Development and validation of a supportive programme for family caregivers of children suffering from cystic fibrosis: protocol for a sequential exploratory mixed-methods study

Introduction

Cystic fibrosis (CF) is the most common autosomal recessive, life-restricting condition, with multisystems involvement.1 Approximately 70 000–100 000 people worldwide suffer from this disease, which is most common among white people in northern Europe, and its prevalence is decreasing eastward. The same trend is observed in Asia; CF is more prevalent in West Asia compared with East Asia.2–5 Obtaining accurate statistics on the prevalence of a certain disease in Iran is challenging due to the lack of a comprehensive registration system, the limited availability of diagnostic tests, and the economic and social conditions in certain areas.6 Recent studies have shown that this disease is more common among children under the age of 18 in Iran than previously thought. Unfortunately, only a small percentage of those affected by this disease reach adulthood, which is very different from the situation in other countries, such as the UK and the USA.7 8 This highlights the urgent need to pay attention to this population and their families.9 10

Managing CF involves a complex daily care routine with several oral and inhaled medications, airway clearance techniques, dietary considerations and exercise; this requires multidimensional care and diverse knowledge.11 Despite advancements in CF care, the chronic and progressive nature of lung disease, as well as other complications such as pancreatic insufficiency and endocrine disorders, remain a reality for individuals with CF.12

Until recently, the available treatments for CF only controlled symptoms and restricted complications. However, the field has rapidly evolved with advances in CF transmembrane conductance regulator (CFTR) modulator therapies targeting the basic defect of CF.13 However, CFTR modulators approved for use thus far are extremely expensive. This has raised concerns about the affordability of new treatments and highlighted the significant disparities in health outcomes for CF patients between high-income nations and low-income and middle-income countries.14

Advances in clinical care have been multifaceted and include earlier diagnosis through the implementation of newborn screening programmes, formalised airway clearance therapy and reduced malnutrition through effective pancreatic enzyme replacement and a high-energy, high-protein diet.14–16 Now, drugs are available to address respiratory symptoms targeting airway mucus and airway surface liquid hydration. Additionally, antimicrobial therapies such as antibiotic eradication treatment for early-stage infections and protocols for maintenance therapy for chronic infections are available. Although there has been a recent breakthrough in the use of CFTR modulators for treating CF, it is still important to develop new mucolytic, anti-inflammatory and anti-infective therapies, especially for patients with more advanced stages of lung disease.14 Over the years, various treatments have been adopted to reduce the effects of the disease.17 With each additional treatment component, the time needed for medication preparation and administration, proper disinfection of equipment (such as nebulisers) and different therapeutic regimens and burden of care increased. As a result, patients and their families often have to dedicate several hours of daily care to managing the disease.18 Chronic health conditions in children and adolescents can cause parents to experience a heavy caregiving burden.19 This burden is caused by the need for home-based treatments and the impact on caregivers of patients who are hospitalised and other healthcare contacts.20 The ability of families to cope with their health conditions is largely determined by the level of caregiver burden they experience.21

According to theoretical models of caregiving, the concept of burden includes objective and subjective elements.22 Stress theory suggests that caregivers experience primary stressors, such as the level of care needed, disease severity, patient health and behavioural issues. This can lead to a subjective feeling of burden, causing caregivers to feel overwhelmed and strained.23 As a result, caregivers may be at risk of developing anxiety or depression, which can lead to poor mental and physical health outcomes.24 Additionally, caregiver depression can negatively affect patient adherence to treatment and contribute to disease progression.25

The occurrence of a chronic disease in one family member can impact the entire family and affect all family members.26 According to research, a child’s illness can alter the family’s lifestyle, and parents of such children often feel responsible for their child’s condition, leading to feelings of anxiety, guilt, helplessness and incapacity.27

