Peanut allergy burden survey: Factors associated with health-related quality of life in adolescents.

BACKGROUND: Patients with peanut allergy (PA) experience significant burden of illness, which impacts health-related quality of life (HRQoL), particularly in adolescence. There is a paucity of research evaluating drivers of HRQoL scores. METHODS: A prospective, online survey of adolescents with self-reported, provider-diagnosed PA completed from November 2018 to January 2019 was used to explore drivers of the real-world impact of PA on HRQoL using the Pediatric Quality of Life Inventory 4.0 (PedsQL) and other measures. Univariate and multivariate analyses were used to identify potential factors associated with PedsQL scores and to understand the level of association. RESULTS: A total of 102 adolescents were included. The final model included 10 variables: race, reported strict peanut avoidance, satisfaction with prophylaxis, moderate-to-severe reaction within the past 12 months, touching peanut as cause of most severe reaction, fear of reaction, age, gender, comorbidities, and daily life limitations. In total, three items were shown to be strong predictors of the PedsQL total score including cause of severe reaction was touching peanut (yes), level of agreement with avoiding peanut (completely agree), and satisfaction with prophylaxis (not very much/not at all). CONCLUSIONS: There is substantial heterogeneity in the impact of the burden of PA on PedsQL scores across patients. This indicates the importance of shared and individualized decision making for PA management to optimize outcomes and improve HRQoL.

Real-world experience of pediatric patients treated with peanut (Arachis hypogaea) allergen powder-dnfp.

BACKGROUND: Peanut (Arachis hypogaea) allergen powder-dnfp (PTAH) is the first oral immunotherapy indicated for children aged 4 to 17 years with peanut allergy. There are limited real-world data on patients treated with PTAH. OBJECTIVE: To characterize pediatric patients treated with PTAH and associated treatment patterns in US clinical practice. METHODS: US-based physicians with allergy and immunology training treating patients with peanut allergy aged 4 to 17 years with PTAH were recruited from an existing physician panel and completed an online case report form (October to December 2021) with data abstracted from patient medical charts. Physician practice circumstances, patient characteristics, and PTAH treatment patterns were reported. Time to reach the 300-mg dose and treatment persistence were assessed using Kaplan-Meier analysis. RESULTS: A geographically balanced sample of 43 physicians contributed data for 118 demographically diverse pediatric patients. Patients had heterogeneous diagnostic test results, with a wide range of peanut-specific immunoglobulin E levels; 6.8% received an oral food challenge. During the updosing phase, there were no temporary interruptions and 5.1% of the patients required downdosing. Patients reached the 300-mg dose at a median of 21.3 weeks post-initiation. The rate of PTAH persistence at 24 weeks was 93.4%. Only 1 patient discontinued treatment because of treatment-related systemic allergic symptoms, and the remaining discontinuations were for reasons other than treatment-related symptoms. Prophylactic antihistamines were used by 33.9% of the patients to prevent PTAH adverse effects. CONCLUSION: PTAH was prescribed in demographically diverse patients with a wide range of peanut-specific immunoglobulin E levels. Treatment persistence with PTAH was high in this study population, with a small number of patients experiencing treatment modification.

Qualitative interviews to understand health care providers' experiences of prescribing licensed peanut oral immunotherapy.

OBJECTIVE: This research sought to explore health care providers' (HCPs) experiences of delivering the first US Food and Drug Administration (FDA) and European Commission (EC) approved peanut oral immunotherapy (peanut OIT; Palforzia). Semi-structured qualitative interviews with HCPs who had initiated treatment with ≥ 3 patients in the first nine months following FDA approval sought to identify challenges faced and successful implementation strategies. RESULTS: Eight allergists and three nurse practitioners from eight sites based in the United States participated. The HCPs included in this research were motivated to implement this novel treatment, however, entered the process with some reservations. HCPs described how successful implementation of peanut OIT requires them to be thoughtful about their clinic's abilities to integrate complex, time-consuming treatments into their daily practice. Prior experience of OIT was deemed beneficial, but not essential for implementation and learning from others' experience was suggested as a way of helping new prescribers overcome perceived and actual implementation challenges. Delivering licensed peanut OIT during the COVID-19 pandemic posed both challenges and unexpected opportunities for implementation. The experiences described have the potential to benefit the wider allergy community by providing practical solutions, successful implementation strategies and opportunities to enhance training and resources.

The Risk Reduction of Accidental Exposure-Related Systemic Allergic Reactions Extrapolated Based on Food Challenge Data After 1 Year of Peanut Oral Immunotherapy.

