Study protocol of a prospective, interventional non-randomised trial investigating the impact of asthma education on specific disease understanding, health literacy and therapy outcome in childhood

Introduction

Worldwide, approximately 262 million person suffer from asthma, a condition that stands as the most common chronic disease in childhood globally.1 In Germany, the lifetime prevalence of asthma is reported to be around 8.8% in the adult population and around 6.0% among children.2 3 The treatment of asthma includes inhalations, oral therapeutics and biologicals, as well as patient education to increase disease awareness, to improve the quality of life of affected children and to enable patients to live as symptom-free as possible.4 Factors such as specific knowledge about the disease and health literacy seem to play relevant roles in the course of the disease.5 6 Furthermore, patients with a lower socioeconomic status (SES) seem to suffer more frequently from asthma.7 On the other hand, structured patient education programmes are associated with better asthma outcomes and reduced emergency visits and hospitalisation in children with asthma significantly.8 Therefore, the national healthcare guideline (Nationale Versorgungsleitlinie Asthma) in Germany strongly recommends the participation of paediatric patients in asthma education programmes.9 In addition, children’s regular participation in asthma education training has been associated with less emergency visits due to uncontrolled symptoms.8 However, the relationship between health literacy, specific disease understanding, SES and outcomes of patient education programmes requires further investigation.10 Furthermore, it is important to analyse how health literacy and specific disease understanding can be strengthened through patient education and how disease-related disadvantages due to lower SES can be appropriately managed.

To address these questions, an interventional clinical trial was designed to identify strategies to support affected children with specific risk profiles and to increase the awareness and availability of patient education programmes throughout Europe. The trial focuses on two main aspects, which will be prospectively investigated in two subprojects. The first subproject aims to examine the influence of asthma education on health literacy and disease understanding while the second subproject will analyse the role of SES on healthcare outcomes among children participating in asthma education programmes, defined as relevant improvements in symptoms and the risk for suffering exacerbations. Through this study trial, we aim to achieve valuable insights into the specific influence and benefits of asthma education programmes for young children with asthma and their parents. The specific study objectives are detailed in box 1.

Box 1

Primary and secondary objectives of the trial

Objectives

Primary objective: to investigate

  1. Whether a standardised asthma education leads to better specific disease understanding in children with asthma compared with routine patient education as part of the initial visits in outpatient clinics.

Secondary objectives: to investigate

  1. The impact of asthma education on health literacy among children with asthma.

  2. The correlation between improved specific disease understanding and enhanced health literacy in children with asthma.

  3. Whether improvements in health literacy and specific disease understanding lead to (a) a milder symptom course as measured by the ‘Children Asthma Control Test in subjects with asthma, (b) reduced number of emergency department visits and (c) reduced number of physician visits.

  4. Retention rates of disease-specific information in children with low socioeconomic status (SES) following asthma education and informational discussions.

  5. Whether or not children with low SES benefit from asthma education, measured as less exacerbations compared with children without asthma education.

  6. Whether or not children with low SES experience more frequent exacerbations compared with children with high SES.

Methods

Study design and setting

The trial is designed as a comparative, interventional, non-randomised study of an intervention group and a comparison group investigating the relationship between asthma training and educational parameters while considering various individual factors that may influence patients and their families.

The intervention group will be recruited from practices and training centres from the network of the Arbeitsgruppe Asthmaschulung im Kindes- und Jugendalter e.V. (AGAS), mostly based in North Rhine-Westphalia, Germany, and the state of Berlin, Germany. In addition, the control group will be recruited from the paediatric pulmonology departments of the University Children’s Hospital of Witten/Herdecke University, Germany (Klinikum Dortmund and Vestische Children’s Hospital Datteln) or potentially from private practices.

