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
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.
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