Cystic Fibrosis Questionnaire-Revised Research Articles

Validation of the Integrated Palliative Care Outcome Scale (IPOS) in adults with cystic fibrosis.

A primary palliative care model for cystic fibrosis (CF) recommends using the Integrated Palliative Care Outcome Scale (IPOS) for screening. Validation of the IPOS is needed. This secondary analysis utilized baseline data from a multisite trial of the palliative care model, Improving Life with CF. Adults with CF completed the IPOS, the Memorial Symptom Assessment Scale-CF (MSAS-CF), the CF Questionnaire-Revised (CFQ-R), the Patient Health Questionnaire (PHQ-8), the Generalized Anxiety Disorder (GAD-7), and the Perceived Stress Scale (PSS). IPOS structure was assessed using Cronbach α coefficients and a factor analysis. Construct validity was evaluated through bivariate relationships between IPOS scores and other questionnaire scores, and linear regressions assessing the extent to which the IPOS explains variance in quality-of-life domains. The sample comprised 256 adults with complete IPOS data. α coefficients were .86 for the IPOS total score, .81 for the Physical Symptoms subscale, .79 for the Emotional Symptoms subscale, and .63 for the Communication/Practical Issues subscale. A two-component factor structure best aligned with the current subscales. IPOS scores were significantly associated with other measures; associations with MSAS-CF and CFQ-R subscales differentiated the IPOS Physical and Emotional subscales. The IPOS total score provided unique information about the variance in the CFQ-R Physical Functioning and Respiratory Symptoms domain scores. In adults with CF, the IPOS has acceptable internal consistency and there is evidence of construct validity. These findings support adoption of the IPOS in the primary palliative care model for CF.

Impact of Elexacaftor–Tezacaftor–Ivacaftor Therapy on Body Composition, Dietary Intake, Biomarkers, and Quality of Life in People with Cystic Fibrosis: A Prospective Observational Study

Background: The combination of elexacaftor–tezacaftor–ivacaftor modulators (ETI) has improved clinical outcomes for people with cystic fibrosis (pwCF). Objectives: This study aimed to evaluate changes in nutritional and morphofunctional assessments, as well as anxiety, depression symptoms, and quality of life, in pwCF after starting ETI therapy. Methods: This was a prospective observational study. We measured body composition (fat mass [FM] and fat-free mass [FFM]) using bioelectrical impedance analysis (BIA) and skinfold thickness measurements (SMs). We also assessed hand grip strength, dietary intake via surveys, blood and stool biomarkers, symptoms of anxiety and depression using the Hospital Anxiety and Depression Scale [HADS], and quality of life through the Cystic Fibrosis Questionnaire—Revised (CFQR). Results: A total of 31 pwCF were evaluated. Significant improvements were observed in respiratory function and quality of life, alongside an average weight increase of approximately 5 kg (60% FM and 40% FFM). The prevalence of malnutrition, based on BMI and the FFM index, decreased significantly, while the rate of overweight/obesity increased. Biomarker analysis indicated better nutrient absorption and reduced intestinal inflammation, as evidenced by significant changes in faecal calprotectin, nitrogen, and fat levels, as well as blood lipid and vitamin profiles. Conclusions: Despite a reduction in caloric intake, an increase in weight was observed one year after initiating ETI. This increase was attributed to gains in both FM and FFM, suggesting improved metabolic efficiency and nutrient absorption. Both SM and BIA were found to be useful assessment tools. These findings indicate the need to modify the nutritional approach, focusing on the quality rather than the quantity of intake, and aiming for an appropriate body composition (FFM) rather than solely focusing on BMI.

Evaluation of elexacaftor–tezacaftor–ivacaftor treatment in individuals with cystic fibrosis and CFTRN1303K in the USA: a prospective, multicentre, open-label, single-arm trial