After the diagnosis of asthma, the intervention group undergoes standardised medical, physical and psychoeducational asthma training according to the standards of the AGAS. The training is provided at AGAS-certified training centres associated with paediatricians in private practice, all of which are part of the AGAS network, which is a national certified education working group. These standards are defined by the Quality Management Manual of the AGAS in its fifth edition, which provides a detailed description of the prerequisites, structure and content of the qualified patient training courses.11

The AGAS considers asthma training as part of a long-term treatment concept, following a disease management programme (DMP). The DMP aims to optimise standardised care process for chronically ill patients and includes three central components: (a) evidence-based treatment guidelines, (b) structured and evaluated patient education and (c) regular training and education for the professional and care providers.11 In this context, the AGAS defines schedules for asthma trainings for different age groups, including 5–12 (children), as well as 12–18 year of age (adolescents), with predefined topics, contents, goals, methods and materials. Furthermore, these trainings must be performed by qualified asthma trainers.11

In contrast, the comparison group undergoes a significantly shorter period of education or instruction with only limited time focused on the use of inhalation medication as part of the standard care after diagnosis at one of the outpatient clinics associated with hospitals, respectively, ‘Klinikum Dortmund’ or ‘Vestische Kinder- und Jugendklinik Datteln’. Both hospitals are academic university children’s hospitals of Witten/Herdecke University, Germany. Notably, these two hospitals do not offer AGAS-certified asthma education trainings. Paediatric pulmonologists, who have years of experience in managing patients with asthma, provide care as usual for the control group. While the treatment of children with asthma should be standardised and oriented on national guidelines, there is a probability that different treatment approaches for children with asthma could exist, varying between clinics and practices. This underscores the importance of the comparison group in highlighting the significance of asthma education sessions. Differences may exist in the form of therapy and the frequency of visits. Children in the intervention group will receive standardised information about handling asthma, using medication, behaviour during physical activity, recognising emergency situations, preventive measures and dealing with health information in general are among others central elements of AGAS-certified asthma education sessions. These aspects should be discussed during the initial consultation and diagnosis, and appropriate informational material should be provided.

As with any type of study, biases can occur, affecting comparability within and between cohorts. The careful selection of participating centres and outpatient clinics based on quality criteria such as clinical experience, patient numbers and facilities is crucial to ensure adequate comparability and minimise biases. Standardised implementation in this context is challenging and represents an important limiting factor that should be discussed in future publications.

Due to fact that asthma education programmes at present are only provided in centres associated with paediatricians in private practice and not in outpatient clinics associated with hospitals, randomisation will not be feasible in this study. The intervention and control groups will be recruited from inpatient and outpatient clinics associated with hospitals and the ambulant sectors with private practices within the German healthcare system. To undergo asthma education, patients must be enrolled in DMP. This enrolment can only be facilitated by physicians in private practice, who then either provide their patients with their own education programme or refer them to specialised training centres. Although capacity of these standardised asthma education programmes is limited due to available resources, the aim is to include as many children as possible in these programmes. Patients receiving care in hospital outpatient clinics undergo diagnostics, therapy and longitudinal monitoring with regular follow-ups; however, they are not enrolled in DMP and thus cannot participate in standardised asthma education programmes.

Participants

The study includes patients between the ages of ≥7 and <14 years, who have received an asthma diagnosis from a paediatrician and/or paediatric pulmonologist, following national and international recommendations, and who have not previously undergone an asthma education/training. Exclusion criteria are insufficient language and literacy skills of the parents and children to fill out questionnaires and to provide informed consent for study participation.

The survey includes three measurement points: (1) before education, (2) right aftereducation and (3) 6 months after education. Both subproject 1 (~health literacy) and subproject 2 (~SES) will collect data through a questionnaire-based survey in both groups (intervention group and control group).

Outcomes (primary and secondary)

The primary outcome for both subprojects is the specific disease understanding in children with asthma. Secondary outcomes are health literacy of children with asthma, children’s asthma control, number of emergency doctor visits and visits to the emergency room due to acute asthma symptoms in children. Subproject 1 will investigate the effect of different types of education on these outcomes. Subproject 2 will investigate the impact of socioeconomic factors, in particular SES, on these outcomes.

Detailed data on parameters, tools and surveys used in the asthma trial are summarised in figure 1.

Figure 1
Figure 1

Time flow of recorded data, as well as investigated parameters and tools used for this reason. AGAS, Arbeitsgruppe Asthmaschulung im Kindes- und Jugendalter e.V.

Instruments

For capturing specific disease understanding, a modified version of the Quality Management Questionnaire for Children and Adolescents by AGAS will be used, which includes specific items on disease knowledge via 19 items.11 Based on training materials, a blueprint for the items was created and checked for a balanced distribution across the training content and adjusted if necessary. To reduce the chance of guessing, the items were changed from a true-false answer to a single-choice format with different answer options.

