Phase II studies of nebulised Arikace in CF patients with Pseudomonas aeruginosa infection.

RATIONALE: Arikace is a liposomal amikacin preparation for aerosol delivery with potent Pseudomonas aeruginosa killing and prolonged lung deposition. OBJECTIVES: To examine the safety and efficacy of 28 days of once-daily Arikace in cystic fibrosis (CF) patients chronically infected with P aeruginosa. METHODS: 105 subjects were evaluated in double-blind, placebo-controlled studies. Subjects were randomised to once-daily Arikace (70, 140, 280 and 560 mg; n=7, 5, 21 and 36 subjects) or placebo (n=36) for 28 days. Primary outcomes included safety and tolerability. Secondary outcomes included lung function (forced expiratory volume at one second (FEV1)), P aeruginosa density in sputum, and the Cystic Fibrosis Quality of Life Questionnaire-Revised (CFQ-R). RESULTS: The adverse event profile was similar among Arikace and placebo subjects. The relative change in FEV1 was higher in the 560 mg dose group at day 28 (p=0.033) and at day 56 (28 days post-treatment, 0.093L±0.203 vs -0.032L±0.119; p=0.003) versus placebo. Sputum P aeruginosa density decreased >1 log in the 560 mg group versus placebo (days 14, 28 and 35; p=0.021). The Respiratory Domain of the CFQ-R increased by the Minimal Clinically Important Difference (MCID) in 67% of Arikace subjects (560 mg) versus 36% of placebo (p=0.006), and correlated with FEV1 improvements at days 14, 28 and 42 (p<0.05). An open-label extension (560 mg Arikace) for 28 days followed by 56 days off over six cycles confirmed durable improvements in lung function and sputum P aeruginosa density (n=49). CONCLUSIONS: Once-daily Arikace demonstrated acute tolerability, safety, biologic activity and efficacy in patients with CF with P aeruginosa infection.

Supporting cystic fibrosis disease management during adolescence: the role of family and friends.

BACKGROUND: Successful management of a complex disease, such as cystic fibrosis (CF), requires support from family and friends; however, few studies have examined social support in adolescents with CF. METHODS: Twenty-four adolescents were interviewed about the support they receive from family and friends. Interviews were transcribed, coded and analysed to determine the types, frequency and perceived supportiveness of specific behaviours. RESULTS: Both family and friends provided treatment-related support to adolescents with CF. Family provided more tangible support and friends provided more relational support. Adolescents also reported that the manner, timing and context of support behaviours influenced their perceptions of the behaviours' supportiveness. A subset of adolescents (17%) chose not to disclose their diagnosis to their friends. CONCLUSIONS: The provision of support appears to be distinct from adolescent's perception of support and there may be some behaviours, such as treatment reminders, that are important to disease management but viewed as less supportive by adolescents. Facilitating increased social support holds the promise of improving disease management during adolescents, but more work is need to understand which aspects of support are related to management outcomes.

Associations between adherence, depressive symptoms and health-related quality of life in young adults with cystic fibrosis.

BACKGROUND: Cystic fibrosis (CF) is a life shortening disease, however prognosis has improved and the adult population is growing. Most adults with cystic fibrosis live independent lives and balance the demands of work and family life with a significant treatment burden. The aim of this study was to examine the relationships among treatment adherence, symptoms of depression and health-related quality of life (HRQoL) in a population of young adults with CF. METHODS: We administered three standardized questionnaires to 67 patients with CF aged 18-30 years; Morisky Medication Adherence Scale, Major Depression Inventory, and Cystic Fibrosis Questionnaire-Revised. RESULTS: There was a response rate of 77 % and a majority of the young adults (84 %) were employed or in an education program. Most participants (74 %) reported low adherence to medications. One third (32.8 %) of the participants reported symptoms of depression. HRQoL scores were especially low on Vitality and Treatment Burden, and symptoms of depression were associated with low HRQoL scores (p < 0.01) with medium to large deficits across on all HRQoL domains (Cohen's d 0.60-1.72) except for the domain treatment burden. High depression symptom scores were associated with low adherence (r = -0.412, p < 0.001). CONCLUSIONS: Despite improved physical health, many patients with CF report poor adherence, as well as impaired mental wellbeing and HRQoL. Thus, more attention to mental health issues is needed.

Erratum to: Associations between adherence, depressive symptoms and health-related quality of life in young adults with cystic fibrosis.

[This corrects the article DOI: 10.1186/s40064-016-2862-5.].

Depression and anxiety symptoms in Spanish adult patients with cystic fibrosis: associations with health-related quality of life.