CFTR modulators are approved for approximately 90% of people with cystic fibrosis in the USA and provide substantial clinical benefit. N1303K (Asn1303Lys), one of the most common class 2 CFTR defects, has not been approved for these therapies by any regulatory agency. Preclinical investigation by our laboratories showed N1303K CFTR activation with elexacaftor-tezacaftor-ivacaftor (ETI). In this trial, we evaluate whether ETI improves CFTR function, measured by sweat chloride and other clinical outcomes, in people with cystic fibrosis and CFTRN1303K. In this prospective, open-label, single-arm trial, participants aged 12 years or older with cystic fibrosis encoding at least one N1303K variant and at least one CFTRN1303K allele who were ineligible for modulator therapy by US Food and Drug Administration labelling were given ETI for 28 days followed by a 28-day washout period at two cystic fibrosis centres in the USA. Participants received two orally administered pills of 100 mg elexacaftor, 50 mg tezacaftor, and 75 mg ivacaftor once daily in the morning, and 150 mg ivacaftor once daily in the evening. The primary endpoint was mean change in sweat chloride from baseline up to day 28 compared with mixed-effects models. Secondary endpoints were changes in percentage of predicted FEV1 (ppFEV1), Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain, BMI, and weight after ETI therapy. Safety was assessed in all participants who received at least one dose of the study drug and primary and secondary analyses were performed in all participants who took the study drug per protocol. The trial was registered at ClinicalTrials.gov (NCT03506061) and remains open for reporting purposes. Between June 7, 2022, and Oct 20, 2023, 20 participants (ten male and ten female) were enrolled and received ETI treatment. One participant was lost to follow-up but was included in intention-to-treat analyses. At 28 days, the mean sweat chloride reduction was -1·1 mmol/L (95% CI -5·3 to 3·1; p=0·61) with only one participant showing a sweat chloride decrease greater than 15 mmol/L. There was a mean increase in ppFEV1 from baseline at day 28 of 9·5 percentage points (6·7-12·3; p<0·0001) with 15 (75%) participants showing at least a 5% increase in ppFEV1. Improvements were also identified in mean CFQ-R respiratory domain score (20·8 increase [95% CI 11·9-29·8]; p<0·0001), BMI (0·4 kg/m2 increase [0·2-0·7]; p=0·0017), and weight (1·0 kg increase [0·4-1·7]; p=0·0020) after 28 days of ETI treatment. 14 (70%) of 20 participants had adverse events (12 [60%] mild, one [5%] moderate), with one (5%) serious adverse event of hospitalisation attributed to pneumonia. No deaths were recorded in the study. Individuals with CFTRN1303K showed no change in sweat chloride after 28 days of treatment with ETI. However, there were improvements in secondary clinical endpoints, which suggest clinical efficacy. Our approach provides support for the use of in vitro model systems to inform clinical trials for rare CFTR variants. The Cystic Fibrosis Foundation and the US National Institutes of Health.

Impact of sociodemographic status and sex on chronic rhinosinusitis and olfaction in people with cystic fibrosis.

Sociodemographic status (SDS) including race/ethnicity and socioeconomic status as approximated by education, income, and insurance status impact pulmonary disease in people with cystic fibrosis (PwCF). The relationship between SDS and chronic rhinosinusitis (CRS) remains understudied. In a prospective, multi-institutional study, adult PwCF completed the 22-Question SinoNasal Outcome Test (SNOT-22), Smell Identification Test (SIT), Questionnaire of Olfactory Disorder Negative Statements (QOD-NS), and Cystic Fibrosis Questionnaire-Revised (CFQ-R). Lund-Kennedy scores, sinus computed tomography, and clinical data were collected. Data were analyzed across race/ethnicity, sex, and socioeconomic factors using multivariate regression. Seventy-three PwCF participated with a mean age of 34.7±10.9 years and 49 (67.1%) were female. Linear regression identified that elexacaftor/tezacaftor/ivacaftor (ETI) use (β=‒4.09, 95% confidence interval [CI] [‒6.08, ‒2.11], p<0.001), female sex (β=‒2.14, 95% CI [‒4.11, ‒0.17], p=0.034), and increasing age (β=‒0.14, 95% CI [‒0.22, ‒0.05], p=0.003) were associated with lower/better endoscopy scores. Private health insurance (β=17.76, 95% CI [5.20, 30.32],p=0.006) and >16 educational years (β=13.50, 95% CI [2.21, 24.80], p=0.020) were associated with higher baseline percent predicted forced expiratory volume in one second (ppFEV1). Medicaid/Medicare insurance was associated with worse endoscopy scores, CFQ-R respiratory scores, and ppFEV1 (all p<0.017), and Hispanic/Latino ethnicity was associated with worse SNOT-22 scores (p=0.047), prior to adjustment for other cofactors. No other SDS factors were associated with SNOT-22, QOD-NS, or SIT scores. Differences in objective measures of CRS severity exist among PwCF related to sex, age, and ETI use. Variant status and race did not influence patient-reported CRS severity measures or olfaction in this study. Understanding how these factors impact response to treatment may improve care disparities among PwCF. NCT04469439.

