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Growth hormone treatment before the age of 4 years prevents short stature in young girls with Turner syndrome.

Abstract：

OBJECTIVE:Adult height deficit seen in Turner syndrome (TS) originates, in part, from growth retardation in utero and throughout the first 3 years of life. Earlier diagnosis enables earlier therapeutic intervention, such as with recombinant human GH (r-hGH), which may help to prevent growth retardation. In this open-label, multicentre phase III study, we investigated efficacy and safety in r-hGH treatment in young girls with TS. SUBJECTS AND METHODS:Girls (n=61) aged <4 years with TS receiving 0.035-0.05 mg/kg per day r-hGH for 4 years were compared with an historical control group (n=51) comprising untreated, age- and height-matched girls with TS. The main outcome measure was change in height SDS (H-SDS). Other measures included changes in height velocity SDS, IGF1 levels and glucose metabolism. RESULTS:After 4 years, a gain in mean H-SDS of 1.0 SDS (from -2.33±0.73 to -1.35±0.86 SDS) was observed with r-hGH treatment, in contrast to the decrease in mean H-SDS of 0.3 SDS in the control group (from -2.09±0.81 to -2.44±0.73 SDS; P<0.0001). r-hGH treatment was the main predictor of H-SDS gain and accounted for 52% of variability (multivariate analysis). r-hGH was well tolerated. As expected, IGF1 levels rose with treatment. A case of transient glucose intolerance resolved after dietary adaptation. CONCLUSION:Early treatment with r-hGH helps to prevent natural evolution towards short stature in most girls with TS. IGF1 levels and glucose metabolism should be monitored routinely during r-hGH therapy.

journal_name

Eur J Endocrinol

journal_title

European journal of endocrinology

authors

Linglart A,Cabrol S,Berlier P,Stuckens C,Wagner K,de Kerdanet M,Limoni C,Carel JC,Chaussain JL,French Collaborative Young Turner Study Group.

doi

10.1530/EJE-10-1048

subject

Has Abstract

pub_date

2011-06-01 00:00:00

pages

891-7

issue

6

eissn

0804-4643

issn

1479-683X

pii

EJE-10-1048

journal_volume

164

pub_type

杂志文章,多中心研究

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