Development and Content Validity of a Novel Myelodysplastic Syndromes Symptom Daily Diary

Abstract Background Myelodysplastic syndromes (MDS) are associated with the reduction in healthy blood cells produced in the bone marrow, which subsequently lead to a variety of symptoms. A review of existing patient-reported outcome (PRO) questionnaires used in MDS or acute myeloid leukemia found that few if any were developed to measure symptoms of higher-risk MDS in the context of clinical trials. An MDS symptom daily diary has been developed following conduct of a targeted review of peer-reviewed literature and advice meetings with three clinical experts who treat patients with higher-risk MDS. The primary goal of this study was to evaluate the content validity of the novel MDS symptom daily diary among adults with higher-risk MDS. Methods Qualitative interviews were conducted via telephone with 15 adults with higher-risk MDS. The interviews were composed of two parts. During the concept elicitation portion, participants were asked to spontaneously describe their experience of higher-risk MDS, with particular focus on the symptoms they experience and the impact on their lives. Participants then completed the MDS symptom daily diary and provided feedback on the instructions, items, and response options in order to evaluate its readability, comprehensibility, relevance, and comprehensiveness. Interviews were conducted in three waves. Following each wave, developers reviewed summaries of results based on detailed interviewer notes. Revisions to the daily diary were made based on interim results prior to the next wave of interviews. Following the completion of all interviews, audio-recordings of the interviews were transcribed, coded, and analyzed. Results A total of 15 participants (53.3% female, ages 36 - 81 [mean=66, standard deviation=11.3] years) completed an interview: 7 in Wave 1, 3 in Wave 2, and 5 in Wave 3. All had begun treatment with a hypomethylating agent within six months prior to screening or were treatment-naïve. Participants reported experiencing 20 different symptoms of higher-risk MDS; the most frequently reported symptoms were fatigue (n = 13, 86.6%), bruising (n = 11, 73.3%), shortness of breath (n = 11, 73.3%), lack of energy (n = 10, 66.6%), heaviness in the arms or legs (n = 9, 60.0%), weakness (n = 9, 60.0%), bleeding (n = 8, 53.3%), and bone pain (n = 4, 26.6%). Participants identified fatigue and lack of energy as the most bothersome symptoms and the most important to improve with treatment. Based upon participant feedback in Wave 1, two items were removed from the draft diary due to their overlap with other items. Additionally, one item was revised for clarity. Following Wave 2, a single item to assess shortness of breath was retained while alternate items assessing shortness of breath were removed based on participant feedback. No further revisions to the diary were made based on participant feedback in Wave 3. The final version of the MDS symptom daily diary consists of eight items assessing fatigue, weakness, lack of energy, shortness of breath, bruising, bleeding, bone pain, and heaviness in arms or legs. All participants interpreted the instructions, items, and response options as intended by the developers. Participants found the diary easy to complete and relevant to their experience, and no participants suggested removing any items from the final version. Conclusion The MDS symptom daily diary has been developed as a tool to characterize the symptom burden of MDS and to evaluate the efficacy of study medications in clinical trials. The qualitative interviews provide evidence of its content validity; in future research, the psychometric properties of the daily diary and interpretation of its scores will be evaluated using data from an upcoming Phase 3 clinical trial. Disclosures Vallow: Novartis Pharmaceuticals: Current Employment. Brandt: Novartis Pharmaceuticals: Current Employment.

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Symptoms, impacts, and suitability of the Pulmonary Arterial Hypertension – Symptoms and Impact (PAH-SYMPACT™) questionnaire in patients with chronic thromboembolic pulmonary hypertension (CTEPH): a qualitative interview study

Journal of Patient-Reported Outcomes ◽

10.1186/s41687-021-00327-9 ◽

2021 ◽

Vol 5 (1) ◽

Author(s):

Brooke Currie ◽

Evan Davies ◽

Amélie Beaudet ◽

Larissa Stassek ◽

Leah Kleinman

Keyword(s):

