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Sheffield, United Kingdom

The rate of technological improvement continues to accelerate. Regulators in every field dealing with consumer products continue to set ever higher standards to protect consumers from adverse events and use 'recalls' to remove products that prove to be harmful from the market. In the field of medical products in general the issues of 'human factors' and 'usability' are now, quite rightly, a major issue at least among regulators in the USA. The elephant in the inhaled therapy room is of course the continued use of obsolete, portable inhalers which few patients can use effectively for the treatment of asthma. Countless studies have demonstrated that the inability of patients to use these devices effectively is a major factor in perpetuating unnecessarily high levels of morbidity. They fail to meet basic usability standards and do not incorporate the facility to provide feedback to patient and clinician. More than 20 years ago regulators deemed that pressurised metered dose inhalers containing chlorofluorocarbons should be removed from the market on environmental grounds even though their use accounted for less than 0.5% of chlorofluorocarbon use. Surely asthmatic patients require the same level of protection. Unfortunately regulators appear determined to fossilise the field in a 19500s time warp by ensuring that the failings of obsolete technology are perpetuated in any 'generic' device. The time has come for regulators to meet their obligations to 'protect the public health by assuring the safety, effectiveness, and security of drugs, vaccines and other biological products, medical devices...' and mandate the phasing out of these antiquated devices within the next decade in order to reduce the unacceptably high burden of preventable morbidity and death associated with their use. Source

Izraeli S.,Sheba Medical Center | Vora A.,Sheffield Childrens Hospital | Zwaan C.M.,Pediatric Oncology Hematology | Whitlock J.,University of Toronto

Children with Down syndrome are at high risk for developing B-cell precursor acute lymphoblastic leukemia (DSALL) associated with poor outcome due to both a high relapse rate and increased treatment-related mortality (TRM) from infections. Biologically, these heterogeneous leukemias are characterized by under-representation of the common cytogenetic subgroups of childhood ALL and overrepresentation of CRLF2-IL7RJAK-STAT activating genetic aberrations. Although relapse is the major determinant of poor outcomes in this population, deescalation of chemotherapy intensity might be feasible in the 10% to 15% DS-ALL patients with ETV6-RUNX1 or high hyperdipoidy in whom TRM is the major limiting event. As infection-associated TRM occurs during all treatment phases, including the maintenance period, increased surveillance and supportive care is required throughout therapy. Improvement in outcome will require better understanding of the causes of treatment failure and TRM, incorporation of new therapies targeting the unique biological properties of DS-ALL, and enhanced supportive care measures to reduce the risk of infection-related TRM. To facilitate these goals, an international collaboration plans to establish a prospective DS-ALL registry and develop specific supportive care recommendations for this at-risk population. © 2014 by The American Society of Hematology. Source

Sprigg A.,Sheffield Childrens Hospital
Archives of Disease in Childhood

Temporary brittle bone disease has been proposed again as an alternative explanation for suspected non-accidental injury. This is still not considered a real entity by mainstream opinion. The recent publications remind us to look carefully for alternative explanations and to investigate for predisposing bone disorders thoroughly. Source

Goddard J.M.,Sheffield Childrens Hospital
Current Opinion in Supportive and Palliative Care

Clinicians need to be aware of the therapeutic effect of the psychosocial approach to the management of chronic pain in children and young people. Further research is required into the pharmacological and physical aspects of management, which remain important. © 2011 Wolters Kluwer Health | Lippincott Williams & Wilkins. Source

Olpin S.E.,Sheffield Childrens Hospital
Journal of Inherited Metabolic Disease

Fatty acids are a major fuel for the body and fatty acid oxidation is particularly important during fasting, sustained aerobic exercise and stress. The myocardium and resting skeletal muscle utilise long-chain fatty acids as a major source of energy. Inherited disorders affecting fatty acid oxidation seriously compromise the function of muscle and other highly energy-dependent tissues such as brain, nerve, heart, kidney and liver. Such defects encompass a wide spectrum of clinical disease, presenting in the neonatal period or infancy with recurrent hypoketotic hypoglycaemic encephalopathy, liver dysfunction, hyperammonaemia and often cardiac dysfunction. In older children, adolescence or adults there is often exercise intolerance with episodic myalgia or rhabdomyolysis in association with prolonged aerobic exercise or other exacerbating factors. Some disorders are particularly associated with toxic metabolites that may contribute to encephalopathy, polyneuropathy, axonopathy and pigmentary retinopathy. The phenotypic diversity encountered in defects of fat oxidation is partly explained by genotype/phenotype correlation and certain identifiable environmental factors but there remain many unresolved questions regarding the complex interaction of genetic, epigenetic and environmental influences that dictate phenotypic expression. It is becoming increasingly clear that the view that most inherited disorders are purely monogenic diseases is a naive concept. In the future our approach to understanding the phenotypic diversity and management of patients will be more realistically achieved from a polygenic perspective. © 2013 SSIEM and Springer Science+Business Media Dordrecht. Source

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