Measuring recovery in health-related quality of life during and after pulmonary exacerbations in patients with cystic fibrosis

BackgroundWe explored the time-dependent impact of pulmonary exacerbations (PEx) on health-related quality of life (HRQoL) using Cystic Fibrosis Questionnaire–Revised (CFQ-R) data from 2 large cystic fibrosis (CF) trials.MethodsThis exploratory post-hoc analysis evaluated the impact of PEx on CFQ-R domains of functioning in 80 patients with CF (homozygous for F508del-CFTR), aged ≥14 years randomized to placebo in the TRAFFIC and TRANSPORT trials who experienced 1 PEx.ResultsScores on the CFQ-R were significantly lower within 1 week of PEx start in 8 out of 12 domains (Respiratory Symptoms, Physical Functioning, Emotional Functioning, Health Perceptions, Role Functioning, Social Functioning, Eating, and Vitality). Patients whose PEx was treated with hospitalization or intravenous antibiotics had greater reductions in some domains of HRQoL compared with those treated with oral antibiotics. In the immediate weeks post-PEx, improvement was seen on Emotional Functioning, Respiratory Symptoms, and Health Perceptions, while further decline was seen for Eating, Physical Functioning, Role Functioning, Vitality, and Weight. For some measures (Physical Functioning, Vitality), full recovery to pre-PEx levels took several weeks.ConclusionsPulmonary exacerbations have significant effects on multiple domains of HRQoL, and recovery across multiple domains post-PEx can take several weeks. These findings provide insight into the impact of PEx on patient HRQoL and recovery post-PEx.Clinical trial registration:ClinicalTrials.gov identifiers, NCT01807923 and NCT01807949     

Quality of life is poorly correlated to lung disease severity in school-aged children with cystic fibrosis

BackgroundThere is no data exclusively on the relationship between health-related quality-of-life (HRQOL) and lung disease severity in early school-aged children with cystic fibrosis (CF). Using data from the Australian Respiratory Early Surveillance Team for Cystic Fibrosis (AREST CF) we assessed the relationships between HRQOL, lung function and structure.Methods125 children aged 6.5-10 years enrolled in the AREST CF program were included from CF clinics at Royal Children's Hospital (RCH), Melbourne (n = 66) and Perth Children's Hospital (PCH), Perth (n = 59), Australia. Demographics, HRQOL measured by Cystic Fibrosis Questionnaire-Revised (CFQ-R), spirometry, multiple-breath washout (MBW) and chest CT were collected across two years. Correlation between CFQ-R scores and lung structure/function parameters and agreement between parent-proxy and child-reported HRQOL were evaluated.ResultsNo correlation was observed between most CFQ-R domain scores and FEV1 z-scores, excepting weak-positive correlation with parent CFQ-R Physical (rho = 0.21, CI 0.02-0.37), and Weight (rho = 0.21, CI 0.03-0.38) domain and child Body domain (rho = 0.26, CI 0.00-0.48).No correlation between most CFQ-R domain scores and LCI values was noted excepting weak-negative correlation with parent Respiratory (rho = -0.23, CI ^?0.41-^?0.05), Emotional (rho = -0.24, CI ^?0.43-^?0.04), and Physical (-0.21, CI ^?0.39-^?0.02) domains. Furthermore, structural lung disease on CT data demonstrated little to no association with CFQ-R parent and child domain scores. Additionally, no agreement between child self-report and parent-proxy CFQ-R scores was observed across the majority of domains and visits.ConclusionHRQOL correlated poorly with lung function and structure in early school-aged children with CF, hence clinical trials should consider these outcomes independently when determining study end-points.     

Health-related quality-of-life in children with cystic fibrosis aged 5-years and associations with health outcomes