P096 Qualitative interviews confirming the faithful electronic migration of the Preschool Pictorial Cystic Fibrosis Questionnaire-Revised (CFQ-R) and Parent Preschool CFQ-R

P096 Qualitative interviews confirming the faithful electronic migration of the Preschool Pictorial Cystic Fibrosis Questionnaire-Revised (CFQ-R) and Parent Preschool CFQ-R

Evaluating the contribution of the Glittre-ADL test in adults with cystic fibrosis.

Cardiopulmonary and skeletal muscle impairment and poor physical activity are potential contributors to reduced functional capacity in cystic fibrosis (CF). The Glittre-ADL test (TGlittre) has great potential for clinical use in adult CF adults, as it meets the need for a comprehensive assessment of physical function using tasks similar to activities of daily living. This study aimed to evaluate the performance of TGlittre in CF adults compared to the 6-min walk test (6MWT) and, secondarily, to quantify the associations of their results with pulmonary function, muscle strength, and health-related quality of life (HRQoL). This cross-sectional study evaluated 34 CF adults and compared them with 34 subjects from a control group. The participants underwent the following assessments: functional capacity using TGlittre and 6MWT; spirometry; respiratory muscle strength; handgrip strength (HGS); and HRQoL using the Cystic Fibrosis Questionnaire-Revised (CFQ-R). While CF patients showed a longer time to perform TGlittre compared to controls (134 (119-150) versus 107 (95-126) % of the predicted time p=0.0002), no difference between these groups was observed in the 6MWT. When the second TGlittre was compared to the first TGlittre, there was a significant decrease in total time for both CF patients (p<0.0001) and controls (p=0.0001). TGlittre time correlated with 6MWT distance (6MWD) (rs=-0.641, p<0.0001), HGS (rs=-0.364, p=0.034), peripheral oxygen saturation at the end of the test (rs=-0.463, p=0.006) and the "digestive symptoms" domain of CFQ-R (rs=0.376, p=0.028). TGlittre time was shorter in patients who engaged in regular physical activity (3.10 (2.49-3.39) min versus 3.28 (2.95-3.53) min, p=0.016). TGlittre is more effective than the 6MWT in detecting limitations during exercise. There is an important learning effect of TGlittre in adult CF patients. TGlittre time was correlated with 6MWD, HGS, oxygen saturation level, and the patient's level of physical activity.

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

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. 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. 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). 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.

The impact of elexacaftor/tezacaftor/ivacaftor on cystic fibrosis health-related quality of life and decision-making about daily treatment regimens: a mixed methods exploratory study.

Elexacaftor/tezacaftor/ivacaftor (ETI) has reduced many symptoms of cystic fibrosis (CF). We sought to identify the impact of ETI on both symptoms and treatment decisions among adults with CF. Participants were enrolled in a cross-sectional study. Surveys were sent via a RedCap link. Semistructured interviews were administered remotely via Microsoft Teams. Interviews were audio recorded and professionally transcribed. We assessed Cystic Fibrosis Questionnaire-Revised (CFQ-R) subscales for physical, respiratory, emotion, and treatment, and analyzed semistructured interviews covering CF treatment regimens and daily living. Quantitative and qualitative results were analyzed separately and via a mixed-methods convergence coding matrix. Twenty-four adults with CF taking ETI were included. CFQ-R subscale scores (mean scores/standard deviation) were physical (82.1/22.8), respiratory (83.7/11.2), emotion (65.3/14.2), and treatment (57.5/20.1). Three themes about decision-making for non-ETI-treatments emerged: (1) How I'm feeling, (2) Not noticing a difference, and (3) Uncertainty about long-term impact of modifying treatment regimens, and we found participants weighed each of these factors in their treatment decisions. Key findings from mixed-methods analysis show that among individuals experiencing higher CFQ-R scores for physical and respiratory compared to emotion and treatment, there were statements indicating that while those participants were experiencing better physical health, many continued their burdensome treatment regimens. With little long-term data on the impact of reducing non-ETI treatments, participants weighed how they were feeling, treatment efficacy beliefs, and risk tolerance when making treatment decisions.

