Immediate-release granule formulation of hydrocortisone, Alkindi®, for treatment of paediatric adrenal insufficiency (Infacort development programme)

References

• EU. State of paediatric medicines in the EU 10 years of the EU Paediatric Regulation COM 2017; 626.
   Google Scholar
• Fluck CE. Mechanisms in endocrinology: update on pathogenesis of primary adrenal insufficiency: beyond steroid enzyme deficiency and autoimmune adrenal destruction. Eur J Endocrinol. 2017;177:R99–R111.
   PubMed Web of Science ®Google Scholar
• Han TS, Walker BR, Arlt W, et al. Treatment and health outcomes in adults with congenital adrenal hyperplasia. Nat Rev Endocrinol. 2014;10:115–124.
   PubMed Web of Science ®Google Scholar
• Speiser PW, Azziz R, Baskin LS, et al. Congenital adrenal hyperplasia due to steroid 21-hydroxylase deficiency: an Endocrine Society clinical practice guideline. J Clin Endocrinol Metab. 2010;95:4133–4160.
   PubMed Web of Science ®Google Scholar
• Whitaker MJ, Spielmann S, Digweed D, et al. Development and testing in healthy adults of oral hydrocortisone granules with taste masking for the treatment of neonates and infants with adrenal insufficiency. J Clin Endocrinol Metab. 2015;100:1681–1688.
   PubMed Web of Science ®Google Scholar
• Neumann U, Whitaker MJ, Wiegand S, et al. Absorption and tolerability of taste-masked hydrocortisone granules in neonates, infants and children under 6 years of age with adrenal insufficiency. Clin Endocrinol (Oxf). 2018;88:21–29.
   PubMed Web of Science ®Google Scholar
• Neumann U, Burau D, Spielmann S, et al. Quality of compounded hydrocortisone capsules used in the treatment of children. Eur J Endocrinol. 2017;177:239–242.
   PubMed Web of Science ®Google Scholar
• Webb EA, Watson C, Kerr S, et al. Hydrocortisone tablets: human factors in manipulation and their impact on dosing accuracy. Endocrine Abstracts. 2017;51:OC8.1.
   Google Scholar
• EMA. Note for guidance on the manufacture of the finished dosage form April 1996. 1996
   Google Scholar
• Saimbi S, Madden V, Stirling H, et al. Comparison of hydrocortisone 10 mg tablets: tablet hardness optimised for adult use has negative consequences for paediatric use. Arch Dis Child. 2016;101:e2.
   PubMedGoogle Scholar
• Barillas JE, Eichner D, Van Wagoner R, et al. Iatrogenic Cushing syndrome in a child with congenital adrenal hyperplasia: erroneous compounding of hydrocortisone. J Clin Endocrinol Metab. 2018;103:7–11.
   PubMed Web of Science ®Google Scholar
• Hartman MF, Bötcher C, Wudy S, et al. Incorrect dosage of hydrocortisone in individually manufactured capsules by drugstores: a dilemma in the therapy of children with CAH. Hormone Research in Paediatrics. 2010;74:163.
   Google Scholar
• Xavier AF, Moura EF, Azevedo WF, et al. Erosive and cariogenicity potential of pediatric drugs: study of physicochemical parameters. BMC Oral Health. 2013;13:71.
   PubMed Web of Science ®Google Scholar
• EMA. Guideline on pharmaceutical development of medicines for paediatric use 1 August 2013 EMA/CHMP/QWP/805880/2012 Rev. 2 Committee for Medicinal Products for Human Use (CHMP) Paediatric Committee (PDCO). 2013
   Google Scholar
• Neumann U, Whitaker MJ, Wiegand S, et al. Absorption and tolerability of taste-masked hydrocortisone granules in neonates, infants and children under 6 years of age with adrenal insufficiency. Clin Endocrinol (Oxf). 2018 Jan;88(1):21-29.
   Web of Science ®Google Scholar
• Buttgereit F, Burmester GR, Straub RH, et al. Exogenous and endogenous glucocorticoids in rheumatic diseases. Arthritis Rheum. 2011;63:1–9.
