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Growth hormone treatment in a girl with Prader Willi syndrome
S N Pandey1, R A Vaidya, A Irani
1Bhavan's SPARC Center for Disorders of Growth & Puberty 13th N.S. Road, JVPD, Mumbai-400 049. bhaspa@bom5.vsnl.net.in
Insights
Prader Willi syndrome (PWS) patients may benefit from growth hormone (GH) therapy. This treatment improved linear growth and metabolic function, with some lasting effects after discontinuation.
Area of Science:
- Endocrinology
- Metabolic Disorders
- Genetics
Background:
- Prader Willi syndrome (PWS) is a rare genetic disorder affecting multiple endocrine and metabolic functions.
- Key features include hypotonia, hyperphagia, obesity, short stature, hypogonadism, and behavioral issues.
- A significant percentage of individuals with PWS develop diabetes mellitus and often exhibit growth hormone (GH) deficiency.
Observation:
- Growth hormone (GH) replacement therapy has shown promise in addressing PWS characteristics.
- Recent research suggests GH therapy benefits linear growth and metabolic dysfunctions.
- This case study examines the therapeutic outcome in a girl with PWS treated with recombinant GH (rGH).
Findings:
- Treatment with recombinant growth hormone (rGH), specifically Genotropin, was administered to a girl with PWS.
- The therapy demonstrated positive effects on the patient's condition.
- Notably, some therapeutic benefits persisted even after the cessation of rGH treatment.
Implications:
- Growth hormone replacement therapy may be a valuable therapeutic option for managing PWS.
- Further research into the long-term effects and optimal use of GH in PWS is warranted.
- Understanding carry-over benefits could inform treatment strategies and improve patient outcomes.
Abstract:
Prader Willi syndrome (PWS) is a rare endocrine-metabolic disorder that is characterised by neonatal hypotonia, hyperphagia, marked obesity, short stature, hypogonadism and behavioural problems. 7-20% percent of these children develop diabetes mellitus. A large number of individuals with PWS show growth hormone (GH) deficiency. Recent studies indicate beneficial effects of GH replacement therapy not only for their linear growth but also for correction of metabolic dysfunction. In the present communication this article details about the therapeutic outcome in a girl with PWS who received recombinant growth hormone (rGH), Genotropin. Some carry-over therapeutic benefits have been observed even after discontinuation of rGH.
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