INTRODUCTION: The phase 3 trial PALISADE, comparing peanut (Arachis hypogaea) allergen powder-dnfp (PTAH) oral immunotherapy versus placebo in peanut-allergic children, reported that a significantly higher percentage of PTAH-treated participants tolerated higher doses of peanut protein after 1 year of treatment. This study used PALISADE data to estimate the reduction in the risk of systemic allergic reaction (SAR) after accidental exposure following 1 year of PTAH treatment. METHODS: Participants (aged 4-17 years) enrolled in PALISADE were included. Parametric interval-censoring survival analysis with the maximum likelihood estimation was used to construct a real-world distribution of peanut protein exposure using lifetime SAR history and highest tolerated dose (HTD) from a double-blind, placebo-controlled food challenge conducted at baseline. The SAR risk reduction was extrapolated using the exposure distribution and the HTD were collected at baseline and trial exit for PTAH- and placebo-treated participants. RESULTS: Assuming a maximum peanut protein intake of 1500 mg, participants were estimated to have < 1% probability of ingesting > 0.01 mg during daily life. The mean annual SAR risk at trial entry was 9.25-9.98%. At trial exit, the relative SAR risk reduction following accidental exposure was 94.9% for PTAH versus 6.4% for placebo. For PTAH-treated participants with exit HTD of 600 or 1000 mg without dose-limiting symptoms, the SAR risk reduction increased to 97.2%. The result was consistent in the sensitivity analysis across different parametric distributions. CONCLUSION: Oral immunotherapy with PTAH is expected to result in a substantially greater reduction in risk of SAR following accidental exposure compared to placebo among children with peanut allergy.

The Peanut Allergy Burden Study: Impact on the quality of life of patients and caregivers.

BACKGROUND: Peanut allergy (PA) places significant burden on peanut-allergic individuals and their families, yet limited research in the United States has quantitatively examined the impact on peanut-allergic individuals and their families' health-related quality of life (HRQoL). The Peanut Allergy Burden Study (PABS) aimed to quantify the impact of PA on the general and disease-specific HRQoL of children, adolescents, and adults with PA, as well as caregivers of children with PA. METHODS: A cross-sectional survey design was employed to examine the real-world impact of PA in children, adolescents, and adults with PA, and caregivers of children with PA. RESULTS: Of 153 adult patients, 102 adolescents, and 382 caregivers of peanut-allergic children (n = 382), 6.8% and 24.8% of participants indicated being dissatisfied or somewhat dissatisfied, respectively, with current approaches to avoid or prevent PA reactions. Approximately two-thirds of patients and caregivers indicated that PA interferes at least somewhat with daily living. In terms of general HRQoL, adolescents, adult patients, and caregivers indicated that mental/psychosocial health was more problematic than physical health. PA patients and caregivers indicated worse HRQoL in all domains compared to healthy samples, and worse overall HRQoL, psychosocial, emotional, and social functioning than a sample of chronically ill patients. Results from the allergy-specific HRQoL measures showed that adolescents experienced greater impairment in overall HRQoL due to PA and in allergen avoidance and dietary restriction than adults. CONCLUSION: PA negatively affects the general and PA-specific HRQoL of both patients and caregivers. The high emotional and psychosocial burden, in particular, demonstrates significant unmet need for patients with PA and their caregivers. Future work on treatment and preventive options to improve HRQoL for PA patients, particularly adolescents and their families, is needed.

Exploring the implementation of patient-reported outcome measures in cancer care: need for more real-world evidence results in the peer reviewed literature.

BACKGROUND: To explore the existing evidence of the real-world implementation of patient-reported outcomes (PROs) in oncology clinical practice and address two aims: (1) summarize available evidence of PRO use in clinical practice using a framework based on the International Society for Quality of Life Research (ISOQOL) PRO Implementation Guide; and (2) describe reports of real-world, standardized PRO administration in oncology conducted outside of scope of a research study. METHODS: A Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) protocol was developed to guide the systematic literature review (SLR) that was conducted in MEDLINE and Embase databases. A two step search strategy was implemented including two searches based on previously completed reviews. Studies published from 2006 to 2017 were synthesized using a framework based on the ISOQOL PRO Implementation Guide. RESULTS: After screening 4427 abstracts, 36 studies met the eligibility criteria. Most elements of the ISOQOL PRO Implementation Guide were followed. Two notable exceptions were found: 1) providing PRO score interpretation guidelines (39% of studies); and 2) providing patient-management guidance for addressing issues identified by PROs (25% of studies). Of the 22 studies with an intervention component, 19 (86%) reported intervention effects on study outcomes. The European Organisation for Research and Treatment of Cancer Quality-of-Life Questionnaire-Core 30 (EORTC QLQ-C30) was the most commonly used PRO (n = 10, 28%); use of 38 other PRO measures was also reported. Only three studies (8%) reported real-world PRO implementation. CONCLUSION: Reports of real-world PRO implementation are limited. Reports from studies conducted in clinical settings suggest gaps in information on PRO score interpretation and the use of PRO results to inform patient management. Before the promise of practice-based PRO assessment in oncology can be truly realized, investigators need to advance the state-of-the-art of real-time PRO score interpretation as well as developing guidance on how to use PRO insights to drive clinically-meaningful patient-management strategies.

