Determining the Relationship Between Seizure-Free Days and Other Predictors of Quality of Life in Patients with Dravet Syndrome and Their Carers from FFA Registration Studies.

INTRODUCTION: Dravet syndrome (DS) is a rare, lifelong epileptic encephalopathy characterised by frequent and severe seizures associated with premature mortality. Typically diagnosed in infancy, patients also experience progressive behavioural, motor-function and cognitive decline. Twenty percent of patients do not reach adulthood. Quality of life (QoL) is impaired for both patients and their carers. Reducing convulsive seizure frequency, increasing convulsive seizure-free days (SFDs) and improving patient/carer QoL are primary treatment goals in DS. This study explored the relationship between SFDs and patients' and carers' QoL to inform a cost-utility analysis of fenfluramine (FFA). METHODS: In FFA registration studies, patients (or their carer proxies) completed the Paediatric QoL inventory (PedsQL). These data were mapped to EuroQol-5 Dimensions Youth version (EQ-5D-Y) to provide patient utilities. Carer utilities were collected using EQ-5D-5L and mapped to EQ-5D-3L to align patient and carer QoL on the same scale. Linear mixed-effects and panel regression models were tested and Hausman tests identified the most appropriate approach for each group. On this basis, a linear mixed-effects regression model was used to examine the relationships between patient EQ-5D-Y and clinically relevant variables (age, frequency of SFDs per 28 days, motor impairments and treatment dose). A linear panel regression model examined the relationship between SFDs and carer QoL. RESULTS: After adjustment for age and underlying comorbidities, the patient regression model showed that SFDs per 28 days was a significant predictor of QoL. Each additional patient-SFD increased utility by 0.005 (p < 0.001). The carer linear panel model also showed that increasing SFDs per 28 days was a significant predictor of improved QoL. Each additional SFD increased carer utility by 0.014 (p < 0.001). CONCLUSION: This regression framework highlights that SFDs are significantly correlated with both patients' and carers' QoL. Treatment with effective antiseizure medications that increase SFDs directly improves QoL for patients and their carers.

Key considerations when involving children in health intervention design: reflections on working in partnership with South Asian children in the UK on a tailored Management and Intervention for Asthma (MIA) study.

Participatory research is an empowering process through which individuals can increase control over their lives, and allows researchers/clinicians to gain a clearer understanding of a child's needs. However, involving children in participatory research is still relatively novel, despite national and international mandates to engage children in decision making. This paper draws on the learnings from designing the Management and Intervention for Asthma (MIA) study, which used a collaborative participatory method to develop an intervention-planning framework for South Asian children with asthma. There are currently 1 million children in the UK receiving treatment for asthma, making it one of the most prevalent chronic childhood illnesses. Symptoms of asthma are often underrecognized in children from South Asian communities in the UK, contributing to increased disease severity and increased attendance at the emergency department compared to White British children. Despite this, ethnic minorities are often excluded from research and thus absent from the 'evidence base', making it essential to hear their perspectives if health inequalities are to be successfully addressed. We worked alongside healthcare professionals, community facilitators, parents, and children to identify the key concerns and priorities they had and then designed the framework around their needs. Reflecting on the process, we identified several key considerations that need to be addressed when co-developing interventions with children. These include the power dynamics between the parent/researcher and child; navigating the consent/assent process; how parental involvement might affect the research; establishing a convenient time and location; how to keep children engaged throughout the process; tailoring activities to different levels of ability; and accounting for cultural differences. These factors were considered by the researchers when designing the study, however, implementing them was not without its challenges and highlighted the need for researchers to develop expertise in this field. Tailoring existing research methods allowed us to explore children's perceptions, priorities, and experiences of illness more effectively. However, involving children in participatory research is a complex undertaking, and researchers need to ensure that they have the expertise, time, and resources necessary to be able to fully support the needs of child participants before deciding to commit to this approach.

