Questionnaire-based screening of adolescents and young adult women can identify markers associated with endometriosis.

STUDY QUESTION: Do adolescents and young adult women (YAW) with histologically proven endometriosis present a specific clinical history? SUMMARY ANSWER: Questionnaire screening of adolescents and YAW can identify clinical markers associated with histologically proven endometriosis. WHAT IS KNOWN ALREADY: Some validated questionaries can contribute to an earlier endometriosis diagnosis in adults. None of these scores, however, have been validated for adolescents or YAW. STUDY DESIGN, SIZE, DURATION: This was an observational cross-sectional study using prospectively recorded data performed between January 2005 and January 2020 in a single university tertiary referral centre for endometriosis diagnosis and management. After a thorough surgical examination of the abdomino-pelvic cavity, women with histologically proven endometriosis were allocated to the endometriosis group, and symptomatic women without evidence of endometriosis were allocated to the endometriosis-free control group. The endometriotic patients were allocated into two sub-groups according to their age: adolescent (≤20 years) and YAW (21-24 years). PARTICIPANTS/MATERIALS, SETTING, METHODS: Adolescents and YAW ≤24 years of age were operated for a symptomatic benign gynaecological condition with signed informed consent. A standardized questionnaire was prospectively completed in the month before the surgery and included epidemiological data, pelvic pain scores, family history of endometriosis, and symptoms experienced during adolescence. The study searched for correlations by univariate analysis to determine clinical markers of endometriosis in adolescents and YAW compared with endometriosis-free control patients. MAIN RESULTS AND THE ROLE OF CHANCE: Of the 262 study participants, 77 women were adolescents (≤20 years of age) and 185 patients (70.6%) were YAW. The endometriosis group included 118 patients (45.0%) and 144 (55.0%) were assigned to the control group. A family history of endometriosis, absenteeism from school during menstruation, history of fainting spells during menstruation, and prescription of oral contraceptive pills for intense dysmenorrhea were significantly more frequently observed in the endometriotic patients. The prevalence and mean pain scores for dysmenorrhea, deep dyspareunia, non-cyclic chronic pelvic pain and gastrointestinal and lower urinary tract symptoms were significantly greater in the endometriosis group, as was experienced rectal bleeding. LIMITATIONS, REASONS FOR CAUTION: The study was performed in a single referral centre that treats patients with potentially more severe disease. This questionnaire was evaluated on a population of patients with an indication for endometriosis surgery, which can also select patients with more severe disease. Women with asymptomatic endometriosis were not considered in this study. These factors can affect the external validity of this study. WIDER IMPLICATIONS OF THE FINDINGS: Patient interviews are relevant to the diagnosis of endometriosis in adolescents and YAW. Combined with imaging and clinical examination, this approach will enable earlier diagnosis and treatment, while remaining non-invasive and rapid. STUDY FUNDING/COMPETING INTEREST(S): The study received no funding from external sources. There are no conflicts of interest. TRIAL REGISTRATION NUMBER: N/A.

Patient perceptions and experiences of dental fear of different dental specialties: a mixed-method study.

OBJECTIVE: The primary objective of this study was to validate an Urdu translation of Kleinknecht's Dental Fear Survey (DFS) for use in Pakistan and to explore which items contribute the most to the variance in dental fear scores based on patient perceptions and lived experiences during dental care. METHODOLOGY: This mixed-method study was conducted at Dow Dental Hospital from February 2022 to June 2022. For quantitative analysis, a total of 273 participants were enrolled through convenience sampling. After obtaining signed consent, participants were asked to self-report their dental fear. In-depth interviews with 25 patients displaying moderate to high dental fear were conducted to clarify the elements of dental fear scores through the lens of individual perceptions and experiences. RESULTS: The prevalence of moderate dental fear was significantly higher among female participants than males. The mean dental fear score was higher among females (39.47 ± 14.23) as compared to males (30.83 ± 10.50). Most of the female participants reported an increase in breathing rate and heartbeat during dental treatment. The highest mean fear score was reported by participants who underwent oral surgical treatment (42.98 ± 14.21), followed by participants who received restorative care (36.20 ± 12.60). Approaching the dentist's office was the significant factor that contributed the most to the variance in dental fear scores. Four themes were generated through the content analysis of the interviews: physical reactions to dental procedures, perceptions and fears about surgical and restorative procedures, and gender and environmental factors in dental fear and interaction with dentists. CONCLUSION: The Urdu translation of DFS is a reliable and valid instrument for assessing dental fears in Pakistan based on the findings of this study. Patients perceive surgical and restorative procedures as unpleasant and threatening. It was noted that "the heart beats faster" and "the breathing rate increases." were the top two physiological responses.

