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Study design for final height determination in Turner syndrome: pros and cons
1Lilly Research Laboratories, Indianapolis, Ind. 46285.
Hormone Research
|January 1, 1993
Summary
Early research shows human growth hormone (hGH) treatment for Turner syndrome requires higher doses than for growth hormone deficiency (GHD) and yields less consistent results. Long-term trials are crucial for determining final height and optimal study designs.
Area of Science:
- Pediatric Endocrinology
- Genetics and Genetic Diseases
Background:
- Early studies investigated human growth hormone (hGH) for Turner syndrome treatment.
- Higher hGH doses were used compared to those for growth hormone deficiency (GHD).
Purpose of the Study:
- To review early hGH treatment studies in Turner syndrome.
- To evaluate study designs for assessing hGH efficacy in Turner syndrome.
- To discuss ethical considerations for long-term trials.
Main Methods:
- Review of early human growth hormone (hGH) treatment studies.
- Comparison of hGH dosage and response in Turner syndrome versus growth hormone deficiency (GHD).
- Analysis of different clinical trial designs (placebo-controlled, non-treatment-controlled, historical-controlled).
Main Results:
- Patients with Turner syndrome showed less consistent responses to hGH than GHD patients.
- Predicting final height in Turner syndrome using bone age remains uncertain.
- Long-term trials are essential to definitively answer final height questions.
Conclusions:
- Randomized placebo-controlled trials are deemed the most appropriate study design in the USA for Turner syndrome.
- Ethical considerations, including risk-benefit analysis and the necessity of a placebo group, are vital for long-term trials.