Primary response in GHD children treatment as a predictor for long-term 
therapy effectiveness

Jan M Kapała; Tomasz Maroszczuk; Aleksandra Sitarz; Anna Kącka; Dorota Charemska

doi:10.5114/pedm.2024.139270

eISSN: 2083-8441
ISSN: 2081-237X

Pediatric Endocrinology Diabetes and Metabolism

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abstract:

Original paper

Primary response in GHD children treatment as a predictor for long-term therapy effectiveness

Jan M Kapała

¹

,

Tomasz Maroszczuk

¹

,

Aleksandra Sitarz

¹

,

Anna Kącka

¹

,

Dorota Charemska

¹

Department of Clinical Pediatrics, Medical Faculty, University of Warmia and Mazury, Olsztyn, Poland

Pediatr Endocrinol Diabetes Metab 2024; 30

DOI: https://doi.org/10.5114/pedm.2024.139270

Online publish date: 2024/05/03

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Introduction
Short stature in growth hormone deficiency (GHD) can be treated with recombinant human growth hormone (rhGH), which is proven to be both safe and effective. However, a considerable number of patients does not achieve satisfying therapy outcomes.

Aim of the study
To evaluate the predictive effect of height increase in the first year of rhGH treatment on long-term therapy outcomes.

Material and methods
165 short-stature children (mean age 10.72 ±3.33 years; 63% males), diagnosed with GHD, treated with rhGH for at least one year (mean follow-up 4.32 ±1.80 years), divided into 2 groups according to the change in height standard deviation score (SDS) after the first year of rhGH treatment: good responders (GR) and poor responders (PR). Then, in one-year intervals, patient's chronological age, bone age, height, weight, insulin-like growth factor level, and rhGH dose were all assessed.

Results
In the GR group, mean height velocity SDS up to five years of observation was 1.19 ±0.41/year and in the PR group 0.59 ±0.38/year. The differences were statistically significant (p < 0.05).

Conclusions
The primary response to the rhGH treatment in GHD children seems to be a good predictor for long-term therapy outcomes.

keywords:

Primary response in GHD children treatment as a predictor for long-term therapy effectiveness

Jan M Kapała 1 , Tomasz Maroszczuk 1 , Aleksandra Sitarz 1 , Anna Kącka 1 , Dorota Charemska 1

children, retrospective cohort study, growth hormone deficiency, recombinant human growth hormone, primary response

Jan M Kapała

¹

,

Tomasz Maroszczuk

¹

,

Aleksandra Sitarz

¹

,

Anna Kącka

¹

,

Dorota Charemska

¹