Pulmonary Hypertension ◽

Pulmonary Arterial Hypertension ◽

Arterial Hypertension ◽

Chronic Thromboembolic Pulmonary Hypertension ◽

Shortness Of Breath ◽

Response Options ◽

Qualitative Interview Study ◽

Thromboembolic Pulmonary Hypertension ◽

Pulmonary Arterial ◽

The Impact

Abstract Background Chronic thromboembolic pulmonary hypertension (CTEPH) is a rare form of pulmonary hypertension caused by blood clots and scar tissue in the blood vessels of the lungs. Health-related quality of life is often significantly impaired in patients with CTEPH. However, a better understanding of how CTEPH symptoms affect patients’ lives is needed to optimally assess the impact of the disease and treatment. Objectives This qualitative study aimed to better understand the symptoms of CTEPH and how they affect patients’ lives, as well as to determine the appropriateness of the Pulmonary Arterial Hypertension – Symptoms and Impact (PAH-SYMPACT™) questionnaire for use in this patient population. Methods Adults diagnosed with CTEPH, recruited from two clinical sites in the US, participated in one-to-one qualitative telephone interviews. They described their experience of CTEPH symptoms and the impact these symptoms have on their lives. They also provided feedback on the comprehensibility and relevance of the PAH-SYMPACT™‘s instructions, items, and response options. Results Participants (N = 12) had a mean age of 62.5 years. Two thirds were female and most (83%) had undergone pulmonary endarterectomy and/or balloon pulmonary angioplasty. The most frequently endorsed symptoms were shortness of breath (endorsed by all 12 participants), fatigue (11 participants), and lightheadedness (10 participants). All participants identified shortness of breath as an “extremely important” symptom, and seven participants rated fatigue as “extremely important.” The most frequent impacts of CTEPH were on ability to walk quickly (endorsed by all 12 participants), ability to walk up inclines or stairs (11 participants), and ability to carry things (11 participants). The PAH-SYMPACT™ items were relevant to most participants and reflected their experience of CTEPH. All participants indicated that no important CTEPH symptoms were missing from the PAH-SYMPACT™. Overall, the instructions, items, and response options of the PAH-SYMPACT™ were clear and easy to understand. Conclusions The symptoms and impacts experienced by patients with CTEPH align with items included in the PAH-SYMPACT™. The PAH-SYMPACT™ appears to be fit for purpose for assessing disease status in patients with CTEPH.

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Functional Assessment of Chronic Illness Therapy-Fatigue Scale (FACIT-Fatigue): Cognitive Debriefing in Patients with Chronic Lymphocytic Leukemia (CLL)

Blood ◽

10.1182/blood-2019-122849 ◽

2019 ◽

Vol 134 (Supplement_1) ◽

pp. 4777-4777

Author(s):

Helena Chung ◽

Daniel Eek ◽

Calvin Krogh ◽

Matthew Blowfield ◽

Oren Meyers ◽

...

Keyword(s):

