Patient-reported experiences with refractory or unexplained chronic cough: a qualitative analysis.

BACKGROUND: Chronic cough, defined as a cough lasting 8 or more weeks, affects up to 10% of adults. Refractory chronic cough (RCC) is a cough that is uncontrolled despite comprehensive investigation and treatment of comorbid conditions while unexplained chronic cough (UCC) is a cough with no identifiable cause despite extensive evaluation of comorbid conditions. RCC and UCC are often poorly controlled. Understanding individuals' lived experience of the symptoms and impacts of these conditions may guide therapeutic strategies. OBJECTIVES: The primary objectives of this study were to assess respondents' perceptions of the key symptoms of RCC and UCC and the impacts of RCC and UCC and their symptoms on well-being, health-related quality of life, work productivity, and social relationships. DESIGN: Qualitative study. METHODS: This study enrolled 30 adults with physician-diagnosed RCC or UCC. Two trained qualitative researchers conducted individual, in-depth telephone interviews using a semi-structured interview guide. Interviews were audio-recorded, transcribed, coded, and systematically analyzed to identify content themes. RESULTS: A total of 15 respondents with RCC and 15 with UCC were included in the study. Many respondents had RCC or UCC for a long duration (median 9 years, range: 0-24). Half of the respondents reported having a coughing episode at least once daily. Only 40% of respondents reported that medication had improved their symptoms. In over half of the respondents, RCC or UCC hindered communication, caused embarrassment, frustration, and worry, and lowered quality of life. Perceptions of meaningful treatment benefits in RCC or UCC varied widely across respondents. CONCLUSION: RCC and UCC remained poorly managed in many individuals and were associated with a wide range of symptoms and cough triggers that hindered daily activities and reduced emotional well-being. Understanding individuals' lived experiences may inform the development of RCC and UCC therapeutic strategies.

Patient-reported experiences with refractory or unexplained chronic cough: a qualitative analysisChronic cough, particularly refractory and unexplained chronic cough, remain poorly managed in many individuals and are associated with a wide range of symptoms and cough triggers that hinder daily activities and reduce emotional well-being. Currently there are no US Food and Drug Administration-approved treatments for refractory or unexplained chronic cough. Understanding the experience and treatment preferences of individuals with these conditions may help inform the development of new therapies and clarify the potential impact of such therapies on the lives of individuals with chronic cough. Using in-depth interviews, the present study comprehensively evaluated individuals' experience with refractory or unexplained chronic cough and treatment priorities, a research area that has not been well-studied. This study detailed broad-ranging physical, behavioral, and emotional impacts of chronic cough, which hindered individuals' social well-being.

Psychometric evaluation of an electronic Asthma Symptom Diary for young children.

BACKGROUND: Patient-reported outcome measures that facilitate self-report by children are needed to reduce the bias of proxy report. We previously developed an electronic Pediatric Asthma Symptom Diary (ePASD) to assess the severity of daily asthma symptoms and proximal impacts in children aged 6-11 years with mild to severe asthma. The ePASD, administered via a digital application with visuals, sounds, and text, is uniquely designed to minimize the importance of reading skills on children's ability to self-report accurately. Here, we describe the ePASD's psychometric properties. METHODS: Ninety-one children aged 6-11 years with mild to severe asthma and their caregivers participated in 2 study visits, which consisted of training on the provisioned device and completing asthma-specific clinical outcome assessment (COA) questionnaires. The children self-completed the ePASD at home twice daily for 8 consecutive days. The scoring of the ePASD was guided by factor analyses, inter-item correlations, and internal consistencies. Reliability, discriminating ability, construct validity, and responsiveness were evaluated for ePASD items and candidate scores. RESULTS: All COAs included in the study-the ePASD, Asthma Control Questionnaire (ACQ), Childhood Asthma Control Test, Pediatric Asthma Quality of Life Questionnaire-Standardized (PAQLQ[S]), and global ratings-demonstrated that the children exhibited few asthma-related symptoms and impacts at all timepoints, and consequently, showed little change over time. Internal consistencies (all Cronbach's alphas ≥ 0.52) and test-retest reliabilities (all intraclass correlation coefficients ≥ 0.60) were largely satisfactory. Patterns of convergent and divergent correlations supported the construct validity of ePASD scores. The ePASD symptom scores correlated moderately to strongly with PAQLQ(S) Symptom scores (all correlations ≥ - 0.46) and with ACQ scores (all correlations ≥ 0.42), as predicted. Evidence of the discriminating ability of ePASD items and composite scores was demonstrated by known-groups analyses. CONCLUSIONS: The ePASD is a reliable and valid measure of asthma symptoms and proximal impacts in children aged 6-11 years with mild, moderate, or severe asthma. These results lay the psychometric groundwork for use of the ePASD in future clinical trials for the management of pediatric asthma. An ongoing pediatric asthma treatment trial is anticipated to provide evidence of the ePASD's responsiveness to change.

Qualitative assessment of the patient experience of primary hyperoxaluria type 1: an observational study.