BackgroundThe impact of early cystic fibrosis (CF) on health-related quality-of-life (HRQOL) in preschool children is poorly characterised, and data on relationships between HRQOL and health outcomes in young children with CF are limited. We aimed to characterise and compare parent-proxy and child-reported HRQOL and evaluate relationships with clinical outcomes at age 5-years.MethodsSubjects were participating in the multi-centre Australasian Cystic Fibrosis Bronchoalveolar Lavage (ACFBAL) trial investigating BAL-directed versus standard CF therapy. Children aged 5-years and their parents rated HRQOL using the Pediatric Quality of Life Inventory (PedsQL™) and Cystic Fibrosis Questionnaire-Revised (CFQ-R) questionnaires.ResultsPedsQL and CFQ-R questionnaires were completed by 141 primary caregivers and 135 and 130 children, respectively. There were no differences in HRQOL between children randomised to BAL-directed versus standard CF therapy. Children with CF rated worse HRQOL than healthy children and there was poor parent-child concordance across HRQOL domains. Nutritional status, CF-CT scan score, forced expiratory volume in 1-second (FEV₁), and pulmonary exacerbations correlated with HRQOL at age 5-years. FEV₁ z-scores positively correlated with parent-proxy HRQOL in CFQ-R Respiratory (p = 0.018), Physical (<0.001), Emotional (p = 0.007) subscales and PedsQL Total-score (p = 0.021), Physical (p = 0.019) domains. Pulmonary exacerbation rates were inversely associated with parent-proxy CFQ-R Respiratory (p = 0.004), Physical (p = 0.022), PedsQL Total (p = 0.009) and Physical (p = 0.009) scores.ConclusionParent-reported HRQOL is a meaningful clinical endpoint to evaluate interventions in young children. Parent and child HRQOL reports provide different, complementary information. A preschool version of the CFQ-R is needed to assess relationships between HRQOL and clinical outcomes in young children.     

Quality of life in patients with Cystic Fibrosis: Association with anxiety and depression

BackgroundFew studies of patients with CF have looked at the association between patient reported Health-Related Quality of Life (HRQoL) and anxiety and depression. This study investigated whether CF patients with symptoms of anxiety or depression reported lower Health-Related Quality of Life (HRQoL) scores.Methods57 adult CF patients completed the Hospital Anxiety and Depression Scale (HADS) and the Cystic Fibrosis Questionnaire, a CF-specific measure of HRQoL. Analyses of variance with lung function as a covariate were used to investigate differences in HRQoL between groups of patients with and without symptoms of anxiety and depression.ResultsMean age was 26.7 years (SD 8.1), mean FEV1 %predicted was 65.09 (SD 22.18). Anxiety and depression scores were low and similar to normative scores. After controlling for lung function, patients with symptoms of anxiety reported lower on vitality, emotional functioning, social, treatment burden, health perceptions and respiratory symptoms. Those with depressive symptoms reported lower HRQoL scores for emotional functioning, eating disturbances and body image.ConclusionsPreliminary evidence was found of the role of anxiety and depression in different areas of quality of life in CF, which may help in the development of appropriate medical and psychosocial treatment programs.     

The impact of cystic fibrosis-related diabetes on health-related quality of life

Cystic fibrosis-related diabetes (CFRD) is a well-known comorbidity among the CF population. To investigate whether CFRD impacts health-related quality of life (HRQoL), domain scores from the Cystic Fibrosis Questionnaire-Revised for adolescents and adults over 14 years old (CFQ-R 14+) were compared between CF individuals with CFRD on insulin, CFRD not on insulin, impaired glucose tolerance, and normal blood glucose tolerance. The median score for the Treatment Burden domain was significantly worse for individuals with CFRD on insulin (p < 0.001) compared to the other diagnostic groups, and this association remained significant following adjustment for confounding variables. In conclusion, the additional requirement for insulin significantly contributes to treatment burden in adults with CFRD and therefore novel strategies to reduce treatment burden for this group are urgently needed.     