How do studies assess the preventability of readmissions? A systematic review with narrative synthesis.

BACKGROUND: A large number of articles examined the preventability rate of readmissions, but comparison and interpretability of these preventability rates is complicated due to the large heterogeneity of methods that were used. To compare (the implications of) the different methods used to assess the preventability of readmissions by means of medical record review. METHODS: A literature search was conducted in PUBMED and EMBASE using "readmission" and "avoidability" or "preventability" as key terms. A consensus-based narrative data synthesis was performed to compare and discuss the different methods. RESULTS: Abstracts of 2504 unique citations were screened resulting in 48 full text articles which were included in the final analysis. Synthesis led to the identification of a set of important variables on which the studies differed considerably (type of readmissions, sources of information, definition of preventability, cause classification and reviewer process). In 69% of the studies the cause classification and preventability assessment were integrated; meaning specific causes were predefined as preventable or not preventable. The reviewers were most often medical specialist (67%), and 27% of the studies added interview as a source of information. CONCLUSION: A consensus-based standardised approach to assess preventability of readmission is warranted to reduce the unwanted bias in preventability rates. Patient-related and integrated care related factors are potentially underreported in readmission studies.

Clarification of Drug Allergy Information Using a Standardized Drug Allergy Questionnaire and Interview.

BACKGROUND: Vague, incomplete, or inaccurate drug-allergy histories can be detrimental to patient safety and affect patient care. There is an increased chance of medication errors if details of a drug allergy are not documented. Incomplete or inaccurate allergy histories may also result in increased time spent by health care providers to clarify an order in question. Published literature from the early 1990s showed that a patient interview can facilitate collection of more complete allergy information and could lead to the removal of a documented drug allergy in approximately one-third of patients. PURPOSE: This quality-improvement project was conducted to improve the process of collecting drug-allergy information at our institution. DESCRIPTION: The primary objective of this project was to determine the number of patients with a clinically relevant change in allergy history after a standardized drug-allergy questionnaire and interview compared with documentation prior to the interview. This was a descriptive, prospective quality improvement project of a standardized drug-allergy questionnaire and interview. Patients were asked about the history of their allergic reaction, including the drug, route, nature of reaction, treatment of reaction, and time since reaction. RESULTS: A total of 301 patient allergy profiles were reviewed. After screening, 202 patients were included and interviewed, for a total of 509 drug allergies. One hundred twenty-three patients (61%) had a need for a clinically relevant change in allergy history after an interview. A total of 228 changes were made to patient allergy profiles. Adding the allergy reaction accounted for 131 of the changes. Also, there were 185 allergies documented prior to the interview that were categorized as unclear/vague, which was significantly reduced to 112 after the interview (P < 0.0001). CONCLUSION: More complete drug-allergy information was obtained after executing a standardized drug-allergy questionnaire and interview.

Inpatient medical stabilization for adolescents with eating disorders: patient and parent perspectives.

PURPOSE: The serious physical complications of eating disorders in adolescents may necessitate inpatient medical stabilization, yet little is known about how patients and their parents perceive the hospitalization experience. METHODS: We identified 82 patients admitted to a large urban hospital for medical stabilization between January 1, 2010 and June 30, 2013. Twenty-three patients and 32 parents completed directed telephone interviews. Respondents rated components of the inpatient protocol using five-point Likert scales and answered open-ended questions regarding hospitalization. Quantitative and qualitative analyses were performed. RESULTS: The mean age of patients at admission was 14.9 years (range 9-21) and the average stay was 8.4 days (range 2-25). Patients rated "massage therapy" most helpful and "cell phone limits" least helpful. Parents rated "nursing staff" most helpful and "seeing other patients in the hospital" least helpful. Protocol components viewed differently by parents and patients included parents more strongly endorsing "staff supervision of meals" (4.34 vs 2.82, p < 0.001) and "limits on physical activity" (4.34 vs 3.23, p = 0.001). The two most common themes identified in open-ended questions were need for hospitalization as a signifier of eating disorder severity and desire for mental health services on the medical unit. Parents emphasized the value of dietician-directed meal planning. CONCLUSIONS: Inpatient medical stabilization for adolescent eating disorders may play an important role not only in addressing acute medical complications, but also in activating the patient and family regarding the need for ongoing treatment. Parents particularly appreciate staff supervision of meals and having a respite from meal planning.

