Soohoo N, Schneider J A, Kaplan R M
University of California, San Diego 92093-0622, USA.
Med Decis Making. 1997 Apr-Jun;17(2):193-8. doi: 10.1177/0272989X9701700210.
Cysteamine is a recently licensed orphan drug used to treat the inherited metabolic disease cystinosis. The drug delays the onset of renal failure in cystinotic patients and may provide many other significant health benefits. This study examined the cost-effectiveness of the administration of cysteamine to cystinotic patients prior to end-stage renal disease (ESRD).
Decision-tree analysis and cost-effectiveness analysis. Cost data were estimated from current clinical charges and Medicare public-access reports. Life expectancy outcomes were derived from both published and unpublished clinical studies and from the U.S. Renal Data System.
Cysteamine therapy can extend the life of kidneys and delay renal transplantation, thereby increasing life expectancy for patients with cystinosis. Patients receiving cysteamine therapy prior to renal failure have lifetime-treatment drug costs of $234,000, in comparison with $238,000 for those who are not medicated. Costs of cysteamine therapy are offset by savings associated with delaying transplantation and costs of dialysis.
Use of the orphan drug cysteamine both improves health outcomes and reduces health care costs for patients with cystinosis.
半胱胺是一种最近获批的用于治疗遗传性代谢疾病胱氨酸病的孤儿药。该药物可延缓胱氨酸病患者肾衰竭的发作，并可能带来许多其他显著的健康益处。本研究探讨了在终末期肾病（ESRD）之前给胱氨酸病患者使用半胱胺的成本效益。
决策树分析和成本效益分析。成本数据根据当前临床费用和医疗保险公共访问报告估算得出。预期寿命结果来自已发表和未发表的临床研究以及美国肾脏数据系统。
半胱胺疗法可延长肾脏寿命并延迟肾移植，从而提高胱氨酸病患者的预期寿命。在肾衰竭之前接受半胱胺治疗的患者终身治疗药物成本为234,000美元，未接受药物治疗的患者为238,000美元。半胱胺疗法的成本因延迟移植相关的节省和透析成本而得到抵消。
使用孤儿药半胱胺既改善了胱氨酸病患者的健康结局，又降低了医疗保健成本。
