Effects of triple combination therapy on quality of life and psychosocial outcomes: A longitudinal study

Background: Cystic fibrosis (CF) transmembrane conductance regulator (CFTR) modulator triple combination therapy (TCT) is available to approximately 85% of the U.S. CF population. Clinical trials of TCT demonstrate numerous improvements in physical health and healthrelated quality of life (HRQoL), but fewstudies have examined the effects of TCTon mental health and psychosocial outcomes, and little is known about whether gains in HRQoL are sustained over time.We aimed to describe the HRQoL and psychosocial outcomes of people with CF (PwCF) initiating TCT and explored changes in these outcomes up to 1 year after starting TCT. Method(s): This longitudinal study enrolled PwCF aged 14 and older who were followed at a large, combined pediatric and adult CF center. Questionnaires were administered within 6 months of initiating TCT (baseline) and 3, 6, and 12 months later. Study self-report measures evaluated were HRQoL (Cystic Fibrosis Questionnaire-Revised;CFQ-R), optimism, self-efficacy, medication-related beliefs (Medication Beliefs Questionnaire;MBQ), perceived social stigma of illness, and body image. Data were also collected from medical charts on measures of health and mental health screening. Four open-ended questionswere included at each timepoint to elicit qualitative data on experiences starting TCT. Longitudinal data were analyzed using linear mixed-effects models for repeated measures. Result(s): Sixty-three adults and adolescents with CF completed the full set of surveys at baseline. Mean participant age was 30.0 +/- 14.2. Fifty-four percent identified as female, 43% as male, and 2% as nonbinary. Seventyfour percent had private insurance. Mean percentage predicted forced expiratory volume in 1 second (FEV1pp) at baseline was 76.0 +/- 24.1%, and mean body mass index (BMI) was 22.9 +/- 3.1 kg/m2. At 12 months, mean FEV1pp was 80.8 +/- 21.9%, and mean BMI was 24.5 +/- 4.1 kg/m2. On standard measures used in CF mental health screening, mean baseline Patient Health Questionnaire (PHQ-9) score was 3.4 +/- 3.5, and mean General Anxiety Disorder score was 3.4 +/- 3.7. Mean PHQ-9 (3.5 +/- 4.0) and GAD-7 (3.4 +/- 3.7) scores at 12 months were similar to baseline. We found no statistically significant differences between the survey time points in participants' physical, respiratory, or emotional functioning on the CFQ-R, but there was a significant change in social functioning ( p < 0.001). There was no statistically significant change over time in optimism or selfefficacy, but there was a significant difference in CF medication beliefs between the four survey time points ( p = 0.008 for MBQ Importance subscale), with a decrease in perceived importance from baseline to 12 months. Conclusion(s): Whereas lung function and BMI increased in our sample by 12 months, similar improvementswere not seen in standard mental health outcomes. There was no change over time in physical, respiratory, or emotional functioning, optimism, or self-efficacy. Only CFQ-R social functioning had changed by 12 months, perhaps reflecting decreased COVID-related social isolation. There was also a change in medicationrelated beliefs, with a decrease in perceived importance of taking CF medications at 12 months. Future directions include conducting qualitative analyses of open-ended questions and further examining data on social stigma, motivation to take medications, and body image, as well as examining relationships between outcome variables and baseline FEV1 and BMICopyright © 2022, European Cystic Fibrosis Society. All rights reserved

Development of a national cystic fibrosis patient and caregiver experience of care survey

