Evaluating Virtual Patient Interaction Fidelity With Advanced Communication Skills Learners

Previous research in educational medical simulation has drawn attention to the interplay between a simulation’s fidelity and its educational effectiveness. As virtual patients (VPs) are increasingly used in medical simulations for education purposes, a focus on the relationship between virtual patients’ fidelity and educational effectiveness should also be investigated. In this paper, we contribute to this investigation by evaluating the use of a virtual patient selection interface (in which learners interact with a virtual patient via a set of pre-defined choices) with advanced medical communication skills learners. To this end, we integrated virtual patient interviews into a graduate-level course for speech-language therapists over the course of 2 years. In the first cohort, students interacted with three VPs using only a chat interface. In the second cohort, students used both a chat interface and a selection interface to interact with the VPs. Our results suggest that these advanced learners view the selection interfaces as more appropriate for novice learners and that their communication behavior was not significantly affected by using the selection interface. Based on these results, we suggest that selection interfaces may be more appropriate for novice communication skills learners, but for applications in which selection interfaces are to be used with advanced learners, additional design research may be needed to best target these interfaces to advanced learners.

Psycho-social experience of oocyte recipient women: a qualitative study

Background Although associated with many successes, oocyte donation can lead to numerous psychological challenges in recipient women. The identification of these challenges during the treatment process is crucial to improve recipient mental health. Thus, the aim of this study was to gain an understanding of the experiences of oocyte recipient women. Methods This research was conducted using a qualitative approach and inductive content analysis method. The data collection tool was in-depth interviews. Twenty women with the experience of receiving donated oocyte were selected and entered the study using purposive sampling method and considering the maximum variation. Results Three main categories of psychological challenges were extracted from patient interviews, specifically, distressing psychologic symptoms, social stigmatization, and negative coping mechanisms. The category of distressing psychologic symptoms was shaped based on the subcategories of self-esteem destruction, anxiety and stress, depression and spiritual discouragement. The category of social stigmatization included the subcategories of concern about disclosure, judgment of others, and conflict with religious teachings. And the category of negative coping mechanisms was formed based on the subcategories of aggression and denial. Conclusion The results indicated that the process of treatment with donated oocyte is followed by the experiences of distressing psychologic symptoms, social stigmatization, and negative coping mechanisms in recipient women. As such, paying attention to the socio-cultural factors which affect this process seems necessary to maintain the mental health of these women. Plain English summary Although associated with many successes, oocyte donation can lead to numerous psychological challenges in recipient women. The aim of this study was to gain an understanding of the experiences of oocyte recipient women. This research was conducted using a qualitative approach and inductive content analysis method. The data collection tool was in-depth interviews. Twenty women with the experience of receiving donated oocyte were selected and entered the study using purposive sampling method and considering the maximum variation. Three main categories of psychological challenges were extracted from patient interviews, specifically, distressing psychologic symptoms, social stigmatization, and negative coping mechanisms. The category of distressing psychologic symptoms was shaped based on the subcategories of self-esteem destruction, anxiety and stress, depression and spiritual discouragement. The category of social stigmatization included the subcategories of concern about disclosure, judgment of others, and conflict with religious teachings. And the category of negative coping mechanisms was formed based on the subcategories of aggression and denial. The results indicated that the process of treatment with donated oocyte is followed by experience of distressing psychologic symptoms, social stigmatization, and negative coping mechanisms in recipient women. As such, paying attention to the socio-cultural factors which affect this process seems necessary to maintain the mental health of these women.

Symptoms and Impacts Reported By Patients with Acute Myeloid Leukemia (AML) in Remission Post-Stem Cell Transplant

