ESPE Abstracts (2014) 82 WG8.4

Inequities of Treatment Options in Developing Countries: Congenital Adrenal Hyperplasia

Margaret Zacharin

Royal Children’s Hospital, Parkville, Victoria, Australia

Adrenal insufficiency of any cause results in major morbidity and increased lifetime risk for adrenal crisis and potential mortality. A majority of those affected by disorders of either congenital or acquired origin have deficiencies of both glucocorticoid and mineralocorticoid. Preferred replacement treatment for infants, children and adolescents with growth potential is considered to be with hydrocortisone and fludrocortisone.

Both medications are advocated for optimization of linear growth but neither are readily available or able to be procured in many countries including Africa, Eastern Europe, South America, and parts of south East Asia. Despite both being listed on WHO list of essential medicines for the past 6 years, little progress has been made to improve availability and distribution of either.

Where hydrocortisone is not available, appropriate titration of prednisolone can be a satisfactory management strategem for corticosteroid replacement but no alternative is available for fludrocortisone. While pressure is put on governments to provide hydrocortisone, less attention has been paid to mineralocorticoid, although lifetime safety and normalization of linear growth are also dependent upon prevention of hyponatraemia.

Early identification of CAH reduces infant mortality but renders the affected child permanently corticosteroid dependent, another dilemma for families living in resource constrained environments.

Strategic plans directed towards improvement in equitable availability of these essential medicines will require a central model with in principle agreement with the WHO, to reduce morbidity and mortality of children with adrenal insufficiency worldwide.

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