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Advanced therapies in pediatric endocrinology and diabetology
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This article was originally published by Medknow Publications & Media Pvt Ltd and was migrated to Scientific Scholar after the change of Publisher.
This book is the 30th volume of the ongoing series ‘Endocrine Development’. It consists of a compilation of chapters on various advanced therapies in paediatric endocrinology and diabetology. This multi author book, written by experts in their fields, gives a comprehensive updated review of recent advances in endocrinology and diabetes.
The book starts off with a section on automated insulin delivery in type 1 diabetes. The authors take us through the history, types, advantages and limitations of closed loop system of insulin delivery. This is followed by a lucid description of current indications and future perspectives of islet cell transplant in paediatric patients and a chapter on treatment of paediatric obesity with reference to glucagon-like peptide-1 (GLP-1) receptor agonists, metformin and orlistat.
Chapter 4 is a well written account of the role of docosahexaenoic acid in G- protein coupled receptor 120 activation in paediatric non-alcoholic fatty liver disease. This is followed by a chapter on noninvasive prenatal diagnosis of congenital adrenal hyperplasia, which brings forth how using cell-free foetal DNA, in mothers carrying ‘at risk’ foetuses, a diagnosis can be established in as early as six weeks and unnecessary prenatal administration of low dose dexamethasone can be prevented.
Following this is a crisp description of inadequacies of conventional hydrocortisone replacement and the newer options available to replace cortisol in a physiological manner. This includes oral mixed release drug formulations and continuous subcutaneous hydrocortisone infusion to approximate physiological rhythm in adrenal insufficiency and congenital adrenal hyperplasia.
The next chapter deals well with subcutaneous implant of gonadotropin releasing hormone analogue histrelin in management of central precocious puberty. This is followed by a vivid description of medications available for hypogonadotrophic hypogonadism and the recent molecules such as kisspeptin and neurokinin B agonists under evaluation for therapy of delayed puberty. Then there is an update on status of recent formulation of sustained release, long acting recombinant human growth hormone for paediatric use.
The next few chapters deal with potential therapeutic strategies for achondroplasia focusing on C- type natriuretic peptide analogue. This is followed by an exhaustive account of therapeutic neuroendocrine agonist and antagonist analogues of hypothalamic neuropeptides as modulators of hypothalamic-pituitary-gonadal axis. This chapter not only deals with their application in treating numerous conditions like precocious and delayed puberty, prostate cancer, benign prostate hyperplasia, endometriosis, uterine fibroids and in vitro fertilization protocols, but also throws light on kisspeptin and neurokinin B agonists and antagonists which are being developed as newer drugs in this area.
Chapter 12 reviews published findings on the use of aromatase inhibitors in growth with emphasis on treatment efficacy and safety. The last two chapters are on advances in gene therapy for treating adrenoleucodystrophy and metachromatic leukodystrophy and novel therapeutic targets and drug candidates for modifying disease progression in adrenoleucodystrophy, along with their limitations and applications.
The text is well written and to the point to address issues without undue elaboration on a variety of domains in endocrinology. However, it would appear more systematic if arranged in sections like diabetes, obesity, adrenals, hypogonadism, growth and bone disorders.
Overall, this book will be useful for those with a background of either paediatrics or endocrinology, particularly for the super speciality trainees in endocrinology, academicians and researchers.
