PMID- 10509364
OWN - NLM
STAT- MEDLINE
DCOM- 20041025
LR  - 20120605
IS  - 0031-3998 (Print)
IS  - 0031-3998 (Linking)
VI  - 46
IP  - 4
DP  - 1999 Oct
TI  - Treatment of achondroplasia with growth hormone: six years of experience.
PG  - 435-9
AB  - We describe the effects of recombinant hGH (r-hGH) therapy for up to 6 y on 
      stature and body proportions of 35 children with achondroplasia (Ach). 
      Consecutive height (Ht) measurements were plotted on disease-specific Ach growth 
      curves, but age and sex SD scores (SDS) of Ht, sitting Ht, subischial leg length, 
      and Ht velocity were made with respect to Tanner normal standards. r-hGH was 
      administered by daily subcutaneous injections at a median (range) dose of 30 
      (15.8-40) U/m2 per week [0.06 (0.04-0.08) mg.kg(-1).24 h(-1)]. Patients were 
      treated for 3 (1-6) y from age 2.25 (1.2-9.3) y. Before treatment, Ht SDS was 
      -4.6 (-6.5 to -3.24). Treatment caused a significant increase in Ht SDS year to 
      year until y 4 (ANOVA F = 46.94; p < 0.01) that was subsequently sustained with 
      no significant further change (y 5 and 6 versus y 4, p > 0.05). When the response 
      to r-hGH was also expressed as a change in Ht velocity, there was a significant 
      increase in the first year of therapy that was maintained over subsequent 
      treatment years (ANOVA = 4.28, p = 0.001). Age was the most important variable 
      accounting for the first-year response in Ht SDS (r2 = 0.41, p < 0.001), and dose 
      of r-hGH did not influence this. Increments in sitting Ht SDS were greater than 
      subischial leg length SDS (F = 26.25, p < 0.001; F = 9.04, p < 0.001, 
      respectively). r-hGH treatment improved the Ht position of Ach children relative 
      to their normal and Ach peers without obvious side effects. A young age at 
      initiation of therapy prevented the characteristic Ht deficit from accumulating. 
      The greater increase in spinal Ht accentuated the existing disproportion. The 
      addition of later surgical leg lengthening could offer the possibility of 
      proportionate adult stature just within the normal range.
FAU - Ramaswami, U
AU  - Ramaswami U
AD  - London Centre for Paediatric Endocrinology, Middlesex Hospital, United Kingdom.
FAU - Rumsby, G
AU  - Rumsby G
FAU - Spoudeas, H A
AU  - Spoudeas HA
FAU - Hindmarsh, P C
AU  - Hindmarsh PC
FAU - Brook, C G
AU  - Brook CG
LA  - eng
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
PL  - United States
TA  - Pediatr Res
JT  - Pediatric research
JID - 0100714
RN  - 0 (Receptors, Fibroblast Growth Factor)
RN  - 0 (Recombinant Proteins)
RN  - 12629-01-5 (Human Growth Hormone)
RN  - EC 2.7.10.1 (FGFR3 protein, human)
RN  - EC 2.7.10.1 (Protein-Tyrosine Kinases)
RN  - EC 2.7.10.1 (Receptor, Fibroblast Growth Factor, Type 3)
SB  - IM
MH  - Achondroplasia/*drug therapy/genetics/pathology
MH  - Body Height/drug effects
MH  - Child
MH  - Child, Preschool
MH  - Female
MH  - Human Growth Hormone/*therapeutic use
MH  - Humans
MH  - Infant
MH  - Leg/pathology
MH  - Male
MH  - Middle Aged
MH  - Point Mutation
MH  - Protein-Tyrosine Kinases/genetics
MH  - Receptor, Fibroblast Growth Factor, Type 3
MH  - Receptors, Fibroblast Growth Factor/genetics
MH  - Recombinant Proteins/therapeutic use
EDAT- 1999/10/06 00:00
MHDA- 2004/10/27 09:00
CRDT- 1999/10/06 00:00
PHST- 1999/10/06 00:00 [pubmed]
PHST- 2004/10/27 09:00 [medline]
PHST- 1999/10/06 00:00 [entrez]
AID - 10.1203/00006450-199910000-00012 [doi]
PST - ppublish
SO  - Pediatr Res. 1999 Oct;46(4):435-9. doi: 10.1203/00006450-199910000-00012.