Background: Since 2015, the Cystic Fibrosis Foundation (CFF) has collected and reported experience-of-care (XoC) data. Data collection was discontinued with the onset of the COVID-19 pandemic in 2020. In 2021, CFF convened a steering committee of a person with cystic fibrosis (CF), caregivers, and clinicians to develop a XoC survey to help understand and improve the XoC at CFF accredited programs. Method(s): Using prior CFF patient and family XoC surveys (2015-2020 pediatric and adult versions) [1,2] and a telehealth survey created in 2020 [3], draft pediatric and adult versions of the survey were developed. The steering committee and CFF leaders conducted three rounds of reviewand revision. After the surveys were professionally translated into Spanish, and the CFF Spanish Speakers Committee reviewed them, the surveys were programmed into Qualtrics for data collection. The data collection process was piloted with selected programs before a national launch. Result(s): Pediatric and adult surveys were developed in English and Spanish. The surveys cover in-person and telehealth (phone/video) visits and visits that are a mix of in-person and telehealth. The topics include interactions with care team members, relationship-centered care, care planning, shared decision-making, overall quality of care, race and ethnicity, gender identity, infection, prevention and control, quality of the virtual connection, and experience with remote monitoring. People with CF (PwCF) and their families are invited to complete a survey once every 6 months by text or email. PwCF and family contact data (email and mobile phone number) are stored in CFF's CFSmartReports Patient and Family Tool. After a clinic visit, contact data are electronically transferred to the Qualtrics platform to trigger a survey invitation. Responses are anonymous and reported back to programs via an electronic dashboard in near-real time. The data collection process was tested with three pediatric and three adult care programs for 3 weeks before the national launch on October 25, 2021. More than 2,000 PwCF and their families have completed a survey. Conclusion(s): The new XoC surveys offer PwCF and their families an opportunity to share feedback about their in-person and virtual care experiences. Efforts are underway to create a national report for dissemination and to engage programs with the data reported in their dashboards to celebrate what PwCF and their families appreciate about their care and to work together with them to improve gaps.Copyright © 2022, European Cystic Fibrosis Society. All rights reserved

Health-related quality of life, optimism and self-efficacy among people with CF beginning triple combination therapy

Background: With FDA approval in October 2019 of elexacaftor/ tezacaftor/ivacaftor, a CFTR modulator triple combination therapy (TCT), approximately 85% of the CF population was eligible to initiate this treatment. Clinical trial data indicates numerous improvements in physical health and health-related quality of life (HRQoL). Real-world studies of treatment initiation recommend including mental health as an outcome meaure, not reported in clinical trials. Objective: To describe the QoL and mental health outcomes of people with CF (pwCF) initiating TCT in a real-world setting. Methods: This longitudinal study enrolled pwCF 14 years and older who are followed at a large, combined pediatric and adult CF center. Data will be obtained at the following timepoints: within 6 months of initiating TCT (baseline), and then at 3, 6, and 12 months after baseline. Study self-report measures evaluate: HRQoL (CFQ-R), optimism, perceived social stigma of illness, self-efficacy, medication-related beliefs, and body image. Four open-ended questions were included to elicit qualitative data on experiences starting TCT. Data from the baseline survey are reported here. Results: Sixty-five adults and adolescents with CF completed the full set of surveys at baseline. Mean participant age was 30.2 years (SD= 14.0). Among this group, 57% identified as female, 42% as male, and 2% as nonbinary. With respect to education, 20.8% completed high school or less, 23.6% completed some college, and 45.9% completed college or above. As compared to a large 2010 US sample (Quittner, et al), participants reported higher Physical functioning (t=3.0;p<0.01), lower Emotional functioning (t=-6.7;p<0.001), and lower Social functioning (t=-2.1;p<0.05) on the CFQ-R measure of HRQoL. In terms of mental health, participants reported a mean score of 15.6 (SD= 5.7) on the LOT-R Optimism scale, falling in the Moderate optimism range. Participants had a mean score of 32.4 (SD= 4.3) on the General Self-Efficacy Scale measure, representing a t-score of 56 (73rd percentile). Open-ended questions revealed that patients' expectations regarding initiating TCT ranged from skepticism, to cautious optimism, to high expectations for life-changing results. Common hopes for TCT included reduction in treatment burden and increased quality of life, while collective fears included ineffectiveness and negative side effects. Many patients identified a change in future planning in response to starting TCT, namely increased hope and ambition. Conclusion: On average, pwCF in our sample who were starting TCT reported feeling moderate optimism and self-efficacy. They reported better physical functioning, but worse emotional and social functioning, than a 2010 sample. Whether these differences in HRQoL are due to TCT, COVID-19, or other factors requires further study. Open-ended questions elicited a mixture of positive and negative feelings related to starting TCT. Future analyses for this study include evaluation of key outcomes from the 3-month follow-up timepoint, including data on the mental health impact of COVID-19. Future directions include longitudinal analyses of the impact of TCT on HRQoL and mental health.