As a result, family members need to reorganise their roles, interaction patterns and relationships within and outside the family to adapt to the new situation.28 When parents receive a diagnosis of CF for their child, they are confronted with a future they had not previously imagined. Facing the limitations imposed by a diseasesuch as CF can lead parents to redefine their expectations and reevaluate the importance of specific aspects of their child’s life.29 Some parents may experience grief and mourning, similar to the process of loss. These parents may grieve the loss of their ‘normal’ child or experience an ambiguous loss.26 29 In addition to grief and mourning, parents and children often experience uncertainty about the future. This uncertainty may stem from a lack of knowledge about the disease, its complexity, treatment, side effects and management; the creation of trustworthy relationships with healthcare providers; quality of life and the child’s ability to function.30 This uncertainty is linked to high levels of emotional distress, reduced quality of life and poor psychosocial adaptation because parents and children fear the worst and prepare for it.31 Neglecting a child’s psychological, cognitive and social aspects can negatively affect various dimensions, including physical, social, academic and general well-being.32 33 This can interfere with parents’ ability to perform essential tasks such as monitoring the child’s health, enforcing consistent behavioural expectations, promoting independence and self-management, and caring for and supervising other siblings.34

Families often remain helpless due to a lack of knowledge about the cause of the disease, treatment methods, the burden imposed on their child during the disease, uncertainty and ineffective coping, and they need to be supported in different fields.26 Findings of multiplestudies conducted to examine the behaviours of parents of children with chronic diseases found that these families had an unsuccessful role in providing complete and comprehensive support to their children.35 36

One of the fundamental roles of medical staff, particularly nurses, is to support patients.37 Providing adequate support enhances the quality of patient care and enriches the nursing profession.38 Support is an essential aspect of healthcare delivery that can take many forms, including informational, emotional, spiritual, social and physical support and is necessary at every stage of care.39 In nursing, supporting patients involves representing them, defending their rights, supporting their interests in decision-making and respecting their decisions.40 Currently, supporting patients has expanded to include wider dimensions. Nurses are expected to support patients and their families in various social settings, including economic, educational and research settings. They provide healthcare services, control costs and improve the quality of healthcare.41

Additionally, supporting parents is an essential aspect of providing family-centred care.42 This approach focuses on supporting, respecting and valuing the needs and values of family members.43 Family-centred care emphasises maintaining the integrity and unity of the family while providing proper care for the child and their family.44 Since support includes the components of care and care is part of empowerment, supporting and continuing to care for families with children with CF is essential.45 Therefore, emphasising family support and increasing it in nursing care is a vital and essential role in improving children’s living conditions.46

It is the responsibility of the family to care for children of different age groups, from complete to partial dependency. As treatment advances and life expectancy increases, there is a significant shift from hospital and healthcare systems to home and family care. This change highlights the importance of addressing the various needs of family caregivers in different cultural, economic, and social contexts to ensure proper patient care.47 48 Therefore, identifying these needs is critical in planning and designing a family-centred support programme to provide quality care, improve quality of life, self-efficacy and self-esteem, and reduce stress, anxiety and depression.47 49

The lack of culturally sensitive and current support programmes, along with the unavailability of home care centres and medical teams to provide assistance in Iran, prompted the researcher to conduct a study aimed at developing and validating support programmes for families with children suffering from CF in Iran.

Methods and analysis

Aim and design

This study aims to develop and validate a supportive programme for family caregivers of children with CF in Iran. An exploratory sequential mixed-methods design will be used in the study. The study is scheduled to begin in April 2024 and end in February 2025.

Study-specific aims

During the qualitative phase of the research, the specific aims of the qualitative phase will be to explain the experiences of families of children with CF in supporting them, to understand the experiences of healthcare personnel in supporting these families, and to review the available evidence regarding the supportive care needs of caregivers for children with CF. In the intermediate phase, the focus will be on developing a supportive programme for family caregivers of children with CF in Iran. Finally, during the quantitative phase, the aim will be to validate this family support programme in supporting caregivers of children with CF.

Study setting

We will conduct interviews in a natural setting for this study, following the qualitative research approach. Mardani Azar Children’s Hospital, located in northwestern Iran, as a referral centre, or wherever participants feel comfortable, will be the interview site for parents and patients with CF.

Participation

The sample will consist of parents of children of school age diagnosed with CF, as well as healthcare personnel as key participants. Participants will also be recruited through purposeful sampling, including children of school age and above who will cooperate and provide helpful information.

Inclusion criteria for caregivers

  1. A family member should be the primary caregiver for a child with CF.

  2. Willingness and ability to participate in the study.

  3. Availability of the child’s medical records in a clinic or hospital.

  4. Children under 18 years of age.

Inclusion criteria for healthcare personnel

Having at least 6 months of experience working with families of children with CF.

