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Family physicians commonly find themselves in difficult patient encounters that can result in dissatisfaction for the patient and physician. Successful navigation of these encounters includes recognizing common physician factors, such as systemic pressures, interpersonal communication, and situational issues. The practice of labeling patient types can lead to disparities in care and patient harm and should be avoided. When physicians recognize that they are in a difficult patient encounter, simple mindfulness approaches, such as the Name It to Tame It and CALMER approaches, can improve outcomes. CALMER approaches help physicians acknowledge which situations they can control, alter their thoughts about the situation, and tolerate uncertainty. Physicians working with patients to create a therapeutic bond can focus the encounter to understand the situation that the patient is experiencing and work to recognize and acknowledge strong emotions that are nonproductive. Negotiating an agenda can help manage expectations of what can reasonably be done during each visit. Supporting patients by validating their symptoms and helping them embrace uncertainty can enable them to take control of their diagnosis and focus on managing chronic conditions rather than curing them. Motivational interviewing is a useful tool to help patients take ownership of their illnesses and therapeutic goals. Self-care through reflection groups or personal coaching or counseling can help physicians feel supported and avoid burnout.

Background New treatments are being evaluated for lipodystrophy; however, limited information is available on the patient experience. Results of a prior patient panel showed that hunger and temperature-related symptoms were an issue for participants. Therefore, evaluation of any changes in these symptoms is recommended for inclusion in new treatment options. The objective of this study was to further understand the patient experience and to evaluate newly developed items of hunger and temperature regulation. Methods Individual, in-depth telephone interviews were conducted via semi-structured discussion guide. Telephone interviews were conducted with 21 US patients with generalized lipodystrophy (GLD) or partial lipodystrophy (PLD). Eligibility requirements included self-reported PLD or GLD. Interviews included open-ended concept elicitation followed by a review of newly developed items assessing hunger, temperature sensations, and patient globals. Interviews were conducted in two rounds, with the newly developed items assessing hunger revised after each round of interviews based on participant feedback. Results Results indicated that hunger-related symptoms were considered a current issue for greater than half (N = 11) of participants, and all but one reported this as an issue at some point in their lives. Specifically, participants most often reported symptoms of increased appetite and not feeling full. The cognitive debriefing process indicated that the hunger-related symptoms, temperature, and global impression of change and severity items were correctly interpreted and easily completed by the participants. While not a focus of the interviews, the concept elicitation results demonstrated that pain was a frequently reported and bothersome symptom in this patient population. Conclusions This qualitative research provided evidence to support the use of clinical outcomes assessments such as hunger and temperature-related items in clinical trials.

Pulmonary hypertension resulting from chronic lung disease such as chronic obstructive pulmonary disease and interstitial lung disease is categorized by the World Health Organization as Group 3 pulmonary hypertension. To identify the symptoms and impacts of World Health Organization Group 3 pulmonary hypertension and to capture data related to the patient experience of this disease, qualitative research interviews were undertaken with 3 clinical experts and 14 individuals with pulmonary hypertension secondary to chronic obstructive pulmonary disease or interstitial lung disease. Shortness of breath, fatigue, cough, and swelling were the most frequently reported symptoms of pulmonary hypertension due to chronic obstructive pulmonary disease or interstitial lung disease, and shortness of breath was further identified as the single most bothersome symptom for most patients (71.4%). Interview participants also described experiencing a number of impacts related to pulmonary hypertension and pulmonary hypertension symptoms, including limitations in the ability to perform activities of daily living and impacts on physical functioning, family life, and social life as well as emotional impacts, which included frustration, depression, anxiety, isolation, and sadness. Results of these qualitative interviews offer an understanding of the patient experience of pulmonary hypertension due to chronic obstructive pulmonary disease or interstitial lung disease, including insight into the symptoms and impacts that are most important to patients in this population. As such, these results may help guide priorities in clinical treatment and assist researchers in their selection of patient-reported outcome measures for clinical trials in patients with pulmonary hypertension due to chronic obstructive pulmonary disease or interstitial lung disease.

