Safety and efficacy of oxandrolone in growth hormone-treated girls with Turner syndrome: evidence from recent studies and recommendations for use
Open accessAbstract
There has been no consensus regarding the efficacy and safety of oxandrolone (Ox) in addition to growth hormone (GH) in girls with Turner syndrome (TS), the optimal age of starting this treatment, or the optimal dose. This collaborative venture between Dutch, UK and US centers is intended to give a summary of the data from three recently published randomized, placebo-controlled, double-blind studies on the effects of Ox. The published papers from these studies were reviewed within the group of authors to reach consensus about the recommendations. The addition of Ox to GH treatment leads to an increase in adult height, on average 2.3–4.6 cm. If Ox dosages<0.06 mg/kg/day are used, side effects are modest. The most relevant safety concerns are virilization(including clitoromegaly and voice deepening) and a transient delay of breast development. We advise monitoring signs of virilization breast development and possibly blood lipids during Ox treatment, in addition to regular follow-up assessments for TS. In girls with TS who are severely short for age, in whom very short adult stature is anticipated,or in whom the growth rate is modest despite good compliance with GH, adjunctive treatment with Ox at a dosage of 0.03–0.05 mg/kg/day starting from the age of 8–10 years onward scan be considered.
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- Authors
- Sas, T. C. J., Gault, E. J., Zeger Bardsley, M., Menke, L. A., Freriks, K., Perry, R. J., Otten, B. J., de Muinck Keizer-Schrama, S. M. P. F., Timmers, H., Wit, J. M., Ross, J. L., & Donaldson, M. D. C.
- Affiliations
- Albert Schweitzer Ziekenhuis (NL)
- Royal Hospital for Children (GB)
- Leiden University Medical Center (NL)
- Radboud University Medical Center (NL)
- Thomas Jefferson University (US)
- Venue
- Hormone Research in Paediatrics
- Publisher
- S. Karger AG
- Published
- 2014
- Volume
- 81
- Issue
- 5
- Pages
- 289-297
- ISSN
- 1663-2818 (ISSN-L)
- 1663-2826
- OpenAlex
- W2102501531
- Cited by
- 55
- References
- 24
- Citations by year
- 2026: 1 · 2025: 1 · 2024: 6 · 2023: 2 · 2022: 2 · 2021: 8 · 2020: 4 · 2019: 10 · 2018: 4 · 2017: 4 · 2016: 4 · 2015: 9
- FWCI
- 3.71
- Type
- journal article
- Language
- en
- Open access
- green
- pure.amsterdamumc.nl
- Locations
- Hormone Research in Paediatrics (publishedVersion)
- PubMed (publishedVersion)
- Pure Amsterdam UMC (publishedVersion, other-oa)
- Data Archiving and Networked Services (DANS) (submittedVersion, other-oa)
- EUR Research Repository (Erasmus University Rotterdam) (submittedVersion)
- Data Archiving and Networked Services (DANS) (submittedVersion)
- Leiden Repository (Leiden University) (submittedVersion)
- ENLIGHTEN (Jurnal Bimbingan dan Konseling Islam) (submittedVersion)
- Radboud Repository (Radboud University) (submittedVersion)
- Topics
- 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
- Health Sciences › Medicine › Endocrinology, Diabetes and Metabolism › Growth Hormone and Insulin-like Growth Factors
- Keywords
- oxandrolone, Turner syndrome, growth hormone therapy, adult height, masculinization, clitoromegaly, breast development, treatment safety, treatment efficacy, dose recommendations, treatment timing, short stature, serum lipids
- MeSH terms
- Adolescent, Adult, Age Factors, Androgens, Child, Child, Preschool, Double-Blind Method, Female, Humans, Oxandrolone, Turner Syndrome, Randomized Controlled Trials as Topic, Human Growth Hormone
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