Queensland Childrens Medical Research Institute

Brisbane, Australia

Queensland Childrens Medical Research Institute

Brisbane, Australia
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Bell S.C.,Prince Charles Hospital | Bell S.C.,Queensland Childrens Medical Research Institute | De Boeck K.,Catholic University of Leuven | Amaral M.D.,University of Lisbon
Pharmacology and Therapeutics | Year: 2015

With the discovery of the CFTR gene in 1989, the search for therapies to improve the basic defects of cystic fibrosis (CF) commenced. Pharmacological manipulation provides the opportunity to enhance CF transmembrane conductance regulator (CFTR) protein synthesis and/or function. CFTR modulators include potentiators to improve channel gating (class III mutations), correctors to improve abnormal CFTR protein folding and trafficking (class II mutations) and stop codon mutation read-through drugs relevant for patients with premature stop codons (most class I mutations). After several successful clinical trials the potentiator, ivacaftor, is now licenced for use in adults and children (>six years), with CF bearing the class III G551D mutation and FDA licence was recently expanded to include 8 additional class III mutations. Alternative approaches for class I and class II mutations are currently being studied. Combination drug treatment with correctors and potentiators appears to be required to restore CFTR function of F508del, the most common CFTR mutation. Alternative therapies such as gene therapy and pharmacological modulation of other ion channels may be advantageous because they are mutation-class independent, however progress is less well advanced. Clinical trials for CFTR modulators have been enthusiastically embraced by patients with CF and health care providers. Whilst novel trial end-points are being evaluated allowing CFTR modulators to be efficiently tested, many challenges related to the complexity of CFTR and the biology of the epithelium still need to be overcome. © 2014 Elsevier Inc.

Unemo M.,Örebro University | Golparian D.,Örebro University | Limnios A.,Prince of Wales Hospital | Whiley D.,Queensland Childrens Medical Research Institute | And 3 more authors.
Antimicrobial Agents and Chemotherapy | Year: 2012

Clinical resistance to the currently recommended extended-spectrum cephalosporins (ESCs), the last remaining treatment options for gonorrhea, is being reported. Gonorrhea may become untreatable, and new treatment options are crucial. We investigated the in vitro activity of ertapenem, relative to ceftriaxone, against N. gonorrhoeae isolates and the effects of ESC resistance determinants on ertapenem. MICs were determined using agar dilution technique or Etest for international reference strains (n = 17) and clinical N. gonorrhoeae isolates (n = 257), which included the two extensively drug-resistant (XDR) strains H041 and F89 and additional isolates with high ESC MICs, clinical ESC resistance, and other types of clinical high-level and multidrug resistance (MDR). Genetic resistance determinants for ESCs (penA, mtrR, and penB) were sequenced. In general, the MICs of ertapenem (MIC 50 = 0.032 μg/ml; MIC 90 = 0.064 μg/ml) paralleled those of ceftriaxone (MIC 50 = 0.032 μg/ml; MIC 90 = 0.125 μg/ml). The ESC resistance determinants mainly increased the ertapenem MIC and ceftriaxone MIC at similar levels. However, the MIC ranges for ertapenem (0.002 to 0.125 μg/ml) and ceftriaxone (<0.002 to 4 μg/ml) differed, and the four (1.5%) ceftriaxone-resistant isolates (MIC = 0.5 to 4 μg/ml) had ertapenem MICs of 0.016 to 0.064 μg/ml. Accordingly, ertapenem had in vitro advantages over ceftriaxone for isolates with ceftriaxone resistance. These in vitro results suggest that ertapenem might be an effective treatment option for gonorrhea, particularly for the currently identified ESC-resistant cases and possibly in a dual antimicrobial therapy regimen. However, further knowledge regarding the genetic determinants (and their evolution) conferring resistance to both antimicrobials, and clear correlates between genetic and phenotypic laboratory parameters and clinical treatment outcomes, is essential. Copyright © 2012, American Society for Microbiology. All Rights Reserved.

