Near adult height in prepubertal children treated with growth hormone: differences between definite growth hormone deficiency (dGHD), short stature unresponsive to stimulation tests (SUS) and idiopathic short stature (ISS)

Gianluca Tamaro; Lanzetta Maria Andrea; Carlsson Martin Ove; Torre Daria La; Tresoldi Alberto Stefano; Pietro Bruschini; Gianluca Tornese

P1-59

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ESPE Abstracts (2024) 98 P1-59

ESPE2024 Poster Category 1 GH and IGFs 1 (11 abstracts)

Near adult height in prepubertal children treated with growth hormone: differences between definite growth hormone deficiency (dGHD), short stature unresponsive to stimulation tests (SUS) and idiopathic short stature (ISS)

Gianluca Tamaro ^1,2 , Maria Andrea Lanzetta ² , Martin Ove Carlsson ³ , Daria La Torre ⁴ , Alberto Stefano Tresoldi ⁴ , Pietro Bruschini ⁴ & Gianluca Tornese ^1,2

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¹Institute for Maternal and Child Health IRCCS Burlo Garofolo, Trieste, Italy. ²University of Trieste, Trieste, Italy. ³Pfizer inc., New York, USA. ⁴Pfizer srl, Rome, Italy

Objective: To understand whether characterization into definite growth hormone deficiency (dGHD; peak GH <8 ug/L and identifiable genetic, functional or anatomical cause), short stature unresponsive to stimulation test (SUS; peak GH <8 ug/L but without any identifiable cause) or idiopathic short stature (ISS; peak GH ≥8) can predict the near adult height (NAH) of prepubertal children treated with recombinant human growth hormone (rhGH).

Study design: Search on the KIGS (Pfizer International Growth Database) observational data set revealed 3,939 children with treatment with rhGH started before puberty.

Results: Data are reported in the Table. At multiple regression analysis, taking into account height SDS at GH start, birth weight, max GH peak at stimulation test, treatment duration, sex and rhGH dose, the increase in height was 0.13 SDS for SUS and 0.12 SDS for dGHD, compared to 0 SDS for ISS.


	dGHD (n = 1,401)	SUS (n = 1,300)	ISS (n = 1,238)	p-value dGHD vs.SUS:	p-value dGHD vs.ISS	p-value SUS vs.ISS
Birth weight (SDS)	-0.46±1.31	-0.69±1.25	-0.81±1.20	<0.01	<0.01	<0.05
Male sex (%)	60.0	65.8	61.1	<0.01	NS	<0.05
MPH (SDS)	-0.69±1.23	-1.19±1.23	-1.38±1.19	<0.01	<0.01	<0.01
Max GH peak (ug/L)	3.25±2.30	4.89±2.12	13.87±8.38	<0.01	<0.01	<0.01
rhGH start Age (yrs)	7.86±3.08	8.29±2.70	8.68±2.47	<0.01	<0.01	<0.01
Height (SDS)	-3.27±1.56	-3.30±1.08	-3.14±0.89	NS	<0.05	NS
Height – MPH (SDS)	-2.60±1.64	-2.10±1.38	-1.75±1.23	<0.01	<0.01	<0.01
IGF-1 (SDS)	-2.44±1.50	-1.95±1.23	-1.79±1.39	<0.01	<0.01	NS
BMI (SDS)	0.17±1.40	-0.36±1.21	-0.49±1.17	<0.01	<0.01	NS
Dose (mcg/kg/day)	29.59±11.49	31.44±10.28	31.26±9.52	<0.01	<0.01	NS
NAH
Age (yrs)	18.00±1.92	17.17±1.51	17.18±1.58	<0.01	<0.01	NS
Duration of treatment (yrs)	9.47±3.11	8.40±2.47	7.91±2.18	<0.01	<0.01	<0.01
Height (SDS)	-1.15±1.44	-1.37±1.20	-1.66±1.17	<0.01	<0.01	<0.01
Delta height (NAH – start) (SDS)	2.13±1.63	1.93±1.08	1.49±0.94	<0.01	<0.01	<0.01
IGF-1 (SDS)	-2.81±2.33	-1.48±2.26	-1.37±1.54	<0.01	<0.01	NS
BMI (SDS)	0.34±1.50	0.03±1.24	-0.18±1.20	<0.01	<0.01	<0.01
Dose (mcg/kg/day)	27.52±8.01	30.73±9.26	32.29±9.52	<0.01	<0.01	<0.01

Conclusions: Children with SUS had almost the same response to rhGH compared to dGHD in terms of NAH, although they differ in baseline characteristics, such as birth weight, MPH, IGF-1 levels and BMI SDS.