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Growth hormone therapy in children: predictive factors and short-term and long-term response criteria
Gabriella Pozzobon1, Cristina Partenope1, Stefano Mora2
1Department of Pediatrics, IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University, Milan, Italy.
Insights
Growth hormone (GH) treatment is effective for short children with GH deficiency (GHD), with most reaching mid-parental height. However, defining poor responders remains challenging due to varying criteria and lack of predictive factors.
Area of Science:
- Pediatric Endocrinology
- Growth Hormone Therapy
- Childhood Growth Disorders
Background:
- The definition of growth response in children treated with growth hormone (GH) is debated.
- Growth hormone deficiency (GHD) affects childhood growth, necessitating effective treatment strategies.
Purpose of the Study:
- To evaluate the short-term and long-term efficacy of GH treatment in short children with GHD.
- To compare different criteria for assessing poor growth response to GH therapy.
- To identify factors that predict growth response in children with GHD.
Main Methods:
- Ninety-four children with isolated GHD received GH treatment until final height.
- Poor responder criteria included height gain SDS (Bang, Cianfarani) and height velocity (Bakker, Ranke).
- Data were analyzed to compare criteria and identify predictive factors.
Main Results:
- After one year, poor responder rates varied: 55.3% (Bang), 40.9% (Bakker), and 23.4% (Ranke).
- At final height, 22.34% were poor responders by Cianfarani criteria; 97.9% achieved mid-parental height (MPH).
- A negative association was found between height gain and age at diagnosis; no reliable predictive factors were identified.
Conclusions:
- The Bang criterion identified the most poor responders but had low predictive value.
- Ranke and Cianfarani criteria showed similar poor response rates.
- Despite challenges in defining poor responders, GH treatment demonstrated good efficacy, with most children achieving their genetic height potential (MPH).
Purpose:
The definition of growth response in growth hormone (GH)-treated children is controversial. This study aims at: (1) evaluating short-term and long-term efficacy of GH treatment in a cohort of short children with GH deficiency (GHD); (2) assessing and compare various poor response criteria; (3) identifying predictive factors of growth response.
Methods:
Our study included 94 children, affected by isolated GHD and treated with GH until they reached final height. Criteria used for calculating the proportion of poor responders to GH for the first year were gain in height (ΔHt) SDS < 0.5 ("Bang criterion"), <0.3 or <0.4 SDS for less-severe and severe GHD, respectively ("Ranke criterion"), height velocity (HV) < mean -1 SDS ("Bakker criterion"); for adult height "Cianfarani criterion" was total ΔHt < 1 SDS.
Results:
After 1 year of treatment we defined "poor responders" 55.3% of patients according to Bang criterion, 40.9% according to Bakker criterion and 23.4% according to Ranke criterion. At the end of the treatment, poor responders according to Cianfarani criterion were 22.34%; almost everyone in our population (97.9%) achieved mMid-parental height (MPH). Median final Ht was -1.11 SDS. Our analysis revealed a significant negative association between ΔHt and age at diagnosis.
Conclusions:
Bang criterion generated the highest number of poor responders, but had a low negative predictive value (67.5%); Ranke and Cianfarani criteria displayed similar rate of poor response. There is no reliable predictive factor of growth hormone response. However, almost all children treated reached MPH, suggesting good treatment efficacy.
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