title: Successful treatment of congenital hyperinsulinism with long-acting release octreotide.
abstract: BACKGROUND
  Congenital hyperinsulinism (HI) is a common cause of hypoglycemia in infancy. The medical treatment of diazoxide-unresponsive HI is based on a somatostatin analogue.
  
  
  OBJECTIVE
  This study aims at replacing three daily s.c. octreotide (Sandostatin, Novartis) injections by a single and monthly i.m. injection of long-acting release (LAR) octreotide (Sandostatin LP, Novartis) in HI patients.
  
  
  METHODS
  LAR octreotide was injected every 4 weeks during 6 months and s.c. octreotide injections were stopped after the third injection of LAR octreotide. After this 6-month study, LAR octreotide was continued, with an average follow-up of 17 months. Ten HI pediatric patients unresponsive to diazoxide and currently treated with s.c. octreotide were included in the trial. Glycemias and other parameters (HbA1c, IGF1, height, weight, quality of life (QoL), and satisfaction) were monitored at each monthly visit.
  
  
  RESULTS
  For all ten patients, glycemias were maintained in the usual range, HbAlc (mean 5.5%; 95% CI: 4.6-6.2) and IGF1 (mean 89.7 ng/ml; 95% CI: 26-153) were unchanged. Patients gained height significantly (mean 2.7 cm; 95% CI: 1.9-3.4) and no side effect was noted during the study and the later follow-up. Plasma octreotide levels were stable under LAR octreotide. Parents' questionnaires of general satisfaction were highly positive whereas children's QoL evaluation remained unchanged.
  
  
  CONCLUSIONS
  In these diazoxide-unresponsive HI patients, LAR octreotide was efficient, well tolerated and contributed to a clear simplification of the medical care.
authors: Centre de Référence des Maladies Héréditaires du Métabolisme, Hôpital Necker-Enfants Malades, AP-HP, Université Paris Descartes, 149 Rue de Sèvres, 75015 Paris, France.
authors: Kim-Hanh
authors: KH
authors: Le Quan Sang
authors: Jean-Baptiste
authors: JB
authors: Arnoux
authors: Asmaa
authors: A
authors: Mamoune
authors: Cécile
authors: C
authors: Saint-Martin
authors: Christine
authors: C
authors: Bellanné-Chantelot
authors: Vassili
authors: V
authors: Valayannopoulos
authors: Anais
authors: A
authors: Brassier
authors: Honorine
authors: H
authors: Kayirangwa
authors: Valérie
authors: V
authors: Barbier
authors: Christine
authors: C
authors: Broissand
authors: Jean-Roch
authors: JR
authors: Fabreguettes
authors: Brigitte
authors: B
authors: Charron
authors: Jean-Christophe
authors: JC
authors: Thalabard
authors: Pascale
authors: P
authors: de Lonlay
mesh_terms: D018528:ATP-Binding Cassette Transporters / Q000235:genetics; D002648:Child; D002675:Child, Preschool; D044903:Congenital Hyperinsulinism / Q000097:blood / Q000188:drug therapy* / Q000235:genetics / Q000378:metabolism; D003692:Delayed-Action Preparations; D005260:Female; D005500:Follow-Up Studies; D005765:Gastrointestinal Agents / Q000008:administration & dosage / Q000009:adverse effects; D006801:Humans; D007223:Infant; D007273:Injections, Intramuscular / Q000009:adverse effects; D007279:Injections, Subcutaneous / Q000009:adverse effects; D008297:Male; D015282:Octreotide / Q000008:administration & dosage* / Q000009:adverse effects / Q000097:blood / Q000493:pharmacokinetics; D024661:Potassium Channels, Inwardly Rectifying / Q000235:genetics; D011955:Receptors, Drug / Q000235:genetics; D064233:Sulfonylurea Receptors; D016896:Treatment Outcome
chemical_list: D018528:ATP-Binding Cassette Transporters; D003692:Delayed-Action Preparations; D005765:Gastrointestinal Agents; D024661:Potassium Channels, Inwardly Rectifying; D011955:Receptors, Drug; D064233:Sulfonylurea Receptors; D015282:Octreotide