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Neonatal screening for sickle cell disease: a cost-effectiveness analysis
J Tsevat1, J B Wong, S G Pauker
1Department of Medicine, New England Medical Center, Boston, MA 02111.
Insights
Screening newborn infants for sickle cell disease (SCD) is cost-effective for Black infants, saving lives at a minimal additional cost. However, screening non-Black populations with low SCD prevalence is not justified due to extremely high costs per life saved.
Area of Science:
- Public Health
- Genetics
- Pediatrics
Background:
- Sickle cell disease (SCD) is a genetic blood disorder.
- Early detection and intervention, such as penicillin prophylaxis, can prevent serious complications like pneumococcal sepsis.
- Current screening practices and their cost-effectiveness vary across different populations.
Purpose of the Study:
- To evaluate the cost-effectiveness of screening newborn infants for sickle cell disease.
- To compare two strategies: universal screening with penicillin prophylaxis versus symptomatic diagnosis with penicillin treatment.
- To analyze cost-effectiveness across diverse neonatal populations with varying hemoglobin S gene prevalence.
Main Methods:
- A decision-analytic model was developed to compare screening versus no screening strategies.
- Data on penicillin prophylaxis effectiveness, sepsis risk, and disease prevalence were sourced from published literature.
- Costs associated with screening, prophylaxis, and hospitalization were calculated using actual variable costs.
Main Results:
- For Black infants, screening and penicillin prophylaxis resulted in a cost of $3100 per life saved compared to no screening.
- Screening non-Black infants in high hemoglobin S gene prevalence populations had a cost-effectiveness of $1.4 million per life saved.
- Screening non-Black infants in low hemoglobin S gene prevalence populations was prohibitively expensive at $450 billion per life saved.
Conclusions:
- Newborn screening for sickle cell disease is highly cost-effective and recommended for Black infants.
- Screening is not justified in populations with a low prevalence of the sickle cell trait.
- Targeted screening strategies are crucial for optimizing public health resources in SCD prevention.
Purpose:
To determine the cost-effectiveness of screening newborn infants for sickle cell disease.
Design:
We developed a decision model that examined two strategies: (1) screening neonates and administering penicillin to infants found to have sickle cell disease in the hope of preventing pneumococcal sepsis, and (2) not screening but administering penicillin to infants after symptoms of sickle cell disease develop. The model calculates the cost-effectiveness of these strategies during the first 3 years of life. We applied the model to three prototypic populations of neonates--black, nonblack with a relatively high prevalence of hemoglobin S genes, and nonblack with a low prevalence of hemoglobin S genes.
Data Identification:
We obtained from the published literature the effectiveness and risk of penicillin prophylaxis, the risk of pneumococcal sepsis, and the probability that in infants not screened the development of symptoms would lead to the discovery of sickle cell disease within the first 3 years of life; we used the published literature and the Hardy-Weinberg law to determine the prevalence of sickle cell disease. We used actual variable costs of screening, antibiotic prophylaxis, and hospitalization for pneumococcal sepsis or anaphylaxis.
Results:
Screening and then treating affected black infants costs only $3100 more per life saved than not screening. Screening nonblack populations with a high prevalence of hemoglobin S genes would cost $1.4 million per life saved, and screening low prevalence populations would cost $450 billion per life saved.
Conclusions:
Screening black infants is very worthwhile, but screening populations in which the hemoglobin S gene is rare is unjustified.