PurposeFor patients with celiac disease (CeD), the only current management option is adherence to a strict gluten-free diet (GFD); however, many patients on a GFD continue to experience symptoms with a significant impact on quality of life. Potential new treatments for CeD are under development and a validated patient-reported outcome measure is required to evaluate their utility in clinical trials. The purpose of this article is to provide a history of the development of the Celiac Disease Symptom Diary (CDSD) 2.1© for use in clinical trials.MethodsQualitative and quantitative studies were conducted from 2010 to 2021, including concept elicitation and cognitive debriefing interviews with adult and adolescent participants with CeD (N = 93) diagnosed via biopsy and/or serology and input from eight interviews with CeD clinical experts. During these studies, different iterations of the CDSD were presented to the US Food and Drug Administration and the European Medicines Agency, and modifications were made in line with their feedback.ResultsThese studies ultimately led to the development of CDSD 2.1©, a daily diary which focuses on key symptoms of CeD (abdominal pain, bloating, diarrhea, nausea and tiredness). This patient-reported outcome measure was readily understood by adult and adolescent participants with CeD and content validity was demonstrated in both populations.ConclusionCDSD 2.1© is a content-valid patient-reported outcome measure developed in accordance with best practices and regulatory guidance. A thorough exploration of the psychometric properties of CDSD 2.1© for both adult and adolescent participants with CeD is ongoing to support utilization in clinical trials.

Background Molluscum contagiosum is often characterized by persistent lesions and bothersome symptomology. What patients with molluscum contagiosum and/or caregivers consider to be meaningful measures of therapeutic success is unknown. Objective We aimed to collect patient experience data and assess Global Impression of Change from patients and/or caregivers participating in a large phase III molluscum contagiosum interventional trial. Methods The B erdazimer S odium I n M olluscum P atients with LE sions (B-SIMPLE4) phase III study enrolled 891 patients with molluscum contagiosum. Patients were randomly assigned to berdazimer gel, 10.3% or vehicle gel applied once daily for 12 weeks. Assessments of participant and investigator perceptions of complete lesion clearance were collected at weeks 12 and 24 along with Global Impression of Change scores from 1 (very much improved) to 7 (very much worse). A subset of 30 B-SIMPLE4 patients participated in the patient/caregiver experience exit interview to evaluate bothersome signs and symptoms. Results At week 12, among participants with a ≥ 75% molluscum contagiosum lesion count reduction from baseline (as assessed by investigators), 99% (373/376) reported improvement. Perceptions of complete clearance at week 12 were nearly 40% for both participant-reported and investigator-reported Global Impression of Change in berdazimer group vs 20% in the vehicle group: 82% (322/392) of participants in the berdazimer group and 60% (237/394) in the vehicle group reported their molluscum contagiosum lesions were either very much improved or much improved at week 12. Similarly, investigators scored 80% (314/393) of berdazimer and 54% (215/396) of vehicle participants as very much improved or much improved. From the exit interview, the mean duration of participant-reported molluscum contagiosum was nearly 2 years. The most frequently reported molluscum contagiosum-related signs and symptoms were itch ( n  = 20), scarring ( n  = 18), and pain ( n  = 13). Visibility and contagiousness of molluscum contagiosum were the most bothersome aspects to participants. The most frequently reported psychosocial impacts were self-consciousness ( n  = 15) and embarrassment ( n  = 14). Lesion clearance was an expectation of 28/30 study participants. Overall, 26/30 reported being very satisfied ( n  = 18) or satisfied ( n  = 8) with the changes in their disease over the duration of the trial; 23/30 stated that the change in lesion count was meaningful. A mean reduction of 18 lesions (76% decrease) from the baseline lesion count was reported by participants ( n = 28). Although 22 of 28 had less than complete lesion clearance, 17 of 22 reported that the reduction in the number of lesions was meaningful. Conclusions Molluscum contagiosum lesion reductions, with or without complete clearance, may be considered a therapeutic “success” by the patient/caregiver. Clinical Trial Registration NCT04535531 (registered 2 September, 2020).

