P116 Development of a new patient-reported outcome measure for patients with complex cryptoglandular fistulas

Abstract Background Complex cryptoglandular fistulas (CCF) arise from infected anal glands and are often associated with substantial morbidity and healthcare utilization. Standarized patient-reported outcome measures (PROMs) for CCF are lacking. Thus, we aimed to develop a disease-specific PROM to assess the burden of symptoms and their impact on daily life in patients with CCF. Methods To develop a conceptual model, a targeted literature review was conducted to identify symptoms and impacts on daily life associated with CCF. Existing PROMs relating to anal fistulas were also sought. Semi-structured interviews with colorectal surgeons (n=5) assessed clinical perspectives on patient experience of CCF. A draft item pool was developed based on the refined conceptual model and using the Quality of Life in Patients with Anal Fistula Questionnaire as a reference. The tool was refined through concept elicitation interviews on the symptoms and impacts of CCF, and cognitive debriefing on the interpretation, understanding and response to each item. Interviews were conducted until concept saturation was achieved and patient feedback suggested no further refinements were required. Results The literature review identified discharge, pain, faecal incontinence and bleeding as the most prevalent symptoms, and embarrassment as the most prevalent impact on daily life. Results from surgeon interviews were then used to revise the initial conceptual model. Twenty US adults (60% female; mean age, 49 years) with clinically confirmed CCF participated in interviews (four waves of n=5). Patients identified 10 salient symptoms and 11 salient impacts on daily life (salient defined as mention by ≥50% of patients and mean disturbance rating ≥5 on a 10-point scale). The final conceptual model included the main symptoms and treatment-related effects, and impacts on daily life. The draft item pool was refined, resulting in a final PROM consisting of 14 items (covering frequency and severity) relating to symptom domains – discharge, incontinence, pain, irritation, odour, abscess – and 6 items relating to health-related quality of life domains – functional, physical, psychological, social health. The PROM features a 7-day recall period, with responses mostly given on a 5-point verbal rating scale. Cognitive debriefing confirmed that the PROM was clear, easy to understand and relevant to patients’ experiences. Conclusion The 20-item CCF questionnaire (CCFQ-20) is a new PROM that has been developed and tested for content validity, following expert guidance and regulatory best practices. It addresses a comprehensive set of salient symptoms and impacts experienced by patients with CCF. Psychometric testing is required to fully evaluate this PROM. Sponsor: Takeda Pharmaceuticals USA, Inc.

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Considering quality of life for children with cancer: a systematic review of patient-reported outcome measures and the development of a conceptual model

Quality of Life Research ◽

10.1007/s11136-013-0482-x ◽

2013 ◽

Vol 23 (3) ◽

pp. 771-789 ◽

Cited By ~ 29

Author(s):

Samantha J. Anthony ◽

Enid Selkirk ◽

Lillian Sung ◽

Robert J. Klaassen ◽

David Dix ◽

...

Keyword(s):

Quality Of Life ◽

Systematic Review ◽

Conceptual Model ◽

Outcome Measures ◽

Patient Reported Outcome Measures ◽

Patient Reported Outcome ◽

Children With Cancer ◽

Patient Reported

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Patient experiences with hypertrophic cardiomyopathy: a conceptual model of symptoms and impacts on quality of life

Journal of Patient-Reported Outcomes ◽

10.1186/s41687-020-00269-8 ◽

2020 ◽

Vol 4 (1) ◽

Author(s):

Erica Zaiser ◽

Amy J. Sehnert ◽

Ashley Duenas ◽

Sara Saberi ◽

Ella Brookes ◽

...