Current cough and sputum assessed by the cough and sputum assessment-questionnaire (CASA-Q) is associated with quality of life impairment in cystic fibrosis

BackgroundCough and sputum are major symptoms in cystic fibrosis (CF) that contribute to the impairment of quality of life.MethodsThis prospective single centre cross-sectional pilot study aimed to evaluate the results of a self-administered questionnaire assessing cough and sputum symptoms (2 domains), and their impact (2 domains) on daily activities in the previous week, named the Cough and Sputum Assessment Questionnaire (CASA-Q) in CF adult patients at stable state, and to analyse associations with clinical, functional, microbiological, radiological data, and two quality of life scales: the Cystic Fibrosis Questionnaire Revised (CFQ-R) and the Saint George Respiratory Questionnaire (SGRQ).ResultsForty-eight patients were included in this analysis (69% men; median age of 27.8 ± 8.1 years; median body mass index of 21.8 + 3.3 kg/m²; mean FEV1 of 64 ± 30% of the predicted value). The mean values of the CASA-Q domains were 58 ± 23 for cough symptoms, 77 ± 24 for cough impact, 62 ± 25 for sputum symptoms and 84 ± 21 for sputum impact. Impairment in CASA-Q cough and sputum domains was associated with dyspnea mMRC scale (p < 0.005 for all 4 domains of CASA-Q) and exacerbations in the previous year (p < 0.05 for CASA-Q symptoms domains). We also found correlations between all domains of the CASA-Q and quality of life questionnaires including SGRQ (p < 0.001) and to a lesser extend CFQ-R. We identified a clinical phenotype (female gender, ΔF508 heterozygous mutation, dyspnea mMRC scale) associated with an impairment of CASA-Q score and quality of life using a 2-step cluster analysis.ConclusionsCASA-Q allows the assessment of cough and sputum in CF adult patients and is associated with quality of life impairment. This simple easy-to-use tool could be used in routine clinical practice and in clinical studies to assess cough and sputum in CF patients.Trial RegistrationThe study was registered on ClinicalTrials.gov (NCT02924818, first posted on 5th October 2016).

Radiological and clinical evaluation of triple combination modulating therapy effectiveness in adult patients with cystic fibrosis

ObjectivesPrevious studies showed the clinical effectiveness of elexacaftor-tezacaftor-ivacaftor (ETI) in cystic fibrosis (CF) patients and a recently published study evaluated twelve CF patients that performed chest and sinus computed tomography (CT) examinations and showed that ETI decreased pulmonary and sinus morphological abnormalities after one year of treatment. The aim of the present study was to evaluate the role of CFTR modulator therapy in improving radiological and clinical scores one year after ETI therapy initiation in a wider CF patient population. Materials and methodsBetween January 2020 and December 2022, 44 CF adult patients received elexacaftor-tezacaftor-ivacaftor (ETI) therapy for at least one year and underwent a chest CT examination at our hospital before and one year after ETI therapy initiation. Experienced radiologists who were blinded to the treatment assessed the images in consensus. The Brody-II score (BSII), the Lund-Mackay score (LM score) and the Sheikh-Lind CT sinus disease severity scoring system (SL score) were evaluated. Clinical scores such as cystic fibrosis clinical score (CFCS), Cystic Fibrosis Questionnaire-Revised (CFQ-R) score, the 22-item SinoNasal Outcome Test (SNOT-22) questionnaire and the CF-specific 28-modal abdominal symptom score (CFAbd-Score) were evaluated. Forced expiratory volume in 1 ​s (FEV1) and forced vital capacity (FVC) were also assessed. Paired samples t-tests were used to compare differences before and after one year of ETI therapy initiation, and Pearson's correlation coefficient was used to evaluate changes in FEV1 and total BSII and in FVC and total BSII. ResultsTotal BIIS one year after ETI initiation showed statistically significant lower scores (−6.0 p, p ​< ​0.0001). In particular, mucous plugging (−15.8 p, p ​< ​0.0001), peribronchial thickening (−16.2 p, p ​< ​0.0001) and parenchyma (−0.3 p, p ​= ​0.0397) showed statistically significant lower scores. LM score, SL score, FEV1, FVC, CFCS, CFQ-R, SNOT-22 and CFAbd-Score showed statistically significant lower scores one year after ETI initiation (p ​< ​0.0001). The correlation between ΔFEV1 and Δtotal BSII was statistically significant and moderate (r ​= ​−0.5188, p ​= ​0.0003), and the correlation between ΔFVC and Δtotal BSII was statistically significant and weak (r ​= ​−0.3160, p ​= ​0.0367). ConclusionEvolution of imaging findings on CT during follow-up closely correlate with improved clinical scores and functional data one year after ETI therapy initiation, indicating that CT may be a useful adjunct during follow-up of CF patients under this treatment as an objective measure of disease improvement.

