Effectiveness of Recombinant Human Growth Hormone in the Treatment of Cystic Fibrosis. Evidence-based Practice Center Systematic Review Protocol

摘要

Cystic fibrosis (CF) is the second most common life-shortening, childhood-onset genetic disease in the United States, affecting approximately 30,000 people in the U.S.1,2 It is most common among Caucasians, occurring in approximately 1 per 2,500 Caucasian births, compared with 1 per 15,100 African-American births and between 1 per 31,000 to 1 per more than 100,000 Asian-American births.3 CF is carried as an autosomal recessive trait in approximately 10 million Americans, and in approximately 3% of the Caucasian population. The gene responsible for CF encodes the cystic fibrosis transmembrane regulator (CFTR) protein, which regulates sodium and chloride transport across epithelial membranes. Defects in the CFTR protein result in a multisystem disorder affecting nearly all exocrine glands, with abnormally viscous mucus and excessive secretions. The dominant clinical features are chronic lung disease and pancreatic insufficiency with poor nutrition and poor growth. Treatment has improved considerably over the past 25 years, resulting in improvements in measures of malnutrition, lung function, and mortality among children and adolescents with CF. The median age of survival has improved consistently from 1955 (5 year survival) to 1969 (14 year survival), 1985 (25 year survival), and to the most recent data in 2006 (37 year survival). The estimated annual direct medical costs per CF patient are more than $40,000, with an estimated $9,000 in secondary costs per year per patient.