ECE2018 Poster Presentations: Pituitary and Neuroendocrinology Developmental endocrinology (1 abstracts)
CNNHC: Preliminary results of treatment with recombinant somatropin
Margarida Bastos 1 , Alice Mirante 1 , Bernardo Marques 2 , Joana Serra Caetano 1 , Caldas Afonso 3 , Carlos Vasconcelos 4 , Conceicão Bacelar 3 , Conceicão Pereira 5 , Lurdes Lopes 6 , Marcelo Fonseca 7 , Miguel Patrício 7 , César Esteves 8 , Florbela Ferreira 9 , Graciete Bragança 10 , Luísa Raimundo 11 , Lurdes Matos 6 , Manuel Foutoura 8 & Teresa Borges 3
1Centro Hospitalar e Universitário de Coimbra, EPE, Coimbra, Portugal; 2Instituto Português de Oncologia de Coimbra FG, EPE, Coimbra, Portugal; 3Centro Hospitalar do Porto, EPE, Porto, Portugal; 4Centro Hospitalar de Lisboa Ocidental, EPE, Lisbon, Portugal; 5Instituto Português de Oncologia de Lisboa FG, EPE, Lisbon, Portugal; 6Centro Hospitalar de Lisboa Central, EPE, Lisbon, Portugal; 7Unidade Unidade Local de Saúde de Matosinhos, Matosinhos, Portugal; 8Centro Hospitalar de São João, Porto, Portugal; 9Centro Hospitalar de Lisboa Norte, Lisbon, Portugal; 10Hospital Fernando da Fonseca, Amadora, Portugal; 11Hospital Garcia de Orta, Almada, Portugal.
Introduction: Treatment with recombinant Somatropin (rSMT) is safe and has greatly improved the approach of children and adolescents with somatropin deficiency (SMTD) and other growth disorders. In our country, rSMT therapy is approved for isolated/multiple somatropin deficiency, small for gestational age (SGA), chronic kidney disease (CKD), Turner syndrome (TS) and Prader Willi syndrome (PWS). A National Commitee (CNNHC) is responsible for the analysis of each case and treatment approval.
Methods/design: We performed a restrospective and comparative study of 111 patients who completed rSMT treatment, monitored by the CNNHC. The parameters evaluated included midparental target height (MTH), sex, diagnosis, duration of treatment and age, height, growth velocity (VC) at the beginning and end of treatment. Children with isolated SMTD and multiple pituitary deficiency (group 1) were compared to children in need of supraphysiological doses of rSMT (group 2). The association between variables was evaluated using paired samples t-test and the independent samples t-test.
Results: Most patients were female (58%) and mean age at the beginning of treatment was 9.8±3.5 years; mean duration of treatment was 6.3±3.4 years. Isolated SMTD was the most common cause (51%), followed by TS (30%), multiple deficiency (7%), CKD (6%), SGA (5%) and PWS (1%). The highest increase of stature was observed in the group of children with isolated SMTD (mean SDS variation of +1.4), followed by multiple pituitay deficiency (+1.38), CKD (+1.2), ST (+0, 5), SGA (+0.47) and SPW (+0.29). When comparing both groups, we found that, prior to treatment, GV is higher in group 2 children (3.6 cm/year vs 4.4 cm/year; P=0.014). In group 1, mean baseline height was higher (122.4±19 cm versus 113.6±18 cm; P=0.019), as was the final height (155.7±12 cm vs 145.8±11.8 cm, P < 0.001) and the mean SDS variation (1.43 vs 0.63, P <0.001). The difference between final height and MTH was higher in group 2 (−5.3 cm vs −13.2 cm; P=0.001).
Discussion: The primary goal of rSMT in children is acceleration of growth velocity to promote normalization of growth and stature appropriate for the child’s genetic potential. This preliminary study suggests that children with isolated/multiple rSMT deficiency show better results with rSMT treatment. However, most patients start treatment quite late and this fact may hinder its efficacy.
Volume 56
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Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
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