Caffery L.J.,University of Queensland | Smith A.C.,University of Queensland | Smith A.C.,Queensland Childrens Medical Research Institute
Studies in Health Technology and Informatics | Year: 2010

A structured analysis of peer-reviewed literature about the delivery of health services by email was undertaken for this review. A total of 185 articles were included in the analysis. These articles were thematically categorised for medical specialty, participants, sub-topic, study design and service-delivery application. It was shown that email-based telemedicine can be practiced in a large number of medical specialties and has application in primary consultation, second opinion consultation, telediagnosis and administrative roles (e.g. e-referral). Email has niche applications in low-bandwidth, image-based specialties (e.g. dermatology, pathology, wound care and ophthalmology) where attached digital camera images were used for telediagnosis. Diagnostic accuracy of these images was the predominant topic of research and results show email as a valid means of delivering these medical services. Email is also often used in general practice as an adjunct for face-to-face consultation. Further, a number of organisations have significantly improved the efficiency of their outpatient services when using email as a triage or e-referral system. Email-based telemedicine provides specialist medical opinion in the majority of reviewed services and is most likely to be instigated by the patient's primary care giver. However, email-consultations between patient and primary care and patient and secondary care are not uncommon. Most email services are implemented using ordinary email. However, a number of organisations have developed purpose-written email applications to support their telemedicine service due to impediments of using ordinary email. These impediments include lack of management tools for: the allocation and auditing of cases for a timely response and the co-ordination of effort in a multi-clinician, multi-disciplinary service. The ability to encrypt ordinary email thereby securing patient confidentiality is also regarded as difficult when using ordinary email. Hence, alternative web-based email applications where the encryption can be implemented using the more user-friendly HTTPS have become popular. Much of the reviewed literature is descriptive or anecdotal and hence, suffers from lack of conclusive results regarding positive patient outcomes. This may account for email-based telemedicine generally being regarded as underutilised. However, the potential is well recognised. © 2010 The authors and IOS Press. All rights reserved.

Coulthard M.G.,University of Queensland | Coulthard M.G.,Royal Childrens Hospital | Long D.A.,Royal Childrens Hospital | Ullman A.J.,Royal Childrens Hospital | And 2 more authors.
Archives of Disease in Childhood | Year: 2012

Objective: To compare the difference in plasma sodium at 16-18 h following major surgery in children who were prescribed either Hartmann's and 5% dextrose or 0.45% saline and 5% dextrose. Design: A prospective, randomised, open label study. Setting: The paediatric intensive care unit (650 admissions per annum) in a tertiary children's hospital in Brisbane, Australia. Patients: The study group comprised 82 children undergoing spinal instrumentation, craniotomy for brain tumour resection, or cranial vault remodelling. Interventions: Patients received either Hartmann's and 5% dextrose at full maintenance rate or 0.45% saline and 5% dextrose at two-thirds maintenance rate. Main outcomes measures: Primary outcome measure: plasma sodium at 16-18 h postoperatively; secondary outcome measure: number of fluid boluses administered. Results: Mean postoperative plasma sodium levels of children receiving 0.45% saline and 5% dextrose were 1.4 mmol/l (95% CI 0.4 to 2.5) lower than those receiving Hartmann's and 5% dextrose (p=0.008). In the 0.45% saline group, seven patients (18%) became hyponatraemic (Na <135 mmol/l) at 16-18 h postoperatively; in the Hartmann's group no patient became hyponatraemic (p=0.01). No child in either fluid group became hypernatraemic. Conclusions: The postoperative fall in plasma sodium was smaller in children who received Hartmann's and 5% dextrose compared to those who received 0.45% saline and 5% dextrose. It is suggested that Hartmann's and 5% dextrose should be administered at full maintenance rate postoperatively to children who have undergone major surgery in preference to hypotonic fluids.

