scholarly journals A novel patient-reported outcome instrument assessing the symptoms of paroxysmal nocturnal hemoglobinuria, the PNH-SQ

2021 ◽  
Vol 5 (1) ◽  
Author(s):  
R. Paola Daly ◽  
Jessica J. Jalbert ◽  
Shannon Keith ◽  
Tara Symonds ◽  
Jamile Shammo
Keyword(s):  
Abdominal Pain ◽  
Conceptual Model ◽  
Paroxysmal Nocturnal Hemoglobinuria ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Response Options ◽  
Patient Interview ◽  
Outcome Instrument ◽  
Patient Reported ◽  
Bothersome Symptom

Abstract Background Patient-reported outcome measures (PROs) used to measure symptoms of patients with paroxysmal nocturnal hemoglobinuria (PNH) in trials do not measure PNH symptoms comprehensively and do not assess daily fluctuations in symptoms. Following a literature review and consultation with a PNH expert, we drafted the PNH Symptom Questionnaire (PNH-SQ) and a patient-centric conceptual model of PNH symptoms and impacts. We then interviewed 15 patients with PNH to assess comprehensiveness of symptom capture from the patient perspective and to cognitively debrief the PNH-SQ. Patient interview data were also used to finalize the PNH conceptual model. Results Participants mentioned 27 signs or symptoms of PNH spontaneously or after being probed; 93% reported experiencing ≥ 1 PNH symptom. Concept saturation was reached for all PNH symptoms. Further, interviews confirmed the instrument captured the most common PNH symptoms, including fatigue (87%), abdominal pain (60%), and difficulty swallowing (47%), with fatigue ranked as the most bothersome symptom. The interviews demonstrated that participants understood the items of the PNH-SQ (90–100%); considered the symptoms relevant (> 50– > 90%); the recall period appropriate (> 80–100%); and the response options suitable (> 80–100%). Participants also suggested changes regarding item redundancy and relevance; this feedback was used to finalize the instrument. Conclusions The finalized PNH-SQ assesses the presence and severity of 10 symptoms—abdominal pain, chest discomfort, difficulty sleeping, difficulty swallowing, difficulty thinking clearly, fatigue, headache, muscle weakness, pain in the legs or back, and shortness of breath—over 24 h. The PNH-SQ is a content-valid questionnaire suitable for assessing daily symptom presence and severity in PNH clinical trials.

scholarly journals A Novel Patient Reported Outcome Instrument Assessing the Symptoms of Paroxysmal Nocturnal Hemoglobinuria

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 385-385
Author(s):  
R Paola Daly ◽  
Jessica J Jalbert ◽  
Shannon Keith ◽  
Tara Symonds ◽  
Jamile M. Shammo
Keyword(s):  
Abdominal Pain ◽  
Back Pain ◽  
Content Validity ◽  
Research Funding ◽  
Paroxysmal Nocturnal Hemoglobinuria ◽  
Patient Reported Outcome ◽  
Shortness Of Breath ◽  
Response Options ◽  
Patient Reported ◽  
Bothersome Symptom