   PubMed Web of Science ®Google Scholar
• Johnson TN, Whitaker MJ, Keevil B, et al. Bioavailability of oral hydrocortisone corrected for binding proteins and measured by LC-MS/MS using serum cortisol and salivary cortisone. J Bioequiv Availab. 2017;9:585–587.
   Google Scholar
• Melin J, Parra-Guillen ZP, Hartung N, et al. Predicting cortisol exposure from paediatric hydrocortisone formulation using a semi-mechanistic pharmacokinetic model established in healthy adults. Clin Pharmacokinet.
   Web of Science ®Google Scholar
• Finken MJ, Andrews RC, Andrew R, et al. Cortisol metabolism in healthy young adults: sexual dimorphism in activities of A-ring reductases, but not 11beta-hydroxysteroid dehydrogenases. J Clin Endocrinol Metab. 1999;84:3316–3321.
   PubMed Web of Science ®Google Scholar
• Brykalski J, Papierska L, Zaluska M. Acute psychosis in the course of treatment of acute adrenal crisis with hydrocortisone in the patient with secondary adrenal insufficiency - a case study. Psychiatr Pol. 2015;49:673–681.
   PubMed Web of Science ®Google Scholar
• Hogler W, Wudy SA, Luef G, et al. Oxcarbazepine accelerates cortisol elimination via cytochrome P450 3A4 induction. Arch Dis Child. 2010;95:1065.
   PubMed Web of Science ®Google Scholar
• Kara C, Ucakturk A, Aydin OF, et al. Adverse effect of phenytoin on glucocorticoid replacement in a child with adrenal insufficiency. J Pediatr Endocrinol Metab. 2010;23:963–966.
   PubMed Web of Science ®Google Scholar
• Kyriazopoulou V, Parparousi O, Vagenakis AG. Rifampicin-induced adrenal crisis in addisonian patients receiving corticosteroid replacement therapy. J Clin Endocrinol Metab. 1984;59:1204–1206.
   PubMed Web of Science ®Google Scholar
• Maguire AM, Ambler GR, Moore B, et al. Prolonged hypocortisolemia in hydrocortisone replacement regimens in adrenocorticotrophic hormone deficiency. Pediatrics. 2007;120:e164–e171.
   PubMed Web of Science ®Google Scholar
• Peters CJ, Hill N, Dattani MT, et al. Deconvolution analysis of 24-h serum cortisol profiles informs the amount and distribution of hydrocortisone replacement therapy. Clin Endocrinol (Oxf). 2013;78:347–351.
   PubMed Web of Science ®Google Scholar
• Han TS, Conway GS, Willis DS, et al., United Kingdom Congenital Adrenal Hyperplasia Adult Study E. Relationship between final height and health outcomes in adults with congenital adrenal hyperplasia: United Kingdom congenital adrenal hyperplasia adult study executive (CaHASE). J Clin Endocrinol Metab. 2014;99:E1547–E1555.
   PubMed Web of Science ®Google Scholar
• Simpson A, Hunter A. A congenital adrenal hyperplasia: parents’ experiences of treating their child’s condition. Endocrine Abstracts. 2016;41:GP140.
   Google Scholar
• Porter J, Blair J, Ross RJ. Is physiological glucocorticoid replacement important in children? Arch Dis Child. 2017;102:199–205.
   PubMed Web of Science ®Google Scholar
• Mallappa A, Sinaii N, Kumar P, et al. 2 study of Chronocort(R), a modified-release formulation of hydrocortisone, in the treatment of adults with classic congenital adrenal hyperplasia. J Clin Endocrinol Metab.
   Web of Science ®Google Scholar
• Jones CM, Mallappa A, Reisch N, et al. Modified-release and conventional glucocorticoids and diurnal androgen excretion in congenital adrenal hyperplasia. J Clin Endocrinol Metab. 2017;102:1797–1806.
   PubMed Web of Science ®Google Scholar
• Auchus RJ, Buschur EO, Chang AY, et al. Abiraterone acetate to lower androgens in women with classic 21-hydroxylase deficiency. J Clin Endocrinol Metab. 2014;99:2763–2770.
   PubMed Web of Science ®Google Scholar