Exclusion criteria

Intellectual disability of the child’s caregiver (in health record).

Determination of addiction of the child’s parents (in health record).

Requirement

After receiving permission from the Ethics Committee of Tabriz University of Medical Sciences, the researcher will go to Mardani Azar Children’s Educational Hospital in East Azerbaijan, Tabriz. The researcher will receive a list of families who have a hospitalised child with CF or other families who have a child with a registered case in the hospital. To participate in the study, the researcher will contact families and healthcare personnel, explain the purpose of the study and guide them through the process. The researcher will ask individuals willing to participate to complete an informed consent form before the interview.

Study design

This exploratory sequential mixed-methods study will be divided into three stages, described below (figure 1).

Figure 1
Figure 1

Study visual diagram.

Procedures

Stage 1: qualitative study

The initial stage includes the qualitative phase. The qualitative phase consists of two substudies:

Qualitative interviews.

Scoping review.

Qualitative interviews

In this phase, the qualitative interviews will be conducted. Data will be collected via semistructured interviews using open-ended questions to gain a deeper understanding of the respondents’ experiences. The process will continue until we reach data saturation, ensuring a comprehensive understanding of the topic.

The qualitative interviews will investigate the parents’ experience of the support received while caring for a child with CF disease and healthcare personnel’s experience of support for families of children with CF.

The researcher will select participants purposefully after referring to the research field and checking the existing conditions. The participation of the participants will be voluntary. If they agree, written informed consent will be obtained, and they will enter the study as participants. To coordinate the interview session, the researcher will introduce herself, explain the research objectives and invite the participants to participate. Confidentiality of participant information for both families of children with CF and healthcare personnel will be emphasised. Contact will be made with the selected participants by agreeing to a previous time and providing interview equipment, including a digital audio recorder, paper and pen, and specifying the appropriate place to conduct the interview and the approximate time shared with the participants (ie, between 30–90 min). If participants agree, the interview will be audio recorded. The audio file will be deleted if participants withdraw at any stage.

To enhance the credibility of the data, the researcher will use an interview guide based on previous qualitative studies50 51 and discussions among the research team members. The interviews will start with a general question such as ‘What is your experience of support?’ or ‘What support did you need while caring for your child?’ After that, prompt questions including ‘Can you give an example?’ or ‘Do you have relevant experience?’ will be used. The face-to-face interview will be conducted in a calm and suitable environment. During the interview, the researcher will document non-verbal data such as the participants’ tone of voice, facial expressions and body posture. The date and place of the interview will also be recorded. Confidentiality of participant information, including families of children with CF and healthcare personnel, will be emphasised. To ensure that the researcher has accurately interpreted the participants’ statements, the transcripts will be sent to each participant along with a summary of the key themes of each interview (member check). Each interview will be transcribed verbatim. Data collection will continue until data saturation is reached, meaning that no new information or codes are added to the existing data.

Data analysis

In this stage, conventional content analysis based on the Bengtsson method will be used.52 Content analysis is a process of coding and classifying data; during this process, the classes may be changed to provide a complete and clear picture of the studied phenomenon by obtaining the patterns and themes related to the studied phenomenon.53 In the conventional type of content analysis, the codes are obtained directly from the data text and inductively.53 The main advantage of conventional content analysis is that it allows us to obtain direct information from the study without imposing predetermined concepts or theories. Due to its lack of predefined structure, it leads to the discovery of new findings.54

The audio recordings and transcripts will be reviewed thoroughly to comprehensively understand the interviews and gain deep insight into the data. Cross-checking the audio files and transcripts will address any inconsistencies or uncertainties. At this stage, the 2020 MAXQDA software will organise the interview texts and codes. For this purpose, after conducting the interviews on paper and preparing the electronic data file, the data will be coded using 2020 MAXQDA software. Coding is the method of breaking data and grouping them based on related and inferred concepts, and the codes ultimately represent what is happening in the data.53 We will take notes during the initial analysis, which will help us lay the groundwork for the emergence of codes. During this process, we extract code labels directly from the data text. After coding, the obtained related codes will be collected in their classes according to their similarities, differences, agreements and contradictions. As the interviews progress, the classes will expand and expand; in this process, sometimes new classes appear or some merge. Five criteria, credibility and confirmability, dependability, transferability55 and authenticity,56 have been proposed for evaluating the rigour of qualitative studies, which will be used in this study to increase the study’s accuracy.

