ESPE2024 Poster Category 2 GH and IGFs (22 abstracts)
Short and long-term response to rhGH therapy in short children born at very low birth weight.
Alexsandra Christianne Malaquias 1,2 , Thais Kataoka Homma 1,3 , Naiara Castelo Branco Dantas 1,3 , Bruna Lucheze Freire 3 , Edoarda Vasco de Albuquerque Albuquerque 1,3 , Ivo Jorge Prado Arnhold 1,3 , Renata Cunha Scalco 1,3 & Alexander Augusto de Lima Jorge 1,3
1Unidade de Endocrinologia Genética, Laboratório de Endocrinologia Celular e Molecular LIM25, Disciplina de Endocrinologia da Faculdade de Medicina da Universidade de Sao Paulo (FMUSP), Sao Paulo, Brazil. 2Unidade de Endocrinologia Pediátrica, Departamento de Pediatria, Irmandade da Santa Casa de Misericórdia de São Paulo, Faculdade de Ciências Médicas da Santa Casa de Sao Paulo, Sao Paulo, Brazil. 3Unidade de Endocrinologia do Desenvolvimento, Laboratório de Hormônios e Genética Molecular LIM42, Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo (FMUSP), Sao Paulo, Brazil
Introduction: Extrauterine growth retardation is a common characteristic among children born at very low birth weight (VLBW). Over time, recombinant human growth hormone (rhGH) has been used in this group based on indications for children born small for gestational age (SGA). However, this group represents an extreme phenotype to the majority of children born SGA, and data on the outcomes of this therapy in this group are limited.
Objective: To report the short and long-term response to rhGH therapy in a cohort of patients born at VLBW, comparing according to size, gestational age, and causal factors associated with VLBW.
Materials and Methods: We described 33 patients born at VLBW treated with rhGH, with 16 also receiving GnRHa. Data analysis of medical records and the rhGH treatment was performed from baseline and after one year. Data on adult height SDS and height SDS changes during the treatment from 23 patients were also collected. Growth velocities and height standard deviation scores (SDS) changes were calculated, alongside differences between observed and predicted growth velocities.
Results: The first-year growth velocity (7.5±2.1cm/y) aligned with predictions based on SGA children. After one year of treatment, height SDS improved from -3.0±1.1 to -2.6±1.3, with no significant differences among subgroups. Among patients reaching adult height, 73.9% remained short-statured (height SDS -2.6±1.3) after long-term therapy (6.7±3.3y). Initial height SDS (P <0.001), height SDS change in the first year of treatment (P <0.001), and target height SDS (P = 0.003) were key independent predictors of height gain.
Conclusion: The response to rhGH treatment is suboptimal in the VLBW group, independent of size, gestational age, or etiological diagnosis. However, it may still be superior to not receiving treatment. This underscores the need for tailored protocols and further investigations to optimize outcomes in this population.
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