[The admission interview in addictology, nursing practices and skills]. / L'entretien d'accueil en addictologie, pratiques et compétences infirmières.

The reception of a patient in addictology determines how the care will proceed. The setting up of nurse-patient admission interviews adapted to the request for addiction treatment helps professionals meet the patients' needs in terms of listening, guidance, consideration and care. How should the nurse approach this call for help, this "desire" or this necessity for addicted patients to get help? What skills does she need to use?

The dog that didn't growl: the interactional negotiation of momentary confabulations.

We examine from a discursive perspective momentary confabulations generated by patients in clinical interviews based on confabulation questionnaires. Commonly, neuropsychology treats such confabulations solely as evidence of patients' inabilities or deficits. Here we argue that patients' descriptions indicate the interaction of memory deficits with preserved interactional skills. More than this, however, patients' descriptions can be seen in part to arise out of the interviews themselves, in that the interviewer's turns (a) signal agreement rather than disagreement or challenge, and (b) lead to further development of descriptions instead of marking these out as problematic. These features mark out the clinical interviews as different from (i) everyday conversation, and (ii) other clinical settings such as the administration of verbal memory tests, and as a result the interviews allow scope for patients to develop unchallenged autobiographical narratives. Thus, ironically, interviews that rely on the standard use of confabulation questionnaires can provide settings that are especially conducive to the generation of the momentary confabulations that they aim to study.

A Brief Patient-Recorded Audio File Called TIMS (This Is My Story) Improves Communication and Empathy for Healthcare Teams in the Hospital.

Our objective was to assess the impact of a 4-question patient audio interview (this is my story [TIMS]) on medical staff empathy and communication with hospitalized patients and loved ones. We recorded a 4-question audio interview with patients and posted it to the electronic health record. We used a cross-sectional, mixed methods design to pilot this patient version of the TIMS intervention. To evaluate the intervention we collected a brief evaluation survey and conducted semistructured interviews with medical staff. Fifty-three participants responded to our TIMS evaluation survey. Fifty of 51 respondents reported the TIMS file contained useful information. Twenty-four respondents reported listening to the file decreased their distress. Most responded that they either did not have distress or the TIMS file did not change their distress. Of concern, 3 people reported that listening to the file increased their distress. Importantly, most respondents reported feeling greater empathy for the patient after listening (53%) and most reported listening improved their communication with family members (63%, n = 9/13). Qualitative analysis revealed most participants had positive impressions about TIMS. We conclude that empathy and communication were both improved with use of the 4-question TIMS recording.

Patient experience of the use of continuous positive airway pressure for the treatment of obstructive sleep apnea with or without telemedicine during COVID-19: a qualitative approach.

STUDY OBJECTIVES: Continuous positive airway pressure (CPAP) is considered effective treatment for moderate-severe obstructive sleep apnea (OSA). Clinicians measure compliance to treatment by the number of hours CPAP is used per 24 hours. There have been very few studies examining the patients' lived experience of CPAP and the barriers to use. This study examined the patients' experience of OSA, CPAP use and treatment during COVID-19. METHODS: This CPAP patient experience qualitative study was part of a larger project. The overarching study was a three-armed nonblinded randomized controlled trial of patients on CPAP for treatment of OSA using standard care or one of two telemedicine interventions. Patients who had completed the RCT study were invited to be interviewed via telephone, about their OSA diagnosis, CPAP experiences, their experience of using telemedicine and the impact of SARS-CoV-2 (COVID-19). RESULTS: Fifteen patients (five from each arm of the trial) took part in a semi-structured interview. Thematic analysis identified three themes: day to day living, standard hospital care versus telemedicine management and living though COVID-19 with CPAP. CONCLUSIONS: All participants described significant symptoms caused by their OSA before diagnosis. While CPAP treatment was often described as challenging, patients discussed the improvement in their symptoms with treatment. There were differing opinions on virtual appointments, however the consensus was the use of telemedicine to support CPAP treatment was well received. COVID-19 appeared to have little effect on the patient experience.