To assess health literacy, a modified version of the European Health Literacy Survey Questionnaire (HLS-EU-Q) will be used.12 This tool was initially developed and validated as part of a study in 2020. Here, the HLS-EU-Q elements comprise a total of 26 items to capture health literacy based on three health domains (healthcare, disease prevention and health promotion) and the four stages of information processing (access, understanding, appraisal and application), using a Likert scale.

Children’s asthma control is assessed using the Children Asthma Control Test (C-ACT).13 The C-ACT is a validated instrument consisting of seven items (four items for children and three items for parents). The resulting score ranges from 0 to 27 points, where a high score indicating better asthma control.

Furthermore, the number of emergency doctor visits and visits to the emergency room due to acute asthma symptoms in children over the past 6 months (between assessment periods 2 and 3) will be recorded using a simple frequency query.

To measure the SES, a modified questionnaire from the KiGGS study ‘Messung des sozioökonomischen Status und des subjektiven sozialen Status in KiGGS Welle 2’ (Engl. Measurement of Socioeconomic Status and Subjective Social Status in KiGGS Wave 2) is used.14 Questions considering subjective perception, as well as two additional questions, were omitted as they were irrelevant for calculation, which makes the content more concise. The KiGGS questionnaire includes aspects of SES in three areas: ‘education’, ‘profession’ and ‘income’, where the modified form includes both the father’s and mother’s status. These terms can also apply to individuals who have taken the place of biological parents and live with the child in the same household. Based on the individually collected point value, families are categorised into low, medium or high SES. To control potential biases, age, gender, migration background and residential region will be also collected via different questionnaires.14

Investigating the impact of asthma education on the development of health literacy, specific disease understanding and disease progression

For subproject 1, we hypothesise that asthma education will strengthen specific disease understanding in these children, as suggested by the results of the AGAS questionnaire at survey points 2 and 3 relative to survey point 1. Moreover, we anticipate that children diagnosed with asthma attending asthma education will also demonstrate significant improvements in health literacy because understanding general health information will also be addressed during the asthma educational, though not in the same depth and to the same extent as the understanding of asthma. This expectation is based on measurements from the German version of the European Health Literacy Survey Questionnaire for 9 years and 10 years, comparing results from survey point 3 to those from survey point 1. Furthermore, we hypothesise a direct correlation between enhanced health literacy and a deeper understanding of the specific disease in asthmatic children. Such improvements will result in milder symptoms, as evaluated by C-ACT. In addition, we expect these advancements in health literacy and disease comprehension to reduce the number of emergency outpatient visits and unplanned consultations due to acute asthma exacerbations, as measured by comparing the numbers of such visit in the intervention group and in the comparison group at survey point 3.

Subproject 2 aims to address the role of SES, assuming SES influences the retention of information and understanding of asthma following asthma educational sessions or informative discussions. In addition, the subproject explores the relationship between SES and asthma exacerbations in these children. We hypothesise that children with lower socioeconomic backgrounds may exhibit less retention of information after asthma education and informational sessions compared with children with high SES, potentially affecting the progression of their asthma.

Consequently, we expect that children from disadvantaged socioeconomic backgrounds who undergo asthma education may show a reduction in asthma exacerbations compared with their peers without asthma education. A secondary objective is to investigate whether children from lower socioeconomic backgrounds experience more frequent exacerbations than those from higher socioeconomic environments. Therefore, as demonstrated in figure 2, we hypothesise that participation in asthma education will lead to improved asthma management, improved asthma control and decreased hospitalisations through enhanced health literacy and specific disease understanding.

Figure 2
Figure 2

Hypothesis on the influence of asthma training on the parameters under study. Based on current studies, asthma training may lead to enhanced asthma control and a reduction in emergency hospitalisations and physician visits. Moreover, a strong health literacy and comprehensive understanding of the disease appear to be associated with improved asthma control and fewer emergency hospitalisations and physician visits. Thus, we aim to explore the direct impact of asthma education on health literacy and specific disease understanding, and subsequently, its effects on patient outcomes. In addition, project 2 will investigate the influence of the socioeconomic status on the parameters, for example, asthma control, number of emergency visits and their potential correlations.

