title: Long-term medical treatment in congenital hyperinsulinism: a descriptive analysis in a large cohort of patients from different clinical centers.
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%).
  
  
  CONCLUSIONS
  Severe side effects are rare and a causal relation remains disputable. We conclude that long-term conservative treatment of CHI is feasible.
authors.affiliation: Department of General Pediatrics, Neonatology and Pediatric Cardiology, University Children's Hospital Duesseldorff, Moorenstrasse 5, Duesseldorf, D-40225, Germany. Alena.Welters@med.uni-duesseldorf.de.
authors.forename: Alena
authors.initials: A
authors.lastname: Welters
authors.affiliation: Cochrane Metabolic and Endocrine Disorders Group, Institute of General Practice, Duesseldorf University Hospital, Duesseldorf, Germany.|Department of Pediatric Kidney, Liver and Metabolic Diseases, Hannover Medical School, Hannover Medical School, Germany.
authors.forename: Christian
authors.initials: C
authors.lastname: Lerch
authors.affiliation: Department of General Pediatrics, Neonatology and Pediatric Cardiology, University Children's Hospital Duesseldorff, Moorenstrasse 5, Duesseldorf, D-40225, Germany. Sebastian.Kummer@med.uni-duesseldorf.de.
authors.forename: Sebastian
authors.initials: S
authors.lastname: Kummer
authors.affiliation: Department of General Pediatrics, Neonatology and Pediatric Cardiology, University Children's Hospital Duesseldorff, Moorenstrasse 5, Duesseldorf, D-40225, Germany. j.marquard@gmx.com.
authors.forename: Jan
authors.initials: J
authors.lastname: Marquard
authors.affiliation: Department of General Pediatrics, Neonatology and Pediatric Cardiology, University Children's Hospital Duesseldorff, Moorenstrasse 5, Duesseldorf, D-40225, Germany. burak.salgin@gmail.com.|Neonatal Intensive Care Unit, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK. burak.salgin@gmail.com.|University Department of Obstetrics & Gynaecology, University of Cambridge, Cambridge, UK. burak.salgin@gmail.com.
authors.forename: Burak
authors.initials: B
authors.lastname: Salgin
authors.affiliation: Department of General Pediatrics, Neonatology and Pediatric Cardiology, University Children's Hospital Duesseldorff, Moorenstrasse 5, Duesseldorf, D-40225, Germany. mayatepek@med.uni-duesseldorf.de.
authors.forename: Ertan
authors.initials: E
authors.lastname: Mayatepek
authors.affiliation: Department of General Pediatrics, Neonatology and Pediatric Cardiology, University Children's Hospital Duesseldorff, Moorenstrasse 5, Duesseldorf, D-40225, Germany. Thomas.Meissner@med.uni-duesseldorf.de.
authors.forename: Thomas
authors.initials: T
authors.lastname: Meissner
mesh_terms: D001786:Blood Glucose / Q000187:drug effects / Q000378:metabolism; D015331:Cohort Studies; D044903:Congenital Hyperinsulinism / Q000097:blood / Q000175:diagnosis* / Q000188:drug therapy*; D003981:Diazoxide / Q000008:administration & dosage; D006801:Humans; D013004:Somatostatin / Q000008:administration & dosage; D013997:Time Factors; D016896:Treatment Outcome
chemical_list: D001786:Blood Glucose; D013004:Somatostatin; D003981:Diazoxide