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: The rapid development and deployment of SARSCoV- 2 vaccines elevated reliance on pharmacovigilance to inform benefit-risk assessments and national vaccine policy recommendations. During the mass vaccination program against pandemic coronavirus, the VAERS received an unprecedented number of adverse event reports. The VAERS database doubled in size in 2021 and accumulated 856,340 SARS-CoV-2 vaccine reports (54.3% of all VAERS reports) as of May 6, 2022 (HHS 2022). This publicly available resource has been heavily relied upon to inform US vaccine policy. Objective(s): To understand how VAERS has been used to study the safety of the SARS-CoV-2 vaccines. Method(s): Publications containing the terms "Vaccine Adverse Event Reporting System" or VAERS and COVID-19 or SARS-CoV-2 were identified from PubMed. Non-research articles, publications that did not use VAERS or did not study SARS-CoV-2 vaccines, and withdrawn publications were excluded. Key data fields were ed from the remaining articles and summarized through descriptive statistics. Result(s): 88 publications were identified with 27 excluded upon review;1 was withdrawn, 9 were commentaries/editorials, and the remainder did not study SARS-CoV-2 vaccines in VAERS. Approximately one-half of the 61 included publications focused on one or more Adverse Events of Special Interest (e.g., anaphylaxis, facial nerve palsy, Guillain-Barre syndrome, myocarditis/pericarditis, etc.) or death rather than all events or signal detection. Several special populations were studied including children, adolescents, and "pregnant persons." Methods ranged from constructing a case series for clinical review to modeling. Adverse event reporting rates were calculated in 36 studies (58%). Denominators were derived from state or national vaccine administration data (CDC 2022), and included estimates of doses administered, number of persons vaccinated, and person-years. In more than one-third of the publications that calculated adverse event reporting rates, these values were misreported or misinterpreted by the study authors as estimates of incidence rates or cumulative incidence (risk). Conclusion(s): The rapid nationwide SARS-CoV-2 vaccine rollout resulted in an unprecedented volume of VAERS reports, which have been relied upon to investigate rare adverse events and inform vaccination policy. The methods and scientific rigor of vaccine adverse event studies varied considerably. Despite the inability to calculate incidence or risk using voluntary adverse event reports, these terms were frequently used instead of, or interchangeably with, reporting rate. Where a causal relationship exists, relying on reporting rates as a proxy for incidence may substantially distort estimates of harms (Weiss 2022).
RESUMEN
Introduction: The rapid development and deployment of SARSCoV-2 vaccines elevated reliance on pharmacovigilance to inform benefit-risk assessments and national vaccine policy recommendations. During the mass vaccination program against pandemic coronavirus, the VAERS received an unprecedented number of adverse event reports. The VAERS database doubled in size in 2021 and accumulated 856,340 SARS-CoV-2 vaccine reports (54.3% of all VAERS reports) as of May 6, 2022 (HHS 2022). This publicly available resource has been heavily relied upon to inform US vaccine policy. Objective: To understand how VAERS has been used to study the safety of the SARS-CoV-2 vaccines. Methods: Publications containing the terms "Vaccine Adverse Event Reporting System" or VAERS and COVID-19 or SARS-CoV-2 were identified from PubMed. Non-research articles, publications that did not use VAERS or did not study SARS-CoV-2 vaccines, and withdrawn publications were excluded. Key data fields were ed from the remaining articles and summarized through descriptive statistics. Results: 88 publications were identified with 27 excluded upon review;1 was withdrawn, 9 were commentaries/editorials, and the remainder did not study SARS-CoV-2 vaccines in VAERS. Approximately one-half of the 61 included publications focused on one or more Adverse Events of Special Interest (e.g., anaphylaxis, facial nerve palsy, Guillain-Barre syndrome, myocarditis/pericarditis, etc.) or death rather than all events or signal detection. Several special populations were studied including children, adolescents, and "pregnant persons." Methods ranged from constructing a case series for clinical review to modeling. Adverse event reporting rates were calculated in 36 studies (58%). Denominators were derived from state or national vaccine administration data (CDC 2022), and included estimates of doses administered, number of persons vaccinated, and person-years. In more than one-third of the publications that calculated adverse event reporting rates, these values were misreported or misinterpreted by the study authors as estimates of incidence rates or cumulative incidence (risk). Conclusion: The rapid nationwide SARS-CoV-2 vaccine rollout resulted in an unprecedented volume of VAERS reports, which have been relied upon to investigate rare adverse events and inform vaccination policy. The methods and scientific rigor of vaccine adverse event studies varied considerably. Despite the inability to calculate incidence or risk using voluntary adverse event reports, these terms were frequently used instead of, or interchangeably with, reporting rate. Where a causal relationship exists, relying on reporting rates as a proxy for incidence may substantially distort estimates of harms (Weiss 2022).