Patients' views on usefulness and effects of a risk communication tool for cardiovascular disease: a qualitative analysis.

BACKGROUND: Failing to comprehend risk communication might contribute to poor treatment adherence. Using hypertension as a case, we investigated how a risk communication tool for patients with an elevated risk of cardiovascular disease was perceived. METHODS: As part of a large project featuring a randomised controlled trial in a general practice setting in the Region of Southern Denmark, we conducted a semi-structured individual interview study. The study included patients with hypertension who had used an intervention comprising a visual and dynamic cardiovascular risk communication tool, along with receiving recurring emails providing advice on a healthy lifestyle. The analyses were based on Malterud's Systematic Text Condensation. RESULTS: This article focuses solely on the results of the interview study, which comprised a total of 9 conducted and analysed interviews. The IT setup had a major impact on adherence to the intervention. A positive impact was found when the IT setup was perceived as easy to use and accessible, while a negative impact was noted when it malfunctioned. The intervention increased patients' self-reported insight into risk of cardiovascular disease. Patients reported the intervention and their risk of cardiovascular disease to become less important to them when they had more severe comorbidities. The involved health professional was very important for treatment adherence when communicating risk visually. Patients expressed trust in their general practitioners, and the general practitioners' attitudes toward the intervention affected patients' perceptions of its usefulness. While the informants reported an increased awareness of their risk of cardiovascular disease, none of them felt more concerned. CONCLUSIONS: Patients reported an increase in their perceived insight into the risk of cardiovascular disease but not an increased concern. Our findings align with previous studies emphasizing the importance of patients' motivation as well as risk perception for adherence. General practitioners have an important role when implementing new tools for patients.

Patient-reported impact of symptoms in adrenoleukodystrophy (PRISM-ALD).

BACKGROUND: Adrenoleukodystrophy (ALD) is a multifaceted, X-linked, neurodegenerative disorder that comprises several clinical phenotypes. ALD affects patients through a variety of physical, emotional, social, and other disease-specific factors that collectively contribute to disease burden. To facilitate clinical care and research, it is important to identify which symptoms are most common and relevant to individuals with any subtype of ALD. METHODS: We conducted semi-structured qualitative interviews and an international cross-sectional study to determine the most prevalent and important symptoms of ALD. Our study included adult participants with a diagnosis of ALD who were recruited from national and international patient registries. Responses were categorized by age, sex, disease phenotype, functional status, and other demographic and clinical features. RESULTS: Seventeen individuals with ALD participated in qualitative interviews, providing 1709 direct quotes regarding their symptomatic burden. One hundred and nine individuals participated in the cross-sectional survey study, which inquired about 182 unique symptoms representing 24 distinct symptomatic themes. The symptomatic themes with the highest prevalence in the overall ALD sample cohort were problems with balance (90.9%), limitations with mobility or walking (87.3%), fatigue (86.4%), and leg weakness (86.4%). The symptomatic themes with the highest impact scores (on a 0-4 scale with 4 being the most severe) were trouble getting around (2.35), leg weakness (2.25), and problems with balance (2.21). A higher prevalence of symptomatic themes was associated with functional disability, employment disruption, and speech impairment. CONCLUSIONS: There are many patient-relevant symptoms and themes that contribute to disease burden in individuals with ALD. These symptoms, identified by those having ALD, present key targets for further research and therapeutic development.

Survey of Potential Drug Interactions, Use of Non-Medical Health Products, and Immunization Status among Patients Receiving Targeted Therapies.

