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Effectiveness of Recombinant Human Growth Hormone (rhGH) in the Treatment of Patients With Cystic Fibrosis [Internet]

Effectiveness of Recombinant Human Growth Hormone (rhGH) in the Treatment of Patients With Cystic Fibrosis [Internet]

Comparative Effectiveness Reviews - Agency for Healthcare Research and Quality (US)

Version: October 2010

Summary and Discussion

A succinct summary of evidence on benefits and harms of using rhGH therapy in patients with CF in addition to usual care is presented in Table 31. More elaborate discussions are provided at the end of the results for each Key Question. More detailed assessments of strength of evidence for major clinical outcomes and harms are summarized in an EPC grading table of evidence (Appendix Tables F9–). Key Questions 1 and 2 focus on benefits while Key Question 4 focuses on nonmalignant harms and Key Question 6 focuses on malignancy. Benefits evaluated for included: pulmonary function (percent predicted FEV1 and change in FEV1), growth (height, weight, lean body mass, protein turnover), exercise tolerance, bone mineralization, frequency of required intravenous antibiotic treatments, frequency of hospitalization, quality of life, bone fracture or development of osteoporosis/osteopenia, or total mortality. Harms evaluated for included: glucose intolerance, diabetes, hypoglycemia, and malignancy. Members of the TEP identified these outcomes as important because they are most likely relevant to patients, clinicians, and policymakers. Key question 3 explored the associations between intermediate outcomes and final health outcomes. Key Questions 6 and 7 focused on factors that might impact the efficacy of rhGh in patients with CF or subpopulations that might receive rhGH therapy.

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