Human Growth Hormone Treatment for Children with Prader-Willi Syndrome: A Review of Clinical Effectiveness, Cost-Effectiveness, and Guidelines

Rob Edge, Philip la Fleur, L Adcock

Research output: Contribution to journalArticle

Abstract

One small, high-quality randomized controlled trial, in addition to moderate quality evidence from four small randomized controlled trials, examining a total of 139 patients, and two cohort studies were identified on human growth hormone treatment of pediatric patients with Prader-Willi Syndrome. Improved body composition, behavioral benefits, and improved quality of life associated with human growth hormone treatment were suggested in the evidence. However, possibly due to small sample sizes, statistically significant treatment benefits were not consistently observed in the identified evidence. Inconsistent evidence for improved cognition was also identified, although the highest quality evidence suggested that, at least in patients with Prader-Willi Syndrome previously treated with human growth hormone until adult height was reached, no cognitive benefits manifested following one year of treatment. Significant uncertainty is therefore associated with the clinical effectiveness evidence in this report. Data on adverse events and risks of human growth hormone treatment was absent from the identified evidence on this patient population. No cost-effectiveness evidence or relevant guidelines were identified in the limited literature search.
Original languageEnglish
JournalCanadian Agency for Drugs and Technologies in Health (CADTH)
Publication statusPublished - Jan 25 2018

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Prader-Willi Syndrome
Human Growth Hormone
Cost-Benefit Analysis
Guidelines
Randomized Controlled Trials
Therapeutics
Body Composition
Sample Size
Cognition
Uncertainty
Cohort Studies
Quality of Life
Pediatrics
Population

Cite this

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title = "Human Growth Hormone Treatment for Children with Prader-Willi Syndrome: A Review of Clinical Effectiveness, Cost-Effectiveness, and Guidelines",
abstract = "One small, high-quality randomized controlled trial, in addition to moderate quality evidence from four small randomized controlled trials, examining a total of 139 patients, and two cohort studies were identified on human growth hormone treatment of pediatric patients with Prader-Willi Syndrome. Improved body composition, behavioral benefits, and improved quality of life associated with human growth hormone treatment were suggested in the evidence. However, possibly due to small sample sizes, statistically significant treatment benefits were not consistently observed in the identified evidence. Inconsistent evidence for improved cognition was also identified, although the highest quality evidence suggested that, at least in patients with Prader-Willi Syndrome previously treated with human growth hormone until adult height was reached, no cognitive benefits manifested following one year of treatment. Significant uncertainty is therefore associated with the clinical effectiveness evidence in this report. Data on adverse events and risks of human growth hormone treatment was absent from the identified evidence on this patient population. No cost-effectiveness evidence or relevant guidelines were identified in the limited literature search.",
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AB - One small, high-quality randomized controlled trial, in addition to moderate quality evidence from four small randomized controlled trials, examining a total of 139 patients, and two cohort studies were identified on human growth hormone treatment of pediatric patients with Prader-Willi Syndrome. Improved body composition, behavioral benefits, and improved quality of life associated with human growth hormone treatment were suggested in the evidence. However, possibly due to small sample sizes, statistically significant treatment benefits were not consistently observed in the identified evidence. Inconsistent evidence for improved cognition was also identified, although the highest quality evidence suggested that, at least in patients with Prader-Willi Syndrome previously treated with human growth hormone until adult height was reached, no cognitive benefits manifested following one year of treatment. Significant uncertainty is therefore associated with the clinical effectiveness evidence in this report. Data on adverse events and risks of human growth hormone treatment was absent from the identified evidence on this patient population. No cost-effectiveness evidence or relevant guidelines were identified in the limited literature search.

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