Exploring the content validity of the EQ-5D-5L and four bolt-ons (skin irritation, self-confidence, sleep, social relationships) in atopic dermatitis and chronic urticaria.

Background: The objective of this study was to assess the content validity of the EQ-5D-5L and four bolt-ons: skin irritation, self-confidence, social relationships and sleep, for people with atopic dermatitis (AD) and chronic urticaria (CU).

Methods: Adults with AD or CU in the United Kingdom, with varying levels of severity, participated in either online or in-person semi-structured interviews. During the interviews, participants were first asked about the symptoms and impacts of their condition.

Estimating the Health-Related Quality of Life Benefit of Prophylactic Treatment for COVID-19 in Immunocompromised People: A Multimethod Valuation Study.

Pre-exposure prophylaxis (PrEP) for COVID-19 provides additional protection, beyond vaccines alone, for individuals who are immunocompromised (IC). This may reduce the need for preventative behavioral modification, such as shielding-a behavioral restriction limiting an IC individual to minimize face-to-face interactions and/or crowded places. Therefore, PrEP may improve psychosocial well-being and health-related quality of life (HRQoL) for individuals with IC conditions.

Estimating utility values for non-alcoholic steatohepatitis health states: a discrete choice experiment.

This study estimated utility values for non-alcoholic steatohepatitis (NASH). Previous studies have assumed that health-related quality of life does not vary between the early stages of NASH. Discrete choice experiment (DCE) surveys estimated the value of avoiding fibrosis progression.

Exploring the impact of caring for an individual with neurogenic orthostatic hypotension: a qualitative study.

This study aimed to explore the impact of caring for an individual with neurogenic orthostatic hypotension (nOH). Informal caregivers of individuals with nOH and either Parkinson's disease, multiple system atrophy, pure autonomic failure or dementia with Lewy bodies completed semi-structured interviews. Data were analyzed using thematic analysis; the identified concepts were used to develop a conceptual model.

Quality of Life with Late-Onset Pompe Disease: Qualitative Interviews and General Public Utility Estimation in the United Kingdom.

Late-onset Pompe disease (LOPD) is a rare, progressive neuromuscular condition typically characterized by weakness of skeletal muscles, including those involved in respiration and diaphragmatic dysfunction. Individuals with LOPD typically eventually require mobility and/or ventilatory support. This study aimed to develop health state vignettes and estimate health state utility values for LOPD in the United Kingdom.

Health-related quality-of-life and burden for caregivers of individuals with neurogenic orthostatic hypotension.

This study explores the burden of caring for an individual with neurogenic orthostatic hypotension (nOH) and an underlying neurodegenerative disease (Parkinson's disease, multiple system atrophy, pure autonomic failure or dementia with Lewy bodies). A survey including several validated instruments was conducted with informal caregivers of individuals with Parkinson's disease, multiple system atrophy, pure autonomic failure or dementia with Lewy bodies. Caregivers of patients with nOH (n = 60) reported greater burden across all outcomes compared with those without nOH (n = 60).

Real-World experience of interictal burden and treatment in migraine: a qualitative interview study.

Background: The debilitating nature of migraine attacks is widely established; however, less is known about how the interictal burden (i.e., how patients are affected in-between migraine episodes) of migraine impacts on patients' health-related quality of life (HRQL).

Exploring Health-Related Quality of Life Burden in Peanut Allergy and the Potential Benefit of Oral Immunotherapy: Estimation of Health State Utility Values for Children and Adolescents and Their Caregivers.

Purpose: This study aimed to estimate utility values for health states relating to oral immunotherapy (OIT) for peanut allergy (PA), for children with PA and their caregivers.

Patients And Methods: Two methods were used: an online survey and structured interviews. Both methods assessed current utility/untreated PA (health-related quality of life, HRQoL) and HRQoL in different health states: "up-dosing phase of treatment", "maintenance phase" and "able to tolerate 6-8 peanuts if accidentally ingested".

Allergy to Peanuts imPacting Emotions And Life (APPEAL): The impact of peanut allergy on children, teenagers, adults and caregivers in the UK and Ireland.

