Bone and Body Composition after one year of Vosorotide treatment in children with Achondroplasia: a longitudinal prospective study

Alessia Angelelli; Maria Allegri Anna Elsa; Caterina Tedesco; Nadia Maiorano; Alice Zucconi; Agnese Repetto; Gilda Gizzi; Flavia Napoli; Emilio Casalini; Daniela Fava; Maghnie Mohamad; Iorgi Natascia Di

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ESPE Abstracts (2024) 98 RFC2.1

ESPE2024 Rapid Free Communications Bone, Growth Plate and Mineral Metabolism (6 abstracts)

Bone and Body Composition after one year of Vosorotide treatment in children with Achondroplasia: a longitudinal prospective study.

Alessia Angelelli ^1,2 , Anna Elsa Maria Allegri ¹ , Caterina Tedesco ¹ , Nadia Maiorano ² , Alice Zucconi ² , Agnese Repetto ² , Gilda Gizzi ² , Flavia Napoli ¹ , Emilio Casalini ² , Daniela Fava ^1,2 , Maghnie Mohamad ^1,2 & Natascia Di Iorgi ^1,2

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¹Pediatric Endocrinology Unit, IRCCS Istituto Giannina Gaslini, Genova, Italy. ²D.I.N.O.G.M.I., Università degli studi di Genova, Genova, Italy

Background: Bone and body composition data in Achondroplasia (Ach) treated or not with vosorotide are lacking.

Aim: To assess longitudinally body composition and bone markers in children with Ach during Vosorotide treatment (V-Tx).

Method: Twenty-one patients (F=11, M=10) underwent DXA evaluation before (T0) and after 12 months of V-Tx (T12): bone mineral density-BMD (g/cm², Z-score), bone mineral content-BMC (g) at the lumbar spine (LS) and at the total body less head (TBLH), total fat mass and free fat mass (kg) were measured; BMAD (Z-score) at the LS was calculated based on Crabtree-2017. Height (SDS, Merker-2018) and pubertal evaluation (Tanner stage), were collected. Bone alkaline phosphate (bALP, ug/l) and telopeptide-C (CTx, ng/ml) were measured at each time.

Results: Median age at T0 was 8.7 yrs (6.99; 10.32). Three F and 2 M were pubertal at T0 and 7 patients (F=5, M=2) enterd puberty by T12. Overall anthropometric and DXA parameters are shown in the Table. Height SDS increased, whereas weight decreased at T12. Height-SDS increased similarly in M and prepubertal F, instead weight-SDS increased only in prepubertal M (P = 0.015). All bone parameters increased at the TBLH site, while remained stable at the LS except for BMD and BMC. Body fat increased while free fat mass decreased.


	Median (25°-75°) at 0 m	Median (25°-75°) at 12 m	P value
Median height (SDS)	-0.64 (-1.18; 0.12)	-0.33 (-0.75; 0.50)	0.0002
Median weight (SDS)	-0.65 (-1.41; 0.57)	-0.49 (-0.77; 0.72)	0.017
BMD-LS (g/cm²)	0.55 (0.51;0.63)	0.61 (0.54; 0.68)	0.020
BMD-LS (Z-score)	-1.50 (-2.06; -1.11)	-1.49 (-1.83; -1.12)	0.55
BMC-LS (g)	17.42 (12.80; 20.33)	18.08 (15.08; 22.78)	< 0.0001
BMAD (SDS)	-1.73 (-2.35; -1.17)	-1.82 (-2.50; -0.73)	0.91
BMD-TBLH (g/cm²)	0.48 (0.43;0.54)	0.52 (0.46; 0.59)	< 0.0001
BMD-TBLH (Z-score)	-2.44 (-2.86; -1.82)	-2.40 (-2.78; -1.89)	< 0.0001
BMC-TBLH (g)	402.5 (299.2; 551.0)	480.6 (363.5; 645.3)	< 0.0001
Total Fat Mass (kg)	4.84 (3.53; 6.49)	5.61 (4.54; 8.43)	0.0002
Total Lean Mass (kg)	11.30 (9.45; 13.40)	13.63 (10.80; 15.12)	< 0.0001

An increase in bALP was observed overall (P = 0.004) and in prepubertal F (P = 0.04) and post-pubertal F (P = 0.0009), while CTx increased at T12 only in the total cohort (P = 0.013).

Conclusion: Preliminary data show that 12 months V-Tx is associated with an increased height, TBLH bone and body composition DXA parameters and bone turnover markers in children with Ach; some gender differences might be present, however more longitudinal studies on larger numbers of subjects are warranted.