ESPE Abstracts (2014) 82 P-D-3-2-830

Prader-Willi Syndrome and GH Therapy: valuable Effects and Adverse Events

Magdalena Wilkb, Katarzyna Wzorekb, Ewa Tobórb, Barbara Dejniakb, Anna Wedrychowicza, Katarzyna Dolezal-Oltarzewskaa, Agata Zygmunt-Gorskaa & Jerzy Starzyka


aDepartment of Pediatric and Adolescent Endocrinology Polish-American Pediatric Institute, Medical College, Jagiellonian University in Cracow, Cracow, Poland; bStudents’ Scientific Group, Department of Pediatric and Adolescent Endocrinology, Polish-American Pediatric Institute, Medical College, Jagiellonian University in Cracow, Cracow, Poland


Background: Prader–Willi syndrome (PWS) is a genetic disorder with hypothalamic–pituitary dysfunction, in which obesity, excess fat to lean body mass cause metabolic complications. For the purpose of these disorders normalization, PWS patients have been treated with recombinant human GH (rhGH). Long-term tolerance in PWS children treated with rhGH is not well known and the data are still required.

Objective and hypotheses: To evaluate effects of rhGH treatment in PWS patients.

Method: Twelve patients (pts) with genetically confirmed PWS: eight boys, median age 13.4±5.64 years (5–17 years), treated with rhGH in 2013, were included to the study. Treatment duration was 5.9±2.62 years (2–11years). The study was conducted on a basis of data from patients medical records.

Results: During treatment weight SDS increased from 0.16 to 0.93 and height SDS from −1.38 to 0.55. Weight SDS raised after median 3 years of treatment (to 1.15) and noted downturn (0.93) during next year. BMI-SD at the beginning of the treatment was 1.31, now totals 1.29. Most pts required decreasing of rhGH dose: 8/12 due to high IGF1 values, 2/12 due to glucose intolerance and one pt due to the rapid acceleration of growth. One pt discontinued therapy due to an excessive increase in body weight (weight-SDS from 0.99 to 2.53 during 3 years). Mean pts HbA1c level was 5.37% (57 mmol/mol), no diabetes was observed. Glucose intolerance was observed in two pts, who developed obesity (BMI-SD from 1.025 to 2.522 during 6 years of treatment and from 0.01 to 0.556 during 7 years), Lipids profiles was within normal ranges in all cases except this one who discontinued rhGH therapy. In 2/12 pts tonsillar hypertrophy and in 1/12 pt the progression of scoliosis were observed.

Conclusion: In PWS pts treated with rhGH, BMI-SD should be strictly monitored, not to allow develop obesity and severe metabolic disorders as a result.

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