Long-term medical treatment in congenital hyperinsulinism: a descriptive analysis in a large cohort of patients from different clinical centers

Alena Welters¹^*, Christian Lerch²³, Sebastian Kummer¹, Jan Marquard¹, Burak Salgin¹⁴⁵,Ertan Mayatepek¹ and Thomas Meissner¹

*Corresponding author: Alena Welters Alena.Welters@med.uni-duesseldorf.de

Author Affiliations

¹Department of General Pediatrics, Neonatology and Pediatric Cardiology, University Children’s Hospital Duesseldorff, Moorenstrasse 5, Duesseldorf D-40225, Germany

²Cochrane Metabolic and Endocrine Disorders Group, Institute of General Practice, Duesseldorf University Hospital, Duesseldorf, Germany

³Department of Pediatric Kidney, Liver and Metabolic Diseases, Hannover Medical School, Hannover Medical School, Germany

⁴Neonatal Intensive Care Unit, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK

⁵University Department of Obstetrics & Gynaecology, University of Cambridge, Cambridge, UK

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Orphanet Journal of Rare Diseases 2015, 10:150 doi:10.1186/s13023-015-0367-x

The electronic version of this article is the complete one and can be found online at:http://www.ojrd.com/content/10/1/150

Received:	14 August 2015
Accepted:	15 November 2015
Published:	25 November 2015

Open AccessThis article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated.

Abstract

Background

Up to now, only limited data on long-term medical treatment in congenital hyperinsulinism (CHI) is available. Moreover, most of the drugs used in CHI are therefore not approved. We aimed to assemble more objective information on medical treatment in CHI with regard to type and duration, dosage as well as side effects.

Methods

We searched MEDLINE (from 1947) and EMBASE (from 1988) using the OVID interface for relevant data to evaluate medical treatment in a large cohort of patients with CHI from different clinical centers. Randomized, controlled trials were not available. We evaluated case reports and case series. No language restrictions were made.

Results

A total number of 619 patients were medically treated and information regarding conservative treatment was available. Drugs used were diazoxide (in 84 % of patients), somatostatin analogues (16 %), calcium channel antagonists (4 %) and glucagon (1 %). Mean dose of diazoxide was 12.5 (±4.3) mg/kg ⋅ d (range 2–60 mg/kg ⋅ d), mean duration of diazoxide treatment until remission was 57 months. Side effects of diazoxide were usually not severe. The causal relation between diazoxide and severe side effects, e.g. heart failure (3.7 %) remains doubtful. Mean dose of octreotide was 14.9 (±7.5) μg/kg ⋅ d (range 2.3–50 μg/kg ⋅ d), of lanreotide 67.3 (±39.8) mg ⋅ month (range 10–120 mg ⋅ month). Mean duration of treatment with somatostatin analogues until remission was 49 months. Frequent side effects included tachyphylaxis and mild gastrointestinal symptoms. The risk of persistent growth deceleration was low (<5 %).