Keyword(s):

Quality Of Life ◽

Chest Pain ◽

Hypertrophic Cardiomyopathy ◽

Literature Review ◽

Conceptual Model ◽

Left Ventricular ◽

Shortness Of Breath ◽

Patient Reported ◽

One To One

Abstract Background Hypertrophic cardiomyopathy (HCM) is a primary myocardial disorder defined by left ventricular hypertrophy that cannot be explained by another cardiac or systemic disease. There is a general lack of knowledge about patients’ perspectives on the symptoms and day-to-day limitations they experience as a result of HCM. We therefore sought an in-depth understanding of patients’ experiences of obstructive (oHCM) and nonobstructive (nHCM) forms of the disease, including symptoms and their quality of life impacts, and to develop a conceptual model to capture them. Methods Development of the HCM conceptual model involved a web-based survey to capture patients’ insights, a targeted literature review (which included relevant guidelines and patient advocacy websites), one-to-one interviews with clinical experts, and one-to-one qualitative concept elicitation interviews with patients. Key symptoms and their impacts most important to patients’ experiences were identified and used to develop a conceptual model of the patient experience with HCM. Results The HCM symptoms reported by patient interviewees (n = 27) were largely consistent with findings from the patient web survey (n = 444), literature review, and interviews with three expert clinicians. The symptoms most commonly reported in patient interviews included tiredness (89%), shortness of breath (89%), shortness of breath with physical activity (89%), and dizziness/light-headedness (89%). Other symptoms commonly reported included chest pain (angina) (70%), chest pain (angina) with physical exertion (70%), and palpitations (fluttering or rapid heartbeat) (81%). The most commonly reported impacts of HCM symptoms on patients’ lives included limitations to physical activities (78%), emotional impacts, including feeling anxious or depressed (78%), and impacts on work (63%). Symptoms and impacts were similar for both oHCM and nHCM. Conclusions A conceptual model was developed, which identifies the core symptoms that patients with oHCM and nHCM reported as most frequent and most important: shortness of breath, palpitations, fatigue/tiredness, dizziness/light-headedness, and chest pain, as well as the impacts those symptoms have on patients’ lives. This HCM conceptual model reflecting patients’ experiences and perspectives was used in the development of a patient-reported outcomes instrument for use in clinical trials and it may also help inform the clinical management of HCM.

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Incorporating Quality of Life Metrics in Interventional Oncology Practice

Seminars in Interventional Radiology ◽

10.1055/s-0037-1608826 ◽

2017 ◽

Vol 34 (04) ◽

pp. 313-321 ◽

Cited By ~ 3

Author(s):

David Li ◽

David Madoff

Keyword(s):

Quality Of Life ◽

Cancer Patients ◽

Rating Scale ◽

Numerical Rating Scale ◽

Brief Pain Inventory ◽

Patient Reported Outcome ◽

Life Questionnaire ◽

European Organization ◽

Patient Reported

AbstractInterventional radiologists care for a large number of cancer patients with the breadth of palliative intent minimally invasive procedures that we provide. Understanding our meaningful impact on patients' quality of life is essential toward validating our role in the palliation of cancer patients. As such, it is critically important for interventional radiologists to understand common instruments used for the reporting of patient's quality of life measures. Common instruments used to measure pain and quality of life for cancer patients include the numerical rating scale, visual analog scale, brief pain inventory, the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire, and the Functional Assessment of Cancer Therapy. An ideal quality of life instrument should be a patient reported outcome measure across multiple domains (e.g., physical health, psychological, social), and be both validated and reliable.

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PMH41 QUALITY OF LIFE, EMOTIONAL AND PHYSICAL SYMPTOMS IN DEPRESSION: A LITERATURE REVIEW OF SYMPTOMATOLOGY AND PATIENT REPORTED OUTCOME (PRO) INSTRUMENTS

Value in Health ◽

10.1016/s1098-3015(10)63076-8 ◽

2005 ◽

Vol 8 (3) ◽

pp. 397

Author(s):

A Garcia-Cebrian ◽

V Staniek ◽

L Hugonot-Diener ◽

B Arnould ◽

B Monz

Keyword(s):

Quality Of Life ◽

Literature Review ◽

Physical Symptoms ◽

Patient Reported Outcome ◽

Patient Reported ◽

Pro Instruments

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Patient experiences with obstructive and nonobstructive hypertrophic cardiomyopathy: a conceptual model of symptoms and impacts on quality of life

10.21203/rs.3.rs-29996/v1 ◽

2020 ◽

Author(s):

Erica Zaiser ◽

Amy J. Sehnert ◽

Ashley Duenas ◽

Sara Saberi ◽

Ella Brookes ◽

...

