A Multicenter Experience with Long-Acting Somatostatin Analogues in Patients with Congenital Hyperinsulinism

Ivo van der Steen*, Mirjam E. van Albada, Klaus Mohnike, Henrik Thybo Christesen, Susann Empting, Maria Salomon-Estebanez, Amalie Greve Rasmussen, Annemarie Verrijn Stuart, Annelieke A. A. van der Linde, Indraneel Banerjee, Annemieke M. Boot

*Bijbehorende auteur voor dit werk

    Onderzoeksoutput: ArticleAcademicpeer review

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    Samenvatting

    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. (C) 2017 S. Karger AG, Basel

    Originele taal-2English
    Pagina's (van-tot)82-89
    Aantal pagina's8
    TijdschriftHormone Research in Paediatrics
    Volume89
    Nummer van het tijdschrift2
    DOI's
    StatusPublished - feb-2018

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