Table 1.

Current clinical endpoints, surrogate endpoints and biomarkers in GH therapy and acromegaly.

Patients and treatments	Clinical efficacy endpoints	Surrogate endpoints	Biochemical biomarker
Paediatric GH treatment
GH deficiency	Adult height	Change in height SDS/growth velocity	IGF-I
GH deficiency in the transition period	Body composition	Peak bone mass, DXA Z score, LBM, and FM	IGF-I
CRI, Noonan syndrome, SHOX, Turner syndrome, SGA, SRS, ISS	Adult height	Change in height SDS/growth velocity	IGF-I
Prader–Willi syndrome	Body compositionAdult height	Neuro-cognitionMuscle tone	IGF-I
Adult GH treatment	Body compositionQuality of life	Anthropometry, DXA Z score, LBM and FMQuestionnaires	IGF-I
Acromegaly treatment	Serum IGF-I and GH reductionSymptom relief	IGF-I, GHSymptom scores	IGF-I

CRI, chronic renal insufficiency; DXA, dual X-ray absorptiometry; FM, fat mass; IGF-I, insulin-like growth factor I; ISS, idiopathic short stature; LBM, lean body mass; SDS, standard deviation score; SGA, small for gestational age; SHOX, short stature homeobox deficiency; SRS, Silver–Russell syndrome.