Endocrine Abstracts

Introduction: National Institute of Health and Care Excellence (NICE) has provided guidance for the use of human recombinant GH in the treatment of growth failure in children. An ongoing National GH Audit was established in 2013 by BSPED to maintain a central database and gather information regarding trends in prescribing and facilitate future long-term follow up. This part of the audit looked at the trends of off label prescribing of GH.

Method: Data were collected from 79 centres across the UK who prescribed GH therapy for children <16 years of age. The data were collected on a quarterly basis and collated centrally for a year. Data collected included indication for starting GH, age, sex, GH dose, and ongoing prescriptions (GP/hospital).

Results: Of the total 816 children who were commenced on GH in 2013, 11% (93) were for off label indications. Boys (58%) were more likely to receive GH treatment for indications outside NICE recommendations. Short stature (24%) was the most common indication followed by syndromic or genetic causes of extreme short stature (18%) and Noonan’s syndrome (10%). Majority of the off label prescriptions were from England (80%) followed by Scotland (19%) with none reported from Northern Ireland. In England most of the off label prescriptions were from Merseyside (37%), followed by Central Manchester (16%), North East England (11%), and West Midlands (9%). 57 (60%) of these prescriptions were funded by the hospital and the remainder (40%) by clinical commissioning groups.

Conclusion: A significant number of prescriptions for GH therapy in children are outside NICE recommendations. Long-term follow up is essential to establish the actual benefit and effects of GH in this cohort of patients.