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Robin Michelet, Johanna Melin, Zinnia P. Parra-Guillen, Uta Neumann, J Martin Whitaker, Viktoria Stachanow, Wilhelm Huisinga, John Porter, Oliver Blankenstein, Richard J. Ross, and Charlotte Kloft

Context:

Accurate hydrocortisone dosing in children with adrenal insufficiency is important to avoid the risks of over and under treatment including iatrogenic Cushing’s syndrome and adrenal crisis.

Objective:

To establish a population pharmacokinetic model of hydrocortisone in children and use this to refine hydrocortisone replacement regimens.

Design and methods:

Pharmacokinetic study of hydrocortisone granules, available in 0.5, 1, 2 and 5 mg dose strengths, in 24 children with adrenal insufficiency aged 2 weeks to 6 years. Cortisol concentrations quantified by LC-MS/MS were used to refine an adult pharmacokinetic model to a paediatric population model which was then used to simulate seven different hydrocortisone treatment regimens.

Results:

Pre-dose cortisol levels were undetectable in 54% of the 24 children. The developed pharmacokinetic model had good predictive performance. Simulations for the seven treatment regimens using either three- or four-times daily dosing showed treatment regimens delivered an AUC0- 24h within the 90% reference range for healthy children except in neonates where two regimens had an AUC below the 5th percentile. Cortisol concentrations at individual time points in the 24 h were outside the 90% reference range for healthy individuals in 50%, 55–65% and 70–75% for children, infants and neonates, respectively, with low cortisol levels being most prevalent.

Conclusions:

Current paediatric hydrocortisone treatment regimens based on either three- or four-times daily administration replicate cortisol exposure based on AUC0- 24h, but the majority of cortisol levels are above or below physiological cortisol levels with low levels very common before the next dose.

Free access

Ann M Maguire, Geoffrey R Ambler, Bin Moore, Kay Waite, Mark McLean, and Christopher T Cowell

Objective: The aim of glucocorticoid replacement therapy in ACTH-deficient patients is to mimic the normal diurnal variation of cortisol. However, current hydrocortisone (HC) replacement results in prolonged episodes of hypocortisolaemia and supraphysiological peaks. Plasma cortisol profiles are an accurate yet labour-intensive method of assessing HC replacement. Salivary and bloodspot cortisol sampling methods are less invasive and may be useful tools for assessing glucocorticoid replacement, particularly in children. Therefore, we aimed to define normal salivary and bloodspot cortisol levels in children and their correlations with the gold standard (plasma cortisol).

Design: Cross-sectional study in a paediatric teaching hospital.

Methods: Plasma, saliva and bloodspot cortisol profiles were performed on 30 ACTH-deficient children and 22 healthy siblings.

Results: In ACTH-deficient patients taking oral HC, the bloodspot–plasma correlation (ρ = 0.90) was stronger than the salivary–plasma correlation (ρ = 0.49). Using target ranges for salivary and bloodspot cortisol levels based on normal data from control subjects, the less invasive sampling methods had low rates of agreement with plasma cortisol target ranges (saliva 65% and bloodspot 75%). Using the plasma–bloodspot correlation regression equation to convert bloodspot to calculated plasma cortisol, there was a high concordance between calculated and actual measured plasma cortisol (88%).

Conclusion: Bloodspot cortisol sampling is a feasible and accurate method for monitoring oral HC replacement in paediatric patients without necessitating hospital admission, but salivary sampling is not useful.