Keyword(s):

Quality Of Life ◽

Chest Pain ◽

Hypertrophic Cardiomyopathy ◽

Literature Review ◽

Conceptual Model ◽

Left Ventricular ◽

Shortness Of Breath ◽

Patient Reported ◽

One To One

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Identifying Salient Quality of Life Domains in Patients Living with High-Risk MDS and AML

Blood ◽

10.1182/blood-2021-150821 ◽

2021 ◽

Vol 138 (Supplement 1) ◽

pp. 4982-4982

Author(s):

Anthony J. Messina ◽

Sabrina Figueiredo ◽

John H Powers ◽

Mary A Corcoran

Keyword(s):

Quality Of Life ◽

Conceptual Model ◽

Treatment Satisfaction ◽

Research Study ◽

Patient Reported Outcome ◽

Individual Factors ◽

Life Domains ◽

Patient Interviews ◽

Patient Reported

Abstract Background Valuing patients as the experts in their experience, how they live with myelodysplastic disease, and how to reframe assessments of efficacy, patient-reported outcomes (PROs) can complement traditional measurements of response. Therefore, when used correctly, PROs serve as a valued instrument to assess a patient's Quality of Survival (QoS) throughout the course of their treatment. This study aimed to address the need for improved quality of life measurement in MDS and AML research requires identifying instruments that are appropriate for this population and ones that accurately represent the goals, needs, and concerns of these patients. Therefore research efforts should be directed to providing confirmatory data of the suitability for utilizing existing patient-reported outcome measurements (PROMs) for this population or identify the need to develop and use highly sensitive disease-specific measurements when evaluating new treatments. Identification of representative, influential, patient-reported quality of life domains first needed to be established in order to direct discovery of appropriate PROMs for this population in this two part research study (part-one: patient interviews/surveys, part two: Delphi panel/identification of appropriate PROMs [forthcoming]). Methods Between January and June 2021, 17 participants (AML, n = 8; HR-MDS, n = 9) were enrolled in an IRB-research study participating in concept-elicitation, semistructured interviews and surveys aimed to identify the lived experience of those being treated for HR-MDS and AML. An interpretive descriptive methodology was chosen to produce coherent conceptual descriptions aimed to expose thematic patterns and commonalities that characterize individuals with HR-MDS and AML. Using these insights, a conceptual model was derived to establish the basis for PROM content validity evaluation and to improve the accuracy of the captured quality of life measurements of this patient population. Results Seven (7) patient-reported themes emerged from qualitative analysis; social wellbeing, physical wellbeing, emotional wellbeing, management of side effects, treatment satisfaction, autonomy, and individual factors. Treatment Satisfaction, Autonomy, and Individual Factors (such as education, financial wellbeing, incoming treatment goal, health status, outlook, and prognosis) were found to be the most salient related to quality of life and overall wellbeing. The conceptual model highlights the main areas of impact and the relationships among concepts. Significant overlap was observed between the influential domains identified amongst those with HR-MDS and AML. Surveys, which were administered after patient interviews, were analyzed for consistency, reliability (α=0.8405), and variable correlation. Conclusion A clear understanding of the influential health-related quality of life domains that make up the foundation of this patient population's experience has proven fundamental in the process of identification of appropriate PROMs to measure quality of life. The patient-reported influence treatment satisfaction (confidence in treatment/provider, quality of care, compassion received, provided support, knowledge of treatment, burden of care, information of prognosis, etc) has on a patient's quality of life may lead to a more holistic approach to patient-reported outcome item-banks and instrument selection. The importance of the maintenance of autonomy during and after treatment was apparent in the reported anxiety associated with the loss of the ability of self-care, and independence. Additionally, the absence of divergent themes amongst those with HR-MDS and AML further adds to the evidence that these sub-populations share similar patient experiences. Disclosures Powers: Arrevus, Celularity, Corbus, DaVolterra, Eicos, Eli Lilly, Evofem, Eyecheck, Fuji, Gilead, GSK group of companies, Johnson & Johnson, Microbion, Mustang, OPKO, Otsuka, Romark, Shiniogi, Vir: Consultancy.