General Population ◽

Content Validity ◽

Cognitive Debriefing ◽

Employment Equity ◽

Response Options ◽

Concept Elicitation ◽

The Past ◽

Fit For Purpose ◽

Equity Ownership ◽

The Impact

Introduction: The FACIT-Fatigue is a 13-item patient-reported outcome instrument (PRO) that was designed to assess fatigue-related symptoms and impacts on daily functioning. This study assessed the content validity of the FACIT-Fatigue in patients with CLL to help determine if it is fit-for-purpose in this population. Methods: Forty adults with first-line (1L) or relapsed or refractory (R/R) CLL took part in an interview study comprising concept elicitation and cognitive debriefing. All participants had experienced CLL-related symptoms (fatigue, weight loss, fever or night sweats) in the past week. For concept elicitation, interviewers used a standardized semi-structured interview guide with open-ended and prompted questions to explore patients' experiences of the signs, symptoms and impacts of CLL and its treatments. Patients were asked to provide a disturbance rating of these concepts on a 0-10 scale of severity. All interviewed patients completed the FACIT-Fatigue. Item scores can range from 0 ("not at all") to 4 ("very much"), and the total score from 0 to 52; lower scores indicate greater fatigue. The recall period for each item is the past 7 days. The general population mean score is 43 (Cella et al. 2002; Montan et al. 2018). During cognitive debriefing, the clarity of the FACIT-Fatigue items, their relevance to the patients, and the comprehensiveness of the response options were assessed. Interviews were carried out by telephone and lasted about 60-75 minutes. De-identified audio-recorded interviews were transcribed, coded and analyzed using qualitative data analysis software. Results: Median age of participants was 58 years (range: 28-73 years), and gender distribution was equal. During concept elicitation, fatigue was identified as the most prominent and impactful concept. It was the only concept mentioned by 100% of patients, and almost all (1L: 95%; R/R: 85%) mentioned it spontaneously without prompting. Fatigue had a high mean disturbance rating (1L: 7.1; R/R: 6.9). Seven fatigue-related sub-components were identified from the way that patients described their fatigue, covering symptoms (tiredness/need for sleep, lack of energy, weakness, cognitive fatigue) and impacts (decreased ability to maintain social/familial/professional role, decreased physical functioning, frustration). Mean (standard deviation) FACIT-Fatigue score was 28.9 (13.6) for patients with 1L CLL and 29.3 (11.5) for those with R/R CLL, indicating greater fatigue than the general population and providing further evidence that fatigue is a core component of CLL. The items that scored highest were: "I feel fatigued" (item 1; mean score: 2.4) and "I feel tired" (item 4; mean score: 2.4). The items that scored lowest were: "I am too tired to eat" (item 10; mean score: 0.4) and "I need help doing my usual activities" (item 11; mean score: 1.0). During cognitive debriefing, 100% of patients confirmed that the FACIT-Fatigue was reflective of their experiences with fatigue resulting from CLL. All patients found the response options provided by the FACIT-Fatigue to be sufficient. Patients confirmed that most of the FACIT-Fatigue terminology was clear. An exception was the item "I feel listless/washed out" (item 3), which was not consistently understood: most patients linked it to an absence of both physical and mental energy, but some interpreted it just as a lack of physical energy. Overall, patients considered the FACIT-Fatigue items to be relevant and distinct from each other, and found that the impact items captured both the mental and physical impacts of fatigue. One item, "I am too tired to eat", was not considered to be highly pertinent because, although respondents could imagine that some patients with CLL might be too tired to eat, they themselves could only recall being too tired to prepare a meal, not being too tired to eat. However, respondents felt that the item was still relevant and that it did not detract from the applicability or clarity of the FACIT-Fatigue. Conceptual relevance of the FACIT-Fatigue was supported by mapping of its items to the 7 fatigue-related sub-components identified during concept elicitation (Table). Conclusions: Results from concept elicitation and cognitive debriefing interviews demonstrated content validity of the FACIT-Fatigue in patients with CLL with 1L or R/R disease, thus providing evidence that it is fit-for-purpose in this population. Disclosures Chung: AstraZeneca: Employment, Equity Ownership. Eek:AstraZeneca: Employment, Equity Ownership. Eyre:Janssen: Honoraria; Gilead: Consultancy, Honoraria, Other: commercial research support; Abbvie: Honoraria; Roche: Honoraria.

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

Blood ◽

10.1182/blood-2019-125844 ◽

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.

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Development of a novel PRO instrument for use in familial chylomicronemia syndrome

Journal of Patient-Reported Outcomes ◽

10.1186/s41687-021-00347-5 ◽

2021 ◽

Vol 5 (1) ◽

Author(s):

David Davidson ◽

Christina Slota ◽

Montserrat Vera-Llonch ◽

T. Michelle Brown ◽

Andrew Hsieh ◽

...

Keyword(s):

Clinical Trials ◽

Content Validity ◽

Burden Of Illness ◽

Genetic Disorder ◽

Qualitative Interviews ◽

Psychometric Evaluation ◽

Patient Reported Outcome ◽

Regulatory Guidance ◽

Patient Reported ◽

Illness Study

Abstract Background Familial chylomicronemia syndrome (FCS), a rare genetic disorder characterized by high levels of circulating triglycerides, negatively impacts multiple organs, including the liver and pancreas. Objective The objective of this study was to develop and support the content validity of a novel patient-reported outcome (PRO) measure addressing FCS symptoms and impacts. To facilitate use in clinical trials of new treatments, evidence supporting the new measure needed to be consistent with regulatory guidance and requirements. Methods A pool of items addressing symptoms and impacts of FCS was initially developed based on data from a large burden-of-illness study with patients with FCS as well as a review of available literature and existing PRO measures. Two rounds of qualitative interviews were conducted with patients with FCS (N = 10) to refine the draft items and support the measure’s content validity. Each interview began with concept elicitation followed by cognitive debriefing of the draft FCS measure. Results Patient-reported symptoms and impacts of FCS were generally consistent with those identified by the burden-of-illness study; abdominal pain was particularly prevalent and salient for patients. Suggested changes to the draft item pool were generally minor. Comprehensibility and ease of completion for the final instrument were confirmed during the second set of interviews. Conclusion The content validity of the final 17-item FCS Symptoms and Impacts Scale is strongly supported by patient input gathered through both a large burden-of-illness study and qualitative research. To further support use in clinical trials, psychometric evaluation of the measure is underway.