Development and validation of the Ulcerative Colitis patient-reported outcomes signs and symptoms (UC-pro/SS) diary.

BACKGROUND: The clinical course of ulcerative colitis (UC) and the effects of treatment are assessed through patient-reported signs and symptoms (S&S), and endoscopic evidence of inflammation. The Ulcerative Colitis Patient-Reported Outcomes Signs and Symptoms (UC-PRO/SS) measure was developed to standardize the quantification of gastrointestinal S&S of UC in clinical trials through direct report from patient ratings. DESIGN: The UC-PRO/SS was developed by collecting data from concept elicitation (focus groups, and individual interviews), then refined through a process of cognitive interviews of 57 UC patients. Measurement properties, including item-level statistics, scaling structure, reliability, and validity, were evaluated in an observational, four-week study of adults with mild to severe UC (N = 200). RESULTS: Findings from qualitative focus groups and interviews identified nine symptom items covering bowel and abdominal symptoms. The final UC-PRO/SS daily diary includes two scales: Bowel S&S (six items) and Abdominal Symptoms (three items), each scored separately. Each scale showed evidence of adequate reliability (α = 80 and 0.66, respectively); reproducibility (intraclass correlation coefficient = 0.81, 0.71) and validity, including moderate-to-high correlations with the Partial Mayo Score (0.79; 0.45) and Inflammatory Bowel Disease Questionnaire (IBDQ) total score (- 0.70; - 0.61). Scores discriminated by level of disease severity, as defined by the Partial Mayo Score, Patient Global Rating, and Clinician Global Rating (p < 0.0001). CONCLUSIONS: Results suggest that the UC-PRO/SS is a reliable and valid measure of gastrointestinal symptom severity in UC patients. Additional longitudinal data are needed to evaluate the ability of the UC-PRO/SS scores to detect responsiveness and inform the selection of responder definitions.

Development and validation of the Crohn's disease patient-reported outcomes signs and symptoms (CD-PRO/SS) diary.

BACKGROUND: The clinical course of Crohn's disease (CD) and the effect of its treatment are monitored through patient-reported signs and symptoms (S&S), and endoscopic evidence of inflammation. The Crohn's Disease Patient-reported Outcomes Signs and Symptoms (CD-PRO/SS) measure was developed to standardize the quantification of gastrointestinal S&S of CD through direct report from patient ratings. METHODS: The CD-PRO/SS was developed based on data from concept elicitation (focus groups, interviews; n = 29), then refined through cognitive interviews of CD patients (n = 20). Measurement properties, including item-level statistics, scaling structure, reliability, and validity, were examined using secondary analyses of baseline and two-week clinical trial data of adults with moderate-to-severe CD (n = 238). RESULTS: Findings from qualitative interviews identified nine S&S items covering bowel and abdominal symptoms. The final CD-PRO/SS daily diary includes two scales: Bowel S&S (three items) and Abdominal Symptoms (three items), each scored separately. Each scale showed evidence of adequate reliability (α = 0.74 and 0.67, respectively); reproducibility (intraclass correlation coefficient > 0.80), and validity, with the last including moderate correlations with the Inflammatory Bowel Disease Questionnaire bowel symptom score and select items (ranging from r = 0.43-0.54). Scores distinguished patients categorized by patient global ratings of disease severity (p < 0.0001). CONCLUSIONS: Results suggest the CD-PRO/SS is a reliable and valid measure of gastrointestinal symptom severity in CD patients. Additional longitudinal data are needed to evaluate the ability of the CD-PRO/SS scores to detect responsiveness and inform the selection of responder definitions.

A randomized intervention to improve heart failure outcomes in community-based home health care.

This study examines the effects of a home health intervention designed to standardize nursing care, strengthen nurses' support for patient self-management and yield better CHF patient outcomes. Participants were 371 Medicare CHF patients served by 205 nurses randomized to intervention and control groups in a large urban home healthcare agency (HHA). The intervention consisted of an evidence-based nursing protocol, patient self-care guide, and training to improve nurses'teaching and support skills. Outcome measures included home care,physician and emergency department (ED) use, hospital admission, condition-specific quality of life (QoL), satisfaction with home care services and survival at 90 days. The intervention was associated with a marginally significant reduction in the volume of skilled nursing visits (p = .074), and a reduction variation in the typical number of visits provided (p < .05), without a significant increase in physician or ED use or patient mortality. Hypothesized improvement in other outcomes did not occur.