Prevalence of unmet palliative care needs in adults with cystic fibrosis

BackgroundPhysical and emotional burdens impair quality of life (QoL) in many adults with cystic fibrosis (CF). Palliative care (PC) improves QoL in other serious illnesses, yet the full array of palliative needs amenable to PC are unknown in CF.MethodsWe surveyed 164 adults with CF using the Supportive Care Needs Survey 34 (SCNS-34) to assess unmet PC needs across five domains, the Edmonton Symptom Assessment System (ESAS) to assess symptom burden, and the Cystic Fibrosis Questionnaire—Revised (CFQ-R) to assess CF-specific QoL. We assessed associations between SCNS-34 domain scores and respondent characteristics, including symptom burden and FEV₁.ResultsMedian age was 29 years; 56% of respondents were male. Median FEV₁ was 57% predicted. 78% of respondents reported ≥1 unmet PC need; physical and daily living (72%) and psychological (66%) needs were most prevalent. Symptom burden was correlated with all SCNS-34 domains scores, and strongly correlated with the physical (r = 0.79) and psychological (r = 0.72) domain scores. FEV₁ was moderately inversely correlated with the physical domain score (r = −0.41). Forty-four of the 45 inverse correlations between SCNS-34 domain scores and CFQ-R domain scores were significant. Patient-reported depressive and anxiety symptoms were significantly associated with higher scores in five and four SCNS-34 domains, respectively.ConclusionsAdults with CF have substantial unmet PC needs. Patient-reported symptom burden is more strongly associated with reporting unmet PC needs than FEV₁. Routine screening of unmet PC needs, using tools such as the SCNS-34, may enable CF care teams to optimize the provision of primary and specialist PC.     

Antisense oligonucleotide eluforsen is safe and improves respiratory symptoms in F508DEL cystic fibrosis

BackgroundEluforsen is an antisense oligonucleotide designed to bind to the mRNA region around the F508-encoding deletion and restore the cystic fibrosis transmembrane conductance regulator (CFTR) protein function in the airway epithelium. We assessed the safety and tolerability, pharmacokinetics and exploratory measures of efficacy of inhaled eluforsen in cystic fibrosis (CF) patients homozygous for the F508del-CFTR mutation.MethodsThis randomised, double-blind, placebo-controlled, dose escalation 1b study recruited adult CF subjects with a FEV₁ > 70% predicted in four single ascending dose cohorts and four multiple ascending dose cohorts. Primary objectives were safety and tolerability. Secondary endpoints included pharmacokinetics, percent predicted forced expiratory volume in 1 s (ppFEV₁), and Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Symptom Score (RSS).ResultsSingle and multiple doses of inhaled eluforsen up to 50 mg were safe and well tolerated. A maximum tolerated dose was not established. Systemic exposure was low in all cohorts and lung function remained stable throughout the study. Three of four eluforsen-treated groups in the MAD study demonstrated an improvement in CFQ-R RSS at end of treatment with adjusted mean change from baseline values ranging from 6.4 to 12.7 points. In comparison, there was a mean decrease of 6.5 points in the placebo group from baseline to end of treatment.ConclusionsInhaled eluforsen up to 50 mg dosed 3 times per week for 4 weeks was safe and well tolerated, showed low systemic exposure, and demonstrated improvement in CFQ-R RSS, a relevant measure of clinical benefit in CF patients.     

Screening for ADHD in adults with cystic fibrosis: Prevalence,health-related quality of life,and adherence

Background

International guidelines recommend depression and anxiety screening in individuals with cystic fibrosis (CF), but Attention-Deficit Hyperactivity Disorder (ADHD) remains understudied.

Treatment complexity in cystic fibrosis: Trends over time and associations with site-specific outcomes

BackgroundPatients with cystic fibrosis (CF) have increasing treatment complexity and high treatment burden. We describe trends in treatment complexity and evaluate its relationship with health outcomes.MethodsUsing Epidemiologic Study of Cystic Fibrosis (ESCF) data, we developed a treatment complexity score (TCS) from 37 chronic therapies and assessed change by age group (6–13, 14–17, and 18 + years) over a three year period. Differences in average site TCS were evaluated by quartiles based on FEV1, BMI, or Treatment Burden score on the Cystic Fibrosis Questionnaire-Revised (CFQ-R).ResultsTCS scores were calculated for 7252 individual patients (42% child, 16% adolescent, 43% adult) across 153 sites. In 2003, mean TCS was 11.1 for children, 11.8 for adolescents, and 12.1 for adults. In all 3 age groups, TCS increased over 3 years; the increase in TCS from 2003–2005 for children was 1.25 (95% CI 1.16–1.34), for adolescents 0.77 (0.62–0.93), and for adults 1.20 (1.08–1.31) (all P < 0.001 for trend over time). At the site level, there were no significant differences in mean TCS based on FEV₁ quartile. Mean TCS was higher in the highest BMI z-score quartile. Across all 3 versions of the CFQ-R, mean TCS was lower at sites in the highest quartiles (lowest burden) for CFQ-R treatment burden scores.ConclusionTreatment complexity was highest among adults with CF, although over 3 years, we observed a significant increase in treatment complexity in all age groups. Such increases in treatment complexity pose a challenge to patient self-management and adherence. Future research is needed to understand the associations between treatment complexity and subsequent health outcomes to reduce treatment burden and improve disease management.     