Background Little is known about the burden of symptoms and impacts on quality of life in patients with AML in the posttransplant period. As treatment options for AML increase, it is vital that the patient experience be considered when making treatment decisions. Qualitative methods, largely underutilized in hematologic malignancy research, help capture the patient voice and provide insights into signs, symptoms, and impacts that are most relevant to the patient experience and yield valid results with small sample sizes. Aim/Objective To gain a deeper understanding of the patient experience in the AML posttransplant period, we conducted patient interviews to identify the most bothersome symptoms and impacts on patients' lived experiences with AML. Methods Patients diagnosed with AML and currently in remission ≥90 days posttransplant and <1 year from the transplant date were recruited for the study. The number of patients interviewed was estimated based on the projected number of patients needed to reach concept saturation (ie, the point at which no new symptoms or concepts are identified during patient interviews). An interview guide was developed that included a list of bothersome symptoms and life impacts that were identified during a prior study enrolling patients with relapsed/refractory AML and refined for the posttransplant population based on results from a literature review and discussions with clinicians. One-on-one concept elicitation interviews were conducted via telephone in 4 waves. Patient disease experiences were explored, particularly those symptoms and impacts most important to patients during the posttransplant period. Interviews lasted 60 to 90 minutes and were conducted in line with the International Society for Pharmacoeconomics and Outcomes Research Good Research Practices Task Force recommendations. Interviewers asked a series of open-ended questions to elicit spontaneous responses and included prompts to probe patient understanding and experiences in greater depth. When interviewed, patients were asked to rate their symptoms/impacts on a scale of 0 to 10, with 10 being the most bothersome/impactful. Salient symptoms and impacts were those reported by ≥50% of respondents as relevant either spontaneously or upon probing and rated most impactful (average rating of ≥5). Results Twenty patients who were in complete remission posttransplant were interviewed. Median age of patients was 59.5 years and 55% of patients were female. Seventeen patients were confirmed FLT3-mutation-positive. Symptom saturation was reached in wave 1 (42 total concepts), with 13 new symptoms identified. Impact saturation was reached in wave 2 (28 total concepts), with 12 new impacts identified. During interviews, patients mentioned both positive and negative impacts. Mean peak symptom scores are shown in Figure A. Salient symptoms identified were fatigue, weakness, nausea, pain, and diarrhea. The disturbance ratings for most salient symptoms decreased from the peak to the current rating, suggesting that symptoms were less bothersome over time. Mean peak impact scores are shown in Figure B. Salient impacts included 1 positive impact (life outlook) and 6 negative impacts (fear, decreased ability to maintain roles, anxiety, appetite loss, decreased ability to function, and change in appearance). Impacts were generally more stable over time than symptoms in terms of their disturbance to patients. Conclusions Bothersome symptoms and impacts continued in patients with AML in the posttransplant period. In general, most salient symptoms were less bothersome over time. Conversely, fear, anxiety, decreased ability to maintain familial roles, decreased ability to function, and change in appearance remained impactful during the posttransplant period, suggesting that fears/concerns about relapse and disease sequelae persist in the posttransplant period. Patients did report positive changes on life outlook, possibly reflecting completion of a stem cell transplant, a potentially curative therapy. The number of salient impacts suggests that while drugs prolong remission and decrease symptoms, emotional impacts continue and are central to the patient experience posttransplant. Figure 1 Figure 1. Disclosures Cella: FACIT: Membership on an entity's Board of Directors or advisory committees. LeBlanc: Otsuka: Consultancy, Honoraria, Other; AbbVie: Consultancy, Honoraria, Other: Advisory board; Travel fees, Speakers Bureau; Agios: Consultancy, Honoraria, Other: Advisory board; Travel fees, Speakers Bureau; BMS/Celgene: Consultancy, Honoraria, Other: Travel fees, Research Funding, Speakers Bureau; Daiichi-Sankyo: Consultancy, Honoraria, Other: Advisory board; Duke University: Research Funding; NINR/NIH: Research Funding; Amgen: Consultancy, Other: travel; Jazz Pharmaceuticals: Research Funding; UpToDate: Patents & Royalties; Seattle Genetics: Consultancy, Other: Advisory board, Research Funding; Astellas: Consultancy, Honoraria, Other: Advisory board; Helsinn: Consultancy, Research Funding; AstraZeneca: Consultancy, Honoraria, Other: Advisory board, Research Funding; Heron: Consultancy, Honoraria, Other: advisory board; CareVive: Consultancy, Other, Research Funding; Flatiron: Consultancy, Other: Advisory board; Pfizer: Consultancy, Other: Advisory Board; American Cancer Society: Research Funding. Shah: Astellas Pharma, Inc.: Current Employment; University of Michigan School of Public Health Department of Health Management and Policy Alumni Board: Other: Chair-Elect. de la Motte: Astellas Pharma, Inc.: Consultancy. Toublan: Astellas Pharma, Inc.: Consultancy. Kelly: Astellas Pharma, Inc.: Consultancy.

