coordinating committee, a group of statisticians and epidemiologists involved in the design and conduct of RCTs, led a comprehensive literature review to identify time-to-event endpoints, events of interest and the existence of guidelines in adjuvant and metastatic settings. The steering committee, which included additional medical experts, validated the list and prepared the questionnaire sent for rating to an independent expert committee. Results: The consensus process involved 2 rounds of scoring (31 experts) and 1 in-person meeting (in parallel to ASCO'12). Each expert had to rate on a 1-9 scale if s/he agreed or not for including events (e.g. death from breast cancer) in the definition of time-to-event endpoints (e.g. progression-free survival). 150 events had to be scored for the 11 selected endpoints. Consensus was reached for 57% of the events after the 2 rounds of scoring. After the in-person meeting, consensus was reached for all the remaining events except one. Conclusions: The DATECAN guidelines should help standardizing definitions of commonly used endpoints. This process should (i) facilitate the comparison of RCTs and (ii) improve the quality of future RCTs by providing better estimation of sample size and treatment effect.

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Guidelines for the definitions of time-to-event endpoints in randomized clinical trials: Results of the DATECAN Project for Sarcomas and GISTs.

Journal of Clinical Oncology ◽

10.1200/jco.2013.31.15_suppl.10570 ◽

2013 ◽

Vol 31 (15_suppl) ◽

pp. 10570-10570

Author(s):

Carine A. Bellera ◽

Monia Ouali ◽

Nicolas Penel ◽

Sylvie Bonvalot ◽

Paolo Giovanni Casali ◽

...

Keyword(s):

Randomized Clinical Trials ◽

Progression Free Survival ◽

Steering Committee ◽

Consensus Method ◽

Expert Committee ◽

Time To Event ◽

Delphi Consensus ◽

Consensus Process ◽

Number Of Patients ◽

Independent Expert

10570 Background: With the necessity of reducing randomized clinical trial (RCT) duration, cost and number of patients, surrogate endpoints of overall survival (OS) are increasingly being used as replacement for OS in cancer RCTs. However, most of these endpoints currently lack of standardized definition enabling a comparison of RCT results. Some recommendations have been proposed for specific cancer sites but they do not rely on a formal consensus methodology. The objective of the Definition for the Assessment of Time-to-event Endpoints in Cancer trials (DATECAN) project is to provide guidelines to standardize definitions of time-to-event endpoints in RCTs for different cancer sites. We present results for sarcomas and GISTs. Methods: We relied on the modified Delphi consensus method, a validated formalized consensus process for the development of practice guidelines. International experts with various backgrounds and expertise were involved. First, the coordinating committee, a group of statisticians and epidemiologists involved in the design and conduct of RCTs, led a comprehensive literature review to identify time-to-event endpoints and events of interest. The steering committee, which included additional medical experts, validated the list and prepared the questionnaire sent for rating to an independent expert committee. Results: The consensus process is finalized. It involved 2 rounds of scoring (28 experts) and 1 in-person meeting (in parallel to ASCO'12). Each expert had to rate on a 1-9 scale if s/he agreed or not for including events (e.g. death related to primitive cancer) in the definition of time-to-event endpoints (e.g. progression-free survival). 16 events were rated for inclusion or not in the definitions of 14 time-to-event endpoints. Consensus was reached for 73% of the events after the 2 rounds of scoring. Consensus was finalized at the in-person meeting for the remaining events. Conclusions: The DATECAN guidelines should help standardizing definitions of commonly used endpoints. This process should (i) facilitate the comparison of RCTs and (ii) improve the quality of future RCTs by providing better estimation of sample size and treatment effect.

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Assessing the impacts of having a child with achondroplasia on parent well-being

Quality of Life Research ◽

10.1007/s11136-020-02594-3 ◽

2020 ◽

Author(s):

Kathryn M. Pfeiffer ◽

Meryl Brod ◽

Alden Smith ◽

Jill Gianettoni ◽

Dorthe Viuff ◽

...

