RESUMEN
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
RESUMEN
Introduction: Cystic Fibrosis (CF) centers globally increased the number of telehealth clinics during the Covid-19 pandemic and developed processes utilizing telemedicine to replace a standard clinic visit. Whether these new approaches provide an opportunity to improve patients' clinical condition need further investigations. Aims and objectives: To assess the short-term clinical impact of using the NuvoAir Home platform to monitor children and adolescents with CF at home for six months as part of a virtual model of care. Method(s): The NuvoAir Home platform consists of a smartphone application, Bluetooth spirometer and clinician portal. Patients and/or parents were trained to use the platform and asked to do home spirometry monthly. Cystic Fibrosis Questionnaire-Revised (CFQ-R) was collected at the time of study entry and after six months. We calculated the percentage of change between "pre" and "post" conditions of the variables. Result(s): Sixteen children and adolescent CF patients from Federico II Hospital, Naples, Italy (9 females;mean age 16.3+/-0.9;5 homozygous for delta F508;FEV1 79.5+/-26.2 % predicted;FVC 91.6+/-23.6 % predicted;BMI 21.5+/-3.8) were recruited from June 2021. All patients had completed six months follow-up. CFQ-R revealed higher scores indicating a higher patient-reported quality of life with regard to "health perception" + 3%, "social limitations" + 11%, "digestive symptoms" + 6%. We observed a 2 % improvement from baseline in FEV1 % predicted. There was no difference in pulmonary exacerbation versus the previous year. No changes of medical treatment were reported during that time. Conclusion(s): Digital technology for home monitoring in children and adolescents with cystic fibrosis led to improved quality of life and lung function.