BACKGROUND: Cystic fibrosis (CF) is a chronic disease with an impact on the quality of life. Self-reported symptoms of depression and anxiety were assessed in the Spanish cohort of the International Epidemiological Study on Depression and Anxiety in patients with CF (International Depression-Anxiety Epidemiological Study) and their relationship with health status and health-related quality of life (HRQoL) was evaluated. METHODS: This cross-sectional study recruited adult patients with CF at 10 Spanish centers. Patients completed the Hospital Anxiety and Depression Scale (HADS) and the Revised Cystic Fibrosis Questionnaire. Demographic and health data were recorded from medical charts. Logistic regression was used to determine the predictors of elevated symptoms of depression and anxiety (HADS≥8). RESULTS: Of the 336 participants recruited (mean age, 28.1years; 48.2% women), 41 (12.2%) had elevated depression-related scores, and 100 (29.7%) had elevated anxiety-related scores (HADS≥8). After adjusting for confounders, only less education, intravenous antibiotics, psychiatric medications and psychotherapy were significantly associated with elevated psychological symptoms. Specifically, regardless of lung function, patients who were depressed or anxious reported worse HRQoL. CONCLUSIONS: The prevalence of elevated symptoms of depression and anxiety was high in Spanish adult patients with CF, and these symptoms were associated with a decreased HRQoL.

Cystic Fibrosis Foundation and European Cystic Fibrosis Society Survey of cystic fibrosis mental health care delivery.

BACKGROUND: Psychological morbidity in individuals with cystic fibrosis (CF) and their caregivers is common. The Cystic Fibrosis Foundation (CFF) and European Cystic Fibrosis Society (ECFS) Guidelines Committee on Mental Health sought the views of CF health care professionals concerning mental health care delivery. METHODS: An online survey which focused on the current provision and barriers to mental health care was distributed to CF health care professionals. RESULTS: Of the 1454 respondents, many did not have a colleague trained in mental health issues and 20% had no one on their team whose primary role was focused on assessing or treating these issues. Insufficient resources and a lack of competency were reported in relation to mental health referrals. Seventy-three percent of respondents had no experience with mental health screening. Of those who did, they utilized 48 different, validated scales. CONCLUSIONS: These data have informed the decision-making, dissemination and implementation strategies of the Mental Health Guidelines Committee sponsored by the CFF and ECFS.

Role strain in couples with and without a child with a chronic illness: associations with marital satisfaction, intimacy, and daily mood.

This study examined marital role strain in 33 couples caring for a child with cystic fibrosis (CF) and 33 couples with a healthy child. The relationship between role strain, marital satisfaction, and psychological distress was tested. Couples completed a structured interview, questionnaires, a card sort procedure, and 4 daily diaries assessing activities and mood. Couples in the CF versus comparison group reported greater role strain on measures of role conflict, child-care tasks, and exchanges of affection. They also spent less time in recreational activities, but no reliable group differences were found in marital satisfaction or depression. Regression analyses indicated that role strain was related to marital satisfaction and depression and that recreation time accounted for additional variance. Path analysis suggested that recreation mediated the negative relationship between role strain and distress. The importance of using a contextual, process-oriented approach is discussed.

Assertiveness training for disabled adults in wheelchairs: self-report, role-play, and activity pattern outcomes.

Thirty-four physically disabled adults participated in an 11-week assertiveness training (AT) program. Ss were randomly assigned to either AT or a waiting-list (WL) condition. Each was asked to complete three self-report measures, a role-play test, and a social and recreational activity diary. The AT Ss showed significant improvements on both self-reported assertiveness and role-play performance from pre- to posttreatment, whereas WL controls showed no changes on these measures. No significant changes in frequency of social or recreational activities were found at posttest for either AT or control Ss. A mixed pattern of results was shown at 6-month follow-up. Posttest gains were maintained across all self-report measures of assertiveness and acceptance of disability, whereas role-play performance approached baseline levels at 6-month follow-up. Overall results support the use of AT in enhancing perceived social efficacy and interpersonal skill of physically disabled adults in wheelchairs.

Chronic parenting stress: moderating versus mediating effects of social support.

The present study contrasted the widely cited "buffer" model of social support with an alternative mediator model. Distinctions were drawn between the functions of social support under chronic vs. acute stress conditions, and between situation-specific stressors and major life events. Ongoing parenting stress was assessed in 96 mothers of deaf children and 118 matched controls. Tests of the competing models showed no moderating effects for social support. However, path analyses suggested that social support mediated the relationship between stressors and outcomes. Chronic parenting stress was associated with lowered perceptions of emotional support, and greater symptoms of depression and anxiety. Furthermore, parenting stress accounted for a substantial proportion of the variance in psychological distress scores in contrast to life event stress, which was only weakly related to psychological outcomes. The implications of mediational models for understanding adaptation to chronic stress are discussed.

Psychological interventions for cystic fibrosis.