Changes in the parent cystic fibrosis questionnaire-revised (CFQ-R) with respiratory symptoms in preschool children with cystic fibrosis

IntroductionThe Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory score is a validated and widely used patient-reported outcome. This study aimed to establish changes in the score with acute respiratory events in preschool children with CF and to investigate its’ relationship with physiological outcomes.MethodsThe Parent CFQ-R, multiple breath washout test and spirometry were performed at six study visits over one year. The clinical status of participants, stable or symptomatic, was defined by the patient's physician. Linear regression and distribution-based statistical methods were used to examine the changes in the CFQ-R from the last stable visit and to investigate its relationship with physiological outcomes.ResultsThere were 272 stable and 115 symptomatic visits from 78 participants. The mean CFQ-R Respiratory score did not change between consecutive stable visits (-0.73, SD 20.4). The mean (SD) score deteriorated by 15.5 (20.7) points between stable and symptomatic visits and improved by 14.8 (20.1) points between symptomatic and stable follow-up visits. When a clinically important change is defined as 0.5SD change (10-points), the positive predictive value (PPV) was 45% and the negative predictive value (NPV) was 84%. For visits with a 10-point worsening in the CFQ-R Respiratory score and a 15% increase in LCI, the PPV was better (81%) than using either measure alone.ConclusionThe CFQ-R Respiratory score is responsive to acute respiratory events in preschool children with CF and its utility to monitor individual patients is improved when combined with LCI.     

Effect of highly effective modulator treatment on sinonasal symptoms in cystic fibrosis

BackgroundElexacaftor-tezacaftor-ivacaftor is a highly effective modulator for cystic fibrosis (CF) patients homozygous or heterozygous for F508del. Effects of the drug on sinonasal symptoms have not been studied.MethodsAdult participants were prospectively evaluated at baseline and after three months of treatment using validated questionnaires assessing sinonasal symptoms (SNOT-22) and CF-related quality of life (CFQ-R).ResultsForty-three participants completed the study; 23 were taking other CF transmembrane conductance (CFTR) modulators at the time of study participation. There was a significant improvement in mean SNOT-22 from 34.8 (29.4–40, 95% confidence interval) to 24.4 (19.9–29.0) (p = 0.000003) and in the Respiratory domain of the CFQR from 60.6 (57.1–64.1) to 83.3 (79.4–87.2) (p = 0.0000002), both achieving a minimal clinically important difference. Patients previously taking CFTR modulators experienced a greater benefit in sinonasal and respiratory symptoms.ConclusionsElexacaftor-tezacaftor-ivacaftor is associated with significant improvement in sinonasal symptoms; previous use of CFTR modulators is associated with greater benefit.     

Amikacin liposome inhalation suspension for chronic Pseudomonas aeruginosa infection in cystic fibrosis

BackgroundShortcomings of inhaled antibiotic treatments for Pseudomonas aeruginosa infection in patients with cystic fibrosis (CF) include poor drug penetration, inactivation by sputum, poor efficiency due to protective biofilm, and short residence in the lung.MethodsEligible patients with forced expiratory volume in 1 s (FEV₁) ≥25% of predicted value at screening and CF with chronic P. aeruginosa infection were randomly assigned to receive 3 treatment cycles (28 days on, 28 days off) of amikacin liposome inhalation suspension (ALIS, 590 mg QD) or tobramycin inhalation solution (TIS, 300 mg BID). The primary endpoint was noninferiority of ALIS vs TIS in change from baseline to day 168 in FEV₁ (per-protocol population). Secondary endpoints included change in respiratory symptoms by Cystic Fibrosis Questionnaire-Revised (CFQ-R).ResultsThe study was conducted February 2012 to September 2013. ALIS was noninferior to TIS (95% CI, −4.95 to 2.34) for relative change in FEV₁ (L) from baseline. The mean increases in CFQ-R score from baseline on the Respiratory Symptoms scale suggested clinically meaningful improvement in both arms at the end of treatment in cycle 1 and in the ALIS arm at the end of treatment in cycles 2 and 3; however, the changes were not statistically significant between the 2 treatment arms. Treatment-emergent adverse events (TEAEs) were reported in most patients (ALIS, 84.5%; TIS, 78.8%). Serious TEAEs occurred in 17.6% and 19.9% of patients, respectively; most were hospitalisations for infective pulmonary exacerbation of CF.ConclusionsCyclical dosing of once-daily ALIS was noninferior to cyclical twice-daily TIS in improving lung function.ClinicalTrials.gov Identifier: NCT01315678     