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Evaluation of International Working Group 2006 Response Criteria in Patients with Higher-Risk Myelodysplastic Syndromes (HR-MDS) Treated with Hypomethylating Agent Monotherapy in the Frontline Setting

Blood ◽

10.1182/blood-2021-146331 ◽

2021 ◽

Vol 138 (Supplement 1) ◽

pp. 3701-3701

Author(s):

Jan Philipp Bewersdorf ◽

Wei Wei ◽

Anna Jaiani ◽

Prital Patel ◽

Rajni Mehta ◽

...

Keyword(s):

Clinical Trial ◽

Overall Survival ◽

Clinical Trials ◽

Myelodysplastic Syndromes ◽

Working Group ◽

Research Funding ◽

Response Criteria ◽

The Impact ◽

Support Research

Abstract Introduction: Monotherapy with hypomethylating agents (HMA) remains the standard of care for patients (pts) with myelodysplastic syndromes (MDS). Response in MDS is based on the modified International Working Group (IWG) 2006 criteria. Prior studies focusing on unselected MDS pts showed that achieving a complete remission (CR) was associated with favorable overall survival (OS). However, the association of other outcomes with OS was less clear and only 20% of HMA-treated MDS pts achieve a CR. For example, pts who achieved <5% bone marrow (BM) blasts are currently classified as marrow CR (mCR), which has not been associated with OS improvement. Therefore, interpreting the significance of mCR reported in various clinical trials is challenging. Clinically meaningful reduction in bleeding or infectious complications can occur at improvements in absolute neutrophil count (ANC) and platelet counts that do not meet the current thresholds used for CR (ANC ≥1.0 × 10 9/L, platelets ≥100 × 10 9/L, and Hb >11 g/dL). To avoid missing clinically meaningful benefits when studying new drugs in clinical trials, a clearly defined response criterion that is less stringent than CR but still captures clinically meaningful hematologic improvement (HI) is needed. Here we sought to evaluate the impact of current IWG 2006 response criteria as well as CRh on OS of pts with HR-MDS treated with frontline HMA monotherapy. Methods: We included all adult (≥18 years) MDS pts treated with frontline HMA (azacitidine [AZA], decitabine [DEC], or ASTX727) monotherapy between 1/1/2012 and 12/31/2020 at Yale University. We decided to use HMA monotherapy as it is the standard care for HR-MDS and to minimize the impact of therapy choice confounding the association of achieved response with OS. Pts were excluded if they received prior treatments for MDS aside from erythropoiesis-stimulating agents and if no baseline with at least one follow-up BM study were available for response assessment. We collected patient and disease characteristics (transfusion burden, IPSS/IPSS-R score, cytogenetics, molecular studies) at baseline. Best responses were assessed based on IWG 2006 criteria for MDS. We defined CRh as <5% BM blasts, platelets ≥50 × 10 9/L, ANC ≥0.5 × 10 9/L and no peripheral blood blasts. We followed pts until death or last follow-up and recorded dates of allogeneic hematopoietic cell transplant (HCT) if applicable. Date of data cut-off for survival status was 5/31/2021. We performed Kaplan-Meier analysis to estimate the duration of overall survival and we used log rank test to test the difference in OS between subgroups of pts. Multiple comparisons were adjusted using the Bonferroni method. Results: A total of 100 pts was included in this analysis (Table 1). Median age was 68 years (yrs; range, 23 - 86), 60% were males, and 79% and 18% of pts received AZA and DEC, respectively. Median number of HMA cycles was 6 (interquartile range [IQR]: 4-10), and 33 pts (33%) underwent HCT. During follow-up, 46 pts (48%) progressed to AML. At a median follow-up of 1.5 yrs (IQR: 0.9 - 2.3 yrs), median OS for the entire pt cohort was 1.9 yrs (Figure 1). OS by response category is shown in Table 2. Median OS was not reached for patients who achieved a CR (95% CI: not reached [NR] - NR) as compared to 1.9 yrs (95% CI: 1.5 yrs - NR) and 2.0 yrs (95% CI: 1.2 yrs - NR) among pts with mCR + HI and mCR without HI, respectively. Median OS among patients with stable disease (SD) was similar (2.0 yrs [95% CI: 1.5 yrs - NR]). Finally, we explored the prognostic value of CRh and found a median OS of 1.9 yrs (95% CI: 1.5 yrs - NR), which appeared comparable to mCR +/- HI or SD. Similar results were found with censoring at time of HCT (Figure 2). Discussion: In this retrospective analysis of MDS pts treated with HMA monotherapy in the frontline setting, achieving CR as best response was associated with improved OS compared with mCR +/- HI and SD. However, as the numbers were small these results should be interpreted with caution, and other clinically relevant outcomes such as freedom of transfusion, infectious or bleeding complications, and patient-reported outcomes were not captured in the current analysis. Our results also apply only to MDS pts treated with HMA monotherapy in the frontline setting. The prognostic implications of CRh need to be evaluated in larger patient cohorts. To overcome these limitations, we are currently in the process of expanding the study to a much larger multi-center, international analysis. Figure 1 Figure 1. Disclosures Neparidze: Eidos Therapeutics: Membership on an entity's Board of Directors or advisory committees; GlaxoSmithKline: Research Funding; Janssen: Research Funding. Shallis: Curis: Divested equity in a private or publicly-traded company in the past 24 months. Podoltsev: PharmaEssentia: Honoraria; Pfizer: Honoraria; CTI BioPharma: Honoraria; Blueprint Medicines: Honoraria; Incyte: Honoraria; Bristol-Myers Squib: Honoraria; Novartis: Honoraria; Celgene: Honoraria. Brunner: GSK: Research Funding; Aprea: Research Funding; Keros Therapeutics: Consultancy; Agios: Consultancy; AstraZeneca: Research Funding; Novartis: Consultancy, Research Funding; Acceleron: Consultancy; Takeda: Consultancy, Research Funding; BMS/Celgene: Consultancy, Research Funding; Janssen: Research Funding. Zeidan: AbbVie: Consultancy, Other: Clinical Trial Committees, Research Funding; Gilead: Consultancy, Other: Clinical Trial Committees; Epizyme: Consultancy; Amgen: Consultancy, Research Funding; BioCryst: Other: Clinical Trial Committees; Incyte: Consultancy, Research Funding; Boehringer Ingelheim: Consultancy, Research Funding; Cardiff Oncology: Consultancy, Other: Travel support, Research Funding; Acceleron: Consultancy, Research Funding; Agios: Consultancy; Novartis: Consultancy, Other: Clinical Trial Committees, Travel support, Research Funding; Genentech: Consultancy; Jasper: Consultancy; ADC Therapeutics: Research Funding; Jazz: Consultancy; Astex: Research Funding; Daiichi Sankyo: Consultancy; Kura: Consultancy, Other: Clinical Trial Committees; Aprea: Consultancy, Research Funding; BMS: Consultancy, Other: Clinical Trial Committees, Research Funding; Geron: Other: Clinical Trial Committees; AstraZeneca: Consultancy; Pfizer: Other: Travel support, Research Funding; BeyondSpring: Consultancy; Ionis: Consultancy; Loxo Oncology: Consultancy, Other: Clinical Trial Committees; Janssen: Consultancy; Astellas: Consultancy.