Keyword(s):

Focus Group ◽

Well Being ◽

The United States ◽

Patient Reported Outcome ◽

Concept Elicitation ◽

Qualitative Evidence ◽

Parent Experience ◽

Patient Reported ◽

Family And Work ◽

Group Interviews

Abstract Purpose This study’s purpose was to develop a better understanding of the experiences of parents of children with achondroplasia and to provide qualitative evidence to support the development of a patient-reported outcome (PRO) measure of parent impacts. Methods Concept elicitation (CE) individual/focus group interviews were conducted with parents of children aged 2 to < 12 years with achondroplasia in the United States and Spain. The qualitative analysis informed the PRO measure development. Cognitive debriefing (CD) interviews were conducted to ensure parent understanding and item relevance. Results Thirty-six parents participated in individual/focus group CE interviews. The analysis identified parent impacts in four domains, including caretaking responsibilities, emotional well-being, family, and work, and results informed the development of the Achondroplasia Parent Experience Measure (APEM). Caretaking responsibilities included managing child’s medical care (92%), helping child with self-care (67%), advocating for child (64%), assisting child (56%), and observing/monitoring child (e.g., to ensure safety; 47%). Impacts on parents’ emotional well-being included worry about the future (75%), worry about child’s physical health (67%), safety concerns (50%), feeling stressed/overwhelmed (44%), and worry about child’s social relationships (42%). Impacts on family and work included family strain (56%), limiting/adapting family activities (42%), and missed work time (50%). CD interviews with an additional 16 parents of children with achondroplasia confirmed understanding and item relevance. Conclusion The results improve our understanding of the experiences of parents of children with achondroplasia and provide qualitative evidence to support the content validity of the APEM. A psychometric study is needed to validate the measure.

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Qualitative study: burden of menopause-associated vasomotor symptoms (VMS) and validation of PROMIS Sleep Disturbance and Sleep-Related Impairment measures for assessment of VMS impact on sleep

Journal of Patient-Reported Outcomes ◽

10.1186/s41687-021-00289-y ◽

2021 ◽

Vol 5 (1) ◽

Author(s):

Marci English ◽

Boyka Stoykova ◽

Christina Slota ◽

Lynda Doward ◽

Emad Siddiqui ◽

...

Keyword(s):

Sleep Disturbance ◽

Content Validity ◽

Short Form ◽

Work Productivity ◽

The United States ◽

Vasomotor Symptoms ◽

Response Options ◽

Short Forms ◽

Patient Reported ◽

The Impact

Abstract Purpose We evaluated the impact of menopause-associated vasomotor symptoms (VMS) on sleep. We also sought to establish the content validity of Patient-Reported Outcomes Measurement Information System (PROMIS) short form Sleep-Related Impairment and Sleep Disturbance measures in postmenopausal women with moderate to severe VMS. Methods Cross-sectional, in-person, qualitative interviews were conducted in the United States (Texas, Illinois) and European Union (UK, France) with women aged 40–64 years experiencing moderate to severe VMS (≥35/wk). Main outcomes were impact of VMS on sleep based on concept elicitation and content validity of PROMIS Sleep-Related Impairment and Sleep Disturbance short forms via cognitive debriefing. Results Thirty-two women (US: n = 16; EU: n = 16) participated. A majority (US: 93.8%; EU: 93.8%) said VMS affected sleep; specifically, they had sleep interrupted by sweating or overheating and had difficulty returning to sleep. Sleep disturbance was the most bothersome aspect of VMS (US: 75%; EU: 50%). VMS-associated sleep disturbance affected next-day work productivity, mood, relationships, daily activities, concentration, social activities, and physical health. Participants found both PROMIS sleep measures relevant and easy to answer; the Sleep Disturbance measure was considered the most relevant. Participants had no difficulty remembering their experiences over the 7-day recall period and found the response options to be distinct. Conclusion VMS associated with menopause significantly interferes with sleep and next-day functioning (e.g., work productivity), supporting assessment of sleep outcomes in studies evaluating treatment of VMS. Women with moderate to severe VMS found that the PROMIS Sleep-Related Impairment and Sleep Disturbance short forms assessed constructs important to understanding sleep in the context of menopause-associated VMS.

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