T. Michelle Brown

A multicenter, non-interventional study to evaluate patient-reported experiences of living with psoriasis

Abstract Background: Moderate to severe plaque psoriasis (with or without psoriatic arthritis) places significant burden on patients’ lives. Objective: Explore and document patients’ experiences of living with psoriasis, including symptoms, treatments, impact on daily lives and patient-reported functioning. Methods: In a US-based, non-interventional study, narrative interviews were conducted at baseline and again within 16 weeks. In interviews, patients with moderate to severe psoriasis indicated symptoms, ranked symptoms according to level of bother and indicated areas of their lives affected by psoriasis. Transcripts of interviews were coded for themes. Measurements of psoriasis severity including BSA, PGA and PASI were recorded. Results: Symptoms reported most frequently included flaking/scaling (non-scalp areas), itching/scratching and rash, while the most bothersome symptoms were itching/scratching, flaking/scaling (non-scalp areas) and skin pain. Frequently reported impact areas were social and emotional. Conclusion: Broad-reaching interviews with patients with psoriasis show that these patients suffer in many aspects of their lives and in ways not indicated by typical psoriasis severity measures. Patients with psoriatic arthritis reported symptoms and disease-related complications at higher rates than those without arthritis. Physicians’ explorations of the effect of psoriasis on patients’ life events could aid in managing these patients.

Development and Validation of the ADHD Benefits of Coaching Scale (ABCS)

Objective: Develop and validate the ADHD Benefits of Coaching Scale (ABCS), a self-report instrument designed to measure benefits of ADHD coaching in college students. Method: Concepts were elicited from interviews with ADHD coaches and college students receiving ADHD coaching. Draft items were tested in cognitive interviews among ADHD students. Item and scale reliability and validity of ABCS were evaluated using web-survey data from students reporting ADHD. Mean ABCS scores for coached and noncoached groups were compared. Results: Five coaches and 14 students participated in concept elicitation interviews; 10 students participated in the cognitive interviews; 209 students completed the web-survey. The 10-item ABCS showed satisfactory internal consistency reliability and validity and differentiated between coached (n = 17) and noncoached groups (n = 192; p < .01). Conclusion: To assess benefits of ADHD coaching in college students, the ABCS was developed and psychometrically validated in accordance with Food and Drug Administration’s (FDA) patient-reported outcome guidance.

The development and initial assessment of the strategy and leadership systems capability evaluation survey

Hospital management and leadership systems are associated with organizational success and quality care. The Strategy and Leadership Systems Capability Evaluation (CE) survey was developed by GE Healthcare to assess management and leadership systems at health care institutions, serve as a benchmark for improvement, and measure progress. To assess the psychometric properties of the 29-item CE survey, including the factor structure, scoring algorithm, reliability, and discriminant validity, an online survey was completed by 3450 employees at 15 US hospitals. Of these employees, 609 worked at a hospital where a leadership and management intervention occurred after the initial survey administration. Data were also collected on job level, number of hospital beds, hospital ownership, location, community type, and the implementation of hospital interventions. Item response frequencies showed no floor or ceiling effects and limited missing data. Interitem correlations were strong without obvious redundancies, and factor analysis suggested a unidimensional scale. The resulting scale had strong internal consistency and was able to discriminate among known groups. The CE survey was developed to evaluate management and leadership systems at health care institutions. This study provides psychometric evidence in support of the reliability, validity, and scoring structure of this survey.