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Transformative Consciousness

Critical Social Work ◽

10.22329/csw.v21i1.6226 ◽

2020 ◽

Vol 21 (1) ◽

pp. 30-53

Author(s):

Alexis Jemal ◽

Sarah R. Bussey ◽

Keith Davis

Keyword(s):

Scale Development ◽

Content Validity ◽

Development Process ◽

Future Research ◽

Response Options ◽

Domain Specific ◽

The Social ◽

Measurement Tools ◽

The Impact ◽

Quantitative And Qualitative Methods

This paper outlines the development of the Transformative Consciousness (TC) of Oppression and Privilege (COAP) Scale created to address the conceptual and measurement limitations of the critical consciousness (CC) construct. Unlike prior CC measures, this scale was developed for the general population, integrates identities that are oppressed and privileged, incorporates the social-ecological framework, assesses cognitions, and attempts to reduce the impact of social desirability bias. Following DeVellis’ (2003) scale development process and Rubio and colleague’s (2003) process for content validity, this paper presents: a review of CC measurement literature and the lack of standardized conceptualization and measurement tools; an overview of TC theory; the COAP scale development process; and, a content validity study of COAP including quantitative and qualitative methods. An outgrowth of the first author’s conceptualization of Transformative Consciousness, the domain-specific and content validated COAP scale demonstrates a unique scaling method combining vignettes, sentence completions, rank ordering of response options, and scoring of ordering patterns. Limitations of the COAP scale and opportunities for future research are discussed.

