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Cardiac examination in children with Laron syndrome undergoing mecasermin therapy

Cardiac examination in children with Laron syndrome undergoing mecasermin therapy AbstractBackground:Laron syndrome (LS), which can be defined as primary growth hormone resistance or insensitivity, is a rare genetic disease inherited by an autosomal recessive trait. Although it is undistinguishable from growth hormone deficiency, LS has high levels of growth hormone, but insulin-like growth factor (IGF-1) cannot be synthesized. Mecasermin treatment is the only option for the patients who suffer from LS. This study aims to research cardiac findings of children with LS, who receive treatment with mecasermin.Methods:The study enrolled five children four males and one female, 4 M/1 F with LS, two of whom were siblings with a mean age of 6.3±2.1 years, a body weight of 13.36±4.74 kg, a height of 88±8.7 cm, and a body mass index (BMI) of 16.47±3.35. Their demographic data were obtained from their family and files. The children received mecasermin via subcutaneous injection at 0.04–0.12 μg/kg doses twice per day. The duration of mecasermin treatment was 8–53 months. All of them were examined clinically by electrocardiogram and echocardiogram.Results:Their cardiac examinations were normal, except for one case, who had systolic murmur at cardiac auscultation. Arrhythmia was not observed on their electrocardiograms. The echocardiograms did not show a significant congenital cardiac anomaly. Their cardiac measure and functions were within normal ranges. The echocardiogram of the child with the murmur showed mitral and tricuspid insufficiency. The Doppler images showed pulmonary hypertension findings. These findings were proven by angiography. The vasoreactivity test results of that patient were negative. No reason could be found for the observed pulmonary hypertension. We diagnosed this finding as a primary pulmonary hypertension and Bosentan therapy was started.Conclusions:In this study, we showed that cardiac findings were consistent with previous studies. To the best of our knowledge, the observed pulmonary hypertension in children with LS, who received treatment with or without mecasermin, is reported for first time in the literature. http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png Journal of Pediatric Endocrinology and Metabolism de Gruyter

Cardiac examination in children with Laron syndrome undergoing mecasermin therapy

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Publisher
de Gruyter
Copyright
©2018 Walter de Gruyter GmbH, Berlin/Boston
ISSN
2191-0251
eISSN
2191-0251
DOI
10.1515/jpem-2017-0473
pmid
29750649
Publisher site
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Abstract

AbstractBackground:Laron syndrome (LS), which can be defined as primary growth hormone resistance or insensitivity, is a rare genetic disease inherited by an autosomal recessive trait. Although it is undistinguishable from growth hormone deficiency, LS has high levels of growth hormone, but insulin-like growth factor (IGF-1) cannot be synthesized. Mecasermin treatment is the only option for the patients who suffer from LS. This study aims to research cardiac findings of children with LS, who receive treatment with mecasermin.Methods:The study enrolled five children four males and one female, 4 M/1 F with LS, two of whom were siblings with a mean age of 6.3±2.1 years, a body weight of 13.36±4.74 kg, a height of 88±8.7 cm, and a body mass index (BMI) of 16.47±3.35. Their demographic data were obtained from their family and files. The children received mecasermin via subcutaneous injection at 0.04–0.12 μg/kg doses twice per day. The duration of mecasermin treatment was 8–53 months. All of them were examined clinically by electrocardiogram and echocardiogram.Results:Their cardiac examinations were normal, except for one case, who had systolic murmur at cardiac auscultation. Arrhythmia was not observed on their electrocardiograms. The echocardiograms did not show a significant congenital cardiac anomaly. Their cardiac measure and functions were within normal ranges. The echocardiogram of the child with the murmur showed mitral and tricuspid insufficiency. The Doppler images showed pulmonary hypertension findings. These findings were proven by angiography. The vasoreactivity test results of that patient were negative. No reason could be found for the observed pulmonary hypertension. We diagnosed this finding as a primary pulmonary hypertension and Bosentan therapy was started.Conclusions:In this study, we showed that cardiac findings were consistent with previous studies. To the best of our knowledge, the observed pulmonary hypertension in children with LS, who received treatment with or without mecasermin, is reported for first time in the literature.

Journal

Journal of Pediatric Endocrinology and Metabolism – de Gruyter

Published: Jun 27, 2018

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  • Managing the child with severe primary insulin-like growth factor-1 deficiency [IGFD]: IGFD diagnosis and management
  • Human conditions of insulin-like growth factor-I [IGF-I] deficiency
  • Extensive personal experience. LS [Primary growth hormone resistance or insensitivity]: the personal experience 1958–2003
  • The GHIS collaborative group. Therapy for 6,5-7,5 years with recombinant insulin-like growth factor 1 in children with growth hormone insensivity syndrome: a clinical research center study
  • Insulin-like growth factor-I deficiency in children with growth hormone insensitivity current and future treatment options
  • Long-term treatment with recombinant insulin-like growth factor [IGF]-I in children with severe IGF-I deficiency due to growth hormone insensitivity
  • IGF-I therapy in growth disorders