PMID- 27104176
OWN - NLM
STAT- PubMed-not-MEDLINE
DCOM- 20160422
LR  - 20200930
IS  - 2287-1012 (Print)
IS  - 2287-1292 (Electronic)
IS  - 2287-1012 (Linking)
VI  - 21
IP  - 1
DP  - 2016 Mar
TI  - Long-term efficacy of recombinant human growth hormone therapy in short-statured 
      patients with Noonan syndrome.
PG  - 26-30
LID - 10.6065/apem.2016.21.1.26 [doi]
AB  - PURPOSE: Noonan syndrome (NS) is characterized by short stature, heart anomalies, 
      developmental delays, dysmorphic features, cryptorchidism, and coagulation 
      defects. Several studies reported the short-term effects of recombinant human 
      growth hormone (rhGH) treatment on the improvement of height. This study was 
      performed to evaluate the long-term efficacy of rhGH in children with NS in 
      Korea. METHODS: This study included 15 prepubertal NS children who received rhGH 
      subcutaneously at a dose of 50-75 microg/kg/day for 6 days a week for at least >3 
      years. Preand posttreatment data, such as height, weight, bone age, insulin-like 
      growth factor 1 (IGF-1), and IGF binding protein 3 (IGFBP-3) levels, were 
      collected every 6 months. RESULTS: Chronologic age and bone age at the start of 
      treatment were 7.97+/-1.81 and 5.09+/-2.12 years, respectively. Height standard 
      deviation score (SDS) was increased from -2.64+/-0.64 to -1.54+/-1.24 years after 3 
      years (P<0.001). Serum IGF-1 SDS levels were elevated from -1.28+/-1.03 to 
      -0.10+/-0.94 (P<0.001). Height SDS was more increased in subjects without PTPN11 
      mutations compared to those with mutations after 3 years (P=0.012). However, the 
      other parameters, including bone age, IGF-1 SDS, and IGFBP-3 SDS, were not 
      significantly different between patients with and without PTPN11 mutations. 
      CONCLUSION: Although this study included a relatively small number of patients, 
      long-term rhGH therapy in NS patients was safe and effective at improving height, 
      growth velocity, and serum IGF-1 levels, in accordance with previous studies. 
      However, the meticulous monitoring of potential adverse events is still needed 
      because of high dose of rhGH and preexisting hyperactivity of RAS-MAPK pathway. 
      Patients with PTPN11 mutations demonstrated a decreased response to rhGH therapy 
      compared to those without mutations.
FAU - Jeong, Insook
AU  - Jeong I
AD  - Department of Pediatrics, Asan Medical Center Children's Hospital, University of 
      Ulsan College of Medicine, Seoul, Korea.
FAU - Kang, Eungu
AU  - Kang E
AD  - Department of Pediatrics, Asan Medical Center Children's Hospital, University of 
      Ulsan College of Medicine, Seoul, Korea.
FAU - Cho, Ja Hyang
AU  - Cho JH
AD  - Department of Pediatrics, Asan Medical Center Children's Hospital, University of 
      Ulsan College of Medicine, Seoul, Korea.
FAU - Kim, Gu-Hwan
AU  - Kim GH
AD  - Medical Genetics Center, Asan Medical Center Children's Hospital, University of 
      Ulsan College of Medicine, Seoul, Korea.
FAU - Lee, Beom Hee
AU  - Lee BH
AD  - Department of Pediatrics, Asan Medical Center Children's Hospital, University of 
      Ulsan College of Medicine, Seoul, Korea.
FAU - Choi, Jin-Ho
AU  - Choi JH
AD  - Department of Pediatrics, Asan Medical Center Children's Hospital, University of 
      Ulsan College of Medicine, Seoul, Korea.
FAU - Yoo, Han-Wook
AU  - Yoo HW
AD  - Department of Pediatrics, Asan Medical Center Children's Hospital, University of 
      Ulsan College of Medicine, Seoul, Korea.
LA  - eng
PT  - Journal Article
DEP - 20160331
PL  - Korea (South)
TA  - Ann Pediatr Endocrinol Metab
JT  - Annals of pediatric endocrinology & metabolism
JID - 101588279
PMC - PMC4835558
OTO - NOTNLM
OT  - Growth hormone
OT  - Noonan syndrome
OT  - PTPN11
COIS- Conflict of interest: No potential conflict of interest relevant to this article 
      was reported.
EDAT- 2016/04/23 06:00
MHDA- 2016/04/23 06:01
PMCR- 2016/03/01
CRDT- 2016/04/23 06:00
PHST- 2015/09/14 00:00 [received]
PHST- 2015/10/12 00:00 [revised]
PHST- 2015/11/26 00:00 [accepted]
PHST- 2016/04/23 06:00 [entrez]
PHST- 2016/04/23 06:00 [pubmed]
PHST- 2016/04/23 06:01 [medline]
PHST- 2016/03/01 00:00 [pmc-release]
AID - 10.6065/apem.2016.21.1.26 [doi]
PST - ppublish
SO  - Ann Pediatr Endocrinol Metab. 2016 Mar;21(1):26-30. doi: 
      10.6065/apem.2016.21.1.26. Epub 2016 Mar 31.