In recent years, several changes have occurred in the management of chronic immunological conditions with the emerging use of targeted therapies. This two-phase cross-sectional study was conducted through structured in-person interviews in 2018-2019 and 2022. Additional data sources included ambulatory medical records and the itemized reimbursement reporting interface of the National Health Insurance Fund. Drug interactions were analyzed using the UpToDate Lexicomp, Medscape drug interaction checker, and Drugs.com databases. The chi-square test was used, and odds ratios (ORs) were calculated. In total, 185 patients participated. In 53% of patients (n = 53), a serious drug-drug interaction (DDI) was identified (mean number: 1.07 ± 1.43, 0-7), whereas this value was 38% (n = 38) for potential drug-supplement interactions (mean number: 0.58 ± 0.85, 0-3) and 47% (n = 47) for potential targeted drug interactions (0.72 ± 0.97, 0-5) in 2018. In 2022, 78% of patients (n = 66) were identified as having a serious DDI (mean number: 2.27 ± 2.69, 0-19), 66% (n = 56) had a potential drug-supplement interaction (mean number: 2.33 ± 2.69, 0-13), and 79% (n = 67) had a potential targeted drug interactions (1.35 ± 1.04, 0-5). Older age (>60 years; OR: 2.062), female sex (OR: 3.387), and polypharmacy (OR: 5.276) were identified as the main risk factors. Screening methods and drug interaction databases do not keep pace with the emergence of new therapeutics.

An Interactive, Case-Based Workshop on the Patient Experience for Internal Medicine Residents.

Introduction: The technological revolution has narrowed the information gap between physician and patient. This has led to an evolution in medicine from paternalistic to patient-centric, with health care systems now prioritizing patient experience to achieve higher satisfaction scores. Therefore, it is imperative to start early in educating trainees on how to best address the holistic needs of the patient while also delivering high-quality care. Methods: We implemented a 1-hour workshop that was repeated weekly over 8 weeks to capture all internal medicine residents in our program. During the workshop, we reviewed the historical evolution of patient care from paternalistic to patient-centered, presented the Hospital Consumer Assessment of Healthcare Providers and Systems survey questions, and discussed evidence-based strategies for physicians to improve their patients' experience utilizing four case-based scenarios. Results: Over the 8-week period, a total of 195 residents participated in the workshop. One hundred thirty-nine residents (71%) completed the pre- and postsession survey. Results demonstrated significant knowledge improvement (p < .001) in all of the topics discussed. Additionally, the majority of residents felt the workshop would be useful in their clinical practice and found the clinical scenarios useful. Discussion: Given the evolution towards patient-centered care, it is important to take a proactive approach in providing residents with the tools to best address their patients' needs. Early understanding of patient satisfaction surveys and the impacts they have on hospital metrics can help trainees in their careers as practicing physicians.

Evaluation of a murder mystery activity to teach patient communication interviewing skills.

INTRODUCTION: While gamification has been used in pharmacy education, more research is needed to establish that these methods are effective. The objective of our study was to evaluate the effectiveness of a murder mystery activity to teach patient communication and interviewing skills to first year pharmacy students in a pharmacy skills laboratory. METHODS: A non-medical murder mystery activity was used to introduce and provide practice on communication techniques needed for obtaining a medical history. These techniques included an introduction, confirmation of patient identity, nonverbal expression, self-expression, empathy, emotional response, question style, organization, and appropriate closure. In groups of three to five, students interviewed five different suspects and were assessed as a group on their second and fifth suspect interviews within one, three-hour laboratory session using a standardized rubric. Assessments were completed by students, standardized patients, and faculty. RESULTS: A total of 161 students completed the murder mystery exercise over three years. Total student scores all significantly improved from the second to fifth interview. In addition to total scores, each subtotal score significantly improved from the second to fifth interview, regardless of the evaluator. CONCLUSIONS: Within the murder mystery laboratory, students' communication scores improved on a standardized communication rubric. Use of a murder mystery is an effective, engaging way to introduce and practice communication skills that could be adapted by other institutions.

Follow-up Rates After Teleretinal Screening for Diabetic Retinopathy: Assessing Patient Barriers to Care.