BACKGROUND: As survival estimates for cystic fibrosis (CF) steadily increase long-term management has become an important focus for intervention. Psychological interventions are largely concerned with emotional and social adjustments, adherence to treatment and quality of life, however no systematic review of such interventions has been undertaken for this disease. OBJECTIVES: To describe the extent and quality of effectiveness studies utilising psychological interventions for CF and whether these interventions provide significant psychosocial and physical benefits in addition to standard care. SEARCH STRATEGY: Relevant trials were identified from searches of Ovid MEDLINE, the Cochrane trial registers for CF and Depression, Anxiety and Neurosis Groups and PsychINFO; unpublished trials were located through professional networks and Listserves. Most recent search: April 2003. SELECTION CRITERIA: This review included RCTs and quasi-randomised trials. Study participants were children and adults diagnosed with CF, and their immediate family members. Psychological interventions were from a broad range of modalities and outcomes were primarily psychosocial, although physical outcomes and cost effectiveness were also considered. DATA COLLECTION AND ANALYSIS: Two reviewers independently selected relevant trials and assessed their methodological quality. For binary and continuous outcomes a pooled estimate of treatment effect was calculated for each outcome. MAIN RESULTS: This review is based on the findings of eight studies, representing data from a total of 358 participants. Studies fell into four conceptually similar groups: (1) gene pre-test education counselling for relatives of those with CF (one study); (2) biofeedback, massage and music therapy to assist physiotherapy (three studies); (3) behavioural intervention to improve dietary intake in children up to 12 years (three studies); and (4) self-administration of treatments to improve quality of life in adults (one study). Interventions were largely educational or behavioural, targeted at specific treatment concerns during the chronic phase. No completed studies concentrating on complex treatment approaches were found. There is some evidence that behavioural interventions can improve emotional outcomes in people with CF and their carers. There was no consistent effect on lung function although one small study showed that biofeedback assisted breathing re-training was associated with improvement in some measures of spirometric lung function. Insufficient evidence is available at this point for interventions aimed at other aspects of the disease process. REVIEWER'S CONCLUSIONS: Multicentre approaches are required to increase the sample sizes of studies in the psychosocial field and to enhance the power and precision of the findings. This has consequent implications for funding.

Audition and visual attention: the developmental trajectory in deaf and hearing populations.

This study examined the development of visual attention in 5- to 13-year-olds who differed in their access to sound. Hearing children, deaf children with cochlear implants, and deaf children without implants participated in a task in which they were to respond to some visual signals and not others. The results of Experiment I indicated that the timing of developmental changes in visual selective attention was similar for all 3 groups, occurring around 8 years. The magnitude of age-related change differed among groups; hearing children and older deaf children using a cochlear implant reached higher levels of performance with age than did deaf children without enhanced access to sound. The results of Experiment 2 suggest that the developmental differences between deaf children with and without cochlear implants begin prior to 8 years and may be related to their use of environmental sounds to organize visual attention.

Aztreonam for inhalation solution (AZLI) in patients with cystic fibrosis, mild lung impairment, and P. aeruginosa.

BACKGROUND: Previous aztreonam for inhalation solution (AZLI) studies included patients with cystic fibrosis, Pseudomonas aeruginosa (PA) airway infection, and forced expiratory volume in 1s (FEV(1)) 25% to 75% predicted. This double-blind, multicenter, randomized, placebo-controlled trial enrolled patients (≥6 years) with FEV(1)>75% predicted. METHODS: AZLI 75 mg (n=76) or placebo (n=81) was administered 3-times daily for 28days with a 14-day follow-up. RESULTS: Day 28 treatment effects were 1.8points for CFQ-R-Respiratory Symptoms Scale (95%CI: -2.8, 6.4; p=0.443; primary endpoint); -1.2 for log(10) sputum PA colony-forming units (p=0.016; favoring AZLI), and 2.7% for relative FEV(1)% predicted (p=0.021; favoring AZLI). Treatment effects favoring AZLI were larger for patients with baseline FEV(1) <90% predicted compared to ≥90% predicted. AZLI was well-tolerated. CONCLUSIONS: Effects on respiratory symptoms were modest; however, FEV(1) improvements and bacterial density reductions support a possible role for AZLI in these relatively healthy patients.

Measurement of quality of life in cystic fibrosis.

Considerable progress has been made over the past 2 decades in defining and measuring health-related quality of life (QOL), and there is a growing recognition that these measures provide unique information about the impact of a chronic illness and its treatment. For patients with cystic fibrosis (CF), health-related QOL measures enable researchers and clinicians to determine the effects of clinical interventions on several aspects of daily living (psychological, emotional, social) that are not reflected in typical health indicators, eg, pulmonary functioning scores. Three types of health-related QOL measures have been developed: 1) utility measures, 2) health profiles, and 3) disease-specific measures. The purpose of each type of health-related QOL measure is described, and its application to patients with CF is reviewed. Although important descriptive information has been obtained from utility measures, eg, the Quality of Well-Being Scale, and health profiles, eg, the Nottingham Health Profile, both of these instruments have serious limitations. Disease-specific measures, similar to those developed for children and adults with asthma, seem to hold the greatest promise for advancing our understanding of the impact of CF on daily life and for evaluating the effectiveness of new clinical interventions. The Cystic Fibrosis Questionnaire is the only published disease-specific measure of health-related QOL for children, adolescents, and adults with CF. Its psychometric properties are briefly reviewed, and directions for future research are suggested.