The effects of aerobic exercises compared to conventional chest physiotherapy on pulmonary function, functional capacity, sputum culture, and quality of life in children and adolescents with cystic fibrosis: a study protocol for randomized controlled trial study

BackgroundCystic fibrosis (CF) is an autosomal recessive disorder caused by abnormal function of the chloride ion channels and characterized by pancreatic insufficiency and chronic endobronchial airway infection. Pulmonary dysfunction is very common and could lead to a reduction in the quality of life. Airway clearance techniques (ACT) and physical exercises are introduced as one of the main components of treatment. Therefore, it will be of interest to examine the effect of aerobic exercises compared to conventional chest physiotherapy (CPT) on pulmonary function, functional capacity, sputum culture, and quality of life in patients with CF.MethodsThirty patients with CF will participate in a double-blind parallel controlled trial containing 18 sessions of treatment. Group A consists of CPT and placebo aerobic exercise, and group B includes aerobic exercise and placebo CPT. Pulmonary function, functional capacity, sputum culture, and quality of life will be evaluated with a spirometry test, 6-min walk test (6MWT), sputum culture test, and the Cystic Fibrosis Questionnaire-Revised (CFQ-R), respectively, before and after the intervention.DiscussionWe will evaluate and compare the effectiveness of aerobic exercises and conventional chest physiotherapy on pulmonary function, functional capacity, sputum culture, and quality of life. Comparing these two treatment patterns can contribute to a better understanding of the effectiveness. Therefore, if there is a significant difference between the two treatments, the superior treatment will be prioritized clinically.Trial registrationhttps://www.irct.ir, IRCT20210505051181N5. Registered on 19 February 2023.

Impact of cystic fibrosis multidisciplinary virtual clinics on patient experience, time commitments and costs.

The experience of outpatient care may differ for select patient groups. This prospective study evaluates the adult patient experience of multidisciplinary outpatient cystic fibrosis (CF) care with videoconferencing through telehealth compared with face-to-face care the year prior. People with CF without a lung transplant were recruited. Patient-reported outcomes were obtained at commencement and 12 months into the study, reflecting both their face-to-face and telehealth through videoconferencing experience, respectively. Three patient cohorts were analysed: (i) participants with a regional residence, (ii) participants with a nonregional including metropolitan residence and (iii) participants with colonised multiresistant microbiota. Seventy-four patients were enrolled in the study (mean age, 37 ± 11 years;50% male;mean forced expiratory volume in the first second of expiration, 60% [standard deviation, 23]) between February 2020 and May 2021. No differences between models were observed in the participants' rating of the health care team, general and mental health rating, and their confidence in handling treatment plans at home. No between-group differences in the Cystic Fibrosis Questionnaire - Revised (CFQ-R) were observed. Travel duration and the cost of attending a clinic was significantly reduced, particularly for the regional group (4 h, AU$108 per clinic;P < 0.05). A total of 93% respondents preferred to continue with a hybrid approach. In this pilot study, participants' experience of care and quality of life were no different with face-to-face and virtual care between the groups. Time and cost-savings, particularly for patients living in regional areas, were observed. Most participants preferred to continue with a hybrid model for outpatient care.