Purpose: To study patient follow-up after they engage in a teleretinal screening program and to understand potential barriers to care. Methods: This was a retrospective analysis and a prospective study of telephone-based patient interviews of outpatients screened for diabetic retinopathy (DR) through a teleretinal referral system. Results: Of 2761 patients screened through a teleretinal referral program, 123 (4.5%) had moderate nonproliferative DR (NPDR), 83 (3.0%) had severe NPDR, and 31 (1.1%) had proliferative DR. Of the 114 patients with severe NPDR or worse, 67 (58.8%) saw an ophthalmologist within 3 months of referral. Eighty percent of interviewed patients reported they were not aware of the need for follow-up eye appointments. Conclusions: Of patients with severe retinopathy or worse, 58.8% presented for in-person evaluation and treatment within 3 months of screening. Although this result was negatively affected by factors related to the COVID-19 pandemic, key elements of patient education and improved referral strategies to facilitate in-person treatment are essential to improving follow-up after patients engage in telescreening.

Validation of a Script to Facilitate Social Determinant of Health Conversations with Adolescent Patients.

Current social determinants of health (SDOH) tools exist to assess patient exposure; however, healthcare providers for the adolescent population are unsure of how to integrate SDOH knowledge into clinical practice. The purpose of this study was to validate a focused history script designed to facilitate SDOH conversations between clinicians and adolescents through the use of the Delphi method. Six individuals (1 clinician, 5 educators/researchers) participated as expert panelists. Panelists provided critical feedback on the script for rounds 1 and 2. For rounds 3-7, panelists received an electronic questionnaire asking them to indicate agreement on a 6-point Likert scale (1 = strongly disagree, 6 = strongly agree). We defined consensus as mean item agreement ≥ 5.0 and percent agreement ≥ 80%. In round 7, panelists rated overall script level of agreement. After seven rounds of feedback, the focused history script achieved content validity with 100% of panelists agreeing on the final 40-item script. A focused history script for the SDOH was content validated to aid conversations between healthcare providers and adolescent patients on factors that affect their life, school, and play. Addressing social determinants of health with adolescent patients will improve cultural proficiency and family-centered care delivered by school healthcare professionals.

Health-Related Quality of Life with Diroximel Fumarate in Patients with Relapsing Forms of Multiple Sclerosis: Findings from Qualitative Research Using Patient Interviews.

INTRODUCTION: Diroximel fumarate (DRF) is an oral fumarate for relapsing multiple sclerosis (MS). Clinical and real-world studies of DRF have demonstrated improved gastrointestinal (GI) tolerability and low (< 1%) GI-related treatment discontinuation versus dimethyl fumarate (DMF) and high rates of treatment adherence. Our aim was to conduct a concept elicitation study to identify treatment-related concepts most meaningful to patients and to evaluate how these concepts shape the patient perspective of DRF. METHODS: In-depth qualitative interviews were conducted with patients from October to December 2020. US adults who had been prescribed DRF through routine clinical care and had taken DRF for ≥ 3 weeks in the past 6 months were eligible to participate. Semi-structured interviews explored patient perceptions on treatment selection and impact. RESULTS: Seventeen patients participated in the study. Mean (SD) age was 49.3 (12.0) years. Sixteen patients reported prior disease-modifying therapy, while 10 (58.8%) had prior DMF. DRF treatment duration ranged from ~ 6 weeks to 10 months. Four key concepts emerged: (1) overall wellness and quality of life, (2) ease of administration, (3) minimal and manageable side effects, and (4) patient optimism due to MS treatments. Mode of administration (82.4%), no/mild side effects (70.6%), convenience over injectable/infusion medications (58.8%), and effectiveness (64.7%) were cited as positive aspects of DRF treatment. Frequent dosing (52.9%) and food requirements (41.2%) were cited as negative attributes; however, 94.1% had no dietary changes since starting treatment. CONCLUSION: The patient perspective is a key aspect when considering a disease-modifying therapy for MS, given the multitude of options currently available. Overall wellness, ease of administration, and minimal and manageable side effects were DRF-related concepts most meaningful to patients on therapy. Acknowledging these patient perceptions in shared decision-making may lead to greater patient adherence and optimal treatment outcomes.