Introduction. Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, life-threatening blood disease. Frequently reported symptoms of PNH are fatigue, dyspnea, hemoglobinuria, abdominal pain, and chest pain. While PNH-related symptoms can impact quality of life, there are no patient-reported outcome (PRO) measures that assess daily occurrence and severity of PNH-specific symptoms. The objective of this study was to gain a deeper understanding of the patient experience related to symptoms of PNH, and to assess the content validity of the newly developed PNH Symptom Questionnaire (PNH-SQ). Methods. The content and design of the PNH-SQ was informed by a review of the empirical literature, review of COAs used in registrational PNH trials to assess symptoms and impacts of PNH, and through discussions with physicians who have experience treating patients with PNH. The first draft of the PNH-SQ allowed the patient to record, on a daily basis, the presence/absence of 12 symptoms in the past 24 hours (fatigue; shortness of breath; muscle weakness; headache; abdominal pain; leg/back pain; chest discomfort; sexual difficulties; difficulty sleeping; difficulty focusing; difficulty thinking clearly; difficulty swallowing), and the severity of each symptom using a 5-point Likert scale. Trained interviewers conducted telephone, video, or in-person qualitative, semi-structured 60-minute interviews with adult patients with a clinician-confirmed PNH diagnosis. The interviews consisted of two distinct parts: concept elicitation (CE), to assess whether the PNH-SQ captured all important symptoms from the perspective of the patient; and cognitive debriefing (CD), to evaluate the patient's ability to understand and respond to the PNH-SQ. During CE, participants discussed symptoms and impacts of PNH. This was followed by CD, in which participants reviewed and completed the PNH-SQ and asked about each part of the PNH-SQ including the instructions, understanding and relevance of items, recall period, and response options. Interviews were audio-recorded, transcribed, coded, and analyzed. Revisions to the PNH-SQ were considered after interviews were completed and analyzed. Results. A total of 15 participants (mean age of 42.8 years; 53.3% women) from across the United States were interviewed; participants had a mean of 13.4 years since PNH diagnosis, and 66.7%, 13.3%, and 20.0% self-reported very mild/mild, moderate, and severe/very severe PNH, respectively. In the CE part of the interview, participants reported experiencing fluctuations in the presence and/or severity of individual PNH symptoms. The most common symptoms mentioned were fatigue (n=13/15), abdominal pain (n=9/15), and difficulty swallowing (n=7/15). Symptoms spontaneously mentioned by at least 5 participants were: muscle weakness; back pain; cognitive difficulties; and shortness of breath. Fatigue was by far considered the most bothersome symptom (n=8/15); other symptoms had ≤3 mentions as most bothersome symptom. Each symptom included in the PNH-SQ was spontaneously mentioned by two or more participants. Concept saturation, the point at which no new or relevant information is expected to emerge from additional interviews, was reached. In the CD segment of the interview, participants demonstrated understanding of all the symptoms included in the PNH-SQ (90-100%); considered the symptoms relevant (>50 - >90%); the recall period appropriate (80-100%); and the response options suitable (>80-100%). Based on interview analysis, two items were removed from the PNH-SQ: sexual difficulties, because it was of limited relevance to most participants (2/15), and was reported mostly by men; and difficulty focusing, because most participants (n=9/15) considered it interchangeable with difficulty thinking clearly. Minor modifications were also made to accommodate electronic administration of the PNH-SQ. Conclusions. The PNH-SQ was developed according to FDA guidance on PRO measures development. Findings from this study support content validity of the PNH-SQ as an instrument to evaluate the daily presence and severity of PNH symptoms. In addition, the PNH-SQ has the potential to enable the evaluation of day-to-day fluctuations in PNH symptom presence and severity. The next step in instrument development is to conduct psychometric testing of the PNH-SQ using data from an ongoing clinical trial (NCT03946748). Disclosures Daly: Clinical Outcomes Solutions: Employment. Jalbert:Regeneron Pharmaceuticals, Inc.: Employment, Equity Ownership. Keith:Clinical Outcomes Solutions: Employment. Symonds:Clinical Outcomes Solutions: Employment. Shammo:Incyte: Consultancy, Honoraria, Research Funding, Speakers Bureau; CTI Pharma: Research Funding; Onconova: Research Funding; Alexion: Consultancy, Honoraria, Research Funding, Speakers Bureau; Celgene: Consultancy, Honoraria, Research Funding, Speakers Bureau; Apellis: Membership on an entity's Board of Directors or advisory committees; Otsuka: Consultancy, Honoraria; Novartis: Consultancy, Honoraria; Astex Pharma: Research Funding; Sanofi: Consultancy, Honoraria, Speakers Bureau.

scholarly journals The role of response domain and scale label in the quantitative interpretation of patient-reported outcome measure response options

2021 ◽  
Author(s):  
Tessa Peasgood ◽  
Jen-Yu Chang ◽  
Robina Mir ◽  
Clara Mukuria ◽  
Philip A. Powell
Keyword(s):  
First Language ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Response Options ◽  
The Public ◽  
Online Panel ◽  
Patient Reported ◽  
Common Response ◽  
Chi Squared

Abstract Purpose Uncertainties exist in how respondents interpret response options in patient-reported outcome measures (PROMs), particularly across different domains and for different scale labels. The current study assessed how respondents quantitatively interpret common response options. Methods Members of the general public were recruited to this study via an online panel, stratified by age, gender, and having English as a first language. Participants completed background questions and were randomised to answer questions on one of three domains (i.e. loneliness (negatively phrased), happiness or activities (positively phrased)). Participants were asked to provide quantitative interpretations of response options (e.g. how many times per week is equal to “often”) and to order several common response options (e.g. occasionally, sometimes) on a 0–100 slider scale. Chi-squared tests and regression analyses were used to assess whether response options were interpreted consistently across domains and respondent characteristics. Results Data from 1377 participants were analysed. There was general consistency in quantifying the number of times over the last 7 days to which each response option referred. Response options were consistently assigned a lower value in the loneliness than happiness and activities domains. Individual differences, such as age and English as a second language, explained some significant variation in responses, but less than domain. Conclusion Members of the public quantify common response options in a similar way, but their quantification is not equivalent across domains or every type of respondent. Recommendations for the use of certain scale labels over others in PROM development are provided.

scholarly journals Development of a conceptual model and patient-reported outcome measures for assessing symptoms and functioning in patients with heart failure