The Allergy to Peanuts imPacting Emotions And Life study (APPEAL) explored the psychosocial burden of living with self-reported peanut allergy experienced by children, teenagers, adults and caregivers in the UK and Ireland. A two-stage (quantitative survey and qualitative interview [APPEAL-1]), cross-sectional study of the psychosocial burden of peanut allergy (APPEAL-2) was conducted. Quantitative data were evaluated using descriptive statistics and qualitative data were analysed using MAXQDA software.

Impact of developmental and epileptic encephalopathies on caregivers: A literature review.

Objectives: Developmental and epileptic encephalopathies (DEEs) are rare neurodevelopmental disorders characterized by early-onset seizures and numerous comorbidities. Due to the complex requirements for the care of a child with a DEE, these disorders would be expected to impact health-related quality of life (HRQL) for caregivers as well as for patients. The objective of this literature review was to describe the impact of DEEs on the HRQL, emotional wellbeing, and usual activities (social, work, relationships, etc.

Allergy to Peanuts imPacting Emotions And Life (APPEAL): the impact of peanut allergy on children, adolescents, adults and caregivers in France.

Background: Peanut allergy (PA) has increased in developed countries and can have a dramatic effect on quality of life but data surrounding this is limited in France. Allergy to Peanuts imPacting Emotions And Life study (APPEAL) investigated the experience and impact of living with PA in France.

Methods: Respondents affected by PA directly (children aged 8-12 years, teenagers aged 13-17 years, or adults aged ≥ 18 years) or indirectly (caregiver) completed either an online questionnaire (APPEAL-1, N = 198), or provided in-depth interviews (APPEAL-2, N = 32).

Psychosocial and productivity impact of caring for a child with peanut allergy.

Background: Limited previous research has assessed the psychosocial burden and productivity impact of caring for a child with peanut allergy and factors associated with burden. The objective of this research was to explore caregiver burden in terms of psychosocial and productivity impact of caring for a child with peanut allergy, the influence of caregiver and child gender on caregiver burden, and factors predicting caregiver burden in peanut allergy.

Methods: A cross-sectional survey of caregivers of children with peanut allergy was conducted in the United Kingdom, and included sociodemographic and clinical questions, EQ-5D, Hospital Anxiety and Depression Scale, Food Allergy Quality of Life-Parental Burden, Food Allergy Independent Measure, and productivity questions.

Peanut allergy impact on productivity and quality of life (PAPRIQUA): Caregiver-reported psychosocial impact of peanut allergy on children.

Background: Limited research has examined the impact of peanut allergy (PA) on children using validated instruments to assess psychosocial burden and the factors influencing burden.

Objective: The PAPRIQUA study aimed to assess the caregiver-reported impact of living with PA on children's health-related quality of life (HRQL), correlations between PA severity and child's sex, and associations of caregivers' sex and anxiety with the proxy report of their child's HRQL and to identify significant predictors of a child's HRQL.

Methods: A cross-sectional survey of caregivers of children with mild, moderate and severe PA, based on caregiver perception, was conducted in the United Kingdom.

APPEAL-2: A pan-European qualitative study to explore the burden of peanut-allergic children, teenagers and their caregivers.

Background: Allergy to Peanuts ImPacting Emotions And Life (APPEAL-1) was a recent European multi-country questionnaire survey that highlighted the negative impacts of peanut allergy (PA) on quality of life. A follow-on qualitative study, APPEAL-2, further assessed the burden of PA and associated coping strategies through semi-structured interviews.

Objective: To gain qualitative insight on the strategies used to cope with and manage PA and the impact of these strategies on the quality of life of children, teenagers and caregivers.

Estimation Of The Quality Of Life Benefits Associated With Treatment For Spinal Muscular Atrophy.

Background: Spinal muscular atrophy (SMA) is a rare, genetic, progressive neuromuscular disorder characterized by severe muscle atrophy and weakness and is a leading genetic cause of death in infants and children. Nusinersen was the first treatment targeting the underlying cause of disease approved by the FDA, EMA and other countries for patients with SMA. There are currently very limited data available on the health-related quality of life (HRQoL) burden of SMA suitable for use in a cost-effectiveness analysis.

Disutility Study for Adult Patients with Moderate to Severe Crohn's Disease.

Background: Crohn's disease (CD) treatments and associated adverse events (AEs) can be burdensome for patients. However, specific values which quantify the impact on health-related quality of life (HRQL) for economic evaluation are lacking.