BACKGROUND: Without effective intervention, primary hyperoxaluria type 1 (PH1) causes oxalate-induced kidney damage, leading to end-stage kidney disease and serious complications throughout the body. Although PH1 carries a heavy burden that impacts quality of life, literature on the experiences of those living with PH1 and caring for patients with PH1 is limited. This study aimed to describe the diagnostic journey in PH1 and characterize patients' and caregivers' self-reported experiences throughout the disease course. METHODS: This was an observational study involving in-depth, semi-structured telephone interviews. Dominant trends were assessed using constant comparative analysis to identify themes in interviewees' descriptions of their experiences. Individuals aged ≥ 12 years and caregivers of children aged 6-17 years with genetically confirmed PH1 were eligible. Informed consent/assent and ability to read and speak English were required. RESULTS: Interviewees (16 patients, 12 caregivers) reported a prolonged diagnostic journey due to low disease awareness, among other factors. Upon diagnosis, PH1 was frequently symptomatic, typically involving kidney stone-related symptoms but also potentially symptoms arising beyond the kidneys. PH1 most commonly led to worry and social impairment in adolescents, impaired physical function in adults, and a range of impacts on caregivers. In late-stage disease, dialysis was the most burdensome aspect of living with PH1 (due to time requirements, limitations from living with a catheter, etc.), and this burden was exacerbated by the COVID-19 pandemic. Benefits desired from PH1 management included reductions in laboratory measures of oxalate burden, kidney stone and urination frequency, and oxalate-related skin ulcers. CONCLUSIONS: PH1 greatly impacts patients' and caregivers' lives, primarily due to burdensome disease manifestations and associated emotional, physical, and practical impacts, as well as disease management challenges - particularly those related to dialysis in late-stage disease.

Patients' satisfaction with long-acting injectable somatostatin analog therapy for neuroendocrine tumors.

BACKGROUND: Long-acting somatostatin analogs (LA SSAs) are approved and recommended for the treatment of patients with advanced neuroendocrine tumors (NETs). Given the long duration of therapy and differences in administration routes, it is important to understand patients' experiences with receiving LA SSA injections. METHODS: We conducted a serial survey, informed by qualitative interviews with eight patients treated with LA SSAs and two nurses who administer LA SSA injections, among patients undergoing LA SSA treatment over a 28-day period (administered at baseline and 14 days and 28 days after injection). Eligible patients, recruited by the Carcinoid Cancer Foundation, self-reported having received an LA SSA injection for physician-diagnosed NET within the 5 days before the survey. RESULTS: 202 patients completed the survey at baseline (82 receiving lanreotide and 120 receiving octreotide), 148 at day 14, and 124 at day 28. Patients reported consistently high satisfaction levels with their most recent LA SSA injection (91.1% at baseline, 85.1% at day 14, and 85.5% at day 28); 68.8% reported that their injection experience differed based on the nursing staff administering the injection. CONCLUSIONS: Satisfaction with LA SSA injections is high among patients in this population, and specific experiences with LA SSA injections varied based on the nursing staff administering the injection. Evaluations of patients' experiences and satisfaction with treatment are increasingly important as patients take more active roles in decision-making for their treatment pathways.

Training and administration of epinephrine auto-injectors for anaphylaxis treatment in US schools: results from the EpiPen4Schools(®) pilot survey.

BACKGROUND: Anaphylaxis is a serious, potentially life-threatening condition. Adequate preparation for anaphylaxis management is imperative for school personnel. This descriptive pilot study assessed preparedness of US schools to manage anaphylactic reactions. METHODS: An exploratory, cross-sectional, web-based, pilot survey assessed the occurrence and characteristics of anaphylactic events, as well as training provided to school personnel for the recognition and treatment of anaphylaxis. Eligible US schools were participants in the EpiPen4Schools(®) program during the 2013-2014 school year. EpiPen4Schools provides EpiPen(®) (epinephrine injection) Auto-Injectors and training materials to qualifying US schools. Survey data were parsed by US Census Bureau region and state and were evaluated using descriptive statistics. RESULTS: Schools from all 50 states and the District of Columbia participated in the survey (N=6,019). Among schools that provided information on anaphylactic events, 11% (607/5,683) reported the occurrence of one or more events, with significant variability in incidence across census regions and among states. A total of 5,613 schools provided information regarding which staff members were trained to recognize the signs and symptoms of anaphylaxis. Thirty-six percent of schools (2,022/5,613) indicated that only the school nurse and select staff were trained in anaphylaxis recognition. The proportion of schools in which most or all school staff received such training differed by region/state (range, 13%-100%). A total of 5,578 schools provided information on which staff were permitted to administer epinephrine. The majority of schools (54%; 3,024/5,578) permitted only the school nurse and select staff to administer epinephrine, although percentages varied by region/state (range, 4%-100%). CONCLUSION: Schools differed substantially in their preparedness to manage anaphylaxis, with significant disparities in staff training and permission to treat. Given the ramifications of delayed treatment, removing barriers to the recognition and treatment of anaphylactic events in schools is an important public health goal.