Multicenter assessment of telehealth experience of care in adult people with cystic fibrosis

Background: The 2019 novel coronavirus pandemic (COVID-19) necessitated a rapid shift from conducting multidisciplinary cystic fibrosis (CF) care from in-person to telehealth-based visits. Given infrequent use of telehealth in the CF chronic care model prior to this time, experience of care in this new paradigm for patients is unknown. Objective: To determine experience of telehealth in the care model of patients in adult CF centers and assess barriers to future visits. Methods: Adults across 3 CF centers were issued online surveys via email and text link within one week of the patient's first telehealth visit in fashion under IRB approval. In the survey, patients identified their CF care site and their age range. The survey focused on three themes to assess telehealth experience of care: convenience of the visit, level of concern with absence of usual in-person assessments (pulmonary function testing (PFT), weight, sputum culture, vital signs and physical exam), and the role of telehealth in their future care. Using a 5-point Likert scale of concern, we defined 1 as not concerning and 5 as very concerning. Low level of concern was defined as a score of 2 or less. Results: Between March and June 2020, 90 adults with CF were surveyed at 3 CF centers following telehealth visits. Participants' ages were consistent with usual range of adult CF patients. The majority (71%) had not participated in a telehealth visit before March 2020. During the multidisciplinary visits, MD/DOs were seen most often (91% of visits), followed by advanced practice providers (32%), registered dietitians (31%), registered nurses (13%), and social workers (12%). Nearly all respondents (93%) were satisfied with the overall treatment experience and found utilizing telehealth services convenient. Almost all (96%) felt that their issues and concerns were addressed during the visit. The concern for lack of usual in-person assessments varied: 68% indicated low concern with lack of vital signs and physical exam, 80% indicated low concern with lack of weight measurement, 52% for lack of throat/sputum culture, while only 41% indicated low concern with lack of PFTs. When asked how many future care visits would be preferred to be telehealth, 7% indicated all visits, 35% indicated most visits, 50% indicated some visits, and 8% indicated none. Nearly all respondents (97%) were interested in home lung function assessment. Conclusions: These data show that telehealth is well-received in the adult CF population, despite lacking hallmarks of routine care, such as PFTs and sputum culture. This study supports the notion that multidisciplinary telehealth care should be a key component in the chronic CF care model, even after the COVID-19 pandemic, but provisions for home assessments are needed. Additional survey and longitudinal data for ongoing patient attitude is being analyzed to gain a larger understanding of the necessary components for the role of longitudinal telehealth in the CF chronic care model.

Improving mental health screening at a large cystic fibrosis center

Background: People with cystic fibrosis (CF) are 2-3 times more likely to experience depression, anxiety, or both. Untreated depression and anxiety can affect both physical and emotional health. Left untreated, people with CF are less likely to manage their treatment plans effectively, have worse lung function, have a lower BMI, and experience more hospitalizations. At Boston Children's Hospital/Brigham and Women's Hospital combined pediatric and adult CF center, we have 598 patients total (274 in the pediatric program and 324 in the adult program). 79% of our patients are over 12 and eligible for depression and anxiety screening. Our pediatric care team consists of 15 MDs/NPs and 2 social workers. Our adult care team consists of 7 MDs/NPs and 1 social worker. There is 1 psychologist that sees both pediatric and adult patients. Objectives: (1) To streamline mental health screening process, (2) improve mental health screening rates for our CF pediatric and adult populations, and (3) identify patients who need further mental health services. Methods: To ensure we address mental health, we developed a new process for distributing the PHQ-9 assessment tool for depression and GAD-7 assessment tool for anxiety. We implemented a screening note template into our electronic medical record for better documentation of completed screens and began including mental health screens as a part of the weekly pre-visit planning amongst clinicians, nurses, and social workers. We refined the process to allow for our clinic administrators to hand out the assessments to patients at check-in and nursing assisted in collecting the screens and responding appropriately depending on the score. Results: Our average mental health screening rate in 2018 was 46% for the pediatric program and 71% for the adult program. After implementing our new process in 2019, our screening rates increased to 71% for the pediatric program and 83% for the adult program. With increased rates of screening, we did not observe a significant difference in the percentage of patients screening at-risk. A majority of patients scored in the minimal-mild range on both the PHQ-9 and GAD-7. On the PHQ-9, 8% scored in the moderate range (vs 7% in 2018) and 2% scored in the severe range (vs 2% in 2018). On the GAD-7, 10% scored in the moderate range (vs 9% in 2018) and 3% scored in the severe range (vs 6% in 2018). On question #9, 98% indicated “not at all” (vs 96% in 2018). Conclusion: Our mental health screening process has become an embedded part of our CF clinic flow. Our process has led to sustained increases in mental health screening rates. Future work will focus on follow-up on mental health treatment referrals. In addition, with the rapid shift to telehealth due to COVID, we are adapting our process to account for telehealth visits.