Involving families in health research gives children the opportunity to have more control over their healthcare and can help researchers/clinicians to better understand a child's needs. However, involving children in research is still an uncommon practice despite international pressures to include children in decision making. This paper reflects on the development of the Management and Intervention for Asthma study, which worked with communities to develop a framework for planning health interventions for South Asian children with asthma. There are currently 1 million children in the UK being treated for asthma, making it one of the most common chronic childhood illnesses. Symptoms of asthma are overlooked in children from South Asian communities in the UK, leading to worse health outcomes and more visits to the emergency department. We worked with the community to identify the key concerns and priorities they had, and then jointly designed the framework around their needs. We identified several key things to consider when co-developing interventions with children: Power dynamics Consent/Assent Parental involvement Time and location Engagement Diversity of needs Ethnicity and Culture Adapting our research and engagement methods to suit children's needs enabled us to involve them in the study in a way that allowed us to effectively explore their views, priorities, and experiences of their illness. However, researchers need to make sure that they have the expertise, time, and resources to be able to support the needs of child participants.

A structured collaborative approach to intervention design using a modified intervention mapping approach: a case study using the Management and Interventions for Asthma (MIA) project for South Asian children.

BACKGROUND: To describe how using a combined approach of community-based participatory research and intervention mapping principles could inform the development of a tailored complex intervention to improve management of asthma for South Asian (SA) children; Management and Interventions for Asthma (MIA) study. METHODS: A qualitative study using interviews, focus groups, workshops, and modified intervention mapping procedures to develop an intervention planning framework in an urban community setting in Leicester, UK. The modified form of intervention mapping (IM) included: systematic evidence synthesis; community study; families and healthcare professionals study; and development of potential collaborative intervention strategies. Participants in the community study were 63 SA community members and 12 key informants; in-depth semi-structured interviews involved 30 SA families, 14 White British (WB) families and 37 Healthcare Professionals (HCPs) treating SA children living with asthma; prioritisation workshops involved 145 SA, 6 WB and 37 HCP participants; 30 participants in finalisation workshops. RESULTS: Two key principles were utilised throughout the development of the intervention; community-based participatory research (CBPR) principles and intervention mapping (IM) procedures. The CBPR approach allowed close engagement with stakeholders and generated valuable knowledge to inform intervention development. It accounted for diverse perceptions and experiences with regard to asthma and recognised the priorities of patients and their families/caregivers for service improvement. The 'ACT on Asthma' programme was devised, comprising four arms of an intervention strategy: education and training, clinical support, advice centre and raising awareness, to be co-ordinated by a central team. CONCLUSIONS: The modified IM principles utilised in this study were systematic and informed by theory. The combined IM and participatory approach could be considered when tailoring interventions for other clinical problems within diverse communities. The IM approach to intervention development was however resource intensive. Working in meaningful collaboration with minority communities requires specific resources and a culturally competent methodology.

Qualitative study to identify ethnicity-specific perceptions of and barriers to asthma management in South Asian and White British children with asthma.

OBJECTIVE: This paper draws on the data from the Management and Interventions for Asthma (MIA) study to explore the perceptions and experiences of asthma in British South Asian children using semi-structured interviews. A comparable cohort of White British children was recruited to identify whether any emerging themes were subject to variation between the two groups so that generic and ethnicity-specific themes could be identified for future tailored intervention programmes for South Asian children with asthma. SETTING: South Asian and White British children with asthma took part in semi-structured interviews in Leicester, UK. PARTICIPANTS: Thirty three South Asian and 14 White British children with asthma and aged 5-12 years were interviewed. RESULTS: Both similar and contrasting themes emerged from the semi-structured interviews. Interviews revealed considerable similarities in the experience of asthma between the South Asian and White British children, including the lack of understanding of asthma (often confusing trigger with cause), lack of holistic discussions with healthcare professionals (HCPs), an overall neutral or positive experience of interactions with HCPs, the role of the family in children's self-management and the positive role of school and friends. Issues pertinent to South Asian children related to a higher likelihood of feeling embarrassed and attributing physical activity to being a trigger for asthma symptoms. CONCLUSIONS: The two ethnicity-specific factors revealed by the interviews are significant in children's self-management of asthma and therefore, indicate the need for a tailored intervention in South Asian children.

A qualitative study to identify parents' perceptions of and barriers to asthma management in children from South Asian and White British families.