Predictors of cochlear implant use in children.

The purpose of this study was to identify predictors of effective use of a cochlear implant in a sample of 29 profoundly deaf children, who had been using their devices an average of 2 years. The relationship between the variables of age of onset of deafness, etiology, and communication mode measured prior to implant surgery were correlated with a behavioral measure of cochlear implant use in everyday situations. In addition, in a subset of the full sample, nonverbal intelligence subtest scores calculated prior to implant surgery were evaluated for their contribution to subsequent use of the cochlear implant. The results suggested that communication mode, time using the implant device, and performance on two subsets from the Wechsler Intelligence Scale for Children-Revised (WISC-R) accounted for substantial proportions of the variance in parent ratings of implant usage. The implications of these findings, as well as directions for future research, are discussed.

Differential treatment of siblings: interview and diary analyses comparing two family contexts.

We assessed parental differential treatment of siblings (maternal time, affection, discipline) in normal and high-risk families. Differential treatment was measured using home interviews, nightly phone ratings, and daily diaries tracking how mothers spent their time. Subjects were 40 mothers of toddlers (average age 2 years) and preschoolers (average age 4 1/2 years): half were caring for a younger child with a chronic illness (i.e., cystic fibrosis), and half were caring for two healthy children. Little evidence of parental differential treatment was found in the home or phone interview data. However, on the diary variables, both quantitative and qualitative differences in parental treatment were found in cystic fibrosis (CF) versus comparison families. Specifically, mothers spent more individual time with younger, chronically ill children in play and mealtime activities than with their older, healthy siblings. Further, mothers in the CF group rated time spent with older children as significantly more negative than time spent with younger children. Convergence between measures of differential treatment and advantages of using a high-risk comparison approach are discussed.

Follow-up difficulty with substance abusers: predictors of time to locate and relationship to outcome.

A sample of 76 alcoholics and 31 drug abusers who had been inpatients at a Veterans Administration Medical Center were followed up 6 months after termination of treatment. Of this population, 100 (93.5%) were located, and additional data were recorded as to both the length of time required for location and the sources which proved useful in the process. Two-thirds of the sample were located within 1 month and most before the fourth month. Multivariate analyses revealed that criminal history, longer time in treatment, and previous treatment each were independent predictors of difficulty in location. Personal addresses provided by the subjects at intake were crucial in the tracking process, as well as female relatives named by addicts. The time to locate a subject was not generally related to outcome. Employed subjects were somewhat harder to find, and those drinking daily-or not at all-were easier to locate.

Cochlear implants in children: a study of parental stress and adjustment.

A contextual model of stress was employed to examine the impact of cochlear implantation on parents' levels of stress and psychological adjustment. This model provided a framework for identifying stressors tied directly to the situation, and pointed to important life roles that may be altered. Twenty-four mothers of children implanted with cochlear implants completed a series of questionnaires assessing stressors related to daily parenting tasks, time demands, and childhood deafness. Standardized measures of depression, anxiety, and somatic complaints were also administered. The results provided preliminary evidence that parents of children receiving cochlear implants experience higher levels of stress and poorer psychological adjustment than parents of normally hearing children. Greater parenting stress and emotional distress were reported by parents of children using multichannel as opposed to single-channel devices, with little evidence that this was related strongly to length of time using the devices. Finally, in analyses controlling for type of device and length of use, parenting stress as measured in the current study, accounted for substantial proportions of the variance in psychological distress. The implications of these findings, as well as directions for future research, are discussed.

Factors influencing elementary school teachers' ratings of ADHD and ODD behaviors.

Examined factors that influence teachers' ratings of children with either attention deficit hyperactivity disorder (ADHD) or oppositional defiant disorder (ODD). 105 teachers watched 2 videotapes--1 depicting a normal child and the other a child with either ADHD or ODD--and rated each child using 2 different questionnaires. Results indicated that teachers accurately rated the child on the ADHD versus ODD tape as having significantly more inattention and hyperactivity but significantly less oppositionality. However, effect sizes indicated the presence of a unidirectional, negative halo effect of oppositional behaviors on ratings of hyperactivity and inattention. Teachers appeared less biased in their judgments when using a well-operationalized rating scale. Finally, knowledge, education, and experience with children with ADHD generally had no effect on the accuracy of teachers' ratings.