2020 ◽  
Vol 29 (10) ◽  
pp. 2835-2848
Author(s):  
Olga Moshkovich ◽  
Katy Benjamin ◽  
Katie Hall ◽  
Ryan Murphy ◽  
Robyn von Maltzahn ◽  
...  
Keyword(s):  
Heart Failure ◽  
Literature Review ◽  
Conceptual Model ◽  
Phase 1 ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Cognitive Testing ◽  
Expert Interviews ◽  
Cognitive Changes ◽  
Patient Reported

Abstract Purpose Heart failure (HF) is a common condition that places considerable burden on patients. We aimed to develop a patient-reported outcome (PRO) measure to assess the symptoms and impacts of HF. Methods Phase 1: a targeted literature review, expert interviews, and concept elicitation (CE) interviews with patients with HF (n = 26) were used to develop a conceptual model of the core symptoms and impacts of HF. To capture these concepts, three new fit-for-purpose PRO questionnaires were constructed in accordance with US Food and Drug Administration PRO guidance. Phase 2: three ‘waves’ of cognitive interviews were conducted with patients with HF (n = 28) to validate and refine the questionnaires. Results Three key symptoms—shortness of breath, oedema, and fatigue—were identified across the literature review, expert interviews and CE interviews. Several additional symptoms, cognitive changes and impacts of HF were reported in the CE interviews and included in the conceptual model. A 10-item symptom questionnaire (Heart Failure-Daily Symptom Diary) was constructed; cognitive testing showed that the final PRO measure was easy to understand/complete and relevant to patients with HF, confirming content validity. Two HF impact questionnaires were developed (Assessing Dyspnoea’s Impact on Mobility and Sleep and Heart Failure-Functional Status Assessment), but required refinement to ensure patient understanding. Conclusions Patient input contributed to the development of a PRO instrument for assessing physical and cognitive symptoms important to patients with HF using novel measurement strategies. Inclusion of daily metrics offers differentiation from other qualified instruments and may provide clinical insight for improving lifestyles. Additionally, two draft PRO measures may, after further validation, be useful to assess the impacts of HF.

scholarly journals Correction to: Development of a conceptual model and patient-reported outcome measures for assessing symptoms and functioning in patients with heart failure

2020 ◽  
Vol 29 (10) ◽  
pp. 2849-2849
Author(s):  
Olga Moshkovich ◽  
Katy Benjamin ◽  
Katie Hall ◽  
Ryan Murphy ◽  
Robyn von Maltzahn ◽  
...  
Keyword(s):  
Heart Failure ◽  
Conceptual Model ◽  
Outcome Measures ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Patient Reported ◽  
Patients With Heart Failure

The article Development of a conceptual model and patient-reported outcome measures for assessing symptoms and functioning in patients with heart failure.

scholarly journals Exploring the impact of Patient Reported Outcome Measures (PROMs) among orthopaedic surgeons in mainland China: systematic review and survey-based study on hip and knee instruments

2021 ◽  
Vol 22 (1) ◽  
Author(s):  
James Reeves Mbori Ngwayi ◽  
Jie Tan ◽  
Ning Liang ◽  
Kenedy Uzoma Obie ◽  
Daniel Edward Porter
Keyword(s):  
Outcome Measures ◽  
Mainland China ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Response Options ◽  
Orthopaedic Surgeons ◽  
Clinical Registries ◽  
Online Social Media ◽  
Patient Reported ◽  
The Impact

Abstract Background Patient Reported Outcome Measures (PROMs) are widely used in Europe and North America in a variety of areas including research, clinical governance, clinical registries and insurance ascertainment. The aim of this study was to assess commonly used knee and hip PROMs among Chinese surgeons and to gain an insight into their impact on evaluation of clinical outcomes. Methods 1. A systematic literature search of databases Medline, EMBASE, CINAHL and CNKI was performed from the earliest records to 22/07/2020 for knee instruments and 22/08/2020 for hip instruments, to retrieve Chinese Mandarin cross culturally adapted and validated knee and hip PROMs. 2. An 11-item electronic questionnaire was then designed under four domain categories. The survey was distributed via a ubiquitous online social media platform to orthopaedic surgeons. Responses were collected and analyzed. Output from 1. was used to populate parts of the survey questionnaire. Results The systematic online search yielded a total of 41 evaluation instruments, (10 hip and 31 knee); all of which were incorporated as response options. 234 viable questionnaires were retrieved with the largest group representing attending surgeons. 59.0% were familiar with the concept of PROMs among which 78.4% reported to have used PROMs themselves. In order of frequency of use, PROMs were purposed for clinical assessment (55.6%), research (40.7%), health regulation policies (18.6%) and insurance service requirements (10.6%). Implementation was prompted by both departmental (43.4%) and institutional policy (34.5%). 89.4% of PROMs users reported difficulties in the use of PROMs, with major barriers including license fees, limited access, inadequate training and burden of fill-out time (all > 40%). Conclusion There is evidence of limited familiarity with knee and hip PROMs among orthopaedic surgeons. Barriers to their use are significant. Development of a Chinese language PROMs database would be helpful.

scholarly journals P494 Can Patients Monitor Response To Ustekinumab In The Real World?