SF-36 scores in degenerative lumbar spine disorders: Analysis of prospective data from 451 patients

Background?When using Health-Related Quality of Life (HRQoL) in assessing outcomes of treatment, normative data for different diagnoses are needed to allow cross-comparisons of existing and future studies. We determined the SF-36 scores in patients with surgical lumbar spine problems.

Methods?This is a prospective observational study of consecutive surgical patients in one institution. In addition to SF-36 questionnaire responses, local pain, radiating pain, analgesic intake and walking ability were recorded, together with several other demographic variables. 451 patients (50% women) with median age 52 (13–88) years, operated from 1998 through 2002, were included in the study.

Results?Preoperative SF-36 scores were significantly lower than those derived from previously published material (the general population, nonspecific low back pain (LBP) patients, other samples of non-LBP patients), also with the use of normbased scoring. Sick-leave and worker's compensation seemed to affect perceived Health-Related Quality of Life (HRQoL), but smoking habits did not. Some SF-36 domains showed a possible discriminating pattern between diagnoses.

Interpretation?HRQoL reported by patients scheduled for lumbar spine surgery was much worse than for the normal population and for LBP patients. The normative SF-36 values provided may be used as a benchmark in future studies.     

Health related quality of life in cystic fibrosis: To work or not to work?

BackgroundThe present study investigated whether patients with CF who are studying or working report a better HRQoL in comparison to non-working/studying patients.Methods57 adult CF patients completed the Cystic Fibrosis Questionnaire—Revised, a CF-specific measure of HRQoL. Medical condition was quantified in terms of FEV1 % predicted, BMI, Pseudomonas aer. status, pancreatic status (PS), having an indwelling catheter device (PAC), CF Related Diabetes Mellitus and nutritional status.ResultsMean age was 26.7 years (SD 8.1), mean FEV1 % predicted was 65.09 (SD 22.18), mean BMI was 21.23 (SD 3.45). FEV1 % predicted was related to HRQoL domains Physical Functioning and General Health (r = 0.27 and 0.38 respectively, p < 0.05). A higher BMI was associated with better scores on Eating Disturbances (= fewer problems; r = 0.44, p < 0.01) and a better perception of Weight (r = 0.43, p < 0.01) and Body Image (r = 0.28, p < 0.05). Analysis of variance showed that specific domains of HRQoL were related to diabetes (Weight), taking caloric supplements (Body Image and Weight) and/or PAC (Physical Functioning, Treatment Burden, Role, Weight). Twenty-four patients worked/studied, these patients had a higher FEV1, and fewer had Pseudomonas aer. or a PAC or took high caloric supplements, compared to non-working/studying patients. After controlling for medical parameters, patients who were working/studying scored higher than non-working/studying patients on Physical Functioning, Role Functioning and Social Functioning.ConclusionsCF patients' HRQoL is related to medical status. The non-working/studying CF patients in this sample had greater disease severity and reported a lower quality of life than their working/studying peers, even after controlling for relevant medical parameters. The decision to stop work/study for CF patients is difficult and affects patients' personal, social and financial well-being.     

Predictors of health-related quality of life in neurosurgical brain tumor patients: focus on patient-centered perspective

Background

In brain tumor (BT) patients, the association between health-related quality of life (HRQoL) and psychological characteristics remains largely unknown. We evaluated the association of personality traits, clinical factors, psychological distress symptoms, and cognitive state with HRQoL in BT patients.

Methods

On admission for BT surgery, 200 patients (69 % women; age 55.8?±?14.5 years) were evaluated for HRQoL (SF-36 scale), Big-Five personality traits (Ten-Item Personality Inventory), psychological distress symptoms (Hospital Anxiety and Depression Scale or HADS), cognitive function (Mini-Mental State Examination or MMSE) and clinical characteristics, including functional status (Barthel index or BI). The most common BT diagnoses were meningioma (39 %) and high-grade glioma (18 %).

