RhIGF-1 treatment increases bone mineral density and trabecular bone structure in children with PAPP-A2 deficiency
Entity
UAM. Departamento de Pediatría; Instituto de Investigación del Hospital de La Princesa (IP)Publisher
S. Karger AG, BaselDate
2018-02-16Citation
10.1159/000486336
Hormone Research in Paediatrics 89.3 (2018): 200-204
ISSN
1663-2818 (Print); 1663-2826 (Online)DOI
10.1159/000486336Funded by
The authors are funded by Fondos de Investigación Sanitaria and FEDER (Grants PI1302195 and PI1600485 to J.A.), Ministerio de Ciencia e Innovación (BFU2014-51836-C2-2-R to J.A.C.), Centro de Investigación Biomédica en Red Fisiopatología de Obesidad y Nutrición (CIBEROBN), Instituto de Salud Carlos III (J.A.), and Fundación Endocrinología y NutriciónProject
Gobierno de España. PI1302195; Gobierno de España. PI1600485; Gobierno de España. BFU2014-51836-C2-2-REditor's Version
http://doi.org/10.1159/000486336Subjects
Bone density; Bone structure; IGF-1; PAPP-A2; RhIGF-1; MedicinaNote
KARGER: "This is the peer-reviewed but unedited manuscript version of the following article: Hormone Research in Paediatrics 89.3 (2018): 200-204 DOI: 10.1159/000486336. The final, published version is available at http://www.karger.com/. http://doi.org/10.1159/000486336]."Rights
© 2018 S. Karger AG, Basel. Copyright: All rights reserved.Abstract
Aim: Our objective was to determine changes in bone mineral density (BMD), trabecular bone score (TBS), and body composition after 2 years of therapy with recombinant human insulin-like growth factor-1 (rhIGF-1) in 2 prepubertal children with a complete lack of circulating PAPP-A2 due to a homozygous mutation in PAPP-A2 (p.D643fs25∗) resulting in a premature stop codon. Methods: Body composition, BMD, and bone structure were determined by dual-energy X-ray absorptiometry at baseline and after 1 and 2 years of rhIGF-1 treatment. Results: Height increased from 132 to 145.5 cm (patient 1) and from 111.5 to 124.5 cm (patient 2). Bone mineral content increased from 933.40 to 1,057.97 and 1,152.77 g in patient 1, and from 696.12 to 773.26 and 911.51 g in patient 2, after 1 and 2 years, respectively. Whole-body BMD also increased after 2 years of rhIGF-1 from baseline 0.788 to 0.869 g/cm2in patient 1 and from 0.763 to 0.829 g/cm2in patient 2. After 2 years of treatment, both children had an improvement in TBS. During therapy, a slight increase in body fat mass was seen, with a concomitant increase in lean mass. No adverse effects were reported. Conclusion: Two years of rhIGF-1 improved growth, with a tendency to improve bone mass and bone microstructure and to modulate body composition.
Files in this item
Google Scholar:Hawkins, Federico G.
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Muñoz-Calvo, María T.
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Martos Moreno, Gabriel Ángel
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Allo-Miguel, Gonzalo
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Del Río, Luis
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Pozo Román, Jesús
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Chowen, Julie Ann
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Pérez-Jurado, Luis A.
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Argente Oliver, Jesús
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