The purpose of this report is to review the clinical effectiveness of human growth hormone (GH) treatment for Prader-Willi syndrome (PWS) in adolescent and adult patients and the evidence based guidelines for the use of GH for treating adolescents and adults with PWS. This report is an update of a previous CADTH Rapid Response Report which included one meta-analysis, two RCTs and 10 non-randomized studies and reported that GH therapy in patients with PWS, had multiple beneficial effects on body composition and growth and a few negative effects.

Contents

• CONTEXT AND POLICY ISSUES
• RESEARCH QUESTIONS
• KEY FINDINGS
• METHODS
  • Literature Search Methods
  • Selection Criteria and Methods
  • Exclusion Criteria
  • Critical Appraisal of Individual Studies
• SUMMARY OF EVIDENCE
  • Quantity of Research Available
  • Summary of Study Characteristics
  • Summary of Critical Appraisal
  • Summary of Findings
  • Limitations
• CONCLUSIONS AND IMPLICATIONS FOR DECISION OR POLICY MAKING
• REFERENCES
• ABBREVIATIONS
• APPENDIX 1 Selection of Included Studies
• APPENDIX 2 Characteristics of Included Publications
• APPENDIX 3 Grading of Recommendations and Levels of Evidence
• APPENDIX 4 Critical Appraisal of Included Publications
• APPENDIX 5 Main Study Findings and Author’s Conclusions
• APPENDIX 6 Guidelines and Recommendations

Disclaimer: The Rapid Response Service is an information service for those involved in planning and providing health care in Canada. Rapid responses are based on a limited literature search and are not comprehensive, systematic reviews. The intent is to provide a list of sources of the best evidence on the topic that CADTH could identify using all reasonable efforts within the time allowed. Rapid responses should be considered along with other types of information and health care considerations. The information included in this response is not intended to replace professional medical advice, nor should it be construed as a recommendation for or against the use of a particular health technology. Readers are also cautioned that a lack of good quality evidence does not necessarily mean a lack of effectiveness particularly in the case of new and emerging health technologies, for which little information can be found, but which may in future prove to be effective. While CADTH has taken care in the preparation of the report to ensure that its contents are accurate, complete and up to date, CADTH does not make any guarantee to that effect. CADTH is not liable for any loss or damages resulting from use of the information in the report.