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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
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The Coronavirus Disease (Covid-19) pandemic is rapidly spreading across the world, representing an unparalleled challenge for health care systems. There are differences in the estimated fatality rates, which cannot be explained easily. In Italy, the estimated case fatality rate was 12.7% in mid-April, while Germany remained at 1.8%. Moreover, it is to be noted that different areas of Italy have very different lethality rates. Due to the complexity of Covid-19 patient management, it is of paramount importance to develop a well-defined clinical workflow in order to avoid the inconsistent management of patients. The Integrated Care Pathway (ICP) represents a multidisciplinary outline of anticipated care to support patient management in the Sant'Andrea Hospital, Rome. The main objective of this pilot study was to develop a new ICP evaluated by care indicators, in order to improve the COVID-19 patient management. The suggested ICP was developed by a multi-professional team composed of different specialists and administrators already involved in clinical and management processes. After a review of current internal practices and published evidences, we identified (1) the activities performed during care delivery, (2) the responsibilities for these activities, (3) hospital structural adaptation needs and potential improvements, and (4) ICP indicators. The process map formed the basis of the final ICP document;160 COVID-19 inpatients were considered, and the effect of the ICP implementation was evaluated over time during the exponential phase of the COVID-19 pandemic. In conclusion, a rapid adoption of ICP and regular audits of quality indicators for the management of COVID-19 patients might be important tools to improve the quality of care and outcomes.
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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.
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OBJECTIVE: SARS-CoV-2 (Severe Acute Respiratory Syndrome Coronavirus 2)-related pneumonia, referred to as COVID-19 (Coronavirus Disease 19), is a public health emergency as it carries high morbidity, mortality, and has no approved specific pharmacological treatments. In this case series, we aimed to report preliminary data obtained with anti-complement C5 therapy with eculizumab in COVID-19 patients admitted to intensive care unit (ICU) of ASL Napoli 2 Nord. PATIENTS AND METHODS: This is a case series of patients with a confirmed diagnosis of SARS-CoV2 infection and severe pneumonia or ARDS who were treated with up to 4 infusions of eculizumab as an off-label agent. Patients were also treated with anticoagulant therapy with Enoxaparin 4000 IU/day via subcutaneous injection, antiviral therapy with Lopinavir 800 mg/day + Ritonavir 200 mg/day, hydroxychloroquine 400 mg/day, ceftriaxone 2 g/day IV, vitamine C 6 g/day for 4 days, and were on Non-Invasive Ventilation (NIV). RESULTS: We treated four COVID-19 patients admitted to the intensive care unit because of severe pneumonia or ARDS. All patients successfully recovered after treatment with eculizumab. Eculizumab induced a drop in inflammatory markers. Mean C Reactive Protein levels dropped from 14.6 mg/dl to 3.5 mg/dl and the mean duration of the disease was 12.8 days. CONCLUSIONS: Eculizumab has the potential to be a key player in treatment of severe cases of COVID-19. Our results support eculizumab use as an off-label treatment of COVID-19, pending confirmation from the ongoing SOLID-C19 trial.