Assessing the burden of treatment-emergent adverse events associated with atypical antipsychotic medications

BackgroundTreatment of schizophrenia and major depressive disorder (MDD) with atypical antipsychotics (AAPs) show improved efficacy and reduced side effect burden compared with older antipsychotic medications. However, a risk of treatment-emergent adverse events (TEAEs) remains. TEAEs are hard to quantify and perspectives on the importance of TEAEs differ across patients and between patients and physicians. The current study is a qualitative assessment that investigates TEAEs of AAPs from both patient and physician perspectives to provide better understanding of the occurrence and burden of TEAEs associated with these medications.MethodsFocus groups comprised of patients with MDD and interviews with patients with schizophrenia were conducted at two qualitative research facilities, along with a physician focus group at one of the facilities. Information collected from patients included an exhaustive list of TEAEs experienced, and the frequency and level of bother of each TEAE; from psychiatrists, information included an exhaustive list of TEAEs based on personal observations and patient report, frequency of TEAEs, clinically important TEAEs, and levels of patient-perceived bother. Standard qualitative analysis methods were used to identify, quantify, characterize, and summarize patterns found in the data collected.ResultsA total of 42 patients (25 with MDD and 17 with schizophrenia) and 4 psychiatrists participated in the study. TEAEs reported as bothersome across both patients groups included cognitive issues, weight gain and/or increased appetite, low energy, extrapyramidal symptoms (EPS), and need to sleep/excessive sleep/excessive sleepiness. TEAEs considered more bothersome by patients with schizophrenia were weight gain, low energy, EPS, mental anxiety, and increased positive symptoms; those considered more bothersome by patients with MDD were cognitive issues, somnolence/sedation, and flat/restricted affect. TEAEs considered most clinically important by psychiatrists included metabolic syndrome, weight gain, neutropenia, hyperglycemia, and QT prolongation; those TEAEs considered most bothersome to patients from physicians’ perspectives included weight gain, reduced sexual desire or performance, EPS, akathisia, and hormonal issues.ConclusionsThe wide range of TEAEs that are both frequent and bothersome and the variation in perceived burden according to diagnosis highlight the need for a tailored TEAE-awareness approach when choosing an AAP.

Development of the 7-Item Binge-Eating Disorder Screener (BEDS-7)

OBJECTIVE Develop a brief, patient-reported screening tool designed to identify individuals with probable binge-eating disorder (BED) for further evaluation or referral to specialists. METHODS Items were developed on the basis of the DSM-5 diagnostic criteria, existing tools, and input from 3 clinical experts (January 2014). Items were then refined in cognitive debriefing interviews with participants self-reporting BED characteristics (March 2014) and piloted in a multisite, cross-sectional, prospective, noninterventional study consisting of a semistructured diagnostic interview (to diagnose BED) and administration of the pilot Binge-Eating Disorder Screener (BEDS), Binge Eating Scale (BES), and RAND 36-Item Short-Form Health Survey (RAND-36) (June 2014-July 2014). The sensitivity and specificity of classification algorithms (formed from the pilot BEDS item-level responses) in predicting BED diagnosis were evaluated. The final algorithm was selected to minimize false negatives and false positives, while utilizing the fewest number of BEDS items. RESULTS Starting with the initial BEDS item pool (20 items), the 13-item pilot BEDS resulted from the cognitive debriefing interviews (n = 13). Of the 97 participants in the noninterventional study, 16 were diagnosed with BED (10/62 female, 16%; 6/35 male, 17%). Seven BEDS items (BEDS-7) yielded 100% sensitivity and 38.7% specificity. Participants correctly identified (true positives) had poorer BES scores and RAND-36 scores than participants identified as true negatives. CONCLUSIONS Implementation of the brief, patient-reported BEDS-7 in real-world clinical practice is expected to promote better understanding of BED characteristics and help physicians identify patients who may have BED.