Multiple sclerosis (MS), an immune-related disease, may present with neurological symptoms that come and go. Diroximel fumarate (DRF) is a next-generation oral treatment for MS, which has been shown in clinical trials to have fewer gastrointestinal side effects compared to dimethyl fumarate (DMF), another oral treatment. Patients' perspective can shed light on what they value when choosing a treatment, so we interviewed 17 people with MS about how DRF treatment affects their daily life and work. The study participants (49.3 years old on average) received DRF for ~ 6 weeks to 10 months. Around 5 in 10 people had positive feelings about their current health following treatment with DRF. Most felt there was either improvement or no negative change in quality of life since starting DRF treatment; DRF did not affect their work or daily obligations. Treatment characteristics of DRF that were perceived as most important included ease of administration, minimal and manageable side effects, and the facilitation of overall wellness and quality of life. While the oral dosing of DRF was more convenient than injectable or infusion therapy options, about half of the respondents preferred a less frequent treatment regimen than the twice daily dosing of DRF which needs to be taken with food. However, those who switched to DRF from DMF (or other oral medications for MS) expressed that the transition was smooth. Understanding factors that are important to patients can guide treatment choices and help patients stay on treatment longer and have better MS outcomes.

Patient experience before and after treatment with idecabtagene vicleucel (ide-cel, bb2121): qualitative analysis of patient interviews in the KarMMa trial.

OBJECTIVE: To understand the experience of patients with relapsed and refractory multiple myeloma (RRMM) receiving idecabtagene vicleucel (ide-cel), a B-cell maturation antigen-directed chimeric antigen receptor T cell therapy, in the pivotal, phase 2 KarMMa trial. METHODS: Optional semi-structured interviews before leukapheresis (pre-treatment) captured expectations and after ide-cel infusion (1, 2, and 3 months post-treatment), assessed treatment experience, ide-cel advantages/disadvantages, and health and well-being. In a mixed-method analysis, treatment experiences were categorized by clinical response status, health and well-being, and self-reported recovery after infusion. RESULTS: Pre-treatment interviews indicated unmet treatment needs. In post-treatment interviews, most patients reported the positives of ide-cel outweighed negatives (69%, n = 27/39). Most common advantages of ide-cel were efficacy (18-64%), favorable side-effect profile (46-68%), and recovery time (13-18%); most common disadvantages were related to side effects (13-20%). When analyzed by clinical response, patients most often had stringent complete or very good partial response and improved health and well-being with mild or severe recovery from the infusion (27/58, 47%). Most patients with minimal clinical response reported mild infusion recovery (5/6, 83%). CONCLUSIONS: Patient interviews before ide-cel treatment showed unmet needs in triple-class exposed RRMM. Post-treatment experiences generally favored ide-cel versus previously received treatments.

Results from Patient Interviews on Fatigue in Progressive Multiple Sclerosis and Evaluation of Fatigue Patient-Reported Outcome (PRO) Instruments.

INTRODUCTION: Fatigue is one of the most common and debilitating symptoms of multiple sclerosis (MS) but is challenging to assess and has not been comprehensively characterized in patients with progressive MS. This study aimed to (1) obtain qualitative evidence from patients with progressive MS to characterize MS-related fatigue concepts and their impacts on health-related quality of life (HRQoL), and (2) evaluate the conceptual frameworks of existing MS-specific fatigue patient-reported outcome (PRO) instruments using study data to determine the most suitable PRO instrument in this population. METHODS: Qualitative interviews were conducted with 30 US participants with confirmed progressive MS and fatigue in the last 6 months to assess their MS-related fatigue. Data were compared with concepts in existing PRO instruments to evaluate their relevance in progressive MS. RESULTS: Physical and mental concepts of fatigue were identified and characterized distinctly from patients with progressive MS. Most patients characterized fatigue as occurring daily and lasting several hours, with negative impacts on HRQoL. Concept mapping to existing MS-specific fatigue PRO instruments supported the Fatigue Severity Impact Questionnaire-Relapsing Multiple Sclerosis (FSIQ-RMS) as the most suitable existing option for assessing fatigue in patients with progressive MS, as it separates physical and mental aspects of fatigue and includes every highly endorsed concept reported by the interviewed patients. CONCLUSIONS: This qualitative study identified meaningful physical and mental fatigue concepts in patients with progressive MS and preliminarily supports the use of the FSIQ-RMS for this population. More research is needed to fully validate this instrument for progressive MS.

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.