Growth hormone treatment in children with familial dysautonomia.
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.


CONCLUSIONS
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.
Kamboj
Manmohan K
MK
Department of Pediatrics, New York University Medical Center, New York, New York 10016, USA.
Axelrod
Felicia B
FB
David
Raphael
R
Geffner
Mitchell E
ME
Novogroder
Michael
M
Oberfield
Sharon E
SE
Turco
John H
JH
Maayan
Channa
C
Kohn
Brenda
B
D000293:Adolescent; D001827:Body Height; D015992:Body Mass Index; D001835:Body Weight; D002648:Child; D004402:Dysautonomia, Familial / Q000188:drug therapy*; D005260:Female; D019382:Human Growth Hormone / Q000627:therapeutic use*; D006801:Humans; D007223:Infant; D008297:Male
D019382:Human Growth Hormone