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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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PURPOSE/HYPOTHESIS: Physical therapy practice depends on valid and reliable patient reported outcomes measures (PROMs). Post exertional malaise (PEM) and post exertional symptom exacerbation (PESE) are features of myalgic encephalomyelitis, Long Covid, and related conditions. They comprise a constellation of symptoms and signs including profound physical fatigue. Existing fatigue-specific instruments often are inappropriate to measure functional effects of accompanying symptoms and signs and demonstrate substantial floor effects. The purpose of this study was to determine the psychometric properties of a novel PROM for people living with PEM/PESE, the PEM/PESE Activity Questionnaire (PAQ). NUMBER OF SUBJECTS: 981 people living with PEM/PESE who completed a web-based questionnaire. MATERIALS AND METHODS: Respondents chose a function from a dropdown menu, consisting of the International Classification of Function core set for myalgic encephalomyelitis, and rated it on 2 different 0 to 10 scales. Each scale was anchored at 0 being Completely unable to perform, and 10 being Can perform at the same level as a time I have good energy and Can perform at the same level as before I became ill, respectively. Respondents also provided an estimate of effort intensiveness on a 0 to 10 scale, anchored at 0 being the activity took No time, effort, and resources at all and 10 being All of my time, effort, and resources. Respondents took the PAQ twice;they completed a demographic questionnaire after the first PAQ and before the second PAQ. Backward navigation was disabled so respondents could not view the first PAQ while completing the second PAQ. Descriptive statistics were calculated for the participants who completed versus did not complete the entire survey. Subgroup analyses by completion status and function were undertaken by chisquare analysis for binomial variables and one-way analysis of variance for continuous variables. Intraclass correlation coefficients were calculated for each scale to assess testretest reliability. Floor effects were the proportion of participants reporting the lowest score. RESULT(S): 981 surveys were available for analysis, including 675 complete surveys. All participants reported PEM/PESE. Respondents reported less frequent flu-like symptoms and pain, shorter recovery times after physical activity (i.e., less than 24 hours), and lower thresholds for physical and mental fatiguability than respondents who did not complete the survey (P< 001). Activities most frequently chosen for rating were Carry out my normal routine, Prepare meals, and Maintain employment for financial reward. Test-retest reliability was generally fair to excellent, depending on function and scale. Floor effects were noted in 9.2% to 24.7% of responses, also with notable variation based on function and scale. CONCLUSION(S): The PAQ provides valid, reliable, and sensitive outcome measure for people living with PEM/PESE. Survey completion may be limited by severity of PEM/PESE. CLINICAL RELEVANCE: Physical therapists could use the PAQ to assess illness severity and intervention efficacy for people living with PEM/PESE.
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Introduction: Adverse consequences of critical illness are well reported, and include physical, psychological and social effects that can last for years post discharge (Needham et al. 2012). Access to physiotherapy and early rehabilitation have helped to address the physical deterioration experienced by patients in intensive care. The role of other multi-disciplinary team members such as occupational therapy (OT), speech and language therapy (SLT) and psychology is less well defined, but has the potential to support and enhance rehabilitation since they offer alternative, complimentary perspectives. Objectives: This service evaluation sought to investigate the impact of a multi-disciplinary rehabilitation team on intensive care length of stay, ventilator days and functional ability at the point of discharge. Methods: The project was registered with the local NHS Trust clinical governance team. Retrospective data were collected at two points, prior to and post implementation of the multi-disciplinary rehabilitation team. Inclusion criteria were adults intubated and ventilated for more than four days requiring active treatment. Those with a long-term tracheostomy or laryngectomy or needing an inter hospital transfer were excluded. Electronic notes were retrospectively extracted to gather demographics, rehabilitation needs, intensive care length of stay, ventilator days and Chelsea Critical Care Physical Assessment (CPAx) scores at admission and discharge. Results: In total, 589 patients were admitted to intensive care across the two time points. OF these 49 were eligible for inclusion in the pre-implementation group (T1) and 45 in the post-implementation group (T2). There were no statistically significant differences in patient characteristics between patient groups. Patients presented with a range of medical, neurological and respiratory pathologies, with a minority admitted for elective or emergency surgery. Patients in T2 only received physiotherapy, compared to those in T2 who were assessed and treated by OT, SLT and care managed by the rehabilitation coordinator. Joint sessions were completed where appropriate. There was no statistically significant difference in intensive care length of stay or ventilator days between the two groups. Median (interquartile range) for length of stay and ventilator days were 13 (8-22.5) vs. 14 (10-30) and 9 (5.5-11.5) vs. 10 (6-28.5) days for pre-implementation and post-implementation groups respectively (p=0.163 and p=0.202). There was a statistically significant difference in functional ability (measured by CPAx) at discharge (p=0.037). Patients in the post-implementation group had a median change in score from baseline of 16, compared with 13 in the pre-implementation group (p=0.037). Multiple regression suggested that patient age, frailty, pathology and rehabilitation group made a significant contribution to variance in CPAx score, explaining 18% variance (p=0.033) Conclusion: The multi-disciplinary rehabilitation team did not significantly impact intensive care length of stay of ventilator days. However, patients receiving multidisciplinary rehabilitation benefited from the improved physical function at discharge. This suggests that the effects of rehabilitation can be amplified when a wider team is involved. This work was undertaken during the Covid-19 pandemic, where discharge and timely patient flow was severely disrupted. Further research is required to explore further the impact of this innovative approach to the rehabilitation in intensive care.
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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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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.