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A qualitative study of barriers and facilitators to enrollment of adolescents and young adults onto cancer clinical trials at NCI community oncology research program (NCORP) sites.

Journal of Clinical Oncology ◽

10.1200/jco.2020.38.15_suppl.e19050 ◽

2020 ◽

Vol 38 (15_suppl) ◽

pp. e19050-e19050 ◽

Cited By ~ 1

Author(s):

Elizabeth Siembida ◽

Holli Ann Loomans-Kropp ◽

Irene Tami-Maury ◽

Lillian Sung ◽

Brad H. Pollock ◽

...

Keyword(s):

Clinical Trials ◽

Young Adults ◽

Qualitative Interviews ◽

Community Setting ◽

Barriers And Facilitators ◽

Adolescents And Young Adults ◽

Cancer Clinical Trials ◽

Stakeholder Views ◽

Oncology Research ◽

The Impact

e19050 Background: Cancer clinical trials (CCTs) contribute to improving patient survival and quality of life; however, adolescents and young adults (AYAs, 15-39 years old), are underrepresented in CCTs, especially in the community setting. We aimed to identify barriers and facilitators to AYA CCT enrollment in the NCORP. Methods: We conducted 43 one-on-one semi-structured qualitative interviews with key stakeholders involved in the enrollment of AYAs across a diverse group of NCORP primary (n = 5) and affiliate (n = 10) sites. Interviews were conducted remotely by 3 trained interviewers using the Zoom platform. Stakeholders were recruited from high and low AYA enrolling sites (AYA/total site enrollments > 10% and < 3%, respectively). Stakeholders were overall NCORP Site PIs (n = 5), lead NCORP administrators (n = 4), clinical research associates (n = 11), medical and pediatric oncologists involved in the enrollment of AYAs (n = 7), regulatory research associates (n = 5), nurse navigators (n = 6), and patient advocates (n = 5). Interviews were audiotaped and transcribed. Thematic analysis was conducted to identify themes and relate them back to our primary research questions regarding barriers and facilitators to AYA CCT enrollment. Results: Stakeholder views on enrollment barriers centered on 5 main themes: (1) lack of site-level prioritization or discussion of AYA enrollment; (2) limited number of clinical trials for AYAs available nationally, with few trials opened locally; (3) insufficient resources and research staff; (4) concerns about the cost effectiveness of opening AYA trials due to low numbers of eligible patients; and (5) patient misconceptions about CCTs. Stakeholder views on enrollment facilitators centered on 3 main themes: (1) presence of an AYA program focused on increasing enrollment; (2) having a designated site AYA “champion”; and (3) having site leadership identify AYA enrollment as a priority. Stakeholders agreed that incentivizing AYA enrollments via increased reimbursement and/or study credits could potentially lead to increased enrollment. Conclusions: In addition to identifying multiple shared barriers to AYA CCT enrollment, our study also identified possible interventions for enrollment improvement, including designation of AYA “champions”, increased reimbursement for AYA enrollments, and improving AYA’s understanding of CCTs. Further studies are needed to assess the impact of interventions aimed at increasing AYA enrollment across the NCORP.

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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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The myelodysplastic syndromes: biology and implications for management.

Journal of Clinical Oncology ◽

10.1200/jco.1990.8.8.1424 ◽

1990 ◽

Vol 8 (8) ◽

pp. 1424-1441 ◽

Cited By ~ 60

Author(s):

A F List ◽

H S Garewal ◽

A A Sandberg

Keyword(s):

Clinical Trials ◽

Myelodysplastic Syndromes ◽

Randomized Clinical Trials ◽

Prolonged Treatment ◽

Clinical Aspects ◽

Hematopoietic Growth Factors ◽

Hematopoietic Stem ◽

Maturation Defect ◽

Extensive Body ◽

The Impact

Since the initial efforts to characterize the myelodysplastic syndromes in 1976, an extensive body of information has accumulated defining biologic features and the relation to clinical aspects of disease. While the pathogenesis of these disorders remains incompletely understood, laboratory investigations indicate that they are clonal disorders affecting hematopoietic stem cells characterized by a progressive imbalance between self-renewal and differentiation. Despite karyotypic resemblance to acute myeloid leukemia, fundamental biologic differences may underly the disappointing results achieved to date with intensive chemotherapy. The recent availability of recombinant hematopoietic growth factors for use in clinical trials has shown that the maturation defect in many instances can be overcome with administration of lineage-restricted recombinant hematopoietins. Routine use of these promising agents must await results of randomized clinical trials to determine the impact of prolonged treatment on leukemic evolution and disease-related morbidity.

