Exploring perceptions of and decision-making about CFTR modulators.

INTRODUCTION: Cystic fibrosis (CF) treatment has increasingly focused on highly effective modulators. Despite measurable benefits of modulators, there is little guidance for CF care team members on providing education and support to patients regarding initiation of these therapies. We aimed to explore patient, caregiver, and clinician perceptions of modulators and influences on decisions about starting cystic fibrosis transmembrane regulator (CFTR) modulators. METHODS: We conducted semistructured interviews with CF clinicians, adults with CF, and caregivers of children with CF. We reviewed audio recordings and coded responses to identify central themes. RESULTS: We interviewed 8 CF clinicians, 9 adults with CF, and 11 caregivers of children with CF. Themes centered on emotional responses to modulator availability, influences on decision-making, concerns about side effects, impact of modulators on planning for the future, the benefits of the multidisciplinary CF care team in supporting treatment decisions, and the unique needs of people with CF who are not eligible for modulators. Clinicians described changes in conversations about modulators since the approval of elexacaftor/tezacaftor/ivacaftor, specifically greater willingness to prescribe with less nuanced conversations with patients and/or caregivers regarding their use. CONCLUSION: Based on perspectives and experiences of CF clinicians, adults with CF, and caregivers of children with CF, we suggest clinicians approach conversations about CFTR modulators thoughtfully and thoroughly, utilizing the multidisciplinary model of CF care in exploring patient and caregiver emotions while filling in knowledge gaps, asking about treatment goals beyond potential clinical benefit, and having compassionate conversations with those who are ineligible for modulators.

Promoting emotional wellness in children with cystic fibrosis, Part I: Child and family resilience.

Attention should be given to individual and family well-being from a child's first interaction with the medical team and continuing throughout development, especially for families who experience chronic illnesses, such as cystic fibrosis (CF). While much attention has been given to the mental health of people with CF 12 years and older, this paper explores various areas for CF teams to assess and provide additional resources during the first 12 years of a child's life to promote child and family wellness. In this paper, we discuss parental mental health, social determinants of health, adherence/self-care, nutrition, attention to family lifestyle factors, engagement with school and peers, and modulator therapy for this age group of people with CF. This is the first of two companion papers which examines emotional wellness of children during the early years. The second paper examines mental health assessment and intervention for children under 12. Both encourage teams to strive to promote optimal child and family emotional health and wellness, emphasizing holistic health promotion and prevention, early identification, and intervention.

Promoting emotional wellness in children with CF, part II: Mental health assessment and intervention.

This is the second of two companion papers that examine the emotional wellness of children with cystic fibrosis (CF) during the early years of life, defined here as the period between birth and age 12. Both papers promote optimal mental health and well-being, with an emphasis on early identification and intervention. The first paper explores child and family resilience. Here, we discuss strategies for pediatric CF teams to provide routine, systematic mental health assessment, anticipatory guidance, brief intervention, and triage to evidence-based treatment when needed, while addressing barriers to accessing care. Many mental health conditions emerge before the age of 12, with the potential for lifelong effects on individuals, their families, and society. Living with a chronic illness such as CF can further increase the risk of mental health concerns and, in a bidirectional manner, their consequences for the quality of life, sustaining daily care, and health outcomes. There has been a significant focus in recent years on the mental health and wellness of adolescents and adults with CF, but less attention to specifics of depression and anxiety in younger children, or to other common pediatric comorbidities including trauma, developmental disorders such as attention-deficit/hyperactivity disorder or autism spectrum disorder, and oppositional behavior. Given the availability of psychometrically sound screening instruments and effective interventions, routinely addressing the mental health of children with CF and their families is feasible to integrate within multidisciplinary CF care, allowing for a personalized approach respecting individual needs, values, and goals.

Exploring knowledge and perceptions of palliative care to inform integration of palliative care education into cystic fibrosis care.

BACKGROUND: Individuals with cystic fibrosis (CF) face the challenges of managing a chronic, progressive disease. While palliative care is a standard of care in serious illnesses, there are no guidelines for its incorporation into CF care. Patients with CF, caregivers, and CF care providers may lack knowledge about palliative care and perceive barriers to integrated care. OBJECTIVES: To: 1) explore knowledge and perceptions of palliative care among patients with CF, caregivers, and CF care providers; 2) solicit opinions about incorporating palliative care into routine CF care; and 3) solicit recommendations for CF-specific palliative care education for patients and caregivers. METHODS: We conducted semi-structured interviews with adult patients with CF, parents of adolescents with CF, and CF care providers to assess knowledge and perceptions of palliative care. Discussion included suggestions for palliative care education and integration into CF care. The sample was characterized using summary statistics. Key themes were identified using qualitative content analysis. RESULTS: Ten patients with CF, ten parents, and eight CF care providers participated. Many had minimal knowledge of palliative care and endorsed the association with end of life as a barrier to palliative care, but after learning more about palliative care, thought it could be helpful, and should be introduced earlier. CONCLUSIONS: In this single center study, many patients with CF, caregivers, and providers lacked knowledge about palliative care. These findings warrant replication in a larger, multisite study to inform palliative care educational interventions as a step toward consistent integration of palliative care into routine CF care.

Difficult conversations: Discussing prognosis with children with cystic fibrosis.

Background Despite the chronic, progressive, and life-threatening nature of cystic fibrosis (CF), there are no guidelines for when and how to communicate prognosis to children with CF. METHODS: Semi-structured interviews with young adults with CF, parents of young adults with CF, and multidisciplinary CF health care providers assessed recall of and practices for communicating about prognosis. Recommendations for improvements were also solicited. RESULTS: Young adults with CF recalled learning that life expectancy is limited by CF between the ages of 8 and 16 years, and that CF is a progressive disease between the ages of 7 and 19 years. They reported that the information often came from CF physicians or from online resources. Patients and parents reported earlier knowledge of prognosis than providers assumed. While learning about prognosis caused sadness and stress for some patients and families, others denied negative feelings. Interestingly, most patients reported that disclosure of prognosis had minimal impact on their adherence and treatment goals. Patients and parents reported wanting physicians to be involved in conversations about prognosis. However, providers noted several barriers to discussing prognosis, including their own reluctance, time limitations, and uncertainty about appropriate timing and content of communication. CONCLUSIONS: Communication about prognosis is important but also difficult for providers, patients, and families. Appropriately timed conversations, using tools to facilitate communication, could ensure patients receive timely, accurate information.