The relationships between upper extremity muscle strength and endurance with respiratory function, functional capacity and quality of life in children and adolescents with cystic fibrosis

Background/Aims The number of studies exploring isolated upper extremity muscle strength and endurance in paediatric patients with cystic fibrosis is limited. The aim of this study was to investigate the relationship between upper extremity muscle strength and endurance with respiratory functions, functional capacity and quality of life in children and adolescents with cystic fibrosis. Methods Children and adolescents with cystic fibrosis performed a flexed arm hang test to measure muscle endurance, movement against a dynamometer to measure muscle strength, a pulmonary function test and 6-minute walk test to measure functional capacity. The Cystic Fibrosis Questionnaire Revised was used to assess quality of life. Results A total of 29 children and adolescents with cystic fibrosis (15 girls, 14 boys) participated in the study. Biceps muscle strength moderately correlated with forced expiratory volume in 1 second predicted percentage, forced vital capacity predicted percentage and the ratio of forced expiratory volume in 1 second to forced vital capacity predicted percentage (r=0.306, P=0.035; r=0.405; P=0.029; r=0.367, P=0.048, respectively). Biceps muscle strength, shoulder abductors muscle strength, hand grip strength and flexed arm hang test correlated with the ratio of forced expiratory volume in 1 second to forced vital capacity predicted percentage. Biceps muscle strength, shoulder abductors muscle strength, hand grip strength and flexed arm hang test correlated with 6-minute walk test distance (r=0.678, p&lt;0.001; r=0.732, p&lt;0.001; r=0.512, P=0.005; r=0.375, P=0.045, respectively). Biceps muscle strength and shoulder abductors muscle strength moderately correlated with emotional functioning and eating disturbances in the Cystic Fibrosis Questionnaire Revised (r=0.451, P=0.014). Conclusions Specific assessment of upper extremity muscle strength and endurance in children and adolescents with cystic fibrosis may be helpful in informing physiotherapists of respiratory function, functional capacity and quality of life status.]

491 The Cystic Fibrosis Questionnaire–Revised and body image in adults in the era of elexacaftor-tezacaftor-ivacaftor

491 The Cystic Fibrosis Questionnaire–Revised and body image in adults in the era of elexacaftor-tezacaftor-ivacaftor

Spanish adaptation and validation of the child- and parent-report cystic fibrosis questionnaire-revised (CFQ-R).

To evaluate the psychometric properties of the Spanish versions of the child- and parent-report cystic fibrosis questionnaire-revised (CFQ-R). A Spanish adaptation of the CFQ-R was performed; 68 children with CF (6-13 years) and their parents completed the child- and parent-report CFQ-R, respectively, and the Revidierter KINDer Lebensqualitätsfragebogen (KINDL) questionnaire. The CFQ-R was completed twice, 7-10 days apart, and its psychometric properties were analyzed. The internal consistency of both CFQ-R versions was adequate (child-report version, Cronbach's α >.60 for all domains except "Treatment Burden" [α = .42] and "Social Functioning" [α = .57]; parent-report version, α > .60 for all domains except "Social Functioning" [α = .58]). For the child-report version, the lowest measurement error was for "Emotional Functioning" (standard error of measurement [SEM]: 8.3%; minimal detectable change [MDC90 ]:19.3%), and the highest was for "Body Image" (SEM:15%; MDC90 : 35%). For the parent-report version, the lowest measurement error was for "Physical Functioning" (SEM:7.1%; MDC90 : 16.5%), and the highest was for "Weight" (SEM:17.2%; MDC90 ; 40.1%). The correlation between the versions showed higher agreement for the domains related to observable signs ("Physical Functioning") and lower agreement for "Emotional Functioning."There was a significant correlation between the CFQ-R and KINDL. Both the child- and parent-report versions of the Spanish CFQ-R have adequate reliability and validity for clinical and research purposes. These versions can be administered before and after starting modulator therapy to assess its effect on daily functioning. The MDC90 can help identify, with a high probability, whether real changes have occurred in the quality-of-life subscales in children with CF.

