Background: The primary goal of treatment is to achieve a final height within the normal range and avoid the physical and psychological consequences of short stature (SS). If after a time, treatment efficacy criteria are not achieved, it is necessary to revalue its usefulness. There is no consensus about which conduct should be adopted in these cases. An alternative, is the suspension of rhGH treatment and evaluate the clinical and biochemical results to decide to continue or discontinue treatment definitively. This discontinuation of rhGH treatment, we have called transient treatment suspension (TTS).
Objective and hypotheses: Determine the characteristics of a group of prepubertals patients undergoing TTS with rhGH and establish its usefulness in clinical practice.
Method: It is a retrospective study of a group of patients, which indicated TTS with rhGH (n: 41).
Results: 78% of patients were male. 70% had diagnosis of IGHD and the remaining SGA. The average age of the onset of treatment with rhGH was 7.4±2.5 years for IGHD and 4.7±1.7 years for SGA. The mean rhGH dose used was 0.22 mg/kg per week. The mean onset of TTS was to 9.5 years. The mean duration of TTS was 11.3 months. Treatment was restored in 76% of patients, because they showed a significant deterioration in their growth during the TTS period respect to Pre-TTS, both in GV, H, and IGF-1 levels. A significant recovery of the three parameters after 12 months of restoration of rhGH treatment was achieved. In the remaining 24%, rhGH was not restarted due to it was not observed significant difference in GV, H and IGF-1, compared to Pre-TTS. Through ROC curve analysis observed that differences in GV (SDS) between Pre-TTS and TTS period is a useful parameter to identify dependent rhGH patients (AUC=0.876, P<0.0001). A decrease ≥0.97 in GV-SDS between the Pre-TTS and TTS period was the best cut-off point associated with the reinstated of treatment (sensitivity 80%, specificity 80%).
Conclusion: Based on our results, we conclude that TTS may help us to decide to continue or definitively suspend treatment. Thus, the costs and inconveniences caused by the daily administration of an ineffective treatment would be reduced.
10 - 12 Sep 2016
European Society for Paediatric Endocrinology