Results of long-term growth hormone replacement therapy in children: when and how to treat?
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1Cochin Medical School, Paris, France.
Insights
Early diagnosis and treatment are crucial for children with growth hormone deficiency (GHD). Optimal growth hormone (GH) replacement doses require further investigation for long-term effectiveness.
Area of Science:
- Pediatric Endocrinology
- Growth Hormone Therapy
- Childhood Growth Disorders
Background:
- Treatment outcomes in growth hormone deficiency (GHD) are suboptimal, often due to delayed diagnosis.
- Delayed diagnosis leads to insufficient height before puberty, limiting catch-up growth potential.
- Growth hormone (GH) replacement therapy's efficacy diminishes over time, highlighting the need for optimized dosing.
Purpose of the Study:
- To emphasize the critical need for early diagnosis and intervention in GHD.
- To investigate the dose-response relationship of GH replacement therapy.
- To present initial findings from a long-term prospective dose-response study.
Main Methods:
- Analysis of height gain in relation to GH dosage.
- Comparison of GH administration frequency (3 vs. 6 times/week).
- Evaluation of dose-response over the initial treatment period.
Main Results:
- Height gain in the first year correlates with GH dose logarithm.
- A better initial dose-response is observed with GH administered 6 times/week.
- Long-term dose-response relationships and final height outcomes remain to be determined.
Conclusions:
- Early diagnosis and treatment initiation are paramount for improving GHD outcomes.
- GH replacement therapy shows initial efficacy that wanes over time.
- Further long-term studies are essential to establish optimal GH dosing strategies.
Abstract:
Up to now, the end results of treatment in GH deficiency (GHD) have not been as good as expected. Probably the main cause has been the delay in diagnosing GHD in short children, resulting in a very insufficient height at the onset of puberty. The need for early diagnosis and treatment must be emphasized. This agrees with the well-documented data demonstrating (1) that catch-up growth obtained in any treatable growth disorder has a limited duration, and (2) that the effect of GH replacement, whatever the doses and modes of therapy, is maximal during the first year and wanes during the following years. There are still many questions regarding the appropriate GH replacement dose. The height gain during the first year of treatment has been correlated with the logarithm (decimal or natural) of the dose in several groups of patients receiving GH 3 times/week. The initial dose-response relationship is better with GH 6 times/week. For the following years, no dose/response ratio has been demonstrated, and the final height-to-dose relations are still to be investigated. A dose increase after 2 years or more may improve the growth rate, but also with secondary waning. Thus, long-term prospective dose-response studies are needed. The first period of one such study is presented.(ABSTRACT TRUNCATED AT 250 WORDS)