Anaphylaxis in Schools: Results of the EPIPEN4SCHOOLS Survey Combined Analysis.

A pilot survey described the characteristics of anaphylactic events occurring in an initial set of participating U.S. schools during the 2013-2014 school year. This survey was subsequently readministered to large school districts, which were underrepresented in initial results. A cross-sectional survey was administered to the U.S. schools that were participating in the EPIPEN4SCHOOLS® program (Mylan Specialty L.P., Canonsburg, PA) to assess characteristics of anaphylactic events. Data from large school districts were added to initial findings in this comprehensive combined analysis. A total of 1,140 anaphylactic events were reported among 6,574 responding schools. Of 1,063 anaphylactic events with data on who experienced the event, it was observed that it occurred mostly in students (89.5%, 951/1,063). For students, anaphylactic events were reported across all grades, with 44.9% (400/891) occurring in high school students, 18.9% (168/891) in middle school students, and 32.5% (290/891) in elementary school students. Food was identified as the most common trigger (60.1%, 622/1,035). A majority of schools (55.0%, 3,332/6,053) permitted only the school nurse and select staff to administer epinephrine to treat anaphylaxis. The unpredictability of anaphylaxis is emphasized by its high occurrence in individuals with no known allergies (25.0%). A majority of schools permitted only the school nurse and select staff to treat anaphylaxis. Thus, individuals experiencing an anaphylactic event may frequently encounter staff members not being permitted to administer potentially life-saving epinephrine. Epinephrine auto-injectors provided by the EPIPEN4SCHOOLS program were used to treat 38.0% of events. Anaphylaxis can occur in children with no previously known allergies, illustrating the importance of public access to epinephrine.

Anaphylaxis Triggers and Treatments by Grade Level and Staff Training: Findings from the EPIPEN4SCHOOLS Pilot Survey.

This pilot survey was designed to evaluate the characteristics of anaphylactic events and epinephrine autoinjector (EAI) use in children in U.S. schools. A cross-sectional, web-based, pilot survey of schools participating in the EPIPEN4SCHOOLS® program (Mylan Specialty L.P., Canonsburg, PA) assessed characteristics of anaphylactic events and EAI use during the 2013-2014 academic year. Respondents reported 757 anaphylactic events experienced by students; student grade level was noted for 724 events. Of these events, 32.3% (234/724) were experienced by students in grade school, 18.6% (135/724) by students in middle school, and 49.0% (355/724) by students in high school. Frequency of food-related triggers was consistently high across grade levels. However, many events experienced by students in high school (22.3%, 79/355), middle school (15.0%, 20/135), and grade school (14.1%, 33/234) had an unknown trigger. In 36.0% of schools (2008/5579), only the school nurse and select staff received training to recognize anaphylaxis; most staff or all staff received training in 28.9% (1610/5579) and 30.9% (1722/5579) of schools, respectively. In a majority of schools (54.2%, 3003/5544), only the school nurse and select staff were permitted to administer epinephrine, whereas most staff or all staff were permitted to administer epinephrine in 15.8% (876/5544) and 21.9% (1212/5544) of schools, respectively. Risk of anaphylaxis may be particularly high during adolescence, and some students encounter staff members who are untrained in anaphylaxis recognition or management, or both. These findings support the need for continued anaphylaxis training for the protection of all students, staff, and visitors.

EpiPen4Schools pilot survey: Occurrence of anaphylaxis, triggers, and epinephrine administration in a U.S. school setting.

BACKGROUND: Although epinephrine is the treatment of choice for anaphylaxis, it remains underused. OBJECTIVE: This study was designed to describe anaphylactic events and epinephrine autoinjector (EAI) use in U.S. schools enrolled in the EpiPen4Schools program. METHODS: This exploratory, cross-sectional, Web-based survey of 6019 schools that participated in the EpiPen4Schools program assessed anaphylactic events and EAI use at responding schools during the 2013-2014 school year. RESULTS: A total of 919 anaphylactic events were reported in 607 schools. Of the 852 anaphylactic events with data on those who experienced an event, most 88.8% (n = 757) occurred in students, and 21.9% of events (n = 187) occurred in individuals with no known allergies. Of the 851 events with data on EAI use, 74.7% (n = 636) were treated with EAIs and 8.5% (n = 54) received a second epinephrine injection. Of the 204 individuals not treated with an EAI, 77.0% (n = 157) received antihistamines, 12.7% (n = 26) received another treatment, and 8.3% (n = 17) received no treatment. Of the 850 events with data on hospital transport, 79.6% of individuals (n = 677) were transported to the hospital. Common triggers varied seasonally, with food listed most frequently overall (62.5%). CONCLUSION: More than one in ten schools that participated in the EpiPen4Schools survey reported an anaphylactic event. Approximately 25% of individuals with anaphylactic events were not treated with EAIs, and 20.4% of patients were not taken to the hospital after an anaphylactic event. Analysis of these data supports the value of stocking EAIs and of providing continuing education regarding the recognition and proper treatment of anaphylaxis for school personnel.