Growth hormone treatment in Aarskog syndrome: analysis of the KIGS (Pharmacia International Growth Database) data.
Aarskog syndrome is an X-linked disorder characterized by faciogenital dysplasia and short stature. The present study set out to determine the effect of growth hormone (GH) therapy in patients with Aarskog syndrome enrolled in KIGS--the Pharmacia International Growth Database. Twenty-one patients (20 males) were evaluated. Median age at start of treatment was 8.3 years (10-90th percentiles, 5.1-14.1 years) and median height SDS was -2.8 (10-90th percentiles, -2.1 to -3.7). The median dose of GH was 0.22 mg/kg/week (10-90th percentiles, 0.15-0.30 mg/kg/week) given at a median frequency of six (4-7) times per week. Prepubertal patients were followed longitudinally for 1 year (n = 13) or 3 years (n = 7). After 1 year, the median height SDS had improved from -2.8 to -2.3 in 13 patients. After 3 years, height SDS had improved significantly (p <0.05) to -1.8 (10-90th percentiles, -2.1 to -1.1) in the seven patients. No adverse events were noted. Although final height data for these patients are still awaited, the present results support the use of GH to promote growth in children with Aarskog syndrome.
Department of Pediatrics, University of Istanbul, Istanbul, Turkey. feyzad@istanbul.edu.tr
Feyza
F
Darendeliler
Pontus
P
Larsson
Olcay
O
Neyzi
Anthony D
AD
Price
Lars
L
Hagenäs
Ilkka
I
Sipilä
Ann Christin
AC
Lindgren
Barto
B
Otten
Bert
B
Bakker
D000015:Abnormalities, Multiple / Q000188:drug therapy* / Q000235:genetics; D000293:Adolescent; D001827:Body Height / Q000187:drug effects / Q000235:genetics / Q000502:physiology; D002648:Child; D019465:Craniofacial Abnormalities / Q000188:drug therapy; D019992:Databases as Topic; D004334:Drug Administration Schedule; D005385:Fingers / Q000002:abnormalities; D005544:Forecasting; D006130:Growth Disorders / Q000188:drug therapy* / Q000235:genetics; D013006:Growth Hormone / Q000008:administration & dosage / Q000097:blood / Q000627:therapeutic use*; D006801:Humans; D007279:Injections, Subcutaneous; D008137:Longitudinal Studies; D008297:Male; D012611:Scrotum / Q000002:abnormalities; D013577:Syndrome; D013997:Time Factors
D013006:Growth Hormone