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Transforming tallness: how sex steroids influence final height in Marfan syndrome
Adriana Banhos Carneiro Yamaguchi1, Adriana Aparecida Siviero-Miachon2, Paola Matiko Martins Okuda3
1Universidade Federal de São Paulo (UNIFESP), Escola Paulista de Medicina (EPM), Setor de Endocrinologia Pediátrica, São Paulo, SP, Brazil.
Objective:
To evaluate the effects of hormonal treatment with sex steroids on the final height of patients with tall stature, including those diagnosed with Marfan Syndrome (MS), over 15 years in an outpatient setting.
Methods:
This retrospective cohort study reviewed the medical records of patients referred for tall stature. Descriptive statistics characterized the samples, while independent and paired t-tests assessed changes in final height (FH) and height at the start of treatment (HTS). One-way analysis of variance (ANOVA) evaluated the impact of chronological age at the initiation of therapy, bone age at the start of treatment, and pubertal stage on FH and HTS.
Results:
A total of 55 individuals with tall stature (51 % male) were included, among whom 35 (64 %) had clinically confirmed MS. Of these, 34 (62 %) received low-dose steroid treatment. Patients treated during pre-puberty exhibited an average height increase of 25.56 cm (95 %CI 20.40-30.73; p < 0.001; d = 2.86), while those treated during puberty showed an average gain of 11.93 cm (95 %CI 8.69-15.18; p < 0.001; d = 1.72). Early treatment before the age of 10 resulted in height gains of 13.92 cm (95 %CI 4.90-22.93; p = 0.006; d = 1.82) with estrogen and 6.8 cm (95 %CI 1.71-11.88; p = 0.010; d = 0.73) with testosterone.
Conclusions:
Early intervention with low doses of steroids significantly reduced final height in individuals with tall stature, including those with MS, while also minimizing dose-dependent adverse effects.
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