In recent years, the US FDA has become more critical of instruments used to measure patient-reported outcomes (PROs) in clinical trials. To facilitate decisions related to the approval of drugs, labels and promotional claims based on PROs, the FDA created the Study Endpoints and Label Development (SEALD) group. SEALD has developed a PRO guidance related to the use of PRO measures used to support drug approvals and label claims, including recommendations for establishing thresholds for meaningful change at the individual level (i.e., defining a responder). This article examines in detail the FDA-recommended methodology for defining a responder and analyzing responder-based PRO measure results. We also present other responder analysis approaches for consideration in furthering the precision and interpretation of this methodology.

Chronic idiopathic constipation (CIC) is a functional gastrointestinal disorder with an estimated prevalence of 16% in the USA; however, the humanistic and economic burden of CIC is poorly characterized. This systematic literature review aimed to assess the humanistic and economic burden of CIC in adults in the USA. Two systematic literature searches of English-language publications on the humanistic and economic burden of CIC in adults in the USA were conducted using electronic databases and other resources. Both searches included the terms \"chronic idiopathic constipation\" and \"functional constipation\". Specific terms used in the search on humanistic burden included \"quality of life\", \"SF-36\", \"SF-12\", and \"PAC-QOL\"; search terms for economic burden included \"cost\", \"resource use\", \"absenteeism\", and \"productivity\". Overall, 16 relevant articles were identified. Health-related quality of life (HRQoL) appeared to be reduced in patients with CIC compared with controls and the general US population. Abdominal ( =0.33-0.49), stool ( =0.23-0.33), and rectal symptoms ( =0.53) appeared to be associated with reduced HRQoL. Younger age and female sex were associated with reduced overall HRQoL and greater symptom severity. Direct outpatient costs were higher in patients with CIC than those without CIC (US$6284 vs US$5254). Patients with CIC and abdominal symptoms reported more days of disrupted productivity per month than those without abdominal symptoms (3.2 days vs 1.2 days). The overall prevalence of complementary and alternative medicine use by patients with CIC was similar to that in the general US population. The reduced HRQoL and increased costs associated with CIC indicate unmet therapeutic need in this disorder. Further research is required to better understand the humanistic and economic burden of CIC in the USA.

OBJECTIVE: To examine the cost‐effectiveness of moving from usual care to more organized management strategies for patients on chronic warfarin therapy. DESIGN: Using information available in the scientific literature, supplemented with data from a large health system and, when necessary, expert opinion, we constructed a 5‐year Markov model to evaluate the health and economic outcomes associated with each of three different anticoagulation management approaches: usual care, anticoagulation clinic testing with a capillary monitor, and patient self‐testing with a capillary monitor. PATIENTS: Three hypothetical cohorts of patients beginning long‐term warfarin therapy were used to generate model results. MAIN RESULTS: Model results indicated that moving from usual care to anticoagulation clinic testing would result in a total of 1.7 thromboembolic events and 2.0 hemorrhagic events avoided per 100 patients over 5 years. Another 4.0 thromboembolic events and 0.8 hemorrhagic events would be avoided by moving to patient self‐testing. When direct medical care costs and those incurred by patients and their caregivers in receiving care were considered, patient self‐testing was the most cost‐effective alternative, resulting in an overall cost saving. CONCLUSIONS: Results illustrate the potential health and economic benefits of organized care management approaches and capillary monitors in the management of patients receiving warfarin therapy.

This study was intended to evaluate the cost-effectiveness of anticoagulation clinic care and self-testing for the management of patients on chronic warfarin therapy. Using a 5-year Markov model, we evaluated the health and economic outcomes associated with each of three different anticoagulation management approaches: (1) usual care, (2) anticoagulation clinic testing with a capillary monitor, and (3) patient self-testing with a capillary monitor. Data available in the published literature and data from a large health system were used to develop model assumptions. Model results indicate that over a 5-year period, compared with usual care, anticoagulation clinic testing results in a total of 1.7 fewer thromboembolic events and 2.0 less hemorrhagic events per 100 patients. Another 4.0 thromboembolic events and 0.8 hemorrhagic events are avoided with patient self-testing compared with anticoagulation clinic testing. In addition to the health advantages of these strategies, both also have cost advantages. When the costs incurred by provider organizations and patients are considered, patient self-testing is the most cost-effective alternative, resulting in an overall cost saving.