Newborn Screening and Treatment of Phenylketonuria: Projected Health Outcomes and Cost-Effectiveness.

摘要: The objective of this study was to evaluate the cost-effectiveness newborn screening and treatment for phenylketonuria (PKU) in context new data on adherence recommended diet a newly available drug (sapropterin dihydrochloride). A computer simulation model developed project outcomes hypothetical cohort newborns with PKU. Four strategies were compared: (1) clinical identification (CI) treatment; (2) (NBS) (3) CI medication dihydrochloride); (4) NBS medication. Data sources included published literature, primary data, expert opinion. From societal perspective, had an incremental ratio $6400/QALY compared treatment. Adding resulted more than $16,000,000/QALY. Uncertainty analyses did not substantially alter results. Newborn PKU yields lower many other childhood prevention programs even if is previously assumed. results unlikely be considered favorable. Future research should consider conditions under which sapropterin dihydrochloride would economically attractive.