Mavorixafor, an Oral CXCR4 Antagonist, for Treatment of Patients with WHIM Syndrome: Results from the Long-Term Extension of the Open-Label Phase 2 Study

Background: WHIM (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis) syndrome is a rare, autosomal-dominant primary immunodeficiency. Gain-of-function (GOF) mutations in the CXCR4 gene are the most common cause of WHIM syndrome, manifesting as panleukopenia with severe neutropenia, lymphopenia, and monocytopenia, recurrent bacterial infections, unusual susceptibility to human papillomavirus infections with intractable mucocutaneous warts, and increased risk of malignancy (McDermott DH, et al. Immunol Rev. 2019;2878:91-102). Mavorixafor is an investigational, small-molecule, selective antagonist of the CXCR4 receptor being developed as an oral, once-daily (QD) treatment for patients with WHIM syndrome (Dale DC, et al. Blood. 2020;136(26):2994-3003). Objective: We present an update on the clinical outcomes of patients with WHIM syndrome who continued in the long-term extension of the phase 2 study, highlighting long-term safety and efficacy. Methods: A long-term extension is ongoing as part of the open-label, prospective, dose-escalation, phase 2 study evaluating the safety and efficacy of mavorixafor (NCT03005327) in adults with WHIM syndrome. Individuals with a pathogenic GOF CXCR4 mutation and absolute neutrophil count ≤400/μL and/or absolute lymphocyte count ≤650/μL were included. All provided written informed consent. The primary objectives were to evaluate safety and tolerability and assess safe dosage. Exploratory efficacy end points included changes in infection rates, number of cutaneous warts, and white blood cell counts, compared to baseline. Researchers completed detailed interviews of 4 participants continuing in the study to assess their overall study experience and perceived treatment effects. Results: Five of 8 patients in the dose-finding phase 2 study entered into the long-term extension (LTE); median treatment duration was 148.4 weeks. One patient left the LTE because of study fatigue, and all 5 patients had dose escalation to 400 mg oral QD as of May 2021. As of the November 2020 data cutoff, annualized infection rates decreased from 5.6/year at study baseline to 2.2/year at 40 months' treatment, providing evidence of persistent reduction of infections over time. At doses of 300 and 400mg QD (n=7), the mean time above threshold for ANC and ALC were 12.7 hours (SD ± 9.8) and 16.9 hours (SD ± 5.9) compared to 2.1 hours (SD ± 3.3) and 11.5 hours (SD ± 5.9) at doses ≤200 mg QD, respectively. One patient experienced a 79% reduction in warts. Safety data review at May 2021 showed that there were 12 minor treatment-emergent adverse events (grade 1) with long-term treatment (46 months), no treatment-related infections of grade 3 or higher, no treatment-related serious adverse events, and no clinically significant laboratory abnormalities with mavorixafor treatment. Patient interviews revealed that all 4 LTE participants experienced good tolerability of mavorixafor and decreased frequency, severity, and duration of infections and decreased hospital/doctor visits. Three of 4 participants reported previous need for prophylactic treatment to prevent infection of minor wounds, but with mavorixafor, minor wounds healed without infection or need for prophylaxis. The mechanism of action of mavorixafor was of interest to 3 of 4 participants, who found it important that treatment address the underlying cause of disease, not simply the symptoms. Two participants reported a QOL improvement, and the other 2 reported that WHIM syndrome never affected their QOL Conclusion: Ongoing long-term treatment of adults with WHIM syndrome with mavorixafor 300 to 400mg shows durable increase in neutrophils and lymphocytes and sustained improvements in infections and warts. Detailed patient interviews for patient global impression of changes are consistent with sustained clinical benefit of long-term treatment. Mavorixafor has the potential to be a safe, effective, and long-term therapy targeting the underlying cause of WHIM syndrome. A global phase 3 registrational study is ongoing. Disclosures Dale: X4 Pharmaceuticals: Consultancy, Honoraria, Research Funding. Firkin: X4 Pharmaceuticals: Research Funding. Bolyard: X4 Pharmaceuticals: Research Funding. Tang: X4 Pharmaceuticals: Current Employment, Current equity holder in publicly-traded company. Jiang: X4 Pharmaceuticals: Current Employment, Current equity holder in publicly-traded company. MacLeod: X4 Pharmaceuticals: Current Employment, Current equity holder in publicly-traded company. Cadavid: X4 Pharmaceuticals: Current Employment, Current equity holder in publicly-traded company. Hu: X4 Pharmaceuticals: Current Employment, Current equity holder in publicly-traded company.