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THE AWESCORE (Alfred Wellness Score) – a patient reported outcome measure (PROM): development, feasibility, reliability, validity and responsiveness for adults with cystic fibrosis

ERJ Open Research ◽

10.1183/23120541.00120-2021 ◽

2021 ◽

pp. 00120-2021

Author(s):

Brenda M. Button ◽

Lisa M. Wilson ◽

Angela T. Burge ◽

Lara Kimmel ◽

Felicity Finlayson ◽

...

Keyword(s):

Quality Of Life ◽

Cystic Fibrosis ◽

Rating Scale ◽

Numerical Rating Scale ◽

Short Form ◽

Patient Reported Outcome ◽

Pulmonary Exacerbation ◽

Item Score ◽

Patient Reported

BackgroundQuality of life has improved dramatically over the past two decades in people with cystic fibrosis (CF). Quantification has been enabled by patient reported outcome measures (PROMs), however many are lengthy and can be challenging to use in routine clinical practice. We propose a short-form PROM that correlates well with established quality of life measures.MethodsWe evaluated the utility of a ten-item score (AWESCORE) by measuring reliability, validity and responsiveness in adults with CF. The questions were developed by thematic analysis of survey questions to patients in a single adult CF centre. Each question was scored using a numerical rating scale zero to 10. Total scores ranged from 0 to 100. Test-retest reliability was assessed over 24 h. To determine validity, comparisons were sought between stable subjects and those in pulmonary exacerbation, and between AWESCORE and CFQ-R. Responsiveness to pulmonary exacerbation in individual subjects was evaluated.ResultsFive domains, each with two questions, were identified for respiratory, physical, nutritional, psychological and general health. A total of 246 consecutive adults attending the Outpatient Clinic completed the AWESCORE. Scores were higher during clinical stability compared to pulmonary exacerbation (mean 73 [sd 11]) versus 48 [11], p<0.001). Each domain scored worse during an acute exacerbation (p<0.001). No differences in reliability were observed in scores on retesting using Bland-Altman comparison. The CFQ-R scores (mean 813 [sd125]) and AWESCORE (81 [13]) were moderately correlated (Pearson's r=0.649; p=0.002).ConclusionsThe AWESCORE is valid, reliable and responsive to altered health status in cystic fibrosis.

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Development of a Cold Agglutinin Disease-Specific Patient-Reported Outcome Symptom Measure

Blood ◽

10.1182/blood-2021-147199 ◽

2021 ◽

Vol 138 (Supplement 1) ◽

pp. 4057-4057

Author(s):

Florence Joly ◽

Pronabesh Dasmahapatra ◽

Jun Su ◽

Dana DiBenedetti ◽

Katherine Kosa ◽

...

