Ivo van der Steen1, Mirjam E van Albada1, Klaus Mohnike2, Henrik Thybo Christesen3, Susann Empting2, Maria Salomon-Estebanez4, Amalie Greve Rasmussen5, Annemarie Verrijn Stuart6, Annelieke A A van der Linde7, Indraneel Banerjee4, Annemieke M Boot1. 1. Department of Pediatric Endocrinology, Beatrix Children's Hospital, University Medical Center Groningen, University of Groningen, Groningen, the Netherlands. 2. Department of Pediatrics, Otto-von-Guericke University, Magdeburg, Germany. 3. Hans Christian Andersen Children's Hospital and Odense Pancreas Center OPAC, Odense University Hospital, Odense, Denmark. 4. Department of Pediatric Endocrinology, Royal Manchester Children's Hospital, Manchester, United Kingdom. 5. Department of Clinical Research, University of Southern Denmark, Odense, Denmark. 6. Department of Pediatric Endocrinology, University Medical Center Utrecht - Wilhelmina Children's Hospital, Utrecht, the Netherlands. 7. Department of Pediatric Endocrinology, Radboud University Medical Center - Amalia Children's Hospital, Nijmegen, the Netherlands.
Abstract
BACKGROUND/AIMS: Congenital hyperinsulinism (CHI) is a rare disease characterized by recurrent severe hypoglycemia. In the diffuse form of CHI, pharmacotherapy is the preferred choice of treatment. Long-acting somatostatin analogues have been used in children as off-label medication. However, the efficacy, outcomes, and adverse effect profiles of long-acting somatostatin analogues have not been described in multicentered studies. The aim of this retrospective study is to summarize the experience with long-acting somatostatin analogues in a large group of children with CHI. METHODS: Data were obtained retrospectively from 27 patients with CHI who received long-acting somatostatin analogues in 6 different centers in Europe. These included information on glycemic stability, auxology, and adverse effect profile in clinical follow-up assessments. RESULTS: Blood glucose control improved in most patients (89%). No life-threatening side effects occurred. Thirteen patients (48%) experienced side effects; in 3 patients (11%), the side effects were the main reason for discontinuation of the treatment. The most frequent side effect was elevated liver enzymes (n = 10, 37%). CONCLUSION: Long-acting somatostatin analogues are effective in glycemic control of patients with CHI. However, in 37% of all patients increased liver enzymes were observed. It is important to monitor liver function in all patients receiving long-acting somatostatin analogue therapy.
BACKGROUND/AIMS: Congenital hyperinsulinism (CHI) is a rare disease characterized by recurrent severe hypoglycemia. In the diffuse form of CHI, pharmacotherapy is the preferred choice of treatment. Long-acting somatostatin analogues have been used in children as off-label medication. However, the efficacy, outcomes, and adverse effect profiles of long-acting somatostatin analogues have not been described in multicentered studies. The aim of this retrospective study is to summarize the experience with long-acting somatostatin analogues in a large group of children with CHI. METHODS: Data were obtained retrospectively from 27 patients with CHI who received long-acting somatostatin analogues in 6 different centers in Europe. These included information on glycemic stability, auxology, and adverse effect profile in clinical follow-up assessments. RESULTS:Blood glucose control improved in most patients (89%). No life-threatening side effects occurred. Thirteen patients (48%) experienced side effects; in 3 patients (11%), the side effects were the main reason for discontinuation of the treatment. The most frequent side effect was elevated liver enzymes (n = 10, 37%). CONCLUSION: Long-acting somatostatin analogues are effective in glycemic control of patients with CHI. However, in 37% of all patients increased liver enzymes were observed. It is important to monitor liver function in all patients receiving long-acting somatostatin analogue therapy.
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