2021 ◽  
Vol 15 (Supplement_1) ◽  
pp. S477-S478
Author(s):  
A J Walsh ◽  
L Matini ◽  
A Kormilitzin ◽  
J Wilson ◽  
S Lyden ◽  
...  
Keyword(s):  
Abdominal Pain ◽  
Real Time ◽  
Decision Support Tool ◽  
Well Being ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Support Tool ◽  
Patient Reported ◽  
Reported Data ◽  
Over Time

Abstract Background Real time monitoring of patients with Crohn’s disease (CD) gives us the opportunity to examine disease trajectory. We have demonstrated the feasibility of using a monitoring platform with patient reported data, collected prospectively and routinely in clinical practice. The question is whether it can be used for specific drugs Methods TrueColours-IBD (TC-IBD)is a real time, web based platform that through email prompts linked to questionnaires, collects longitudinal patient reported outcome measures (for CD, symptoms measured by Harvey Bradshaw Index (HBI) and quality of life by IBD Control-8). It is routinely used by >2000 patients in Oxford. This study examined 114 patients with Crohn’s: 45 males (median age 36, IQR 28–51) and 69 females (median age 34.8, IQR 28–47), treated with ustekinumab for a maximum of 16 months (range -2 months to 14 months). A linear mixed-effects model was used to approximate longitudinal trends of HBI and IBD Control-8. 3 individual items of the HBI scale (general well-being, abdominal pain & stool frequency) were also assessed. The TC-IBD platform has the capacity to analyse and show patients’ data automatically, using customised models and algorithms through the feedback loop (Fig 1). Model coefficients, intercept and slope were estimated using the restricted maximum likelihood (REML) approach. The intercept corresponds to the expected cohort mean value of the scale (e.g. HBI, IBD Control-8) at baseline (time zero) and the slope describes the rate of the change over time Results The models’ coefficients, the intercept and slope of both HBI and IBD Control-8 scales were statistically significant and are summarised in Table 1 with confidence intervals. Both linear trends (slopes) describing HBI (p<0.001) and IBD Control-8 (p<0.001) confirmed improvements for patients. The bowel movement component of the HBI dominated over general well-being and abdominal pain, while keeping a similar downward trend over time ( Fig 2 and Fig 3a,b) Conclusion Patient reported data through the TC-IBD platform can be used for statistical analyses and continuous monitoring of drug effect over time. This analysis is limited by a lack of comparator group, but the models may be used with the TrueColours platform as a decision support tool for other drugs

scholarly journals What matters to children with CFS/ME? A conceptual model as the first stage in developing a PROM

2015 ◽  
Vol 100 (12) ◽  
pp. 1141-1147 ◽  
Author(s):  
Roxanne Parslow ◽  
Aarti Patel ◽  
Lucy Beasant ◽  
Kirstie Haywood ◽  
Debbie Johnson ◽  
...  
Keyword(s):  
Focus Group ◽  
Conceptual Model ◽  
Well Being ◽  
Chronic Fatigue ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Fatigue Syndrome ◽  
Patient Reported ◽  
The Impact ◽  
The Relationship

BackgroundPaediatric chronic fatigue syndrome (CFS)/myalgic encephalomyelitis (ME) is relatively common and disabling. Research is hampered because current patient-reported outcome measures (PROMs) do not capture outcomes that are important to children with CFS/ME.AimThe aim of this study was to explore the aspects of life and health outcomes that matter to children with CFS/ME.MethodsTwenty-five children with CFS/ME were interviewed (11 males, 14 females; mean age 12.9 years (SD 2.2), range 8–17). Twelve were trial participants interviewed during the trial and 13 were recruited as part of a follow-up qualitative study. Parents were present in 19 interviews with their children. Three mothers participated in a focus group. All the interviews and the focus group were audio-recorded and transcribed. Data were analysed thematically using techniques of constant comparison. NVivo was used to structure and categorise data in a systematic way.ResultsChildren identified four key themes (health outcome domains): ‘symptoms’ that fluctuated, which caused an unpredictable reduction in both ‘physical activity’ and ‘social participation’ all of which impacted on ‘emotional well-being’. These domains were influenced by both ‘management’ and ‘contextual factors’, which could be positive and negative. The relationship between healthcare and school was considered pivotal.ConclusionsChildren's descriptions helped to inform a conceptual model that is necessary to develop a new paediatric CFS/ME PROM. Doctors need to be aware of how children conceptualise CFS/ME; the relationship between healthcare and school is fundamental to ameliorate the impact of CFS/ME.Trial registration numberISRCTN81456207.

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