Height outcome of short children with hypochondroplasia after recombinant human growth hormone treatment: a meta-analysis. (November 2015)

• Record Type: Journal Article
• Authors: Massart, Francesco; Miccoli, Mario; Baggiani, Angelo; Bertelloni, Silvano
• Is Part Of: Pharmacogenomics. Volume 16:Number 17(2015)
• Page Start: 1965
• Page End: 1973
• Publication Date: 2015-11
• Subjects: growth hormone therapy -- height outcome -- hypochondroplasia -- IGF-1 -- meta-analysis -- skeletal dysplasia; Pharmacogenomics -- Periodicals; 615.1
• DOI: 10.2217/pgs.15.129
• Languages: English
• ISSNs: 1462-2416

Abstract

Hypochondroplasia (HCH) is a genetic skeletal dysplasia, characterized by rhizomelic short height (Ht) with facial dysmorphology and lumbar hyperlordosis. Albeit there are concerns that HCH children may not achieve optimal long-term outcome in response to recombinant human growth hormone (rhGH), anecdotal experiences suggested at least short-term Ht improvement. After thorough search of published studies, meta-analysis of rhGH use in HCH children was performed. In 113 HCH children, rhGH administration (median 0.25 mg/kg/week) progressively improved Ht pattern with 12 months catch-up growth (p < 0.0001). Then, Ht improvement resulted constant until 36 months (p < 0.0001), but stature remained subnormal. While bone age chronologically progressed, no serious adverse events were reported. In conclusion, our meta-analysis indicates that rhGH treatment progressively improved Ht outcome of HCH subjects.