Whittingham K.,University of Queensland | Wee D.,Queensland Childrens Medical Research Institute | Sanders M.R.,University of Queensland | Boyd R.,University of Queensland | Boyd R.,Queensland Childrens Medical Research Institute
Disability and Rehabilitation | Year: 2013

Purpose: To explore the grieving, coping and resiliency experiences of parents of children with cerebral palsy (CP) and to investigate the suitability of chronic sorrow theory as a framework to understand those experiences. Methods: This study combined focus groups with a web-based cross-sectional survey to explore chronic sorrow in parents of children with CP. Eight parents of children with CP participated in focus groups. The discussion was transcribed verbatim and thematic analysis was performed. A further 94 parents participated in the web-based survey study in which they completed the Adapted Burke Questionnaire on chronic sorrow. A content analysis of responses was used to confirm the primary qualitative analysis. Results: The reports of parents in the focus group were consistent with chronic sorrow theory, as were the responses of parents to the web-based survey. Some parents found the diagnosis itself a distressing time whereas others found it a relief. Parents reported that times of medical and allied health intervention were particularly challenging. Conclusion: Chronic sorry theory is a useful way of understanding the experiences of parents of children with CP. It is recommended that health practitioners are mindful that, even years after diagnosis, parents of children with CP may experience intensified chronic sorrow symptoms following a triggering event and that this is normal. Implications for Rehabilitation This study suggests that chronic sorrow theory is a suitable framework in which to understand the experiences of parents of children with CP. Parents of children with CP may experience intensified grief following a triggering event, even years after diagnosis. This recurring grieving process is healthy and normal with many parents demonstrating resilience by using an array of coping strategies or seeking support from health professionals. © 2013 Informa UK, Ltd.

Armfield N.R.,University of Queensland | Armfield N.R.,Queensland Childrens Medical Research Institute | Bradford M.,University of Queensland | Bradford N.K.,University of Queensland
International Journal of Medical Informatics | Year: 2015

Background: Low-cost and no-cost software-based video tools may be a feasible and effective way to provide some telemedicine services, particularly in low-resource settings. One of the most popular tools is Skype; it is freely available, may be installed on many types of devices, and is easy to use by clinicians and patients. While a previous review found no evidence in favor of, or against the clinical use of Skype, anecdotally it is believed to be widely used in healthcare for providing clinical services. However, the range of clinical applications in which Skype has been used has not been described. Aim: We aimed to identify and summarize the clinical applications of Skype. Methods: We reviewed the literature to identify studies that reported the use of Skype in clinical care or clinical education. We searched three electronic databases using the single search term "Skype". Results: We found 239 unique articles. Twenty seven of the articles met our criteria for further review. The use of Skype was most prevalent in the management of chronic diseases such as cardiovascular diseases and diabetes, followed by educational and speech and language pathology applications. Most reported uses were in developed countries. In all but one case, Skype was reported by the authors to be feasible and to have benefit. However, while Skype may be a pragmatic approach to providing telemedicine services, in the absence of formal studies, the clinical and economic benefits remain unclear. © 2015.

Burgess S.,Materials Childrens Hospital | Sly P.,Queensland Childrens Medical Research Institute | Devadason S.,University of Western Australia
Pulmonary Medicine | Year: 2011

Suboptimal adherence with preventive medication is common and often unrecognised as a cause of poor asthma control. A number of risk factors for nonadherence have emerged from well-conducted studies. Unfortunately, patient report a physician's estimation of adherence and knowledge of these risk factors may not assist in determining whether non-adherence is a significant factor. Electronic monitoring devices are likely to be more frequently used to remind patients to take medication, as a strategy to motivate patients to maintain adherence, and a tool to evaluate adherence in subjects with poor disease control. The aim of this paper is to review non-adherence with preventive medication in childhood asthma, its impact on asthma control, methods of evaluating non-adherence, risk factors for suboptimal adherence, and strategies to enhance adherence. © 2011 Scott Burgess et al.

