Longitudinal Evaluation of Physical Fitness in Adults with Cystic Fibrosis in the Era of CFTR-Modulating-Therapies.

Purpose: Maintaining physical fitness plays an important role in the management of people with cystic fibrosis (pwCF). Longitudinal data on the course of physical fitness and the potential impact of the introduction of highly effective CFTR modulator therapy with elexacaftor/tezacaftor/ivacaftor (ETI) in adult pwCF are scarce. Methods: Health-related and skill-related components of physical fitness were assessed using an incremental cycle test (Wpeak), plus forward bend (FB), prone bent knee hip extension (HE), plank leg raise (PLR), standing long jump (SLJ), and standing on one leg (OLS). Relevant disease-specific clinical data (body mass index [BMI] and forced expiratory volume in 1 second [FEV1]) were recorded. Results: Twenty-eight adult pwCF (age 26.0 ± 7.8 years) were followed over 5.6 ± 0.9 years; 21 started ETI therapy during this period. Significant improvements from baseline were noted in BMI (p < 0.001) and health-related fitness components (HE, p = 0.002; PLR, p = < 0.001), whereas Wpeak and FB remained stable over time (all p > 0.05). Skill-related components (SLJ, OLS) showed no change (all p > 0.05). Subgroup analysis revealed significant improvements in BMI, FEV1, and health-related fitness measures of muscular strength and endurance (HE, p = 0.009; PLR, p < 0.001) only in pwCF using ETI. Conclusion: Despite the improvements, the impact of ETI on the individual parameters was small. Other factors than implementation of ETI alone need to be considered on the way to a high level of physical fitness in adult pwCF.

Longitudinal changes in habitual physical activity in adult people with cystic fibrosis in the presence or absence of treatment with elexacaftor/tezacaftor/ivacaftor.

Background: Habitual physical activity (PA) and exercise training are accepted as important aspects of care for people with cystic fibrosis (pwCF) to improve health-related measures of physical fitness, which in turn have a positive impact on quality of life and prognosis. In the last decade, effective CFTR modulator therapies have become a promising treatment for pwCF by targeting the underlying cause of CF. This highly effective therapy improves clinical outcomes and quality of life in people with specific CFTR mutations. Little is known about the longitudinal pattern of PA or the impact of the highly effective modulator therapy with Elexacaftor/Tezacaftor/Ivacaftor (ETI) on PA in adult pwCF. This study assessed the course of device-based PA measurement in adult pwCF and evaluated the effects of ETI on habitual physical activity in those who were eligible for ETI. Methods: Data from adult pwCF (aged ≥18 years) were analysed at baseline and follow-up, using identical assessments at both time points. Outcome parameters were PA in steps/day and the intensity of PA. The group that received ETI was treated for an average of 33 weeks and not for the entire duration of the period. The data were collected between 2021 and 2022, following the removal of absolute pandemic restrictions/lockdowns. Results: Follow-up duration was 5.6 years in pwCF with ETI (ETI group, n = 21) and 6.5 years in pwCF without ETI (non-ETI group, n = 6). From baseline to follow-up, pwCF treated with ETI had a significant increase in steps/day (+25%, p = 0.019) and a non-significant increase in moderate-to-vigorous intensity time (+5.6%, p = 0.352). Conversely, individuals in the non-ETI group showed a non-significant decrease in both steps/day -3.2%, p = 0.893) and moderate-to-vigorous intensity time (-25%, p = 0.207). The ETI group showed a significant decrease in percent predicted forced expiratory volume in 1 s (ppFEV1) and FEV1 z-score before the start of ETI treatment, both of which improved significantly after therapy initiation. Body weight and body mass index also improved significantly with ETI use. Conclusions: These data suggest that ETI treatment has a positive effect on habitual physical activity behavior in the adult pwCF studied.

Impact of habitual physical activity and exercise capacity on quality of life in adolescents and adults with cystic fibrosis.