Objectives: This study aimed to elicit health utility values for AEs related to biologic treatment and surgical complications for CD in the UK.

NMDA receptor antagonists and pain relief: A meta-analysis of experimental trials.

Objective: We conducted a meta-analysis of controlled trials that used experimental models of acute pain and hyperalgesia to examine the analgesic effects of NMDA receptor (NMDAR) antagonists.

Methods: Six major databases were systematically searched (to March 2018) for studies using human evoked pain models to compare NMDAR antagonists with no-intervention controls. Pain outcome data were analyzed with random-effects meta-analysis.

A Novel Patient-Derived Conceptual Model of the Impact of Celiac Disease in Adults: Implications for Patient-Reported Outcome and Health-Related Quality-of-Life Instrument Development.

Background: Celiac disease is a chronic inflammatory condition with wide ranging effects on individual's lives caused by a combination of symptoms and the burden of adhering to a gluten-free diet (GFD).

Objectives: To further understand patients' experience of celiac disease, the impact it has on health-related quality of life (HRQOL), and to develop a conceptual model describing this impact.

Methods: Adults with celiac disease on a GFD reporting symptoms within the previous 3 months were included; patients with refractory celiac disease and confounding medical conditions were excluded.

Pain perception in Parkinson's disease: A systematic review and meta-analysis of experimental studies.

While hyperalgesia (increased pain sensitivity) has been suggested to contribute to the increased prevalence of clinical pain in Parkinson's disease (PD), experimental research is equivocal and mechanisms are poorly understood. We conducted a meta-analysis of studies comparing PD patients to healthy controls (HCs) in their response to experimental pain stimuli. Articles were acquired through systematic searches of major databases from inception until 10/2016.

Social preference weights for treatments in Fabry disease in the UK: a discrete choice experiment.

Objective: Fabry disease is a rare inherited lysosomal storage disorder caused by deficiency of α-galactosidase A. Effective enzyme replacement therapies are available that are administered intravenously. However, a new oral treatment is being developed as an alternative option for patients with amenable mutations.

Is Pain Perception Altered in People With Depression? A Systematic Review and Meta-Analysis of Experimental Pain Research.

Unlabelled: Although clinical studies suggest depressed patients may be more vulnerable to pain, experimental research is equivocal. This meta-analysis aimed to clarify whether depression is associated with altered pain perception in response to noxious stimulation and to identify factors that might influence this association. A search of major electronic databases was conducted to identify experimental studies investigating pain response in depressed participants versus healthy control participants using established pain outcome measures.

A qualitative evaluation of the validity of published health utilities and generic health utility measures for capturing health-related quality of life (HRQL) impact of differentiated thyroid cancer (DTC) at different treatment phases.

Purpose: This study explored the impact of differentiated thyroid cancer (DTC) on health-related quality of life (HRQL) at different treatment phases and evaluated the validity of published DTC utilities and generic health utility measures (EQ-5D and SF-6D) for economic evaluation of treatments for radio-iodine (RAI) refractory DTC.

Methods: Focus groups and interviews were conducted with DTC patients grouped by treatment phase. Qualitative thematic analysis was conducted on interview/focus group transcripts.

How do children and their caregivers perceive the benefits of inhaled asthma therapy?

Objective: Although well reported in adults, there is relatively little data on how children with asthma and their parents describe their attitudes to the disease, expectations of therapy, and perception of treatment benefit. We investigated this to determine if they differed from reports by adults with asthma.

Methods: We recruited families with an asthmatic child (4-11 years) who had recently been prescribed a change in treatment [starting inhaled corticosteroid monotherapy (ICS) or changing from ICS to inhaled corticosteroid/long-acting β(2)-agonist combination therapy (ICS/LABA)].

Lennox-Gastaut syndrome (LGS): development of conceptual models of health-related quality of life (HRQL) for caregivers and children.

LGS is a severe form of childhood epilepsy which is characterized by multiple seizures and cognitive impairment. Semi-structured interviews were conducted with 40 parents of children with LGS in the US, UK, and Italy. Parents were asked to report on their perceptions of the HRQL of their child and also to describe the impact on their own HRQL.