Burden of Treatments for Respiratory Complications in Extremely Premature Infants: Interviews with Caregivers.

Introduction: Extremely premature (EP) infants (<28 weeks gestational age) with respiratory conditions after discharge from the neonatal intensive care unit (NICU) impose a significant burden on caregivers. This study explored caregiver burden post-NICU discharge and perceptions of meaningful change in infant chronic respiratory morbidity. Methods: Adult primary caregivers of EP infants 3-14 months corrected age were recruited through patient advocacy organizations or hospital centers in the USA, Northern Ireland, Germany, and Japan and interviewed by phone. Interviews explored caregiver experiences with infants with respiratory conditions, associated treatment burden, and meaningful change in infant respiratory morbidity as measured by treatment use. Qualitative analysis of interview data was performed using MAXQDA software. Sociodemographic data were summarized using descriptive statistics. Results: Forty-five caregivers (95.6% female) of EP infants were interviewed. Respiratory morbidities post-NICU discharge included coughing (78%), breathing difficulties (76%), wheezing (58%), and bronchopulmonary dysplasia/chronic lung disease of prematurity (56%). Respiratory medications were required by 87% of infants, 80% used home respiratory technology support (e.g., supplemental oxygen), 38% were re-hospitalized, and 33% had emergency department visits. Caregivers considered visits to the emergency department to be the most burdensome treatment requirement they experienced, and reduction in the number of emergency department visits was considered the most meaningful change in treatment use. Conclusion: These findings underscore the significant burden faced by caregivers of EP infants with respiratory morbidities. Development of treatments for respiratory complications should take into consideration the concerns and preferences of caregivers in order to provide a meaningful benefit.

Development of the PREMature Infant Index (PREMII™), a clinician-reported outcome measure assessing functional status of extremely preterm infants.

BACKGROUND: Comprehensive measures to evaluate the effectiveness of medical interventions in extremely preterm infants are lacking. Although length of stay is used as an indicator of overall health among preterm infants in clinical studies, it is confounded by nonmedical factors (e.g. parental readiness and availability of home nursing support). OBJECTIVES: To develop the PREMature Infant Index (PREMII™), an electronic content-valid clinician-reported outcome measure for assessing functional status of extremely preterm infants (<28 weeks gestational age) serially over time in the neonatal intensive care unit. We report the development stages of the PREMII, including suggestions for scoring. METHODS: We developed the PREMII according to US Food and Drug Administration regulatory standards. Development included five stages: (1) literature review, (2) clinical expert interviews, (3) Delphi panel survey, (4) development of items/levels, and (5) cognitive interviews/usability testing. Scoring approaches were explored via an online clinician survey. RESULTS: Key factors reflective of functional status were identified by physicians and nurses during development of the PREMII, as were levels within each factor to assess functional status. The resulting PREMII evaluates eight infant health factors: respiratory support, oxygen administration, apnea, bradycardia, desaturation, thermoregulation, feeding, and weight gain, each scored with three to six gradations. Factor levels are standardized on a 0-100 scale; resultant scores are 0-100. No usability issues were identified. The online clinician survey identified optimal scoring methods to capture functional status at a given time point. CONCLUSIONS: Our findings support the content validity and usability of the PREMII as a multifunction outcome measure to assess functional status over time in extremely preterm infants. Psychometric validation is ongoing.

Development of a severity scale to assess chronic lung disease after extremely preterm birth.

OBJECTIVE: Chronic lung disease of prematurity (CLDP) is a frequent complication of prematurity. We aimed to identify what clinicians believe are the most important factors determining the severity of CLDP in extremely preterm infants (<28 weeks gestational age) after discharge from the neonatal intensive care unit (NICU) through 12 months corrected age (CA), and to evaluate how these factors should be weighted for scoring, to develop a CLDP severity scale. STUDY DESIGN: Clinicians completed a three-round online survey utilizing Delphi methodology. Clinicians rated the importance of various factors used to evaluate the severity of CLDP, from 0 (not at all important) to 10 (very important) for the period between discharge home from the NICU and 12 months CA. Fourteen factors were considered in Round 1; 13 in Rounds 2 and 3. The relative importance of factors was explored via a set of 16 single-profile tasks (i.e., hypothetical patient profiles with varying CLDP severity levels). RESULTS: Overall, 91 clinicians from 11 countries who were experienced in treating prematurity-related lung diseases completed Round 1; 88 completed Rounds 2 and 3. Based on Round 3, the most important factors in determining CLDP severity were mechanical ventilation (mean absolute importance rating, 8.89), supplemental oxygen ≥2 L/min (8.49), rehospitalizations (7.65), and supplemental oxygen <2 L/min (7.56). Single-profile tasks showed that supplemental oxygen had the greatest impact on profile classification. CONCLUSION: The most important factors for clinicians assigning CLDP severity during infancy were mechanical ventilation, supplemental oxygen ≥2 L/min, and respiratory-related rehospitalizations.

Patient perspectives on the pathway to psoriatic arthritis diagnosis: results from a web-based survey of patients in the United States.