BACKGROUND: Over one million children receive treatment for asthma in the UK. South Asian children experience excess morbidity and higher rates of hospitalization than the White population. This study aimed to explore perceptions and experiences of asthma and asthma management in British South Asian and White British families, to identify barriers to optimal management and to inform culturally appropriate interventions to improve management. METHODS: A qualitative methodology, using semi-structured interviews was adopted. Members of 30 families from six major South Asian ethnic-religious groups were purposively sampled (n = 49). For comparison, 17 White British parents were interviewed. Topics included understandings of asthma; day-to-day management; interactions with health care providers and the perceived quality of healthcare services. Data were analyzed using interpretive thematic analysis, facilitated by NVivo. Similarities and differences between South Asian and White families were analysed across key themes. RESULTS: Many of the problems facing families of a child with asthma were common to South Asian and White British families. Both had limited understanding of asthma causes and triggers and expressed confusion about the use of medications. Both groups reported delays in receiving a clear diagnosis and many experienced what was perceived as uncoordinated care and inconsistent advice from health professionals. No family had received an asthma plan. South Asian families had more difficulty in recognising severity of symptoms and those with limited English faced additional barriers to receiving adequate information and advice about management due to poor communication support systems. South Asian parents reported higher levels of involvement of wider family and higher levels of stigma. Attendance at the emergency department was related to previous experience, difficulties in accessing primary care, lack of knowledge of alternatives and difficulties in assessing severity. CONCLUSIONS: Barriers to optimal asthma management exist at the individual family, community and healthcare systems levels. Culturally sensitive, holistic and collaboratively designed interventions are needed. Improved communication support for families with lower proficiency in English is required. Healthcare professionals need to ensure that families receive an asthma plan and make greater efforts to check families' understandings of asthma triggers, use of medications, assessment of asthma severity and accessing help.

Can previously sedentary females use the feeling scale to regulate exercise intensity in a gym environment? an observational study.

BACKGROUND: Recent research suggests that the Feeling Scale (FS) can be used as a method of exercise intensity regulation to maintain a positive affective response during exercise. However, research to date has been carried out in laboratories and is not representative of natural exercise environments. The purpose of this study was to evaluate whether sedentary women can self-regulate their exercise intensity using the FS to experience positive affective responses in a gym environment using their own choice of exercise mode; cycling or treadmill. METHODS: Fourteen females (24.9 years ± 5.2; height 166.7 ± 5.7 cm; mass 66.3 ± 13.4 kg; BMI 24.1 ± 5.5)) completed a submaximal exercise test and each individual's ventilatory threshold ([Formula: see text]) was identified. Following this, three 20 min gym-based exercise trials, either on a bike or treadmill were performed at an intensity that was self-selected and perceived to correspond to the FS value of +3 (good). Oxygen uptake, heart rate (HR) and ratings of perceived exertion (RPE) were measured during exercise at the participants chosen intensity. RESULTS: Results indicated that on average participants worked close to their [Formula: see text] and increased their exercise intensity during the 20-min session. Participants worked physiologically harder during cycling exercise. Consistency of oxygen uptake, HR and RPE across the exercise trials was high. CONCLUSION: The data indicate that previously sedentary women can use the FS in an ecological setting to regulate their exercise intensity and that regulating intensity to feel 'good' should lead to individuals exercising at an intensity that would result in cardiovascular gains if maintained.

Acute affective responses to prescribed and self-selected exercise sessions in adolescent girls: an observational study.

BACKGROUND: Positive affective responses can lead to improved adherence to exercise. This study sought to examine the affective responses and exercise intensity of self-selected exercise in adolescent girls. METHODS: An observational study where twenty seven females (Age M = 14.6 ± 0.8 years) completed three 20-minute exercise sessions (2 self-selected and 1 prescribed intensity) and a graded exercise test. The intensity of the prescribed session was matched to the first self-selected session. Intensity, affective responses and ratings of perceived exertion were recorded throughout the sessions and differences examined. Repeated measures ANOVAs were conducted to examine differences. RESULTS: There were no significant differences in intensity between the prescribed and self-selected sessions, but affective responses were significantly more positive (p < .01) during the self-selected session. Ratings of perceived exertion were significantly lower (p < .01) during the self-selected session than the prescribed session. On average participants worked at 72% [Formula: see text] peak; well within the intensity recommended by the American College of Sports Medicine. CONCLUSION: Even though the intensity did not differ between the self-selected and prescribed sessions, there was a significant impact on affective responses, with more positive affective responses being elicited in the self-selected session. This highlights the importance of autonomy and self-paced exercise for affective responses and may have potential long-term implications for adherence.