Development and validation of a novel personalized electronic patient-reported outcome measure to assess quality of life (Q-LIFE): a prospective observational study in people with Cystic Fibrosis

Generic and disease-specific patient-reported outcome measures (PROMs) may lack relevance and sensitivity on a patient-level in chronic diseases with differential disease expression and high individual variability, such as Cystic Fibrosis (CF). This study aimed to develop and validate a novel personalized electronic PROM (ePROM) that captures relevant aspects of quality of life in individuals with CF. The Q-Life app was developed as a short personalized ePROM to assess individual quality of life. Psychometric properties were assessed in a single-center cross-sectional study between September 2019 and September 2021 and in a prospective cohort study between September 2021 and September2022. Combined studies included 223 participants (median age: 24 years, IQR: 19.0-32.5 years, range: 12.0-58.0 years). Internal consistency (Cronbach's alpha: 0.83-0.90) and test-retest reliability (intraclass correlation coefficient: 0.90; 95% CI: 0.65-0.92; p<0.001) of quality of life (Q-Life) scores were strong. Q-Life scores were associated with overall Cystic Fibrosis Questionnaire-Revised (CFQ-R) scores (ρ=0.71; p<0.001), CFQ-R respiratory domain scores (ρ=0.57; p<0.001) and forced expiratory volume in 1s (ρ=0.41; p<0.001). Furthermore, Q-Life scores improved from 65.0 (IQR: 45.0-63.3) at baseline to 84.2 (IQR: 75.0-95.0) and 87.5 (IQR: 75.0-100.0) after 3 and 6 months of elexacaftor/tezacaftor/ivacaftor treatment (change: 20.8; 95% CI: 17.5-25.0; p<0.001), comparable to CFQ-R respiratory domain scores (change: 22.2, 95% CI: 19.4-25.0, p<0.001). The Q-Life app is a reliable, valid and sensitive personalized ePROM to measure all aspects of quality of life that really matter to individuals with Cystic Fibrosis. This patient-centered approach could provide important advantages over generic and disease-specific PROMs in the era of personalized medicine and value-based healthcare. Dutch Cystic Fibrosis Foundation, Health-Holland.

Olfaction, body mass index, and quality of life with cystic fibrosis combination therapy.

Triple-combination therapy of elexacaftor-tezacaftor-ivacaftor (ETI) has been shown to reduce morbidity and mortality in people with cystic fibrosis (PwCF). Although patient body mass index (BMI) favorably increases with ETI treatment, factors contributing to this improvement are poorly characterized. Olfaction contributes to appetite stimulation and anticipation of eating, where higher rates of olfactory impairment (OI) in PwCF may contribute to malnutrition and BMI instability in this population. The authors performed a prospective cohort study analyzing 41 CF patient responses to the Cystic Fibrosis Questionnaire-Revised (CFQR) and the 22-Item Sino-Nasal Outcome Test (SNOT-22) and used generalized estimating equations to understand the change in survey variables from being untreated (baseline) to undergoing 3months of ETI therapy (follow-up). Patients reported significant improvement in their sense of smell at follow-up (p=0.0036). Their improvements in sense of smell were not confounded by changes in rhinologic or extranasal rhinologic symptoms. Self-reported quality of life (QoL) improved after 3months of ETI therapy (p=<0.0001) as did BMI (p=<0.0001), but improved sense of smell did not independently mediate these changes in QoL and BMI. Our results support the impression that ETI therapy improves CF-associated rhinologic symptoms and reverses OI, while contributing to improvement in rhinologic QoL. Sense of smell is not an independent mediator of improved QoL and BMI in this population, suggesting that other factors may have a stronger role in these realms. However, given the subjective improvement in sense of smell, additional evaluation of OI using psychophysical chemosensory assessment will clarify the connection between olfaction, BMI, and QoL in PwCF.

Effects of elexacaftor/tezacaftor/ivacaftor therapy on mental health of patients with cystic fibrosis.

