Bailey S.,Great North Childrens Hospital |
Parkes J.,University of Cape Town
Clinical Endocrinology | Year: 2015
A 13-year-old male presents with fluctuating visual disturbance and headaches. Imaging reveals a significant predominantly cystic suprasellar tumour, typical of a craniopharyngioma. The patient has growth hormone deficiency but the rest of the hypothalamic/ pituitary axis is intact. What are the options for therapy in 2014? Specifically, is there a role for local treatment with interferon injected into the cyst cavity? The aim of management in children with craniopharyngiomas is to reduce the impact of the tumour as much as possible, while minimising the morbidity associated with treatment. There are a number of therapeutic options available: surgery, radiotherapy and the insertion of therapeutic agents directly into the tumour cyst. The role of intracystic therapy in the form of interferon is discussed; including when to use this therapeutic option and practical details of its use. © 2014 John Wiley & Sons Ltd.
Cowan M.J.,University of California at San Francisco |
Gennery A.R.,Great North Childrens Hospital
Journal of Allergy and Clinical Immunology | Year: 2015
Defects in DNA cross-link repair 1C (DCLRE1C), protein kinase DNA activated catalytic polypeptide (PRKDC), ligase 4 (LIG4), NHEJ1, and NBS1 involving the nonhomologous end-joining (NHEJ) DNA repair pathway result in radiation-sensitive severe combined immunodeficiency (SCID). Results of hematopoietic cell transplantation for radiation-sensitive SCID suggest that minimizing exposure to alkylating agents and ionizing radiation is important for optimizing survival and minimizing late effects. However, use of preconditioning with alkylating agents is associated with a greater likelihood of full T- and B-cell reconstitution compared with no conditioning or immunosuppression alone. Areduced-intensity regimen using fludarabine and low-dose cyclophosphamide might be effective for patients with LIG4, NHEJ1, and NBS1 defects, although more data are needed to confirm these findings and characterize late effects. For patients with mutations in DCLRE1C (Artemis-deficient SCID), there is no optimal approach that uses standard dose-alkylating agents without significant late effects. Until nonchemotherapy agents, such as anti-CD45 or anti-CD117, become available, options include minimizing exposure to alkylators, such as single-agent low-dose targeted busulfan, or achieving T-cell reconstitution, followed several years later with a conditioning regimen to restore B-cell immunity. Gene therapy for these disorders will eventually remove the issues of rejection and graft-versus-host disease. Prospective multicenter studies are needed to evaluate these approaches in this rare but highly vulnerable patient population. © 2015 American Academy of Allergy, Asthma & Immunology.
Elemraid M.,Great North Childrens Hospital
Nursing children and young people | Year: 2013
To observe and report rates of, and reasons for, parents' refusal to consent to the participation of their children in appropriate clinical research. The parents of children admitted to hospital with a diagnosis of pneumonia or of empyema were asked for informed consent to research involving blood, urine and nasopharyngeal secretion samples from their child. Circumstances and numbers of agreements and refusals were compared and underlying reasons suggested. Of 144 consent requests, ten were refused, which appeared to be linked to: not wanting the child to undergo further tests, lack of interest in participating in studies, research possibly delaying discharge, and anxiety regarding written consent and the length of information sheets. Severity of the child's illness appeared to determine the parent's decision. Involvement and assistance of non-research nursing and medical staff and previous introductions to the researchers are helpful. The timing and setting for the consent process should be selected carefully. Adequate, accessible study information for parents and children contributes to successful recruitment of participants.
Coulthard M.G.,Great North Childrens Hospital
Paediatrics and International Child Health | Year: 2015
It has been argued that the oedema of kwashiorkor is not caused by hypoalbuminaemia because the oedema disappears with dietary treatment before the plasma albumin concentration rises. Reanalysis of this evidence and a review of the literature demonstrates that this was a mistaken conclusion and that the oedema is linked to hypoalbuminaemia. This misconception has influenced the recommendations for treating children with severe acute malnutrition. There are close pathophysiological parallels between kwashiorkor and Finnish congenital nephrotic syndrome (CNS) pre-nephrectomy; both develop proteinenergy malnutrition and hypoalbuminaemia, which predisposes them to intravascular hypovolaemia with consequent sodium and water retention, and makes them highly vulnerable to develop hypovolaemic shock with diarrhoea. In CNS this is successfully treated with intravenous albumin boluses. By contrast, the WHO advise the cautious administration of hypotonic intravenous fluids in kwashiorkor with shock, which has about a 50% mortality. It is time to trial intravenous bolus albumin for the treatment of children with kwashiorkor and shock. © W. S. Maney & Son Ltd 2015.
Anderson M.,Great North Childrens Hospital
Patient Preference and Adherence | Year: 2013
Prolonged seizures and status epilepticus are a common acute neurological presentation in pediatric practice. As a result, there is a need for effective and safe medications that can be delivered to convulsing children to effect rapid seizure termination both in hospital and community settings. The challenges of achieving intravenous access, particularly in young children, mandate alternative routes of administration for these drugs. Over the last ten years, midazolam delivered via the buccal mucosa has been demonstrated to be efficacious, safe, and acceptable to children and their caregivers, and a formulation has recently been licensed for use in Europe. The aim of this article is to review the clinical pharmacology with respect to these issues. © 2013 Anderson, publisher and licensee Dove Medical Press Ltd.