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MO555ASSESSMENT OF HEALTH-RELATED QUALITY OF LIFE (HRQOL) MEASURES FOR PAEDIATRIC PATIENTS WITH ANAEMIA OF CHRONIC KIDNEY DISEASE (CKD)

Nephrology Dialysis Transplantation ◽

10.1093/ndt/gfab085.0018 ◽

2021 ◽

Vol 36 (Supplement_1) ◽

Author(s):

Antonia Morga ◽

Ana Filipa Alexandre ◽

Patricia Koochaki ◽

Alexandros Georgiadis ◽

Celine Desvignes-Gleizes

Keyword(s):

Quality Of Life ◽

Clinical Trials ◽

Conceptual Model ◽

Daily Living ◽

Product Labelling ◽

Shortness Of Breath ◽

Quantitative Studies ◽

Paediatric Patients ◽

The Impact

Abstract Background and Aims Anaemia is a common complication of CKD in children that is associated with adverse clinical outcomes, including poor physical functioning for patients and lower HRQoL for patients and caregivers.1 Given the subjective nature of the disease, instruments that measure patient- and/or observer-reported outcomes (PROs/ObsROs), by collecting information about symptom severity and HRQoL, could be of value to assess treatment benefit. We determined which aspects of anaemia of CKD and its treatments are important for paediatric patients and their caregivers, and examined which PRO/ObsRO instruments comprehensively capture patients’ and caregivers’ experiences. Method A targeted literature review and database search were performed to find a) qualitative and quantitative studies of symptoms and related health outcomes in paediatric patients with anaemia of CKD; b) related clinical practice guidelines; and c) HRQoL information in product labelling. Study outcomes were used to develop a structured representation of health outcomes concepts and issues (‘conceptual model’) capturing the symptoms, perceptions of treatment and impact on HRQoL of anaemia of CKD, from the perspective of both paediatric patients and their caregivers. PRO and ObsRO instruments were identified from published quantitative studies and ongoing clinical trials, and a selection was then critically assessed to determine their content validity and whether their psychometric properties adequately covered the conceptual model domains. Results The conceptual model captured shortness of breath, fatigue and headaches as the most important symptoms experienced by paediatric patients with anaemia of CKD. Concerns regarding disease management included medical interventions, fear associated with interventions and the need for information about the disease. Disease symptoms and their management impacted seven HRQoL domains, namely physical, emotional and social functioning; activities of daily living; and effects on family, work and caregivers (Figure). The quantitative search of published studies identified 20 unique instruments; of these, the most frequently used was Pediatric Quality of Life Inventory™ (PedsQL) Version 4.0. Ongoing clinical trials used 14 unique instruments, of which only EQ-5D-Y was used in more than one study. Two clinical practice guidelines (National Institute for Health and Care Excellence and Kidney Disease: Improving Global Outcomes) supported measurements of HRQoL, symptoms, patient preferences and school attendance/performance, but they did not advocate use of any specific instruments. No HRQoL product labelling information was identified. Based on the conceptual model, the literature review results and age group coverage, five instruments were selected for critical appraisal: PedsQL, PedsQL Infant Scales, PedsQL Multidimensional Fatigue Scale, PedsQL v3.0 End Stage Renal Disease module, and EQ-5D-Y. Between them, these instruments provided complete coverage of the impact of fatigue and headaches, and partial coverage of the effects on physical functioning, activities of daily living, emotional functioning and social functioning. None of these instruments measured the impact of shortness of breath, and there was no coverage of the consequences for work or the family’s/caregivers’ HRQoL. Conclusion Anaemia of CKD affects many HRQoL domains for paediatric patients and their caregivers. While current PRO/ObsRO instruments partially address these impacts, no single instrument in our assessment measured all symptoms and domains of interest to patients and caregivers. Multiple instruments should be included in clinical studies to capture symptoms and HRQoL important to patients, and adequate measurement of the effects of anaemia on work or the family’s/caregivers’ HRQoL might require development of a new instrument.

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