TY - JOUR
T1 - Uniparental disomy and pretreatment IGF-1 may predict elevated IGF-1 levels in Prader-Willi patients on GH treatment
AU - On behalf of the Genetic Obesity Study Group of the Italian Society of Pediatric Endocrinology and Diabetology
AU - Palmieri, Viviana Valeria
AU - Lonero, Antonella
AU - Bocchini, Sarah
AU - Cassano, Gilda
AU - Convertino, Alessio
AU - Corica, Domenico
AU - Crinò, Antonio
AU - Fattorusso, Valentina
AU - Ferraris, Silvio
AU - Fintini, Danilo
AU - Franzese, Adriana
AU - Grugni, Graziano
AU - Iughetti, Lorenzo
AU - Lia, Rosanna
AU - Macchi, Francesca
AU - Madeo, Simona Filomena
AU - Matarazzo, Patrizia
AU - Nosetti, Luana
AU - Osimani, Sara
AU - Pajno, Roberta
AU - Patti, Giuseppa
AU - Pellegrin, Maria Chiara
AU - Perri, Annamaria
AU - Ragusa, Letizia
AU - Rutigliano, Irene
AU - Sacco, Michele
AU - Salvatoni, Alessandro
AU - Scarano, Emanuela
AU - Stagi, Stefano
AU - Tornese, Gianluca
AU - Trifirò, Giuliana
AU - Wasniewska, Malgorzata
AU - Fischetto, Rita
AU - Giordano, Paola
AU - Licenziati, Maria Rosaria
AU - Delvecchio, Maurizio
PY - 2019/10/1
Y1 - 2019/10/1
N2 - Pediatric patients with Prader-Willi syndrome (PWS) can be treated with recombinant human GH (rhGH). These patients are highly sensitive to rhGH and the standard doses suggested by the international guidelines often result in IGF-1 above the normal range. We aimed to evaluate 1 the proper rhGH dose to optimize auxological outcomes and to avoid potential overtreatment, and 2 which patients are more sensitive to rhGH. In this multicenter real-life study, we recruited 215 patients with PWS older than 1 year, on rhGH at least for 6 months, from Italian Centers for PWS care. We collected auxological parameters, rhGH dose, IGF-1 at recruitment and (when available) at start of treatment. The rhGH dose was 4.3 (0.7/8.4) mg/m2/week. At recruitment, IGF-1 was normal in 72.1% and elevated in 27.9% of the patients. In the group of 115 patients with IGF-1 available at start of rhGH, normal pretreatment IGF-1 and uniparental disomy were associated with elevated IGF-1 during the therapy. No difference in height and growth velocity was found between patients treated with the highest and the lowest range dose. The rhGH dose prescribed in Italy seems lower than the recommended one. Normal pretreatment IGF-1 and uniparental disomy are risk factors for elevated IGF-1. The latter seems to be associated with higher sensitivity to GH. In case of these risk factors, we recommend a more accurate titration of the dose to avoid overtreatment and its potential side effects.
AB - Pediatric patients with Prader-Willi syndrome (PWS) can be treated with recombinant human GH (rhGH). These patients are highly sensitive to rhGH and the standard doses suggested by the international guidelines often result in IGF-1 above the normal range. We aimed to evaluate 1 the proper rhGH dose to optimize auxological outcomes and to avoid potential overtreatment, and 2 which patients are more sensitive to rhGH. In this multicenter real-life study, we recruited 215 patients with PWS older than 1 year, on rhGH at least for 6 months, from Italian Centers for PWS care. We collected auxological parameters, rhGH dose, IGF-1 at recruitment and (when available) at start of treatment. The rhGH dose was 4.3 (0.7/8.4) mg/m2/week. At recruitment, IGF-1 was normal in 72.1% and elevated in 27.9% of the patients. In the group of 115 patients with IGF-1 available at start of rhGH, normal pretreatment IGF-1 and uniparental disomy were associated with elevated IGF-1 during the therapy. No difference in height and growth velocity was found between patients treated with the highest and the lowest range dose. The rhGH dose prescribed in Italy seems lower than the recommended one. Normal pretreatment IGF-1 and uniparental disomy are risk factors for elevated IGF-1. The latter seems to be associated with higher sensitivity to GH. In case of these risk factors, we recommend a more accurate titration of the dose to avoid overtreatment and its potential side effects.
KW - Adverse effects
KW - Growth hormone therapy
KW - IGF-1
KW - Prader-Willi syndrome
KW - Uniparental disomy
UR - http://www.scopus.com/inward/record.url?scp=85071645104&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85071645104&partnerID=8YFLogxK
U2 - 10.1016/j.ghir.2019.08.003
DO - 10.1016/j.ghir.2019.08.003
M3 - Article
AN - SCOPUS:85071645104
VL - 48-49
SP - 9
EP - 15
JO - Endocrinology and Metabolism, Supplement
JF - Endocrinology and Metabolism, Supplement
SN - 1096-6374
ER -