Document Detail

Growth hormone treatment in children with familial dysautonomia.
MedLine Citation:
PMID:  14722520     Owner:  NLM     Status:  MEDLINE    
OBJECTIVE: To assess experience with growth hormone (GH) therapy in patients with familial dysautonomia (FD). Study design Of 580 patients with FD registered at the Dysautonomia Center at New York University Medical Center, 13 patients (8 males, 5 females) aged 1.10 to 15.10 years received GH treatment. GH doses ranged from 0.2 to 0.3 mg/kg/wk; one patient received 0.14 mg/kg/wk. Information regarding auxologic data, skeletal age, pubertal status, and spinal deformity before and after GH therapy was obtained from center records and treating endocrinologists. Growth velocity was analyzed before and during GH treatment at 0 to 6 months, 6 to 12 months, 1 to 2 years, and >2 years. RESULTS: Before GH therapy, growth velocity was <5 cm/y in 10 patients and 5 to 6 cm/y in three patients. In the first six months of GH therapy, growth velocity exceeded pretreatment rates in all but one patient; 10 patients achieved an annualized growth rate >7 cm/y. Six of nine patients treated for more than one year grew >5 cm/y. Less than optimal treatment responses were attributed to poor compliance, intercurrent illness, scoliosis, or advanced puberty. CONCLUSION: The data demonstrate that GH treatment in patients with FD may increase growth velocity, at least in the short term. This experiential data supports a future prospective study.
Manmohan K Kamboj; Felicia B Axelrod; Raphael David; Mitchell E Geffner; Michael Novogroder; Sharon E Oberfield; John H Turco; Channa Maayan; Brenda Kohn
Publication Detail:
Type:  Journal Article; Research Support, Non-U.S. Gov't    
Journal Detail:
Title:  The Journal of pediatrics     Volume:  144     ISSN:  0022-3476     ISO Abbreviation:  J. Pediatr.     Publication Date:  2004 Jan 
Date Detail:
Created Date:  2004-01-14     Completed Date:  2004-02-18     Revised Date:  2009-11-03    
Medline Journal Info:
Nlm Unique ID:  0375410     Medline TA:  J Pediatr     Country:  United States    
Other Details:
Languages:  eng     Pagination:  63-7     Citation Subset:  AIM; IM    
Department of Pediatrics, New York University Medical Center, New York, New York 10016, USA.
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MeSH Terms
Body Height
Body Mass Index
Body Weight
Dysautonomia, Familial / drug therapy*
Human Growth Hormone / therapeutic use*
Reg. No./Substance:
12629-01-5/Human Growth Hormone

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