Palliative care needs among outpatient adults with cystic fibrosis: Baseline data from the Improving Life with CF trial.

BACKGROUND: Little is known about the burden of illness experienced by people with cystic fibrosis (pwCF) since the advent of CF transmembrane conductance regulator (CFTR) modulator therapies. Studies that characterize the nature of illness burden are needed to inform the development and implementation of palliative care programs that can serve this population and address quality of life concerns. METHODS: Adults with CF treated at five U.S. CF centers were surveyed to obtain baseline data for the Improving Life with CF primary palliative care implementation trial. Consenting patients completed the Integrated Palliative Care Outcome Scale (IPOS), a multidimensional measure of unmet needs for palliative care. Sociodemographic and clinical information was also obtained. The associations among these variables were examined through bivariate and multivariable analyses. RESULTS: Among 256 adults, the most distressing symptoms included not feeling "at peace", communication difficulties with family/friends, anxiety over illness or its treatment, and a lack of energy. In the multivariable analyses, CFTR modulator use was associated with lower IPOS total and physical symptoms scores; female sex and increased hospitalizations were associated with higher scores. Increased age and history of distal intestinal obstructive syndrome were associated with higher IPOS physical symptoms scores. CONCLUSIONS: These findings illuminate the nature of illness burden for pwCF in the era of CFTR modulator therapies. Although illness burden is positively affected by modulator therapy, there is a continuing need for palliative care to address physical, emotional, and spiritual distress, and the communication and practical needs experienced by adults with CF.

Addressing Symptom Burden and Palliative Care Needs in Cystic Fibrosis: A Narrative Review of the Literature.

Among people with cystic fibrosis (CF), illness burden is multifaceted, and symptoms may fluctuate in intensity across a lifespan. Caregivers of people with CF may also experience distressing symptoms. Recent developments in CF care, including the availability of highly effective modulator therapies (HEMTs) and new palliative care guidelines promoting palliative care screening may help alleviate symptoms. The objective of this review was to present a narrative view of the recent literature on symptom burden in CF, new screening approaches informed by the Cystic Fibrosis Foundation (CFF) palliative care guidelines, and early data from studies examining the impact of HEMTs on CF symptom burden. A review of the relevant literature was conducted using Google Scholar and PubMed. Included articles covered approaches to burden assessment in CF and other chronic illnesses, epidemiology of CF symptom burden, the impact of HEMTs on symptom burden, and the CFF palliative care guidelines. A primary palliative care model implementing the CFF guidelines was also described. Results of this review show that while recent developments in CF care have led to a reduction in physical symptoms, mental health symptoms remain prevalent. Ongoing screening and triage can ensure that physical symptoms, psychological symptoms, social needs, practical problems, and communication concerns are addressed by care teams.

Center-level self-study identifies opportunities to advance equity in cystic fibrosis clinical trial participation.

Clinical trials are a necessary tool for evaluating the effectiveness of newly developed treatments and interventions for cystic fibrosis (CF). Prior work demonstrated a proportional underrepresentation of people with CF (pwCF) identifying as part of a minoritized racial or ethnic group in clinical trials. In order to establish a baseline for improvement efforts, we undertook a center-level self-study to evaluate if the racial and ethnic backgrounds of pwCF participating in clinical trials at our CF Center in New York City reflect our overall patient diversity (N = 200; 55 pwCF identifying as part of a minoritized racial or ethnic group and 145 pwCF identifying as non-Hispanic White). A smaller proportion of pwCF identifying as part of a minoritized racial or ethnic group participated in a clinical trial as compared to pwCF identifying as non-Hispanic White (21.8% vs. 35.9%, P = 0.06). A similar trend was present for pharmaceutical clinical trials (9.1% vs. 16.6%, P = 0.3). When limiting the study population to the pwCF most likely to be eligible for a CF pharmaceutical clinical trial, a larger proportion of pwCF identifying as part of a minoritized racial or ethnic group participated in a pharmaceutical clinical trial as compared to pwCF identifying as non-Hispanic White (36.4% vs. 19.6%, P = 0.2). No pwCF identifying as part of a minoritized racial or ethnic group participated in an offsite clinical trial. Efforts to improve the racial and ethnic diversity of pwCF in clinical trials, both onsite and offsite, will require a shift in how recruitment opportunities are identified and communicated to pwCF.

The clinical impact of the Covid-19 pandemic first wave on patients with cystic fibrosis in New York.

BACKGROUND: People with cystic fibrosis (pwCF) may be at risk of complications from COVID-19 but the impact of COVID-19 on pwCF remains unknown. METHODS: We conducted a multicenter retrospective cohort study to assess the impact of the COVID-19 pandemic first wave on pwCF in the New York metropolitan area (NY) from March 1, 2020 to August 31, 2020. Objectives were to determine (1) the prevalence of COVID-19 by PCR and IgG antibody testing, (2) the clinical characteristics of COVID-19, (3) delay in routine outpatient care, and (4) the effect on anxiety and depression in pwCF. RESULTS: There were 26 COVID-19 cases diagnosed by PCR or antibody testing among the study cohort of 810 pwCF. The prevalence of COVID-19 by PCR (1.6%) and IgG antibody (12.2%) testing was low. 58% of cases were asymptomatic and 82% were managed at home. 8% were hospitalized and 1 person died. 89% of pwCF experienced delay in care. The prevalence of anxiety increased from 43% baseline to 58% during the pandemic (P<0.01). In post-hoc analysis, the proportion of patients with diabetes (38% versus 16%, P<0.01) and pancreatic insufficiency (96% versus 66%, P<0.01) were higher while CFTR modulator use was lower (46% versus 65%, P = 0.05) in pwCF who tested positive for COVID-19. CONCLUSIONS: The prevalence of COVID-19 among pwCF in NY during the pandemic first wave was low and most cases were managed at home. CFTR modulators may be protective. PwCF experienced delay in routine care and increased anxiety.

