title: Height outcome of short children with hypochondroplasia after recombinant human growth hormone treatment: a meta-analysis.
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.
authors.affiliation: Pediatric Unit, Maternal & Infant Department, St. Chiara University Hospital of Pisa, Pisa, Italy.
authors.forename: Francesco
authors.initials: F
authors.lastname: Massart
authors.affiliation: Epidemiology Unit, Department of Experimental Pathology M.B.I.E., University of Pisa, 56126 Pisa PI, Italy.
authors.forename: Mario
authors.initials: M
authors.lastname: Miccoli
authors.affiliation: Epidemiology Unit, Department of Experimental Pathology M.B.I.E., University of Pisa, 56126 Pisa PI, Italy.
authors.forename: Angelo
authors.initials: A
authors.lastname: Baggiani
authors.affiliation: Pediatric Unit, Maternal & Infant Department, St. Chiara University Hospital of Pisa, Pisa, Italy.
authors.forename: Silvano
authors.initials: S
authors.lastname: Bertelloni
mesh_terms: D001827:Body Height / Q000187:drug effects* / Q000235:genetics; D001842:Bone and Bones / Q000002:abnormalities*; D002648:Child; D004392:Dwarfism / Q000175:diagnosis* / Q000188:drug therapy* / Q000235:genetics; D019382:Human Growth Hormone / Q000494:pharmacology / Q000627:therapeutic use*; D006801:Humans; D017880:Limb Deformities, Congenital / Q000175:diagnosis* / Q000188:drug therapy* / Q000235:genetics; D008141:Lordosis / Q000175:diagnosis* / Q000188:drug therapy* / Q000235:genetics; D016032:Randomized Controlled Trials as Topic / Q000379:methods; D011994:Recombinant Proteins / Q000494:pharmacology / Q000627:therapeutic use; D016896:Treatment Outcome
chemical_list: D011994:Recombinant Proteins; D019382:Human Growth Hormone
keywords: IGF-1; growth hormone therapy; height outcome; hypochondroplasia; meta-analysis; skeletal dysplasia