PMID- 24645945
OWN - NLM
STAT- MEDLINE
DCOM- 20140422
LR  - 20220408
IS  - 1533-4406 (Electronic)
IS  - 0028-4793 (Linking)
VI  - 370
IP  - 12
DP  - 2014 Mar 20
TI  - Sirolimus therapy in infants with severe hyperinsulinemic hypoglycemia.
PG  - 1131-7
LID - 10.1056/NEJMoa1310967 [doi]
AB  - Hyperinsulinemic hypoglycemia is the most common cause of severe, persistent 
      neonatal hypoglycemia. The treatment of hyperinsulinemic hypoglycemia that is 
      unresponsive to diazoxide is subtotal pancreatectomy. We examined the 
      effectiveness of the mammalian target of rapamycin (mTOR) inhibitor sirolimus in 
      four infants with severe hyperinsulinemic hypoglycemia that had been unresponsive 
      to maximal doses of diazoxide (20 mg per kilogram of body weight per day) and 
      octreotide (35 mug per kilogram per day). All the patients had a clear glycemic 
      response to sirolimus, although one patient required a small dose of octreotide 
      to maintain normoglycemia. There were no major adverse events during 1 year of 
      follow-up.
FAU - Senniappan, Senthil
AU  - Senniappan S
AD  - From the Developmental Endocrinology Research Group, Clinical and Molecular 
      Genetics Unit, Institute of Child Health, University College London (S.S., P.S., 
      V.A., K.H.), and the Departments of Paediatric Endocrinology (S.S., P.S., V.A., 
      K.H.) and Histopathology (D.P., M.A.), Great Ormond Street Hospital for Children, 
      London, and the Institute of Biomedical and Clinical Science, University of 
      Exeter Medical School, Exeter (S.F., S.E.) - all in the United Kingdom; the 
      Department of Pathology, University of California, San Francisco, San Francisco 
      (S.A.); and the Department of Pathology and Laboratory Medicine, University of 
      Texas Medical School at Houston, Houston (N.T., R.E.B.).
FAU - Alexandrescu, Sanda
AU  - Alexandrescu S
FAU - Tatevian, Nina
AU  - Tatevian N
FAU - Shah, Pratik
AU  - Shah P
FAU - Arya, Ved
AU  - Arya V
FAU - Flanagan, Sarah
AU  - Flanagan S
FAU - Ellard, Sian
AU  - Ellard S
FAU - Rampling, Dyanne
AU  - Rampling D
FAU - Ashworth, Michael
AU  - Ashworth M
FAU - Brown, Robert E
AU  - Brown RE
FAU - Hussain, Khalid
AU  - Hussain K
LA  - eng
GR  - G1001821/MRC_/Medical Research Council/United Kingdom
PT  - Case Reports
PT  - Journal Article
PL  - United States
TA  - N Engl J Med
JT  - The New England journal of medicine
JID - 0255562
RN  - 0 (Blood Glucose)
RN  - EC 2.7.1.1 (MTOR protein, human)
RN  - EC 2.7.11.1 (TOR Serine-Threonine Kinases)
RN  - W36ZG6FT64 (Sirolimus)
SB  - IM
CIN - N Engl J Med. 2014 Jun 19;370(25):2448-9. PMID: 24941187
CIN - N Engl J Med. 2014 Jun 19;370(25):2448. PMID: 24941188
MH  - Blood Glucose/analysis
MH  - Congenital Hyperinsulinism/blood/*drug therapy/genetics
MH  - Female
MH  - Humans
MH  - Infant
MH  - Male
MH  - Mutation
MH  - Sirolimus/adverse effects/*therapeutic use
MH  - TOR Serine-Threonine Kinases/*antagonists & inhibitors
EDAT- 2014/03/22 06:00
MHDA- 2014/04/23 06:00
CRDT- 2014/03/21 06:00
PHST- 2014/03/21 06:00 [entrez]
PHST- 2014/03/22 06:00 [pubmed]
PHST- 2014/04/23 06:00 [medline]
AID - 10.1056/NEJMoa1310967 [doi]
PST - ppublish
SO  - N Engl J Med. 2014 Mar 20;370(12):1131-7. doi: 10.1056/NEJMoa1310967.