Towns S.J.,The Childrens Hospital at Westmead | Towns S.J.,University of Sydney | Bell S.C.,University of Queensland | Bell S.C.,Queensland Childrens Medical Research Institute
Clinical Respiratory Journal | Year: 2011

Transition to adult health care for adolescents with complex chronic illness such as cystic fibrosis (CF) is a challenge for the health care system with increasing numbers of young people requiring lifelong adult health care due to improvements in early diagnosis, treatment and survival. The changing face of CF and current guidelines for transition to adult care will be reviewed with a discussion and case study illustrating effective transition. Key features include (i) early preparation, planning and facilitating self-management skills; (ii) engaging all involved by a coordinated approach, including young people with CF, their families and the paediatric and adult teams; (iii) detailed communication, including comprehensive written referral report and documentation of prior complications of CF; (iv) feedback between the paediatric and the adult health care teams; and (v) ongoing audit of the transition process. The barriers to effective transition will be examined in the context of challenges faced by the paediatric centres, the development and resourcing of appropriate adult services, difficulties for the young person and their family as well as integrating the health care system overall. While acknowledging the development and evaluation of models of care for transition services with CF, continued evaluation of transition services can provide an evidence base to ensure effective systems with allocation of resources, inform training of health professionals and meet the needs of young people with CF and other chronic illnesses as they navigate the health care system. © 2010 Blackwell Publishing Ltd.

Armfield N.R.,University of Queensland | Armfield N.R.,Queensland Childrens Medical Research Institute | Edirippulige S.K.,University of Queensland | Bradford N.,University of Queensland | And 3 more authors.
Medical Journal of Australia | Year: 2014

• A large literature base on telemedicine exists, but the evidence base for telemedicine is very limited. There is little practical or useful information to guide clinicians and health policymakers. • Telemedicine is often implemented based on limited or no prior formal analysis of its appropriateness to the circumstances, and adoption of telemedicine by clinicians has been slow and patchy. • Formal analysis should be conducted before implementation of telemedicine to identify the patients, conditions and settings that it is likely to benefit. • Primary studies of telemedicine tend to be of insufficient quality to enable synthesis of formal evidence. • Methods typically used to assess eff ectiveness in medicine are often difficult, expensive or impractical to apply to telemedicine. • Formal studies of telemedicine should examine efficacy, effectiveness, economics and clinician preferences. • Successful adoption and sustainable integration of telemedicine into routine care could be improved by evidence-based implementation.

Maskell J.,Queensland Childrens Medical Research Institute | Maskell J.,University of Queensland | Newcombe P.,University of Queensland | Martin G.,University of Queensland | And 2 more authors.
Journal of Burn Care and Research | Year: 2013

Burn injury is one of the most traumatic injuries a child or adolescent can experience. When a burn injury occurs, the child can suffer pain, uncertainty, fear, and trauma from acute treatment to rehabilitation and reintegration. He or she can also experience long-term psychosocial and psychological difficulties. The objective of the study was to compare health-related quality of life (HRQoL), psychopathology, and self-concept of children who have suffered a burn injury with a matched sample of healthy controls. Sixty-six children and adolescents with a burn injury, who were aged between 8 to 17 years, and a caregiver were recruited from six burn centers in Australia and New Zealand. Participants completed the Paediatric Quality of Life Inventory, the Strengths and Difficulties Questionnaire, and the Piers-Harris Self-Concept Scale (P-H SCS). Scores were compared with published normative data. As scarring and appearance are a distinct issue, the Paediatric Quality of Life Inventory cancer module perceived physical appearance subscale was also included. Pediatric burn survivors and their caregivers reported significantly higher emotional and behavioral problems and lower HRQoL, but no significant differences in self-concept compared with healthy counterparts. Pediatric burn survivors also reported significantly poorer perceived physical appearance than the matched pediatric cancer sample. Burned children reported lowered quality of life, particularly related to scarring and appearance; however, they reported normative self-concept. This may be because of self-concept being a psychological trait, whereas HRQoL is influenced by societal norms and expectations. Psychosocial support is necessary to build positive coping strategies and manage the unpleasant social experiences that may reduce quality of life. Copyright © 2012 by the American Burn Association.

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