Symptoms of Anxiety and Irritability in Patients with Major Depressive Disorder

Background: Anxiety and irritability often coexist in patients with major depressive disorder (MDD). The Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-5) added criteria for an anxious distress specifier for MDD. This study aimed at understanding the various components of anxiety and irritability, their impact, and relationships among them in MDD; and the association of anxious distress and irritability with treatment response. Methods: Focus groups were conducted with patients with MDD reporting symptoms of anxiety, irritability, agitation, and/or aggression. Clinical study data were pooled from open-label antidepressant treatment (ADT) phases of seven studies in patients with MDD and a history of inadequate response for the current depressive episode to one to three ADTs (N = 5,182). Post hoc criteria using study measures were applied to identify patients with symptoms of anxious distress and irritability. ADT response rates were compared for patients with and without anxious distress and irritability. Results: Symptoms of anxiety and irritability frequently coexisted for the focus group participants who often described symptoms of anxious distress (e.g., nervous, tense, restless, worry, fear, out of control, acting out). In the clinical studies, approximately 50% of patients with inadequate response to ADTs presented with symptoms of anxious distress and irritability. The presence of anxious distress was associated with lower ADT response rates. Conclusions: Various anxiety symptoms, including those comprising anxious distress, are prevalent and meaningful in depression, and frequently associated with irritability. Symptoms of anxious distress are associated with a decreased likelihood of ADT response.

Development of the Impact of Juvenile Metachromatic Leukodystrophy on Physical Activities scale

BackgroundMetachromatic leukodystrophy (MLD) is a rare disease with three forms based on the age at onset of signs and symptoms. The objective of this study was to develop a caregiver-reported clinical outcome assessment that measures impairments in physical functioning related to activities of daily living in patients with juvenile MLD.MethodsA targeted literature review and exploration of proprietary research, including a conceptual model, were conducted. Concept elicitation interviews were conducted to elicit additional concepts related to impairments in patients’ physical functioning with caregivers of five individuals with juvenile MLD. Based on the research review and concept elicitation interviews, the conceptual model was updated and the Impact of Juvenile Metachromatic Leukodystrophy on Physical Activities (IMPA) scale draft items were created. Cognitive debriefing interviews were conducted with six additional caregivers to finalize the conceptual model and to refine the IMPA scale.ResultsInitially, 17 potentially important concepts were identified and addressed in the draft IMPA scale. Following the cognitive debriefing interviews, 15 activities/items remained: brush teeth, comb/brush hair, bathe/shower, dress self, eat, drink, use pencil/crayon, sit upright, use toilet, get on/off toilet, walk, use stairs, get in/out of bed, get in/out of chair/wheelchair, and get in/out of vehicle. Items that did not uniquely contribute to the purpose of the instrument were removed.ConclusionThe IMPA scale, developed according to regulatory standards, provides a means of detecting changes in activities of daily living in individuals with juvenile MLD and can hence be used in future studies to measure benefits of therapeutic interventions.

Assessing Patient and Caregiver Experiences with Symptoms and Behaviors Associated with Schizophrenia

Objective: Obtaining patient and caregiver perspectives may be critical for informing disease management, particularly with conditions like schizophrenia. The primary objective of the current study was to obtain and assess patient and caregiver perspectives on associated symptoms and behaviors of schizophrenia and their impact or burden on patients and their caregivers through patient interviews and caregiver focus groups. Research design and methods: Seven in-depth individual interviews were conducted with individuals with schizophrenia, and four focus groups were conducted with caregivers of adults with schizophrenia (n = 21) in two different locations in the United States. Following a discussion guide, interviewers asked open-ended questions to ascertain specific definitions and experiences related to five associated symptoms and behaviors (anxiety, irritability, agitation, impulsivity, and aggression), the impact of these symptoms, and how they were associated with schizophrenia. Additionally, patients completed the 8-item Birchwood Insight Scale developed to assess insight in patients with schizophrenia. Results: The impacts of anxiety were the most widespread and persistent for the patients, primarily relating to social and family interactions and relationships. For both patients and caregivers, patients’ anxiety comprised a mental and a physical component and contributed to escalation of other emotions and behaviors. Other significant or debilitating impacts, such as emotional functioning, legal issues, and financial distress, were attributed to aggression and impulsivity for both the patients and the caregivers. Conclusion: This study provides evidence of the impact of anxiety in the lives of patients with schizophrenia and their caregivers, and the potential relationship of and escalation from anxiety to aggression. Additionally, the study reinforces the importance of ensuring that treatment is taking into account the highly prevalent anxiety symptoms and behavioral disturbances in patients with schizophrenia as well as the implications of these symptoms and disturbances on the caregivers.