Scoping review

A scoping review is conducted as part of the initial stage of the study, which involves examining quantitative and qualitative studies. A scoping review is an appropriate way to identify research gaps, make recommendations for further research, determine the range of available evidence, bundle and communicate research results, and include all levels and types of evidence.57

For this study, we will use the five-stage framework Arksey and O’Malley suggested for the search process. These stages include identifying the research question, identifying relevant studies, selecting relevant studies, transferring the data, and summarising, synthesising, and reporting the findings.58 The Preferred Reporting Items for Systematic reviews and Meta-Analyses extension for ScopingReviews (PRISMA-ScR) checklist will guide this study. This scoping review will aim to collect and evaluate all available published literature on the supportive care needs of caregivers of children with CF. The Population, Concept, Context framework, which will guide the database searches and eligibility criteria, informs the research questions. The main research question is: What are the supportive care needs of caregivers of children with CF?

As the next step in the scoping review, we will manually search for reference articles to ensure a comprehensive text search. To accomplish this, we will look for relevant articles published between 2000 and 2024 in six electronic databases: Scopus, MEDLINE (PubMed), Science Direct, Web of Science, Magiran and SID. Additionally, we will manually search the reference lists of selected articles. To access the primary sources and full-text versions of the articles, we will use Google Scholar. Once we complete the search process, we will enter all the identified studies into the EndNote software.

The articles will be screened based on their titles and abstracts. Only articles in English related to the needs and support of parents with children with CF will be selected. Articles that are duplicates, not available in full text or are not in English will be excluded from the study. Additionally, articles that focus on CF in adults or will also be excluded.

Stage 2: support programme design

In this stage, we will design and develop an initial support programme from the results of stage 1 (qualitative interviews and scoping review) and the information obtained from their analysis.

This comprehensive and targeted support programme focuses on addressing the issues related to a particular disease and its effects on a specific population. It is designed to meet the specific needs of the population for problem-solving. It employs various methods and approaches to meet their supportive needs as effectively and simply as possible. The programme will involve a significant number of centres and government and private organisations in its implementation. The programme will be designed in three stages, based on the Talbot and Verinder model59 including:

Determining the topic for planning: ((1) needs assessment, (2) initial programme design, (3) finalising the programme (expert panel)).

The research team will discuss the themes extracted from the qualitative interviews and scoping review in line with the supportive needs of caregivers of children with CF. Once the team has identified these needs, they will create an initial supportive care programme for caregivers of children with CF based on the Talbot and Verinder model, which focuses on family-centred care. For a consultation, the programme will be presented to various stakeholders, including nurses, paediatricians, nursing managers and caregivers of children with CF. Finally, The Delphi method will be used to validate the programme, and the final programme will be designed.

This study considers three stages: needs assessment, initial programme design and finalising the programme.

Stage 3: quantitative phase (programme validation through Delphi)

The programme will be based on the content analysis results and findings from the scoping review. The Delphi method, a systematic approach for engaging experts in answering questions not amenable to empirical methods, will be used to validate the programme, and the final programme will be designed. A group of experts will be carefully selected and participate in multiple survey rounds. After each round, a facilitator will summarise the experts’ opinions and feedback without disclosing their identities. This will allow all participants to compare their views. The goal is to reduce the range of responses and gain consensus on one correct answer. The Delphi method is widely used across various fields, including medicine and nursing, for identifying and prioritising complex issues. It is also used for developing evidence-based programmes.60

study design and Delphi method type

The Delphi process is a research method that will be undertaken in this study. The process comprises several stages, including identification and selection of panel members, inviting them to participate, diversifying the panel members based on expertise, educational competence, and work experience, explaining the study’s design and objectives, and obtaining informed consent from the panel members. The number of rounds, response rate and survey distribution method to the panel of experts (online, email or postal) will be determined. In selecting panel members, criteria such as clinical experience, representation of a specific professional role, latest quality publications related to the research field and other factors will be considered. The panel size (15–30 members) will be determined based on the homogeneity or heterogeneity of the participants.60

