| Literature DB >> 26555758 |
Francesco Massart1, Mario Miccoli2, Angelo Baggiani2, Silvano Bertelloni1.
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.Entities:
Keywords: IGF-1; growth hormone therapy; height outcome; hypochondroplasia; meta-analysis; skeletal dysplasia
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Year: 2015 PMID: 26555758 DOI: 10.2217/pgs.15.129
Source DB: PubMed Journal: Pharmacogenomics ISSN: 1462-2416 Impact factor: 2.533