Free access

Vrinda Saraff and Wolfgang Högler

Osteoporosis in children can be primary or secondary due to chronic disease. Awareness among paediatricians is vital to identify patients at risk of developing osteoporosis. Previous fractures and backaches are clinical predictors, and low cortical thickness and low bone density are radiological predictors of fractures. Osteogenesis Imperfecta (OI) is a rare disease and should be managed in tertiary paediatric units with the necessary multidisciplinary expertise. Modern OI management focuses on functional outcomes rather than just improving bone mineral density. While therapy for OI has improved tremendously over the last few decades, this chronic genetic condition has some unpreventable, poorly treatable and disabling complications. In children at risk of secondary osteoporosis, a high degree of suspicion needs to be exercised. In affected children, further weakening of bone should be avoided by minimising exposure to osteotoxic medication and optimising nutrition including calcium and vitamin D. Early intervention is paramount. However, it is important to identify patient groups in whom spontaneous vertebral reshaping and resolution of symptoms occur to avoid unnecessary treatment. Bisphosphonate therapy remains the pharmacological treatment of choice in both primary and secondary osteoporosis in children, despite limited evidence for its use in the latter. The duration and intensity of treatment remain a concern for long-term safety. Various new potent antiresorptive agents are being studied, but more urgently required are studies using anabolic medications that stimulate bone formation. More research is required to bridge the gaps in the evidence for management of paediatric osteoporosis.

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E. Thamdrup

The term premature pubarche is given to conditions in which there is precocious growth of sexual hair (pubes and axillary hair) without any other symptoms of precocious puberty, i. e. without development of the genitals and, in girls, without growth of the breasts. Girls do not become virilized as in the case of the adrenogenital syndrome.

The series of cases comprises 17 patients, 12 girls and 5 boys. The symptoms in the former were observed before the age of 8, and in the latter before the age of 9 years. The patients were from the Dronning Louises Børnehospital (children's hospital, Copenhagen), the Paediatric Department of the University Hospital, Copenhagen, and 5 homes for the mentally defective: Andersvænge, Brejning, Ebberødgård, Ribe and Rødbygård.

Twelve of the patients had severe cerebral disorder, they were all mentally retarded, seven had epilepsy and seven spastic pareses. Four were blind, two had a coloboma of

Free access

Aneta Gawlik and Ewa Malecka-Tendera

Transition in health care for young patients with Turner's syndrome (TS) should be perceived as a staged but uninterrupted process starting in adolescence and moving into adulthood. As a condition associated with high risk of short stature, cardiovascular diseases, ovarian failure, hearing loss and hypothyroidism, TS requires the attention of a multidisciplinary team. In this review paper, we systematically searched the relevant literature from the last decade to discuss the array of problems faced by TS patients and to outline their optimal management during the time of transfer to adult service. The literature search identified 233 potentially relevant articles of which 114 were analysed. The analysis confirmed that all medical problems present during childhood should also be followed in adult life. Additionally, screening for hypertension, diabetes mellitus, dyslipidaemia, and osteoporosis is needed. After discharge from the paediatric clinic, there is still a long way to go.

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Claus H Gravholt, Niels H Andersen, Gerard S Conway, Olaf M Dekkers, Mitchell E Geffner, Karen O Klein, Angela E Lin, Nelly Mauras, Charmian A Quigley, Karen Rubin, David E Sandberg, Theo C J Sas, Michael Silberbach, Viveca Söderström-Anttila, Kirstine Stochholm, Janielle A van Alfen-van derVelden, Joachim Woelfle, Philippe F Backeljauw, and On behalf of the International Turner Syndrome Consensus Group

Turner syndrome affects 25–50 per 100,000 females and can involve multiple organs through all stages of life, necessitating multidisciplinary approach to care. Previous guidelines have highlighted this, but numerous important advances have been noted recently. These advances cover all specialty fields involved in the care of girls and women with TS. This paper is based on an international effort that started with exploratory meetings in 2014 in both Europe and the USA, and culminated with a Consensus Meeting held in Cincinnati, Ohio, USA in July 2016. Prior to this meeting, five groups each addressed important areas in TS care: 1) diagnostic and genetic issues, 2) growth and development during childhood and adolescence, 3) congenital and acquired cardiovascular disease, 4) transition and adult care, and 5) other comorbidities and neurocognitive issues. These groups produced proposals for the present guidelines. Additionally, four pertinent questions were submitted for formal GRADE (Grading of Recommendations, Assessment, Development and Evaluation) evaluation with a separate systematic review of the literature. These four questions related to the efficacy and most optimal treatment of short stature, infertility, hypertension, and hormonal replacement therapy. The guidelines project was initiated by the European Society of Endocrinology and the Pediatric Endocrine Society, in collaboration with the European Society for Paediatric Endocrinology, the Endocrine Society, the European Society of Human Reproduction and Embryology, the American Heart Association, the Society for Endocrinology, and the European Society of Cardiology. The guideline has been formally endorsed by the European Society of Endocrinology, the Pediatric Endocrine Society, the European Society for Paediatric Endocrinology, the European Society of Human Reproduction and Embryology and the Endocrine Society. Advocacy groups appointed representatives who participated in pre-meeting discussions and in the consensus meeting.