BACKGROUND: The influence of habitual physical activity and exercise capacity on health-related quality of life (HRQoL) in people with cystic fibrosis (pwCF) is poorly characterized. This study investigated the influence of habitual physical activity, exercise capacity, lung function, and body mass index (BMI) on HRQoL in adolescent and adult pwCF. METHOD: Subjects were fitted with an accelerometer to determine habitual physical activity (steps/day), including time spent at different intensities, for up to 4 weeks. Then bicycle ergometry (maximal exercise capacity; Wpeak), lung function (percent predicted forced expiratory volume in 1 s, ppFEV1), BMI, and response to the Cystic Fibrosis Questionnaire-Revised (CFQ-R) were determined. RESULTS: Sixty-five pwCF participated in the study. Physically active pwCF had significantly higher ppFEV1 (p < .001) and exercise capacity (p < .001) than inactive pwCF, and had significantly higher scores on the CFQ-R physical (p = .006), emotional (p = .015), role (p = .008), health (p = .006), and weight (p = .004) subscales. On multiple linear regression analysis, ppFEV1 and, to a lesser extent, exercise capacity, were the most important determinants of HRQoL in pwCF. Time spent in moderate-to-vigorous intensity physical activity did not influence any of the CFQ-R subscales, whereas time spent in vigorous-intensity influenced CFQ-R scores for role (p = .007), body (p = .001), health (p = .009), and weight (p = .01). CONCLUSION: HRQoL in adolescent and adult pwCF was influenced by several factors. Avoiding sedentary behavior and spending time in vigorous-intensity levels positively influenced HRQoL, whereas the total number of steps per day played only a minor role in determining HRQoL. Both ppFEV1 and exercise capacity markedly influenced HRQoL.

Long-Term Follow-Up of Health-Related Quality of Life and Short-Term Intervention with CFTR Modulator Therapy in Adults with Cystic Fibrosis: Evaluation of Changes over Several Years with or without 33 Weeks of CFTR Modulator Therapy.

BACKGROUND: Longitudinal data on changes in health-related quality of life (HRQoL) in adult people with cystic fibrosis (pwCF) and the longitudinal effects of Elexacaftor/Tezacaftor/Ivacaftor therapy (ETI) on HRQoL or HRQoL domains are currently scarce. This study aimed to investigate the effects of ETI on HRQoL and compare them with those of pwCF who did not receive highly effective CFTR modulators over a longer period. METHODS: Baseline assessment and follow-up data for 5.6 years in pwCF with (n = 21) and 6.5 years in pwCF without (n = 6) ETI (≥18 years) were evaluated. The assessment of HRQoL and clinical parameters was identical at both time points. HRQoL was assessed using the CFQ-R, and clinical outcomes included BMI, ppFEV1, and FEV1 z-score. RESULTS: ETI was found to improve all HRQoL domains at more than four points over time, and their increases were significant except for vitality, digestion, treatment burden, and social functioning (p < 0.05). Without ETI, psychosocial domains remained almost constant, whereas most physical domains decreased over time. CONCLUSIONS: The results of the present study show that ETI therapy has a positive effect on HRQoL and clinical outcomes over time but not in pwCF without ETI treatment. Furthermore, our results suggest that disease progression over time affects the physical domains of HRQoL more than the psychosocial domains. Due to the small sample size and the heterogeneity of the study population (CFTR mutation genotype), the results should be interpreted with some caution.

Effects of a Long-Term Monitored Exercise Program on Aerobic Fitness in a Small Group of Children with Cystic Fibrosis.