The study will identify critical issues through a domain review and qualitative interviews with parents to formulate a support programme for families of children with CF. The initial programme will be subjected to three Delphi rounds with experts and specialists. In the first round, the initial programme will be sent to the panel members, who will provide their opinions based on their experiences and accumulated knowledge. After review, any findings not identified in the qualitative interviews and scoping review will be added to the initial programme. In the second round, the revised programme will be sent back to the panel members, who will re-evaluate and provide further comments. Any unresolved issues from the first round will be addressed. The third round will aim to attain consensus on the final version of the supportive programme. Panel members will review and provide their final feedback on the programme. The goal is to achieve a high level of agreement among experts on the content and structure of the programme.

The analytical hierarchy process (AHP) will be used to rank the items based on importance and according to quantitative methods. The Delphi phase is a combination of the Delphi technique and the AHP. AHP is a method that combines the Delphi technique with a search for indicators.61 Later, many researchers used AHP combined with the Delphi method to turn them into exploratory methods for examining managerial perspectives on critical factors.62

Statistical methods

SPSS V.24.0 statistical software will be used to process the data and analyse the Delphi results. Data analysis will include calculating the mean, SD of the population mean and per cent agreement for each item.

The AHP is based on pairwise comparisons, leading to elaborating a ratio scale. The AHP uses a hierarchical model for the decision problem, consisting of an overall goal, a group of alternatives and a group of criteria that link the alternatives to the goal. Pairwise comparisons are classically carried out by asking how valuable an alternative A is to criterion c rather than another alternative B. Saaty scales can transform these judgements into numerical values. This information is processed mathematically to transform user information, objective or subjective, into mathematical one. Priorities are then determined thanks to these matrices, and a global consistency test can be performed to evaluate the coherence of the users’ judgements. The final result is a global score for each alternative regarding the single synthesis criterion of the hierarchical structure.63

The degree of coordination of experts’ opinions will indicate whether there is a large difference in the average opinions of all experts on all indicators. The degree of coordination is mainly expressed by the Kendall correlation coefficient W (Kendall W test), and the range of the degree of coordination is generally from 0 to 1. The larger the value of W, the better the agreement of experts’ opinions.64 The AHP will be used for the quantitative scale of the subjective judgement results of experts, and Super-Decision 2.6.0-RC1 statistical software will be used to calculate the weight, combination weight, and consistency coefficient of the indicators.65

Priority setting of the criteria by pairwise comparison (weighing)

For each pair of criteria, the decision-makers will respond to questions such as “How important is criterion A relative to criterion B?” Rating the relative “priority” of the criteria will done by assigning a weight between 1 (equal importance) and 9 (extreme importance) to the more important criterion. In contrast, the reciprocal of this value will assigned to the other criterion in the pair. The weights will then be normalized and averaged to obtain an average weight for each criterion.61

Pairwise comparison of options on each criterion (scoring)

For each pairing within each criterion, the decision-makers will award the better option a score on a scale between 1 (equally good) and 9 (absolutely better). In contrast, the decision-makers will be assigned the other option in the pairing by a rating equal to the reciprocal of this value. Each score will record how well option “x” meets criterion “Y”. Afterwards, the ratings will be normalized and averaged .64

In the final step, the option scores are combined with the criterion weights to produce an overall score for each option. The extent to which the possibilities satisfy the criteria is weighted according to the relative importance of the requirements. This is done by simple weighted summation. Finally, after judgements on the impact of all the elements and priorities have been computed for the hierarchy as a whole, sometimes and with care, the less important elements can be dropped from further consideration because of their relatively small impact on the overall objective. The priorities can then be recomputed with or without changing the judgements.65

Ultimately, the overall interpretation of the qualitative and quantitative stages will yield a support programme for families of children with CF.

Patient and public involvement

Our research focuses on developing and validating a supportive programme for family caregivers of children suffering from CF. The study is based on qualitative interviews and a scoping review. Caregivers, patients or healthcare personnel will not be involved in the study design or recruitment; we will interview them to better understand their expectations and experiences with perceptions of their support in caring for a child with CF and healthcare personnel’s perceptions of support for families of children with CF. We will produce a summary of the study’s results and recommendations, which will be shared with concerned stakeholders, including study participants who opt to receive it at the time of their participation.

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