Growth hormone treatment and adverse events in Prader-Willi syndrome: data from KIGS (the Pfizer International Growth Database).
OBJECTIVE
To evaluate the response to recombinant GH treatment and adverse events in children with Prader-Willi syndrome (PWS) from KIGS, the Pfizer International Growth Database.


METHODS
A total of 328 children (274 prepubertal, median age 6.0 years; 54 pubertal, median age 12.7 years) were treated for 1 year and 161 children were treated for 2 years with GH.


RESULTS
Height standard deviation score (SDS) increased significantly during treatment; the response was greater in prepubertal (-0.7 vs.-1.8 pretreatment) compared with pubertal children (-1.5 vs.-1.8). Predictors of first-year height velocity in multiple regression analysis were GH dose, body weight (positively correlated), height SDS minus mid-parental height SDS and chronological age (negatively correlated), together accounting for 39% of the variation in response to GH. Body mass index (BMI) SDS did not change significantly during 2 years of treatment. Of all the 675 GH-treated PWS patients in KIGS, there were five cases of sudden death (age range 3-15 years). Three were obese (weight for height > 200%) and causes of death included bronchopneumonia, respiratory insufficiency and sleep apnoea. Scoliosis was the most commonly reported adverse event (n = 24), four children developed hyperglycaemia and six had presumptive diabetes (type 2 in five, and one case of type 1).


CONCLUSIONS
Short-term growth improved in response to conventional doses of GH in children with PWS. Prior to commencement of GH, examination of the upper airways and sleep studies should be performed in PWS patients. GH should be used with caution in those with extreme obesity or disordered breathing and all patients should be closely monitored for adverse events.
Craig
Maria E
ME
Institute of Endocrinology and Diabetes, Children's Hospital at Westmead, Westmead, NSW, Australia. mariac@chw.edu.au
Cowell
Christopher T
CT
Larsson
Pontus
P
Zipf
William B
WB
Reiter
Edward O
EO
Albertsson Wikland
Kerstin
K
Ranke
Michael B
MB
Price
David A
DA
D050154:Adiposity / Q000187:drug effects; D000293:Adolescent; D001827:Body Height / Q000187:drug effects; D001996:Bronchopneumonia / Q000150:complications; D002648:Child; D002675:Child, Preschool; D016208:Databases, Factual; D003920:Diabetes Mellitus; D004334:Drug Administration Schedule; D005260:Female; D019382:Human Growth Hormone / Q000009:adverse effects* / Q000627:therapeutic use; D006801:Humans; D008137:Longitudinal Studies; D008297:Male; D011218:Prader-Willi Syndrome / Q000188:drug therapy* / Q000401:mortality; D012044:Regression Analysis; D012131:Respiratory Insufficiency / Q000150:complications; D012600:Scoliosis / Q000150:complications; D012891:Sleep Apnea Syndromes / Q000150:complications
D019382:Human Growth Hormone