How to use medicines in children: principles of paediatric clinical pharmacology

Knowledge of drug administration in children and infants has significantly lagged behind that of adults. Despite several paediatric therapeutic mishaps that have been a catalyst for major regulatory reform, the majority of registered medicines do not have indications or dosing for children. This paper will briefly summarise key issues in paediatric pharmacology, including differences in prescribing for children, off-label and unlicensed prescribing and conduct of paediatric clinical trials. A particular emphasis will placed on the situation in Australia.     

How to manage paediatric intestinal failure

Management of paediatric intestinal failure has come a long way since the advent of parenteral nutrition in the 1960s. Our understanding of the normal physiology of the gastrointestinal tract together with deeper understanding about the pathophysiology of some of the conditions that leads to intestinal failure has meant that management is more targeted than previously. This article aims to focus on the application of pathophysiological understanding of intestinal failure in its management and give some direction in managing some common complications intestine failure. It will cover both surgical as well as medical (including dietetic) treatments that can help children with intestinal failure to achieve enteral autonomy.     

Objective structured clinical examination (OSCE) revisited

Objective structured clinical examination (OSCE) was introduced in 1975 as a standardized tool for objectively assessing clinical competencies — including history-taking, physical examination, communication skills, data interpretation etc. It consists of a circuit of stations connected in series, with each station devoted to assessment of a particular competency using pre-determined guidelines or checklists. OSCE has been used as a tool for both formative and summative evaluation of medical graduate and postgraduate students across the globe. The use of OSCE for formative assessment has great potential as the learners can gain insights into the elements making up clinical competencies as well as feedback on personal strengths and weaknesses. However, the success of OSCE is dependent on adequacy of resources, including the number of stations, construction of stations, method of scoring (checklists and/or global scoring), the number of students assessed, and adequate time and money. Lately, OSCE has drawn some criticism for its lack of validity, feasibility, practicality, and objectivity. There is evidence to show that many OSCEs may be too short to achieve reliable results. There are also currently no clear cut standards set for passing an OSCE. It is perceived that OSCEs test the student’s knowledge and skills in a compartmentalized fashion, rather than looking at the patient as a whole. This article focuses on the issues of validity, objectivity, reliability, and standard setting of OSCE. Presently, the Indian experiences with OSCE are limited and there is a need to sensitise the Indian faculty and students. A cautious approach is desired before it is considered as a supplementary tool to other methods of assessment for the summative examinations in Indian settings.     

Calibration of the paediatric index of mortality in UK paediatric intensive care units

AIM—To test a paediatric intensive care mortality prediction model for UK use.
METHOD—Prospective collection of data from consecutive admissions to five UK paediatric intensive care units (PICUs), representing a broad cross section of paediatric intensive care activity. A total of 7253 admissions were analysed using tests of the discrimination and calibration of the logistic regression equation.
RESULTS—The model discriminated and calibrated well. The area under the ROC plot was 0.84 (95% CI 0.819 to 0.853). The standardised mortality ratio was 0.87 (95% CI 0.81 to 0.94). There was remarkable concordance in the performance of the paediatric index of mortality (PIM) within each PICU, and in the performance of the PICUs as assessed by PIM. Variation in the proportion of admissions that were ventilated or transported from another hospital did not affect the results.
CONCLUSION—We recommend that UK PICUs use PIM for their routine audit needs. PIM is not affected by the standard of therapy after admission to PICU, the information needed to calculate PIM is easy to collect, and the model is free.

     

Calibration of the paediatric index of mortality in UK paediatric intensive care units.

AIM: To test a paediatric intensive care mortality prediction model for UK use. METHOD: Prospective collection of data from consecutive admissions to five UK paediatric intensive care units (PICUs), representing a broad cross section of paediatric intensive care activity. A total of 7253 admissions were analysed using tests of the discrimination and calibration of the logistic regression equation. RESULTS: The model discriminated and calibrated well. The area under the ROC plot was 0.84 (95% CI 0.819 to 0.853). The standardised mortality ratio was 0.87 (95% CI 0.81 to 0.94). There was remarkable concordance in the performance of the paediatric index of mortality (PIM) within each PICU, and in the performance of the PICUs as assessed by PIM. Variation in the proportion of admissions that were ventilated or transported from another hospital did not affect the results. CONCLUSION: We recommend that UK PICUs use PIM for their routine audit needs. PIM is not affected by the standard of therapy after admission to PICU, the information needed to calculate PIM is easy to collect, and the model is free.     

Epidemiology of paediatric renal stone disease in the UK

Background: The previous epidemiological study of paediatric nephrolithiasis in Britain was conducted more than 30 years ago. Aims: To examine the presenting features, predisposing factors, and treatment strategies used in paediatric stones presenting to a British centre over the past five years. Methods: A total of 121 children presented with a urinary tract renal stone, to one adult and one paediatric centre, over a five year period (1997–2001). All children were reviewed in a dedicated stone clinic and had a full infective and metabolic stone investigative work up. Treatment was assessed by retrospective hospital note review. Results: A metabolic abnormality was found in 44% of children, 30% were classified as infective, and 26% idiopathic. Bilateral stones on presentation occurred in 26% of the metabolic group compared to 12% in the infective/idiopathic group (odds ratio 2.7, 95% CI 1.03 to 7.02). Coexisting urinary tract infection was common (49%) in the metabolic group. Surgically, minimally invasive techniques (lithotripsy, percutaneous nephrolithotomy, and endoscopy) were used in 68% of patients. Conclusions: There has been a shift in the epidemiology of paediatric renal stone disease in the UK over the past 30 years. Underlying metabolic causes are now the most common but can be masked by coexisting urinary tract infection. Treatment has progressed, especially surgically, with sophisticated minimally invasive techniques now employed. All children with renal stones should have a metabolic screen.     

Proton beam therapy for paediatric tumours in the UK

Proton beam therapy (PBT) has been available on a limited basis for some paediatric tumours since 2008, but is becoming more widely available. It is a form of radiotherapy, but differs from traditional photon beam radiotherapy in several important aspects. PBT offers the potential for improved outcomes from childhood cancer mainly by reduction of some late effects of treatment, potentially improving both survival and quality of survival. However the delivery of PBT is more complex and burdensome, requiring a significant degree of expertise and co-ordination of care as well as wrap-around support for the child being treated and their family. This short review describes the principles of treatment, some of the current indications and the challenges for paediatricians who care for children either receiving PBT or those who have previously had this treatment.     

Content bias in the neoplastic-related items of the national board of medical examiners part II examination

The Education Evaluation Committee of the American Association for Cancer Education (AACE) attempted to employ the neoplastic-related items from the National Board of Medical Examiners (NBME) Part II examination in a study correlating program characteristics at various medical schools with performance on the neoplastic-related items. All questions on the September, 1977 and April, 1978 NBME Part II examinations were reviewed by board certified medical oncologists who found an approximate 20% discordance between the AACE and NBME classifications. Content analysis of the neoplastic-related items disclosed a preponderance of questions related to gynecologic cancer with few questions concerning the major solid tumors: lung, breast and colorectal cancer. In addition, virtually no questions related to the psychologic impact of cancer or to the therapy of cancer were asked. We conclude that the use of item by item performance scores from the neoplastic-related items is inadequate for program analysis due to disagreements over what constitutes such an item and to content bias in the agreed upon items. We propose a coding system that may allow the results from current NBME examinations to be used in program evaluation.