Addressing the burden of illness in adults with cystic fibrosis with screening and triage: An early intervention model of palliative care.

BACKGROUND: Novel models that improve generalist-level palliative care for cystic fibrosis (CF) are needed to address the burden of this illness. A screening-and-triage model has the potential to identify clinical problems requiring immediate follow-up by CF professionals. This study describes such a model and its immediate impact on care delivery for CF patients during a two-year period. METHODS: Eligible adults completed monthly online screening for sources of distress. If results revealed one or more "indicators of concern" on two consecutive screenings, this triggered an attempted triage by a social worker. Completed triages led to prompt follow-up by CF professionals for clinical problems, if indicated. Process data were summarized and generalized linear mixed models were used to evaluate baseline patient characteristics (symptom distress, quality of life, and sociodemographics) associated with the need for prompt follow-up. RESULTS: A total of 1,015 monthly surveys were completed by 74 patients; 634 (66 patients) had >1 indicators of concern; and 164 surveys (46 patients) had >1 indicators for two consecutive surveys (e.g., global distress, pain, dyspnea, and psychological symptoms). The 164 attempted triages yielded 84 completed triages (51.2%), of which 39 (46.4%) required prompt follow-up. In multivariable analyses, older patients and those with higher symptom distress at baseline were more likely to require prompt follow-up (p < .05). CONCLUSIONS: Web-based screening that assesses varied domains of distress or burden can identify a subset of CF patients whose clinical problems may benefit from immediate medical or psychological attention. Additional investigations should improve screening efficiency.

Trajectories of caregiver burden in families of adult cystic fibrosis patients.

ABSTRACTObjectives:Little is known about the experience of family caregivers of adults with cystic fibrosis (CF). This information is important for the identification of caregivers at risk for burden. METHODS: This was a longitudinal analysis of survey data obtained from caregivers of adult CF patients participating in an early intervention palliative care trial. Caregivers completed the validated Brief Assessment Scale for Caregivers (BASC) repeatedly over a 28-month period. Mixed-effects modeling evaluated multivariate associations with positive and negative caregiver perceptions over time. RESULTS: Of the 54 caregivers, 47.9% were spouses. The mean age was 50.9 years (SD = 13.2); 72.2% were women; 75.9% were married; and 63.0% were employed. At baseline, the BASC revealed large variations in positive and negative perceptions of caregiving. Although average scores over time were unchanging, variation was greater across caregivers than within caregivers (0.49 vs. 0.27, respectively). At baseline, the positive impact of caregiving in the sample was higher than the negative impact. Multivariate analysis revealed that patients' baseline pulmonary function and their full-time employment status predicted caregiver burden over time. SIGNIFICANCE OF RESULTS: Caregivers of CF patients varied in their positive and negative caregiving experiences, although burden levels in individual caregivers were stable over time. When the disease was advanced, caregivers of CF patients experienced more overall burden but also more positive impact. This suggests that the role of caregivers may become more meaningful as disease severity worsens. In addition, full-time patient employment was associated with lower caregiver burden regardless of disease severity. This suggests that burden in CF caregivers may be predicted by financial strain or benefits conferred by patient employment. These associations require further investigation to determine whether highly burdened caregivers can be identified and assisted using tailored interventions.

Web-based symptom screening in cystic fibrosis patients: A feasibility study.

BACKGROUND: Cystic fibrosis (CF) causes high illness burden. Screening may identify patients who could potentially benefit from interventions for symptoms or other sources of distress. We evaluated the feasibility of a web-based system for routine monitoring. METHODS: Adult CF patients enrolled in a study of palliative care service delivery completed web-based assessments every 30 days on global distress, physical and psychological symptom distress, and the perceived benefits of discussing advance care planning (ACP) with CF clinicians. Feasibility was assessed by the rate of survey completion, survey reminders, and missed surveys during a 9-month rolling enrollment period. RESULTS: Of 74 participants (47.3% women, 94.4% white), 36.7% had comorbid diabetes, and 56.9% had an FEV1% predicted score of 40-69. In total, patients completed 456 (80.6%) of 566 surveys every 30 days over the assessment period. Sixty-five (87.8%) completed 405 (79.7%) of 508 surveys online and 9 (12.2%) completed 51 (88.0%) of 58 surveys using a combination of online, telephone, and mail-based methods. Many surveys were completed without reminders (online: 261 [64.4%] of 405; combined methods: 29 [56.9%] of 51), with 166 (36.4%) surveys requiring 1 or more reminders. Further, 23.0% (17) of patients noted elevated global distress; 20.3% (15) endorsed physical symptom distress, 12.2% (9) had psychological distress, and 58.1% (43) reported that discussing ACP would be beneficial. CONCLUSIONS: Repeated web-based screening for symptom distress and ACP preferences is feasible in adult CF patients. Future studies should assess the system's generalizability and staff resources when implementing reminders and non-web methods of completion.