Keyword(s):

Conceptual Model ◽

Patient Perspective ◽

Patient Reported Outcome ◽

Cold Agglutinin ◽

Cognitive Debriefing ◽

Shortness Of Breath ◽

Item Pool ◽

Cold Agglutinin Disease ◽

Publicly Traded ◽

Patient Reported

Abstract Introduction Cold agglutinin disease (CAD) is a rare autoimmune hemolytic anemia in which red blood cells are bound by cold agglutinin autoantibodies. In addition to cold-induced symptoms, patients with CAD experience symptoms of chronic anemia and hemolysis including fatigue, weakness, and shortness of breath (Swiecicki PL et al. Blood 2013;122(7):1114-21). The objectives of this study were to identify disease-related symptoms and impacts most important to patients with CAD, and to evaluate whether existing patient-reported outcome (PRO) measures are appropriate to assess relevant CAD concepts. Methods As part of a larger study, qualitative concept elicitation (CE) interviews were conducted to better understand the disease burden of CAD from the patient perspective. The results from the first set of interviews were reported by Su J et al. Blood 2020;136(1):29-30. Based on the identified symptoms and impacts, standard survey methodological principles were used to develop a draft item pool to address concepts of interest. Items were refined and evaluated during the second set of interviews, which included 3 rounds of 60-minute phone interviews with patients with CAD using a semi-structured interview guide that included a CE section and cognitive debriefing. Pooled results from both sets of interviews were used to develop a conceptual model of the symptoms and impacts of CAD. Physician advisors were also invited to review the concepts identified in the interviews and to select those that they believed were most relevant as part of an advisory board. A PubMed review of literature published between 2010-2020 was also conducted to identify existing PRO measures used in the assessment of symptoms, impacts, and/or quality of life and patient experiences in adults with CAD or similar disease areas. Identified PRO measures were assessed to determine the extent to which they captured CAD-specific concepts identified in the CE interviews. Results In total, 37 participants diagnosed with primary or secondary CAD took part in the interviews (Set 1, n=16; Set 2, n=21). Overall, the mean age of participants was 67.2 years (range: 35-87), and the majority were female (73%). The most frequently reported symptom of CAD was reactions to cold environments (n=36; 97%), e.g., cold or numb feet/hands, and skin discoloration. Other reported symptoms included fatigue (n=35; 95%), shortness of breath (n=28; 76%), and trouble with thinking/concentration (n=21; 57%). The most frequently reported unfavorable impact of CAD was on day-to-day activities (n=32; 87%). Other negative impacts included effects on enjoyable activities (n=29; 78%) e.g., gardening, physical health/activities (n=28; 76%), social/leisure life (n=26; 70%); and mood/emotions (n=24; 65%). Most participants (n=30; 81%) reported they had made lifestyle/behavioral changes to help limit their CAD symptoms, such as wearing extra clothing in places that were likely to be cold. A conceptual model of the symptoms and impacts of CAD was developed based on these data (Figure 1). Physician advisors (n=7) agreed that the patient-reported symptoms and impacts identified during the interviews were relevant concepts to CAD. Following a literature review, no existing PRO measure was found to adequately address concepts deemed critical to CAD from the patient perspective. Owing to the lack of an existing fit-for-purpose measure, 14 concepts were identified from the interviews to develop an initial draft item pool. During the cognitive debriefing in the second set of interviews, participants (n=21: Round 1 n=8; Round 2 n=7; Round 3 n=6) evaluated these concepts. After 3 rounds of interviews, the new item set yielded 11 items relating to: fatigue; cold sensitivity; dyspnea; wearing extra clothing; limited physical, social, and enjoyable activities; difficulty with usual activities; mood; frustration; and anxiety/stress. Participants reported these items were comprehensive of their experiences with CAD, easy to understand, and would be relevant to gaining a better understanding of individuals' experiences with CAD. Conclusion These results support the need for a novel PRO measure(s) that adequately addresses concepts critical to the measurement of CAD symptoms and impacts from the patient perspective. Figure 1 Figure 1. Disclosures Joly: Sanofi: Current Employment. Dasmahapatra: Sanofi: Current Employment, Current equity holder in publicly-traded company. Su: Sanofi: Current equity holder in publicly-traded company, Ended employment in the past 24 months; Astellas US LLC: Current Employment. DiBenedetti: RTI Health Solutions: Current Employment, Other: Employee of RTI Health Solutions, funded by Sanofi Genzyme to conduct the original work which this abstract is based on. Kosa: RTI Health Solutions: Current Employment. Hill: Novartis: Consultancy, Honoraria; Grifols: Consultancy, Honoraria; Sanofi: Consultancy; ReAlta: Consultancy; Alexion: Honoraria; Amgen: Honoraria; Argenx: Consultancy; Apellis: Consultancy, Honoraria.

