Optimal Management of the Cyanotic Neonate With Tetralogy of Fallot-A Clinical Decision Analysis

法洛四联症发绀新生儿的最佳管理——临床决策分析

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Abstract

BACKGROUND: Management options for critically cyanotic neonates with tetralogy of Fallot include primary repair, ductal or right ventricular outflow tract stents, and surgical shunts. However, rigorous comparisons between these strategies are precluded by small numbers, lack of equipoise, and center-specific bias. METHODS: A Markov model decision tree with Monte Carlo microsimulations was constructed to model 2-year outcomes for a hypothetical cohort of 10,000 cyanotic tetralogy of Fallot neonates eligible for all 3 strategies. Input transition state probabilities, utilities, and costs were derived from representative published reports. Outcomes were used to determine quality-adjusted life-years and costs after 50 model iterations. The incremental cost-effectiveness ratio was calculated to determine the preferred strategy. Sensitivity and threshold analysis varied probabilities of 40 factors to identify values at which the preferred strategy would switch. RESULTS: From modeling, immediate mortality from index procedure favored staged approaches, but total mortality favored primary repair after approximately 6 months. Cumulative 2-year mortality from modeling was 8.1%, 11.6%, and 12.4% for primary repair, stenting, and shunting, respectively. Calculated incremental cost-effectiveness ratios identified primary repair as the preferred strategy, followed by stenting and then shunting. Sensitivity and threshold analysis identified total pathway cost to be the only determinant of altered strategy preference with respect to primary repair. For comparisons of staged approaches, several variables reflecting cost and outcomes were identified. CONCLUSIONS: Our modeling suggests that primary repair may be superior to staging with stent or shunt for cyanotic neonates with tetralogy of Fallot, with improved 2-year morbidity, mortality, and cost utility.

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