Cited by 159Growth Hormone and Insulin-like Growth FactorsGenetic and Clinical Aspects of Sex Determination and Chromosomal AbnormalitiesSexual Differentiation and Disorders

Growth hormone and low dose estrogen in Turner syndrome: results of a United States multi-center trial to near-final height

Quigley, Charmian A., Crowe, Brenda J., Anglin, D. Greg, Chipman, John J.·2002·The Journal of Clinical Endocrinology & Metabolism

Abstract

A cardinal clinical feature of Turner syndrome (TS) is linear growth failure resulting in extreme short stature: the median adult height of untreated women with TS is 143 cm, 20 cm (8 in.) below that of the general female population. In the largest multicenter, randomized, long-term, dose-response study conducted in the United States, 232 subjects with TS received either 0.27 or 0.36 mg/kg.wk of recombinant human GH with either low dose ethinyl E2 or oral placebo. The study was placebo-controlled for both GH and estrogen for the first 18 months and remained placebo-controlled for estrogen for its duration. The near-final height of the 99 subjects whose bone age was at least 14 yr was 148.7 +/- 6.1 cm after 5.5 +/- 1.8 yr of GH started at a mean age of 10.9 +/- 2.3 yr; this represents an average increase of 1.3 +/- 0.6 SD scores from baseline (TS standard). Height was greater than 152.4 cm (60 in.) in 29% of subjects compared with the expected 5% of untreated patients. Mean near-final heights of subjects who received the lower GH dose, with or without estrogen, were 145.1 +/- 5.4 and 149.9 +/- 6.0 cm, respectively; those who received the higher GH dose with or without estrogen achieved mean near-final heights of 149.1 +/- 6.0 and 150.4 +/- 6.0 cm, respectively. Factors that most impacted outcome were younger age, lower bone age/chronological age ratio, lower body weight, and greater height SD score at study entry. This study demonstrates significant GH-induced improvement in height SD score, with correction of height to within the normal channels for a significant number of patients, and provides evidence of a GH dose-response effect. These data also indicate that early administration of estrogen, even at relatively low doses, does not improve gain in near-final height in patients with TS.

Integrity signals

Registry

Crossref · 10.1210/jcem.87.5.8477

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  • Only six RCTs have compared GH treatment with concurrent non-treatment or placebo control for at least one year.

    “Despite numerous studies of GH treatment in TS, only six randomized, controlled trials (RCTs) have compared GH treatment with a concurrent non-treatment or placebo control for at least one year157-162”
  • A 2007 Cochrane review concluded that girls treated with GH grew 3 cm/year more than untreated girls in the first 12-18 months.

    “a 2007 Cochrane Center review156 concluded that girls treated with GH grew 3 cm year−1 more than untreated girls in the first 12-18 months of therapy”
  • An additional RCT found no long-term growth benefit from prepubertal estrogen treatment.

    “An additional RCT that used a similar regimen but with higher ethinyl estradiol doses found no long-term growth benefit from prepubertal estrogen treatment.161”

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Authors
Quigley, C. A., Crowe, B. J., Anglin, D. G., & Chipman, J. J.
Affiliations
Eli Lilly (United States) (US)
Venue
The Journal of Clinical Endocrinology & Metabolism
Published
2002-05
Volume
87
Issue
5
Pages
2033-2041
ISSN
  • 0021-972X (ISSN-L)
  • 1945-7197
OpenAlex
W1997601373
Cited by
159
References
28
Citations by year
2026: 5 · 2025: 2 · 2024: 8 · 2023: 3 · 2022: 7 · 2021: 1 · 2020: 4 · 2019: 9 · 2018: 4 · 2017: 5 · 2016: 2 · 2015: 1 · 2014: 5 · 2013: 10 · 2012: 5
FWCI
5.54
Type
journal article
Language
en
Open access
closed
Locations
  • The Journal of Clinical Endocrinology & Metabolism (publishedVersion)
  • PubMed (publishedVersion)
Funders
Eli Lilly and Company
Topics
  • Health Sciences › Medicine › Endocrinology, Diabetes and Metabolism › Growth Hormone and Insulin-like Growth Factors
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Genetic and Clinical Aspects of Sex Determination and Chromosomal Abnormalities
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Sexual Differentiation and Disorders
Keywords
Turner syndrome, growth hormone therapy, recombinant human growth hormone, low-dose estrogen, ethinyl estradiol, short stature, dose-response relationship, bone age
MeSH terms
Body Height, Child, Dose-Response Relationship, Drug, Double-Blind Method, Ethinyl Estradiol, Female, Humans, Longitudinal Studies, Placebos, Recombinant Proteins, Safety, Turner Syndrome, Treatment Outcome, Human Growth Hormone, Estradiol Congeners

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