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

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.

QOLP-06. THE CHALLENGE OF FACING A NEW MALIGNANT GLIOMA DIAGNOSIS: PATIENT EXPERIENCES LEARNING ABOUT THEIR ILLNESS AND COMMUNICATING WITH THEIR PROVIDERS IN THE NEURO-ONCOLOGY CLINIC

BACKGROUND Patients with newly diagnosed malignant gliomas (MG) face the sudden and unexpected news that they have a terminal neurological illness. However, little is known about these patients’ experiences learning about their illness and communicating with their oncology providers. METHODS We conducted semi-structured interviews with patients with MG who had recently completed chemoradiation, to explore patient-clinician communication and evaluate how patients process information about their diagnosis. We excluded patients with marked cognitive dysfunction, with eligible patients required to have a Mini-Mental Status Exam score of 24/30 or greater. We terminated enrollment once we reached thematic saturation, after a total of 9 patient interviews. We thematically analyzed qualitative data to describe patterns common to the patients’ experiences. RESULTS Most patients were male (6/9, 66.7%), with median age of 55 years (range 26-72) and had a diagnosis of glioblastoma (6/9, 66.7%). The majority of patients described a positive experience communicating with their providers, noting that information was delivered clearly, and visits were not rushed. Despite this, patients found it difficult to absorb and process the large volume of new information, particularly in the setting of their shock and dismay about the diagnosis and their ongoing neurological deficits. Emergent themes included concerns about the impact of the disease on their loved ones, struggles in coping with uncertainty, and worries about preserving their quality of life and functioning. Additionally, patients described their efforts to balance their knowledge about their poor prognosis with their desire to maintain hope, with many patients expressing a goal to “beat the odds” or exceed expectations with respect to their survival. CONCLUSION Although patients with newly diagnosed MG describe effective communication with their providers, they face many challenges coping with their terminal diagnosis. Supportive interventions aimed at this population are needed.

Clinical and Imaging Characteristics of Cancer Patients with COVID-19: A Pilot Study

Background: Malignancy is a known risk factor of coronavirus disease 2019 (COVID-19) severe involvement. Information about this infection in patients with cancer is limited. Objectives: This study aimed at reporting the clinical and imaging characteristics of COVID-19 infection in patients with cancer. Methods: All the patients were known cases of a solid tumor with COVID-19 infection in one center, between February and May 2020. Clinical presentation and imaging involvement of COVID-19 infection in addition to cancer features were documented from medical records/patient interviews. Results: Thirty-one patients with solid tumors and COVID-19 involvement were included. The most prevalet presentation was fever, cough, and myalgia. Breast and gastrointestinal malignancies were the most common cancer types. The mortality rate was 22.5% and all deceased patients suffered from stage 4 of their underlying cancer disease. Lung computed tomography scan (CT scan) features in these patients were not different from the non-cancer patients with COVID-19. Conclusions: COVID-19 involvement in patients with cancer seems to be more severe with higher mortality rates especially in patients with other comorbidity and in metastatic cases. Treatment modifications during the pandemic era sound to be logical in decreasing the infection rate.