Introduction: The CFTR modulator drug elexacaftor/tezacaftor/ivacaftor (ETI) was shown to improve CFTR function and clinical symptoms in patients with cystic fibrosis (CF) with at least one F508del allele. Recently, some case reports suggested potential side effects of ETI on mental health with an increase in depressive symptoms and even suicide attempts in patients with CF. However, the general effects of this triple combination therapy on the mental health status of patients with CF remain largely unknown. Methods: We, therefore, performed a prospective, observational study in a real-life setting and investigated the relationship between initiation of ETI therapy and changes in mental health in adult patients with CF. We assessed Cystic Fibrosis Questionnaire-Revised (CFQ-R), Patient Health Questionnaire-9 (PHQ-9), Beck's Depression Inventory - Fast Screen (BDI-FS) and Generalized Anxiety Disorder 7-item Scale (GAD-7) at baseline and 8-16weeks after initiation of ETI. Results: In total, 70 adult patients with CF with at least one F508del allele and a median age of 27.9years were recruited. After initiation of ETI, the CFQ-R respiratory domain score improved by 27.9 (IQR 5.6 to 47.2; p < 0.001). The PHQ-9 score of depressive symptoms decreased by 1.0 (IQR -3.0 to 0.3; p < 0.05) with an increase of 16.9% in the group with a minimal score after initiation of ETI and a decrease in the groups of mild (-11.3%) or moderate (-5.7%) scores compared to baseline. The BDI-FS score of depressive symptoms decreased from 1.0 (IQR 0.0-2.0) at baseline to 0.0 (IQR 0.0 to 2.0; p < 0.05) after initiation of ETI. The group with a minimal BDI-FS score increased by 8.0% after initiation of ETI, whereas the groups with mild (-4.9%), moderate (-1.6%) or severe (-1.6%) scores decreased compared to baseline. The GAD-7 score of anxiety symptoms did not change after initiation of ETI compared to baseline (0.0; IQR -2.0. to 0.0; p = 0.112). Conclusion: Initiation of ETI improves symptoms of depression in adult patients with CF with at least one F508del allele. However, symptoms of anxiety do not change after short-term therapy with ETI.

Clinical characteristics and outcomes of cystic fibrosisin Palestine: Cross sectional study.

To assess the clinical characteristics and outcomes of cystic fibrosis in Palestine by studying the quality of life (QoL) of participants. This cross-sectional study involved the application of Cystic Fibrosis Questionnaire-Revised (CFQ-R) to participants attending the pediatric pulmonology clinic at Caritas Baby Hospital between January and May 2017. Health status was assessed by measuring pulmonary function test (FEV1 ), body mass index (BMI), age of CF diagnosis, and presence of other affected siblings or deaths in the family. There were 77 participants from 58 families: 46.8% (36/77) were males, and 53.3% (41/77) were females. The mean age was 10.7 years (range: 0.5-36 years). The participants were divided into three groups by age in years: group I ( < 6), II (6-13), and III (≥ 14). The highest and lowest CFQ scores were for the eating domain in group III (55.6 ± 22.5) and the body domain in group II (14.5 ± 17.7), respectively. Mean illness severity was 69.6% (range: 33%-111%). The mean BMI was 15.9 (range: 9.6-23.1). The mean age at the time of diagnosis was 4.2 years (± 6.3). The study showed that 1.7%of the families (1/58) had four affected siblings, and 21% (12/58) had death cases related to CF, of which 58.3% (7/12) were from the Hebron district. Finally, all parameters for CF participants in West Bank, Palestine were noticeably lower than those reported in other countries. This study illustrates the need for new therapies for CF participants in Palestine to improve QoL, health status, and longevity.

A systematic review to explore how exercise-based physiotherapy via telemedicine can promote health related benefits for people with cystic fibrosis.

To conduct a systematic review to evaluate the effects of physiotherapy exercises delivered via telemedicine on lung function and quality-of-life in people with Cystic Fibrosis (CF). The databases AMED, CINAHL and MEDLINE were searched from December 2001 until December 2021. Reference lists of included studies were hand-searched. The PRISMA 2020 statement was used to report the review. Studies of any design reported in the English language, included participants with CF, and within outpatient settings were included. Meta-analysis was not deemed appropriate due to the diversity of interventions and heterogeneity of the included studies. Following screening, eight studies with 180 total participants met the inclusion criteria. Sample sizes ranged from 9 to 41 participants. Research designs included five single cohort intervention studies, two randomised control trials and one feasibility study. Telemedicine-based interventions included Tai-Chi, aerobic, and resistance exercise delivered over a study period of six to twelve weeks. All included studies which measured percentage predicted forced expiratory volume in one second found no significant difference. Five studies measuring the Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain found improvements, however, did not meet statistical significance. For the CFQ-R physical domain, measured by five studies, two studies found an improvement, although not statistically significant. No adverse events were reported across all studies. The included studies indicate that telemedicine-based exercise over 6-12 weeks does not significantly change lung function or quality-of-life in people with CF. Whilst the role of telemedicine in the care of pwCF is acceptable and promising; further research with standardised outcome measures, larger sample sizes and longer follow-up are required before clinical practice recommendations can be developed.