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Eileen E. Hill

ABSTRACT

  1. A method for the fractionation of the urinary 17-ketogenic steroids with no oxygen grouping at C11 and those oxygenated at C11, is applied to the clinical problems of congenital adrenal hyperplasia.

  2. In normal children the mean ratio of the non-oxygenated to oxygenated steroids is 0.24. In childrern with congenital adrenal hyperplasia the ratio is 2.3. The reason for this difference in ratio is discussed.

  3. The changes in ratio found under stimulation of the adrenal gland with exogenous or endogenous corticotrophin and the suppression with cortisone therapy are studied.

  4. This test can be applied to isolated samples of urine, a major advantage in paediatric practice, and can be carried out in routine laboratories. It is found to be reliable in the diagnosis and sensitive in the control of congenital adrenal hyperplasia.

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J. M. PARKIN

My interest in children with growth problems was stimulated by hearing the 1964 Windermere Lecture given to the British Paediatric Association (Prader et al). Since these early exciting studies of growth hormone treatment there has been debate about the best way of identifying patients who might benefit from it. When the only preparations available were derived from human pituitaries, and supply was limited, the opinion was readily accepted that only patients with very low serum levels after pharmacological stress should be treated. The demonstration of a serum growth hormone of more than 15 mU/1 after any stress was accepted as an indication that treatment was not indicated.

Recently the situation has changed. Biosynthetic growth hormone is available and, though expensive, in potentially limitless quantities. In addition, there is evidence that many more children than originally thought may be helped by the hormone (Zadik et al 1985). Indeed there may be

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Hans Perrild and Bendt Brock Jacobsen

Thyrotoxicosis during the first two decades of life is rare compared to that in adults, with an incidence of 0.8 per 100000 children per year (1). This limits the experience in the paediatric clinics, and adult treatment strategies are often applied to the thyrotoxic child.

About 95% of thyrotoxic children have Graves' disease (1, 2), in contrast to findings in adults where a significant number of toxic adenomas and multinodular toxic goitres are seen, especially in areas with a relatively low iodine intake (3). It is suggested that Graves' disease in childhood is a self-limiting disorder with a remission rate of 25% every 2 years (4). These observations have to be considered in the choice of therapy.

There are three principal treatments today: antithyroid drugs (ATD), surgery or radioiodine. It is well known that the primary management of thyrotoxicosis in adults is different in Europe, Japan and the USA (5).

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S.M. SHALET

Pituitary adenomas are uncommon in childhood (Zimmerman, 1969). The majority of pituitary adenomas are benign hormonally active tumours arising from the 5 hormonal secreting cell types of the adenohypophysis (Asa and Kovacs, 1983). With the availability of modern immunohistochemical techniques it is apparent that only 25% of pituitary adenomas are non-functioning.

In paediatric practice the important hormonally active adenomas are GH (gigantism), prolactin and ACTH (Cushing's disease). Both functional and non-functional pituitary tumours may be associated with hypopituitarism resulting from compression of the normal pituitary tissue or from tumour interfering with transport and synthesis of hypothalamic releasing hormones. This may lead to impaired growth, hypothyroidism, hypoadrenalism and failure of normal pubertal development.

Gigantism

The excessive secretion of GH before fusion of the epiphyses leads to gigantism. After fusion it produces the typical features of acromegaly. In nearly all cases, gigantism results from a pituitary tumour. Some tumours are mixed and