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"pulmonary function"

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Effectiveness of Questionnaires and Clinical Prediction Rules

Topic Refinement Process: UCONN/HH EPC Example (VII). Questions for Key Informants: How different is a study including people up to 23 years of age from a study limited to 18-year-olds? Consider this given the inverse linear relationship between human growth hormone efficacy and age, as well as the delayed growth and maturation inherent in this population. How readily do you feel we can combine certain end points (e.g., lean body mass measured by different methods [DEXA, skin folds, BIA] in same units) or amino acid catabolism measures (leucine vs. glutamine)?

Topic Refinement Process: UCONN/HH EPC Example (VII)

Topic Refinement Process: UCONN/HH EPC Example (VIII). Questions for Key Informants: In trials/studies that report multiple similar end points (like pulmonary function tests), which surrogate end points would be optimal (i.e., FEV1 percent vs. FEV1 absolute value)? Given the paucity of harms data, could harms data from patients without cystic fibrosis be used to inform the potential risks associated with human growth hormone therapy (i.e., how similar are patients with hypogonadism to those with cystic fibrosis and poor growth progression)? In particular, are you wary of using human growth hormone because of its risk of neoplasms?

Topic Refinement Process: UCONN/HH EPC Example (VIII)

Key Questions (I). KQ 1: In patients with cystic fibrosis, does treatment with rhGH as an adjuvant to usual care improve intermediate outcomes, including pulmonary function (FEV percent and FEV1 absolute value), growth (height, weight, lean body mass, protein turnover), exercise tolerance, and bone mineralization, compared with usual care alone? KQ 2: In patients with cystic fibrosis, does treatment with rhGH as an adjuvant to usual care improve health outcomes, including frequency of required intravenous antibiotic treatments, frequency of hospitalization, quality of life, bone fracture or development of osteoporosis/osteopenia, or mortality, compared with usual care alone? KQ 3: In patients with cystic fibrosis, what is the strength of evidence that intermediate outcomes of pulmonary function, growth, and bone mineralization are associated with improvements in health outcomes of quality of life, bone fracture or development of osteoporosis/osteopenia, or mortality? KQ 4: In patients with cystic fibrosis, what is the frequency of nonmalignant serious adverse effects resulting from treatment with rhGH? Adverse effects of interest include, but are not limited to: glucose intolerance, diabetes, and hypoglycemia.

Key Questions (I)

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