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General Patient-Reported Outcome Measure Tools Non-Reflective of AL Amyloidosis Quality of Life (QoL): A Systematic Literature Review

Blood ◽

10.1182/blood-2021-147021 ◽

2021 ◽

Vol 138 (Supplement 1) ◽

pp. 5007-5007

Author(s):

Sotirios Bristogiannis ◽

Charalampia Kyriakou ◽

Ashutosh Wechelaker ◽

Brendan Wisniowski ◽

Shameem Mahmood ◽

...

Keyword(s):

Quality Of Life ◽

Literature Review ◽

Patient Reported Outcome ◽

Current Therapy ◽

Al Amyloidosis ◽

Health Measurement ◽

Sf 36 ◽

Patient Reported ◽

The Impact

Abstract Background: Systemic AL Amyloidosis is characterized by deposition by amyloid fibrils of light chains produced by clonal plasma cells. New combination therapies have substantially prolonged the life expectancy of patients with AL Amyloidosis . Still the prognosis of the disease in the majority of the patients is dismal and Quality of Life (QoL) issues should be included in clinicians' primary objectives. Nowadays, patient-reported outcome measures (PROMs) are considered one of the most responsive tools that can guide personalized interventions to optimize QoL in parallel with therapeutic interventions. The aim of this metanalysis was to establish which PROMs have been utilized in studies on AL Amyloidosis and evaluate their validity. Methods: Two independent investigators (S.B.; C.K.) systemically reviewed PubMed, Medline and EMBASE databases for publications up to May 2021 on PROMs employed to report QoL outcomes in AL Amyloidosis. The identified PROMs were subsequently assessed for their validity in this context against COSMIN (Consensus-based Standards for the selection of health Measurement Instruments) quality criteria. Results: Of the 246 publications originally retrieved, only 57 were further analysed as 92 were duplicates, 11 were irrelevant to AL Amyloidosis, 56 did nor refer to QOL and 13 reported QOL without using QOLQs, 13 were case-reports and 4 were review articles. These included 47 observational studies and 10 prospective clinical trials. In these, thirteen different PROMs were used on occasion to report QOL outcomes (SF-36; EQ-5D-3L; FACT-G; PROMIS-GH); HPRSS; DT; EORTC QLQ-C30; KCCQ-12; GAF; SWLS; STAI; CESD; MDASI) with SF-36 being the most popular (35/ 58 publications). All of them are traditionally validated in similar to AL Amyloidosis diseases (e.g. Multiple Myeloma) or in its complications (e.g. Congestive Heart Failure). In the absence of face-validity studies, the content validity of these PROMs was assessed against QOL aspects identified in literature by open questionnaires (Table 1). The outcome of the intensive analysis of these studies showed that the QoLQ fail to cover the broad spectrum of disease symptoms and current therapy-related toxicity. Furthermore, there is limited if any evidence for the validation of these tools in this context (Table 2). COSMIN criteria were met only for SF-36 and PROMIS-GH as regards internal consistency (Cronbach's a>70). Conclusions: This literature review reveals that commonly applied PROMs in studies on AL Amyloidosis do not represent the impact of this complex disease and its treatment on QoL issues. Thus, there is a need to develop a new, well-validated, disease-specific PROM that can facilitate the approval of new treatments and the adjustment of therapy-intensity according to its toxicity and QoL. Figure 1 Figure 1. Disclosures No relevant conflicts of interest to declare.

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