No abstract

A significant increase in life expectancy in successive birth cohorts of people with cystic fibrosis (CF) is a result of more effective treatment for the disease. It is also now widely recognized that outcomes for patients cared for in specialist CF Centres are better than for those who are not. Key to the effectiveness of the specialist CF Centre is the multidisciplinary team (MDT), which should include consultants, clinical nurse specialist, microbiologist, physiotherapist, dietitian, pharmacist, clinical psychologist, social worker, clinical geneticist and allied healthcare professionals, all of whom should be experienced in CF care. Members of the MDT are also expected to keep up to date with developments in CF through continued professional development, attendance at conferences, auditing and involvement in research. Specialists CF Centres should also network with other Centres both nationally and internationally, and feed Centre data to registries in order to further the understanding of the disease. This paper provides a framework for the specialist CF Centre, including the organisation of the Centre and the individual roles of MDT members, as well as highlighting the value of CF organisations and disease registries.

Preliminary 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.

RationaleThe lung clearance index (LCI) is a promising endpoint for use in cystic fibrosis (CF) clinical trials, but correlations with validated clinical endpoints have not yet been established. Objective This study aimed to demonstrate that, in young patients with CF, baseline LCI predicts subsequent pulmonary exacerbation (PE) and correlates with the respiratory domain of the CF Questionnaire-Revised (CFQ-R resp ). Methods Baseline LCI, forced expiratory volume in 1 s (FEV 1 ), CFQ-R resp and PEs over the subsequent year were prospectively recorded in 63 patients aged 5-19 years. The ability of baseline LCI to predict PE was assessed using negative binomial regression models and KaplanMeier plots. Results Twenty-six patients (41%) experienced 48 PEs. Baseline LCI and FEV 1 were predictors of PE. Compared with the quartile with the lowest LCI, the annual PE rate in increasing LCI quartiles was 2.9 (95% CI 0.5 to 16.5, p=0.238), 5.4 (95% CI 1.0 to 29.0, p=0.045) and 13.6 (95% CI 2.8 to 67.1, p=0.001). Similarly, time to first PE decreased with worsening LCI quartiles (log-rank test for trend, p<0.001). Furthermore, LCI correlated with CFQ-R resp (r=−0.43, p<0.001). In the subgroup of 53 patients with normal FEV 1 , LCI was a predictor of PE. In this subgroup, LCI also correlated with CFQ-R resp (r=−0.282, p=0.043). Conclusions Baseline LCI predicts PE in young patients with CF and correlates with CFQ-R resp , a validated patient-reported outcome, even in the subgroup with normal FEV 1 . These data further support the use of LCI as a surrogate outcome measure in CF clinical trials.

A total of 21 healthy siblings were interviewed about their experiences when a brother or sister is diagnosed with cancer. Information about the illness, opinions about who should inform siblings, social support, specific worries and worst memories were studied, as well as perceptions of any differences in the way they were treated by their parents. A short General Impact Scale was developed to assess the extent to which the siblings felt their lives had been disrupted by the illness. Results on these measures were compared with scores on four dimensions of the Sibling Perception Questionnaire (Carpenter & Sahler 1991). Siblings who reported some positive effects as a consequence of the illness (they had become more empathic toward others, or valued life more) perceived their interpersonal relations to be more negatively affected, i.e. there were greater difficulties in their relationships with others especially their parents. Higher scores on communication were related to less of an impact of the illness on life generally. However, these siblings also reported heightened concerns that their brother or sister might die. The data suggested that siblings should have opportunities to talk about implications of the disease, especially worries about death, and more efforts should be made to prepare siblings for visits to hospital and seeing the sick child.

Highlights COVID-19 had an impact on the emotional well-being of patients and parents of children with CF. Many patients report more sadness, discouragement, feelings of helplessness, greater criticism of others, perceptions of deterioration of the situation and difficulty with adhering to their routine. Symptoms of posttraumatic stress were elevated, which is worrying, especially if this psychological impact is sustained. Psychological follow-up is necessary to prevent the development of post-traumatic stress disorders. Adult patients with mild CF disease report more worries and stress compared to those with more severe disease. Contingency measures, family responsibilities, fear or deterioration and/or fear of losing control, may explain this unanticipated result. Further research is warranted. Many patients and parents of children with CF report positive changes in home treatment, including ‘eating extra and healthier’, ‘improvement in the quality of nebulizing’ and ‘better physiotherapy’. The challenge for both patients, parents and CF teams, is how to maintain these positive changes over time and promote permanence in resilience. It is important to contextualize the information patients and parents receive about COVID-19 from the media and to support them in balancing perceived risk and true risk. There is a crucial role for multidisciplinary teams, which can pro-actively connect with, inform and support the patients and parents.

CF 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.

The development of a scale to measure perceived illness experience in young people with cancer is described. Areas of concern were first identified through semistructured interviews conducted with children and adolescents. As a result, 78 items were generated to cover the main areas identified (physical appearance, interference with activity, peer rejection, integration in school, manipulation, parental behaviour, disclosure, preoccupation with illness, and impact of treatment). These items were rated (on five point scales) by 41 patients (mean age 14-6 years) and 35 oftheir parents. Measures of physical functioning (symptoms, functional disability, and restrictions) and psychological functioning (symptoms) were included for validation purposes. Test-retest reliability was calculated on the basis of ratings made by a subsample of parents on two separate occasions. A 34 item scale was constructed with four items in each of the areas identified above, except for physical appearance (n= 2). The scale has adequate internal reliability and validity. There were significant correlations between parents and their children on all subscales except for illness disclosure and impact of treatment, suggesting that parents may be less reliable informants for their children in these contexts. The scale has potential use in clinical contexts, in evaluating the psychosocial impact of different treatment regimens, and as an outcome measure in intervention work. (Arch Dis Child 1995; 72: 302-307)