The patient experience of Wilson disease: a conceptual model based on qualitative research

Background Wilson disease (WD) is a rare disease wherein copper accumulates in tissues, leading to hepatic degeneration, neurological impairments, and psychiatric symptoms. This study aimed to characterize the patient experience of WD and develop a conceptual model containing key symptoms and impacts of the disease. Results A targeted literature review was conducted to develop a preliminary conceptual model of WD that was subsequently refined through one-on-one interviews with 3 WD clinicians and finalized following concept elicitation interviews with 11 patients and 1 caregiver. The literature review returned 30 articles, from which 45 concepts (35 signs/symptoms and 10 impacts) were selected for inclusion in the preliminary conceptual model. After interviews with clinicians, the model was expanded to include 45 signs/symptoms and 14 impacts. The final comprehensive conceptual model developed after interviews with patients included 54 symptoms in total (n = 22 hepatic, n = 19 neurological, n = 13 psychiatric), and 21 impacts. Across symptoms, patients reported a high level of bother, with approximately 49% of symptoms reported by patients having an average peak bother rating of ≥ 7 out of 10 (10 = most bothersome). Patient interviews identified 2 subgroups of patients: those who experience neurological, psychiatric, and hepatic symptoms and those who experience mostly hepatic and some psychiatric symptoms, but no neurological symptoms. Conclusions This research underscores the substantial multisystemic symptoms and impacts that patients with WD describe as highly bothersome in their lives. Hepatic symptoms emerged as especially common and important to patients with WD, possibly beyond what is commonly understood in research and clinical practice. Further, the description of 2 distinct patient groups may help to inform patient management and support more targeted drug development processes.

At the Heart of It All: Emotions of Consequence for the Conceptualization of Caregiver-Reported Outcomes in the Context of Colorectal Cancer

Colorectal cancer (CRC) can be demanding for primary caregivers; yet, there is insufficient evidence describing the caregiver-reported outcomes (CROs) that matter most to caregivers. CROs refer to caregivers’ assessments of their own health status as a result of supporting a patient. The study purpose was to describe the emotions that were most impactful to caregivers of patients with CRC, and how the importance caregivers attribute to these emotions changed from diagnosis throughout treatment. Guided by qualitative Interpretive Description, we analyzed 25 caregiver and 37 CRC patient interviews, either as individuals or as caregiver-patient dyads (six interviews), using inductive coding and constant comparative techniques. We found that the emotional aspect of caring for a patient with CRC was at the heart of caregiving. Caregiver experiences that engendered emotions of consequence included: (1) facing the patient’s life-changing diagnosis and an uncertain future, (2) needing to be with the patient throughout the never-ending nightmare of treatment, (3) bearing witness to patient suffering, (4) being worn down by unrelenting caregiver responsibilities, (5) navigating their relationship, and (6) enduring unwanted change. The broad range of emotions important to caregivers contributes to comprehensive foundational evidence for future conceptualization and the use of CROs.

Qualitative interviews to improve patient-reported outcome measures in late-onset Pompe disease: the patient perspective

Background Late-onset Pompe Disease (LOPD) is a rare, heterogeneous disease manifested by a range of symptoms varying in severity. Research establishing the frequency of these symptoms and their impact on patients’ daily lives is limited. The objective of this study was to develop a conceptual model that captures the most relevant symptoms and functional limitations experienced by patients with LOPD, to inform the development of new patient-reported outcome (PRO) tools. Methods A preliminary conceptual model was constructed following a literature review and revised through interviews with expert clinicians to identify important and relevant concepts regarding symptoms and impacts of LOPD. This preliminary model informed the development of a qualitative patient interview guide, which was used to gather the patient perspective on symptoms and impacts relating to LOPD or its treatment (including symptom/impact frequency and levels of disturbance). Patient interviews aided further refinement of the conceptual model. The findings from the patient interviews were triangulated with the literature review and clinician interviews to identify the most relevant and significant effects of LOPD from the patient perspective. Results Muscle weakness, fatigue, pain, and breathing difficulties (especially while lying down) were the most common and highly disturbing symptoms experienced by patients. Limitations associated with mobility (e.g., difficulty rising from a sitting position, getting up after bending) and activities of daily living, (e.g., reduced ability to participate in social/family activities or work/study) were the most frequently reported impacts with the highest levels of disturbance on the patient’s daily life. These identified symptoms and impacts were included in the new conceptual model of disease. Conclusions This qualitative patient interview study, also informed by a literature review and clinician interviews, identified the most frequent and relevant symptoms and the functional impact of LOPD on patients. The study interviews also captured the patient-preferred language to describe symptoms and impacts of LOPD. The results from this study can be used to develop future PRO instruments that are tailored to the specific symptoms and impacts experienced by patients with LOPD.