Results

Only factors significantly associated with SF-36 domains in univariable regression analyses were included in their respective multivariable models and predicted from 6 %–49 % of the total variance of SF-36 scores. Greater TIPI emotional stability score was independently associated with greater SF-36 emotional well-being (β?=?0.23, p?<?0.001) and general health (β?=?0.18, p?=?0.01) scores, and greater TIPI consciousness score, with greater SF-36 emotional well-being score (β?=?0.13, p?=?0.02). HADS-anxiety and HADS-depression scores were the strongest independent determinants of all, except physical functioning, SF-36 scores (β-values range from 0.14 to 0.56; p values?≤?0.03). BI score was the strongest independent determinant of SF-36 physical functioning score (β?=?0.36, p?<?0.001). MMSE score was associated with all but emotional well-being and social functioning SF-36 scores.

Conclusions

Consciousness and emotional stability should be considered important personality-related determinants of HRQoL in BT patients. Psychological distress, functional disability, and cognitive impairment are also important predictors of HRQoL.     

The study of cystic fibrosis transmembrane conductance regulator gene mutations in a group of patients from Romania

BackgroundCystic fibrosis (CF) is produced by mutations in the Cystic Fibrosis Transmembrane Conductance Regulator Gene (CFTR) gene.MethodsOne hundred twenty eight patients with CF were analysed for mutations in the CFTR gene in order to establish the frequency of CF mutations in the Romanian population. The chief methods of analysis were polymerase chain reaction (PCR) of DNA extracted from blood and electrophoresis of PCR products.ResultsThe frequency of F508del in CF chromosomes from Romania is approximately 56.3%. Other frequent mutations noted are: G542X (3.9%), W1282X (2.3%), and CFTRdele2,3(21 kb)(1.6%); the remaining mutations have frequencies below 1%.ConclusionsWe consider that the frequency of F508del in CF patients from Romania is higher than in previous reports, reaching 56.3%, probably owing to more rigorous selection of patients for genetic testing, allowing improved calculation of mutation frequencies.     

Twelve-month self-reported quality of life after retropubic radical prostatectomy: a prospective study with Rand 36-Item Health Survey (Short Form-36)

OBJECTIVES: To evaluate the health-related quality of life (HRQoL) in patients undergoing retropubic radical prostatectomy (RRP) for clinically localized prostate cancer. PATIENTS AND METHODS: From February 2002 to September 2003 all patients undergoing RRP in our department were invited to participate in the study; the data from 75 of them comprised the present analysis. For evaluating HRQoL the RAND 36-Item Health Survey (SF-36) was used. RESULTS: Comparing the baseline scores of the SF-36 domains to those at 3, 6 and 12 months, there was a statistically significant difference in 'physical function', 'role limitations due to physical health problems', 'role limitations due to emotional problems', and 'energy/fatigue'. There were no statistically significant changes in the follow-up values for the other scales of the questionnaire. However, the baseline scores overlapped the 12-month follow-up values for all the SF-36 scales. The mean SF-36 scores reported by incontinent patients were lower than those of the continent patients, although this trend was not statistically significant. At the 12-month follow-up some variables were independent predictors of lower mean scores of some SF-36 scales, i.e. age >65 years, education level less than secondary school, pathological extracapsular extension of cancer and erectile dysfunction. CONCLUSION: At 1 year after RRP, HRQoL levels in each of the SF-36 domains overlapped those of the baseline in >80% of patients. The age, educational level of patients, local extension of the tumour, and erectile dysfunction could significantly affect the HRQoL scores.     

Breathlessness catastrophizing relates to poorer quality of life in adults with cystic fibrosis

