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O0055 IMPACT OF LONG TERM GROWTH HORMONE TREATMENT ON BODY COMPOSITION IN PRADER-WILLI SYNDROME (PWS)

Scheimann, A. O.1; Klish, W. J.2; Hayslett, B.2; Lafuente, N.2; Orellana, D.2; Gordon, B.3; Thompson, R.4; Lee, P. D.5

Journal of Pediatric Gastroenterology and Nutrition: June 2004 - Volume 39 - Issue - p S28-S29
ABSTRACTS: Oral Presentation Abstracts
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1Pediatric Gastroenterology and Nutrition, Johns Hopkins School of Medicine, Baltimore,2Pediatric Gastroenterology and Nutrition,3Pediatrics, Baylor College of Medicine, Houston,4Biostatistics, Johns Hopkins School of Public Health, Baltimore,5Pediatric Endocrinology, David Geffen School of Medicine- UCLA, Los Angeles, United States

Submitted by: ascheima@jhmi.edu

Introduction: PWS is a genetic disorder localized to 15q11.2–13 and characterized by obesity, hypogonadism, and hypopituitarism along with features of growth hormone (GH) deficiency. Prior short-term studies have shown improvement in height, muscle function and body composition. Little long-term data currently exists regarding body composition, growth and complications of GH therapy.

Methods: IRB-approved retrospective chart review of patients with PWS followed in the PWS clinic at Texas Children’s Hospital between 1988–2002. Patients were divided into 2 GH-treated groups (between 0 – 5 years treatment and greater than 5 years GH treatment) and an untreated group. Regression analyses were performed on the outcomes of height z-scores, body mass index z-scores, percent body fat (via total body electrical conductivity (TOBEC)), and percent lean mass (via TOBEC), and clinical evidence for scoliosis utilizing the STATA command regress (StataCorp 2001).

Results: A total of 84 patients (54.8% male) with PWS were enrolled. 53 (63.1%) patients were deletion, 14 patients (16.7%) were UPD, 1 patient (1.1%) had an imprinting mutation, and 16 patients (19.1%) had no genetic information. The age at the initial visit ranged from 0–35 years, with a mean age of 6.19 +/− 6.97 years and median age of 3.90 years. 49 patients received no GH treatment, 25 patients had GH treatment for up to 5 years, 10 patients had GH treatment for at least 5 years. 2 patients had GH treatment discontinued due to development of diabetes. There was a significant difference in BMI slope between long-term GH treated versus untreated controls (p<0.001). There were also significant differences in height z-scores, percent lean mass and percent body fat (39.95% fat untreated vs. 36.07% fat in GH-treated) between treated and controls. When we looked at the association of scoliosis and GH, we found that 18 of 27 (66.7%) in the control group, 10 of 22 (45.5%) in the less than 5 year treated group and 7 of 10 (70%) of the greater than 5 year treated developed scoliosis.

Conclusion: GH treatment appears to have long-term beneficial effects on body composition, and linear growth.

© 2004 Lippincott Williams & Wilkins, Inc.