Published online December 29, 2008
PEDIATRICS Vol. 123 No. 1 January 2009, pp. 262-269 (doi:10.1542/peds.2007-2725)
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ARTICLE

Cost-effectiveness of Early Treatment for Retinopathy of Prematurity

Karen L. Kamholz, MD, MPHa,b, Cynthia H. Cole, MD, MPHa, James E. Gray, MD, MSc,d,e and John A. F. Zupancic, MD, ScDc,d

a Department of Pediatrics, Boston Medical Center, Boston, Massachusetts
b Boston University School of Medicine, Boston, Massachusetts
c Department of Neonatology
e Division of Clinical Computing, Beth Israel Deaconess Medical Center, Boston, Massachusetts
d Division of Newborn Medicine, Harvard Medical School, Boston, Massachusetts

BACKGROUND. The Early Treatment for Retinopathy of Prematurity trial demonstrated that peripheral retinal ablation of eyes with high-risk prethreshold retinopathy of prematurity (early treatment) is associated with improved visual outcomes at 9 months' corrected gestational age compared with treatment at threshold disease (conventional management). However, early treatment increased the frequency of laser therapy, anesthesia with intubation, treatment-related systemic complications, and the need for repeat treatments.

OBJECTIVE. To determine the cost-effectiveness of an early treatment strategy for retinopathy of prematurity compared with conventional management.

DESIGN/METHODS. We developed a stochastic decision analytic model to assess the incremental cost of early treatment per eye with severe visual impairment prevented. We derived resource-use and efficacy estimates from the Early Treatment for Retinopathy of Prematurity trial's published outcome data. We used a third-party payer perspective. Our primary analysis focused on outcomes from birth through 9 months' corrected gestational age. A secondary analysis used a lifetime horizon. Parameter uncertainty was quantified by using probabilistic and deterministic sensitivity analyses.

RESULTS. The incremental cost-effectiveness of early treatment was $14200 per eye with severe visual impairment prevented. There was a 90% probability that the cost-effectiveness of early treatment would be less than $40000 per eye with severe visual impairment prevented and a 0.5% probability that early treatment would be cost-saving (less costly and more effective). Limiting early treatment to more severely affected eyes (eyes with "type 1 retinopathy of prematurity" as defined by the Early Treatment for Retinopathy of Prematurity trial) had a cost-effectiveness of $6200 per eye with severe visual impairment prevented. Analyses that considered long-term costs and outcomes found that early treatment was cost-saving.

CONCLUSIONS. Early treatment of retinopathy of prematurity is both efficacious and economically desirable. Because of the high lifetime costs of severe visual impairment, the early treatment strategy provides long-term cost savings.


Key Words: infant • newborn • retinopathy of prematurity • costs and cost analysis

Abbreviations: ROP—retinopathy of prematurity • ETROP—Early Treatment for Retinopathy of Prematurity • ET—early treatment • QALY—quality-adjusted life-year


Accepted Apr 22, 2008.


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