Growth hormone treatment in a regional centre: licensed and unlicensed indications

Mandy Whitehead; Xanthippi Tseretopoulou; Emily Cottrell-Jane; Sabah Alvi; Talat Mushtaq

doi:10.1530/endoabs.51.P053

Introduction: In the UK, GH therapy is licensed for use in GH deficiency, Turner Syndrome, Small for Gestational Age (SGA), Prader Willi Syndrome (PWS), SHOX deletion and Chronic Renal Failure (CRF). Worldwide there are a number of additional indications. The aim was to review the use of GH prescriptions in relation to indications and to evaluate if there were similarities or differences between the licensed and unlicensed groups.

Methods: All children started on GH over a 4 year period from 2013 to 2017 in a large tertiary hospital were reviewed. The primary indication was recorded and the pre-treatment height, weight and BMI SDS were calculated.

Results: 167 children had GH (94 male, 73 females). 47% were for GHD. The median age for starting GH was 7.2 years. The median height SDS was −3.2 SDS, with the unlicensed group having the lowest median heights of all groups (−3.5 SDS). 32 (19%) of the children (22 male) did not have a licensed indication. Some uses included children with syndromic short stature (7), Russell Silver Syndrome (RSS)(3), Juvenile Idiopathic Arthritis (1), 3M (1), Crohns (1). No cause was apparent in the majority of this group.


Indication	GHD	SGA	Turner	CRF	PWS	Unlicensed	Total
Number (% of total)	79 (47%)	24 (14%)	19 (11%)	8 (8%)	4 (2%)	32 (19%)	167 (100%)
Age. Median (10th, 90th)	6.6 2.8, 15.1	7.9 3.8, 11.7	7.1 3.5, 15.8	11.6	3.5	7.6 4.6, 15.6	7.2 3.3, 14.8
Height SDS. Median (10th:90th)	−3.1 −4.4, −1.4	−3.3 −5.1, −2.2	−2.9 −3.9, −1.2	−3.0	−2.0	−3.5 −4.8, −2.5	−3.2 −4.8, −1.8
Weight SDS. Median (10th:90th)	−1.8 −3.6, 0.9	−3.3 −6.0, −1.2	−1.7 −2.8, 0.0	−1.2	2.4	−2.9 −4.2, −1.7	−2.1 −4.2, 0.7
BMI SDS. Median (10th:90th)	0.8 −1.1, 2.6	−1.0 −2.9, 1.4	0.4 −1.1, 1.9	0.9	4.3	−0.4 −2.0, 1.1	0.5 −1.7, 2.4

Conclusions: The vast majority of GH use is for licensed indications. Thorough evaluations can potentially move children from licensed to unlicensed indications. The unlicensed group had the shortest heights. This indicates that there is judicious use for treatment depending on the individual circumstances in a centre serving a large population with complex pathologies.

Endocrine Abstracts

P053