Background

Cystic Fibrosis (CF) is the most common genetic disease affecting children and young adults in Canada. It is a multi-system disease, however lung disease is largely responsible for mortality. Treatment advances have resulted in increased life expectancy and a subsequent need to better understand psychosocial issues associated with quality of life in adults living with CF. Emerging research suggests that anxiety and depression are related to poorer health-related quality of life (HRQoL) in CF patients, but there is little research examining cognitive processes, such as breathlessness catastrophizing. The present study addresses this gap in the literature. Methods: Participants in this study are based on a convenience sample of patients recruited during their regular CF clinic appointments at a tertiary care center. Forty-five adults (M_age?=?30.73?years) completed measures of lung function, depression, anxiety, pain, breathlessness catastrophizing, and HRQoL at one time point. Results: Results of a hierarchical multiple regression indicate that increased breathlessness catastrophizing was related to poorer HRQoL, after controlling for lung function, depression, anxiety, and pain (p?<?.05). Depression, pain, and breathlessness catastrophizing all emerged as significant unique predictors of HRQoL. Conclusions: Breathlessness catastrophizing is a potential target for clinical intervention and might impact HRQoL. Further research on breathlessness catastrophizing in CF is warranted including longitudinal studies to examine the mechanisms by which breathlessness catastrophizing relates to HRQoL and treatment outcomes in CF.     

“Il faut continuer à poser des questions” patient reported outcome measures in cystic fibrosis: An anthropological perspective

BackgroundPeople with cystic fibrosis (pwCF) are central in the development of patient-led assessment tools. Qualitative analysis of a frequently used CF-specific patient-reported outcome measure (PROM) sought patient recommendations for development of a new quality of life (QoL) tool.MethodsWe performed an inventory of PROMs, symptom-report and QoL tools used in clinical trials within the European Cystic Fibrosis Society Clinical Trial Network (ECFS-CTN) and in routine clinical practice among Cystic Fibrosis Europe and ECFS members. A qualitative study using cognitive interviews with pwCF and their caregivers reviewed the Cystic Fibrosis Questionnaire (CFQ), the French initial form of the Cystic Fibrosis Questionnaire-Revised (CFQ-R).ResultsSurvey results from 33 countries revealed over 70 tools used in routine clinical practice, utilized by clinical specialists (n=124), pwCF/parents/carers (n=49) and other allied health professionals (n=60). The CFQ-R was the main PROM used in clinical trials. The qualitative study enrolled 99 pwCF, 6 to 11 years (n=31); 12 to 18 years (n=38); >18 years (n=30) and 26 parents. Inductive thematic analysis based on the CFQ, revealed 19 key themes. Themes common across all cohorts included burden of treatment, impact of disease on day-to-day life, relationships/family, stress/mood, and nutrition. Themes unique to individual groups included, treatment when not symptomatic for the paediatric group; education/studies and planning for the future for adolescents, impact of anxiety and depression on day-to-day life for adults, and for parents, questions addressing anxiety and their role as carers.ConclusionsPatient-centeredness is paramount in development of an up-to-date PROM in the era of novel therapies.     

A prospective analysis of health-related quality of life in intermediate and high-risk prostate cancer patients managed with intensity modulated radiation therapy,with vs. without hormonal therapy

IntroductionCombined radiotherapy and hormonal treatment are recommended for intermediate- and high-risk prostate cancer (CaP). This study compared the long-term effects on health-related quality of life (HRQoL) of intermediate- and high-risk CaP patients managed with radiation therapy (RT) with vs. without hormone therapy (HT).MethodsPatients with intermediate- and high-risk CaP enrolled in the Center for Prostate Disease Research diagnosed from 2007 to 2017 were included. EPIC and SF-36 questionnaires were completed and HRQoL scores were compared for patients receiving RT vs. RT + HT at baseline (pretreatment), 6, 12, 24, 36, 48, and 60 months after CaP diagnosis. Longitudinal patterns of change in HRQoL were modeled using linear regression models, adjusting for baseline HRQoL, age at CaP diagnosis, race, comorbidities, National Comprehensive Cancer Network (NCCN) risk stratum, time to treatment, and follow-up time.ResultsOf 164 patients, 93 (56.7%) received RT alone and 71 (43.3%) received RT + HT. Both groups reported comparable baseline HRQoL. Patients receiving RT+HT were more likely to be NCCN high risk as compared to those receiving only RT. The RT + HT patients experienced worse sexual function, hormonal function, and hormonal bother than those who only received RT; however, HRQoL recovered over time for the RT + HT group. No significant differences were observed between groups in urinary and bowel domains or SF-36 mental and physical scores.ConclusionCombined RT + HT treatment was associated with temporary lower scores in sexual and hormonal HRQoL compared with RT only. Intermediate- and high-risk CaP patients should be counseled about the possible declines in HRQoL associated with HT.