BACKGROUND: There are limited real-world data on the diagnostic experiences of patients with psoriatic arthritis (PsA), including medical care sought and potential barriers to diagnosis. We aim to describe patient experiences related to receiving a PsA diagnosis. METHODS: Ours was a mixed-method, 2-phase study. Phase 1 comprised concept elicitation and cognitive interviews with clinical experts and adults diagnosed with PsA to develop a cross sectional, web-based survey. US adults with a self-reported PsA diagnosis were recruited through a patient support community (CreakyJoints), an online patient research registry (ArthritisPower), and social media outreach. In Phase 2, the online survey collected data on sociodemographics, clinical symptoms, disease burden, and diagnosis history of survey respondents with PsA. RESULTS: Of the 203 respondents included, 172 (84.7%) were female, and the mean (SD) age was 51.6 (10.8) years. The time between seeking medical attention and receiving a diagnosis was < 6 months for 69 respondents, 6 months to 4 years for 68 respondents, and ≥ 5 years for 66 respondents. Most respondents sought care from general practitioners (79.8%) and rheumatologists (66.5%). Common initial symptoms that led respondents to seek medical attention were joint pain (70.0%) and stiffness (53.7%). Among the initial symptoms that led respondents to seek care, joint pain, swollen joints, and sausage-like fingers or toes (indicating dactylitis) were more common among respondents with shorter time to diagnosis, whereas stiffness, fatigue, enthesitis (indicated by foot problems, tendon and ligament pain), and back pain were more common among respondents with longer time to diagnosis. Common misdiagnoses were psychosomatic issues (26.6%) and osteoarthritis (21.7%). Respondents with shorter times to diagnosis had lower frequencies of misdiagnosis. CONCLUSIONS: Respondents with PsA reported delays in diagnosis and misdiagnoses on their journey to a PsA diagnosis. Symptom differences, such as enthesitis and stiffness, were noted among respondents with shorter vs longer time to diagnosis. Increased understanding of diagnostic barriers may lead to earlier diagnosis and appropriate management to improve outcomes.

The Patient Perspective: Unmet Treatment Needs in Adults With Attention-Deficit/Hyperactivity Disorder.

OBJECTIVE: To characterize impairments in daily life experienced by pharmacologically treated adults with attention-deficit/hyperactivity disorder (ADHD) versus adults without ADHD and to identify unmet needs in ADHD treatment from the perspective of adults with ADHD. METHODS: Adults with ADHD taking prescription medication for ≥ 6 months and adults without ADHD agreed to participate in a cross-sectional online survey during December 2016. Participants with ADHD were stratified by their current ADHD medication: long-acting (LA) once daily, short-acting (SA) ≤ 2 times/d, and augmenters (AU; LA > 1 time/d, SA > 2 times/d, or LA plus SA). RESULTS: A total of 616 adults with ADHD (SA: n = 166, LA: n = 201, AU: n = 249) and 200 adults without ADHD completed the survey. Even with treatment, adults with ADHD reported substantial impairments in their everyday life, particularly at home, at school/work, and in their social life and relationships. Participants with ADHD experienced impairments throughout the day, especially in the afternoon and evening. Signs or symptoms were reported when the ADHD medication was wearing off, resulting in negative effects (including school work, homework, work responsibilities, household responsibilities, emotional responses, mood, and relationships) on the daily life of adults with ADHD. CONCLUSIONS: Adults with ADHD, despite receiving medication, experienced impairments and challenges in many aspects of their daily life. Adults with ADHD described various unmet needs, especially those relating to the duration of treatment effect. When optimizing treatment for adults with ADHD, it is important that the treatment regimen is sufficient to meet the needs of the patient throughout the day.

Real-World Patient Experience on the Path to Diagnosis of Ankylosing Spondylitis.

INTRODUCTION: We describe the journey to diagnosis of ankylosing spondylitis (AS) from the patient perspective and examine differences in this journey by sex. METHODS: US adults aged ≥ 18 years with a self-reported AS diagnosis were recruited online through CreakyJoints, a patient support community, and ArthritisPower, a patient research registry. Respondents completed a web-based survey on sociodemographics, disease burden, and diagnosis history. Results were stratified by sex and time to diagnosis using two-sample t tests and χ2 tests, respectively, to observe differences across the groups; P < 0.05 was considered statistically significant. RESULTS: Among 235 respondents, 174 (74.0%) were female. Mean (SD) ages of female and male respondents were 48.6 (10.6) and 53.1 (10.3) years, respectively. From the time respondents began seeking medical attention, 87 were diagnosed within ≤ 1 year, 71 in 2-9 years, and 77 after ≥ 10 years. Symptoms that led respondents to seek treatment were back pain (73.2%) and joint pain (63.8%); fatigue and difficulty sleeping were more common among respondents with longer times to diagnosis. During the diagnosis process, men with AS tended to receive quicker AS diagnosis compared with women. Overall, commonly reported initial diagnoses among respondents with longer time to AS diagnosis included back problems and psychosomatic disorders. Significantly more women reported misdiagnoses of fibromyalgia (20.7 vs. 6.6%) and psychosomatic disorders (40.8 vs. 23.0%) compared with men. CONCLUSIONS: Diagnosis delays and misdiagnoses were common among respondents with AS. Increasing awareness about AS among referring providers may minimize diagnosis delay. FUNDING: Novartis Pharmaceuticals Corporation. Plain language summary available for this article.