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Newcombe P.A.,University of Queensland | Sheffield J.K.,University of Queensland | Chang A.B.,Queensland Childrens Medical Research Institute
Chest | Year: 2011

Background: The Parent Cough-Specific Quality-of-Life questionnaire (PC-QOL) has relevance and clinical utility as a cough-specific QOL measure for pediatric use. Its validity has been demonstrated. This study sought to determine the minimally important difference (MID) for the PC-QOL completed by parents of young children with chronic cough. Method: Thirty-four children (22 boys, 12 girls; median age, 26.5 months; interquartile range, 17.3-38.8 months) and their mothers participated. Mothers completed a cough-related measure (verbal category descriptive score) and the PC-QOL on two occasions separated by 2 to 3 weeks. Two approaches were used to calculate MID. Results: Distribution-based approaches to estimating MID resulted in ranges of 0.50 to 0.78 (effect size method), 0.30 to 0.48 (SE of measurement method), and 0.60 to 0.69 (one-half SD method) for PC-QOL overall and domain scales. Based on verbal category descriptive score change, an anchor-based approach resulted in an MID estimate of 0.9 for overall PC-QOL change and ranged from 0.71 to 0.95 for individual domain PC-QOL change. Conclusion: An MID for the PC-QOL of 0.9 is recommended in interpreting health status change in children with chronic cough and will aid clinicians and researchers in interpreting health-related QOL changes following treatments and clinical trials. © 2011 American College of Chest Physicians.

Newcombe P.A.,University of Queensland | Sheffield J.K.,University of Queensland | Chang A.B.,Queensland Childrens Medical Research Institute
Journal of Allergy and Clinical Immunology | Year: 2013

Background: Cough is a distressing symptom and has a significant effect on many children and their families. Quality-of-life (QOL) measures provide important outcome indicators for clinicians and aid in evaluating the efficacy of interventions. Objective: The aim of this study was to develop and validate a short cough-specific QOL questionnaire for pediatric use. Method: Two sources provided data to establish a shortened version of the Parent Cough-specific Quality of Life (PC-QOL) questionnaire. The first (n = 240, 137 boys; median age, 29 months [interquartile range, 14-64 months]) was used for development and cross-validation. Stepwise regression was used to select the reduced set of items, and analyses of reliability, validity, and minimally important differences determined psychometric strength and sensitivity to change. The second independent dataset (n = 320, 190 boys; median age, 39.5 months [interquartile range, 16-77 months]) was used as a confirmatory sample. Results: Forward-step regression identified 8 items that accounted for 95% of the variance in the full-scale PC-QOL questionnaire. This shortened version (PC-QOL-8) was internally consistent (Cronbach α = 0.84), had good test-retest reliability (intraclass correlation coefficient = 0.66), and demonstrated strong validity (significant correlations with a cough verbal category descriptor score, cough visual analog scale, and subscales of the Short Form-12 General Health scale, the Pediatric Quality of Life Inventory, and the Depression, Anxiety, and Stress Scale). The reduced scale was responsive to change, and a minimally important difference of 0.9 was suggested. These findings were confirmed with the second dataset. Conclusion: The PC-QOL-8 questionnaire is a short, reliable, and valid instrument for assessing the effect of a child's chronic cough. It demonstrated sensitivity to change, and its length and psychometric properties should enhance its potential uptake and routine use in clinical practice and research. © 2013 American Academy of Allergy, Asthma & Immunology.

Chang A.B.,Charles Darwin University | Chang A.B.,Queensland Childrens Medical Research Institute
Expert Review of Respiratory Medicine | Year: 2011

Studies on cough have come a long way but many shortfalls still exist. These shortfalls can be attributed to: the lack of randomized controlled studies with a focus on cough; studies not using robust cough outcome measures, poor definition of target groups in studies and guidelines, the lack of safe and efficacious treatments; difficulty in defining etiological factors, and the lack of data on the predictors of response to therapies for cough dominant etiologies. Addressing shortfalls in cough therapy that focuses on improving the lives of people with cough requires a systematic approach that includes better medications, high quality studies, improved multidisciplinary guidelines and education (of both health professionals and patients). To achieve new cough therapeutics requires an improved understanding of cough in humans (i.e., not just in animals). Development of new medications without substantial adverse events is long awaited for cough. © 2011 Expert Reviews Ltd.

Moore A.S.,Queensland Childrens Medical Research Institute | Moore A.S.,Institute of Cancer Research and Royal Marsden | Kearns P.R.,University of Birmingham | Knapper S.,University of Cardiff | And 2 more authors.
Leukemia | Year: 2013

Significant improvements in survival for children with acute myeloid leukaemia (AML) have been made over the past three decades, with overall survival rates now approximately 60-70%. However, these gains can be largely attributed to more intensive use of conventional cytotoxics made possible by advances in supportive care, and although over 90% of children achieve remission with frontline therapy, approximately one third in current protocols relapse. Furthermore, late effects of therapy cause significant morbidity for many survivors. Novel therapies are therefore desperately needed. Early-phase paediatric trials of several new agents such as clofarabine, sorafenib and gemtuzumab ozogamicin have shown encouraging results in recent years. Due to the relatively low incidence of AML in childhood, the success of paediatric early-phase clinical trials is largely dependent upon collaborative clinical trial design by international cooperative study groups. Successfully incorporating novel therapies into frontline therapy remains a challenge, but the potential for significant improvement in the duration and quality of survival for children with AML is high. © 2013 Macmillan Publishers Limited.

Plant B.J.,University College Cork | Goss C.H.,University of Washington | Plant W.D.,University College Cork | Bell S.C.,The Prince Charles Hospital | Bell S.C.,Queensland Childrens Medical Research Institute
The Lancet Respiratory Medicine | Year: 2013

Several key advances have been made in the treatment and management of people with cystic fibrosis in the past two decades. Substantial improvements in survival have resulted from the introduction of key drugs, coordinated care packages, improved nutritional support, and the intensive use of antibiotics. The age profile of people with cystic fibrosis has changed greatly during this time-some countries now have more adult than paediatric patients with the disease. With their increasing age and more advanced lung disease, several important sequelae (both pulmonary and extrapulmonary) occur in these adult patients including pulmonary disease, cystic fibrosis-related diabetes, renal disease, metabolic bone disease, cancers, drug allergies and toxic effects, and complications associated with lung transplantation. © 2013 Elsevier Ltd.

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