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Efficacy of intermittent therapy in growth hormone-deficient children
1U 342 INSERM, Service d'Endocrinologie, Hôpital Saint Vincent de Paul, Paris, France.
Insights
Pausing recombinant growth hormone (rGH) therapy in children with growth hormone deficiency did not affect final height gain. Discontinuous rGH treatment may be as effective as continuous therapy, potentially improving patient comfort and reducing costs.
Area of Science:
- Pediatrics
- Endocrinology
- Growth Disorders
Background:
- Growth hormone deficiency (GHD) is a condition affecting childhood growth.
- Recombinant human growth hormone (rGH) is a standard treatment for GHD.
- Previous studies have focused on continuous rGH therapy regimens.
Purpose of the Study:
- To evaluate the impact of interrupted rGH therapy on final height gain in children with GHD.
- To compare the efficacy and dosage requirements of continuous versus discontinuous rGH treatment.
- To explore potential benefits of discontinuous rGH therapy on patient comfort and cost-effectiveness.
Main Methods:
- Retrospective analysis of growth data from 86 children with GHD.
- Comparison of growth outcomes between children receiving continuous rGH and those with a treatment pause.
- Analysis of cumulative rGH dosage and height gain over a 2-year period.
Main Results:
- Children who experienced a pause in rGH therapy achieved similar final height gain compared to those on continuous treatment.
- The discontinuous therapy group required significantly lower cumulative rGH doses (24 U/kg vs. 43 U/kg).
- A notable re-acceleration of growth rate was observed upon re-initiation of rGH therapy after a pause.
Conclusions:
- Discontinuous rGH administration may be a viable and potentially more efficient treatment strategy for GHD.
- Interrupted therapy could lead to reduced treatment costs and improved patient comfort without compromising final height.
- Further prospective randomized trials are warranted to confirm these findings and optimize discontinuous rGH regimens.
Abstract:
Eighty-six growth hormone-deficient children treated with extractive growth hormone were transferred to recombinant growth hormone (rGH): 57 children were transferred directly to rGH, but 29 experienced a 9.7 +/- 1-month pause in growth hormone administration. The retrospective analysis of growth from 1 year before to 1 year after initiation of rGH showed that the interruption of growth hormone administration did not modify the final height gain. During the 2 years, children with continuous therapy gained 12.2 +/- 0.5 cm with a cumulative growth hormone dose of 43 +/- 3 U/kg, while those who paused gained 12.1 +/- 0.3 cm with a cumulative growth hormone dose of only 24 +/- 2 U/kg (p < 0.0005). As expected, during the year preceding the onset of rGH, the children who paused gained less height than those treated continuously, but grew more rapidly during the first year of rGH administration. This was due to an important re-acceleration of growth rate at re-initiation of therapy after the pause. Our observation suggests that regimens of discontinuous rGH administration could be as efficient as continuous treatment. If confirmed in prospective randomized trials, this could have important consequences for improving the clinical efficiency of a given dose of rGH, as well as for the patient's comfort, secondary effects and cost of therapy.