Hypothesised inter-relations of primary and secondary outcomes are illustrated in figure 2.

Patient and public involvement

The survey was piloted as part of an asthma training course with children and parents. It was important for the study team to receive feedback about the lenght of time, comprehensibility and structure of the survey instrument as well as the timing of filling in the survey. As a result, it was decided to use waiting times before the education programme and doctor–patient–parent consultations so that participants were not disadvantaged in terms of time. Parents’ idea of offering the study description in other languages and in the form of a ready-made video will be a major advantage in attracting more participants and allowing a better understanding of study aims and procedures. The children were satisfied with the content and scope of the questions as they reflected the content of the training well. Parents and patients were not involved in the study design. The results of the study will be disseminated to the participants and public via the publication of open-access research papers and relevant dissemination channels, including local and social media and website posts.

Data analysis and sample size estimation

To describe mean differences between the intervention and control group over time (subproject 1), a repeated-measures analysis of variance (ANOVA) will be conducted. Accordingly, the sample size calculation is based on a mixed ANOVA model with two groups and two measurements. With an effect size of η²p=0.06 (corresponding to an f of about 0.253) and a power of 0.8, a total of 126 subjects are needed to test the effect of the between-subjects factor (group) at a significance level of α=0.05.15 Estimation of effect size was based on the results of a meta-analysis of interventions for children who were at the risk of asthma-related emergency attendance, even if the reported statistical heterogeneity was quite high.8

In subproject 2, multivariate linear regression analysis will be used to analyse the variables determining the changes in health literacy and specific disease understanding. With a coefficient of determination of R²=0.13, a statistical power of 0.8 and a significance level of α=0.05, a sample size of n=92 would be required for a significant overall model with 5 predictors.16

The IBM-SPSS V.24.0 statistical package will be used for all analyses.

The lost-to-follow-up is estimated to be 20% over the 6-month study period. We will, therefore, recruit a total of 160 patients (ie, n=80 per group) at baseline.

Ethics, informed consent, data protection

The study including experimental protocols and instruments was approved by the ethics committee of the Witten/Herdecke University, Germany, (S-159, 2023; application submission: 24 June 2023, final vote: 10 July 2023). Furthermore, the study was registered at the German Clinical Trials Register (Deutsches Register für Klinische Studien, DRKS) and received the following project number: DRKS00032423. The application submission was on 3 August 2023 and the final approval was on 4 August 2023.

To request informed consent, the attending asthma trainer or paediatrician will explain the purpose, objective and possible risks of the study to the subjects and their parents. The participation can be withdrawn at any time without any reason. The parents will receive an informed consent form with information on the study details and aims of the trial, and the data protection policy. Consent is given in writing and documented by a standardised consent form with the signature of the legal guardians and/or the patient (from the age of 11), as well as the physician. Patients will be included when inclusion criteria are met, and the patient (11 years of age or older) and/or legal guardians have provided written consent. Patients can request study results at the end of the study.

Due to the methodology of the study, where information collected during the survey will be managed pseudonymously, we anticipate no risks to the privacy of the participants and parents when asking for migration background. Furthermore, both participants and their legal guardians will be informed that their involvement in the study and their responses to questions are entirely voluntary. This approach is designed to ensure that all participants feel their potential sensitivities regarding this subject are thoroughly respected and safeguarded and no feeling of discrimination or disadvantage should arise.

Any data collected during the clinical trial are governed by the German Federal Data Protection Act (=Bundesdatenschutzgesetz). Only the study head/deputy head can associate the initials of the subjects with their personal data. The trial documents will be managed in accordance with data protection regulations and Good Clinical Practice. Data collected during this clinical trial will be recorded on paper forms or electronic platforms, handled with strict confidentiality and without naming and saved with a password. The data will be analysed pseudonymously and will be published anonymously. Study-related documents will be stored for 10 years and stored at the Faculty of Health of Witten/Herdecke University, Germany. An informed consent will be obtained from all subjects and/or their legal guardian(s). The data will be analysed on completion of the trial, and a manuscript including the results and observations of the study will be published in an international, peer-reviewed journal.

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