Uniparental disomy and pretreatment IGF-1 may predict elevated IGF-1 levels in Prader-Willi patients on GH treatment

On behalf of the Genetic Obesity Study Group of the Italian Society of Pediatric Endocrinology and Diabetology

Research output: Contribution to journalArticle

Abstract

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.

Original languageEnglish
Pages (from-to)9-15
Number of pages7
JournalGrowth Hormone and IGF Research
Volume48-49
DOIs
Publication statusPublished - Oct 1 2019

Fingerprint

Uniparental Disomy
Insulin-Like Growth Factor I
Prader-Willi Syndrome
Therapeutics
Italy
Reference Values
Guidelines
Pediatrics

Keywords

  • Adverse effects
  • Growth hormone therapy
  • IGF-1
  • Prader-Willi syndrome
  • Uniparental disomy

ASJC Scopus subject areas

  • Endocrinology, Diabetes and Metabolism
  • Endocrinology

Cite this

On behalf of the Genetic Obesity Study Group of the Italian Society of Pediatric Endocrinology and Diabetology (2019). Uniparental disomy and pretreatment IGF-1 may predict elevated IGF-1 levels in Prader-Willi patients on GH treatment. Growth Hormone and IGF Research, 48-49, 9-15. https://doi.org/10.1016/j.ghir.2019.08.003

Uniparental disomy and pretreatment IGF-1 may predict elevated IGF-1 levels in Prader-Willi patients on GH treatment. / On behalf of the Genetic Obesity Study Group of the Italian Society of Pediatric Endocrinology and Diabetology.

In: Growth Hormone and IGF Research, Vol. 48-49, 01.10.2019, p. 9-15.

Research output: Contribution to journalArticle

On behalf of the Genetic Obesity Study Group of the Italian Society of Pediatric Endocrinology and Diabetology 2019, 'Uniparental disomy and pretreatment IGF-1 may predict elevated IGF-1 levels in Prader-Willi patients on GH treatment', Growth Hormone and IGF Research, vol. 48-49, pp. 9-15. https://doi.org/10.1016/j.ghir.2019.08.003
On behalf of the Genetic Obesity Study Group of the Italian Society of Pediatric Endocrinology and Diabetology. Uniparental disomy and pretreatment IGF-1 may predict elevated IGF-1 levels in Prader-Willi patients on GH treatment. Growth Hormone and IGF Research. 2019 Oct 1;48-49:9-15. https://doi.org/10.1016/j.ghir.2019.08.003
On behalf of the Genetic Obesity Study Group of the Italian Society of Pediatric Endocrinology and Diabetology. / Uniparental disomy and pretreatment IGF-1 may predict elevated IGF-1 levels in Prader-Willi patients on GH treatment. In: Growth Hormone and IGF Research. 2019 ; Vol. 48-49. pp. 9-15.
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AU - Palmieri, Viviana Valeria

AU - Lonero, Antonella

AU - Bocchini, Sarah

AU - Cassano, Gilda

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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

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AU - Giordano, Paola

AU - Licenziati, Maria Rosaria

AU - Delvecchio, Maurizio

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KW - Adverse effects

KW - Growth hormone therapy

KW - IGF-1

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