Background: The aim of this study was to investigate the effects of a monitored exercise program on aerobic fitness in children with cystic fibrosis (CF). Methods: Six children (2f/4m) with ages ranging from 6 to 14 years (11.3 ± 3.3 years.) and a mean ppFEV1 102.5 ± 13.5% pred. participated in the partially monitored 12-month exercise program. VO2peak and Wpeak were used as parameters of aerobic fitness. Incremental Cardio-Pulmonary Exercise Tests (CPETs) were performed before the program began (T1), after 6 months (T3) of monitoring, and after a further 6 months (T4) without monitoring. Habitual physical activity (HPA) was assessed with accelerometry. Results: The values of VO2peak and Wpeak improved slightly from T1 to T3 (p > 0.05), without a further increase after monitoring was stopped (T4). However, the VO2peak and Wpeak values were higher after monitoring was stopped compared to at T1. The exercise program with and without monitoring (p > 0.05) had no or only a slight effect on the FEV1 values, steps/day, and the intensity of HPA. Conclusions: Monitoring seems to facilitate the achievement of beneficial effects on physical fitness in CF children. For that reason, continuous individual exercise monitoring programs that involve close contact with an exercise therapist should be provided to maintain long-term motivation and participation in physical activities and sport activities during leisure time.

Design and Evaluation of an Outdoor Exercise Program for Pediatric Cancer Survivors.

Exercise programs for young people after cancer are not part of regular oncological care. This study describes and evaluates a regional outdoor exercise program and presents data with regard to the promoting and inhibiting factors for participation among pediatric cancer survivors. Exercise options, number of participants, and the cohort were evaluated descriptively for one year. A self-developed questionnaire was used to evaluate satisfaction, mood, motivations, and barriers to exercise. Overall N = 26 survivors (14.6 ± 5.5 years) participated in at least one activity in 2019 including try-out days (N = 10) and active weekend camps (N = 2). No adverse events occurred in 302 physical activity hours. Twenty-one survivors participated in the survey. The largest motivational aspect to participate was "to try out a new sport" (83.9%). Survivors reported "good mood", and 'being happy' after exercising. The largest barrier was concern about 'not being able to keep up with others' (38.1%). Around one-third (try-out day) and 50% (active weekend camp) of survivors did not feel confident to continue exercising outside the supervised exercise oncology program. This survey shows high enthusiasm for this exercise program with different outdoor activities and suggests that similar interventions may be accepted by this population.

Comparative analysis of postural control and vertical jump performance between three different measurement devices.

OBJECTIVES: The aim of this study was to examine the concurrent validity of the HUMAC Balance System (HBS) and Balance Trainer BTG4 (BTG) in comparison to a laboratory-grade force platform (FP) for postural control (PC) and vertical jump performance (VJP) assessment. In addition, reliability of the three devices was measured for PC. METHODS: Overall 22 participants (age = 37.8 ± 13.3 years; gender = 9 male, 13 female; height = 174.1 ± 10.5 cm; body mass = 75.3 ± 17.6 kg) were recruited to participate. Double and single leg standing balance tests with eyes open or closed and counter movement jumps (CMJ) were performed on two separate occasions. Reliability and concurrent validity for COP parameters and VJP were examined using intraclass correlation coefficients (ICC), Bland-Altman plots (BAP), standard error of measurement (SEM) and minimum detectable change (MDC). RESULTS: COP path length test-retest reliability was predominantly good to excellent for all three devices (ICC = 0.80-0.95). SEM and MDC values were high for all plates (SEM% = 8.0-15.2; MDC% = 22.8-44.5), with the HBS MDC values higher than the KIS and BTG in three of the four trials. ICC scores for concurrent validity were good to excellent for the BTG (ICC = 0.76-0.93) and moderate to good for the HBS (0.49-0.83). Band-Altman plots revealed a systematic bias for the HBS towards higher COP path length values under all conditions and for the BTG in two out of four trials towards lower values. Validity of VJP was excellent for the BTG (ICC = 1.0) and poor for the HBS (0.34), with a systematic bias towards lower values. CONCLUSION: The comparative analysis of PC and VJP revealed reliable and valid results for the BTG in comparison to a laboratory-grade force plate. The HBS showed reliable results for PC assessment with restrictions regarding its validity. Results of VJP showed that the HBS revealed deficits in the assessment of activities that require rapid, high force movements such as jumping and running. Due to the variable results of all three devices, it is recommended not to use them interchangeably.