Moore R D, Charache S, Terrin M L, Barton F B, Ballas S K
Johns Hopkins University School of Medicine, Baltimore, Maryland 21205, USA.
Am J Hematol. 2000 May;64(1):26-31. doi: 10.1002/(sici)1096-8652(200005)64:1<26::aid-ajh5>3.0.co;2-f.
The Multicenter Study of Hydroxyurea in Sickle Cell Anemia (MSH) demonstrated the efficacy of hydroxyurea in reducing the rate of painful crises compared to placebo. We used resource utilization data collected in the MSH to determine the cost-effectiveness of hydroxyurea. The MSH was a randomized, placebo-controlled double-blind clinical trial involving 299 patients at 21 sites. The primary outcome, visit to a medical facility, was one of the criteria to define occurrence of painful crisis. Cost estimates were applied to all outpatient and emergency department visits and inpatient hospital stays that were classified as a crisis. Other resources for which cost estimates were applied included hospitalization for chest syndrome, analgesics received, hydroxyurea dosing, laboratory testing, and clinic visits for management of patient care. Annualized differential costs were calculated between hydroxyurea- and placebo-receiving patients. Hospitalization for painful crisis accounted for the majority of costs in both arms of the study, with an annual mean of $12,160 (95% CI: $9,440, $14,880) for hydroxyurea and $17,290 (95% CI: $13,010, $21,570) for placebo. The difference in means was $5,130 (95% CI: $60, $10,200; P = 0.048). Chest syndrome was the next largest cost with a mean difference of $830 (95% CI: $-340, $2,000; P = 0.16). The hydroxyurea arm was also associated with lower costs for emergency department visits, transfusion, and use of opiate analgesics. In total, the annual average cost per patient receiving hydroxyurea was $16,810 (95% CI: $13,350, $20,270) and the annual average costs per patient receiving placebo was $22,020 (95% CI: $17,340, $26,710). The difference in means was $5,210 (95% CI: $-610, $11,030; P = 0.21). The cost of hydroxyurea with the more intensive monitoring required when using this drug appears to be more than offset by decreased costs for medical care of painful crisis and analgesic use. Although the total cost difference was not significant statistically, these results suggest that hydroxyurea therapy is cost-effective compared to placebo in the management of adult patients with sickle cell anemia. If hydroxyurea can prevent development of chronic organ damage, long-term savings may be even greater.
镰状细胞贫血羟基脲多中心研究（MSH）表明，与安慰剂相比，羟基脲在降低疼痛性危象发生率方面具有疗效。我们利用MSH中收集的资源利用数据来确定羟基脲的成本效益。MSH是一项随机、安慰剂对照的双盲临床试验，涉及21个地点的299名患者。主要结局指标——前往医疗机构就诊，是定义疼痛性危象发生的标准之一。成本估算应用于所有被归类为危象的门诊、急诊科就诊及住院治疗。其他应用成本估算的资源包括胸部综合征住院治疗、所接受的镇痛药、羟基脲给药、实验室检查以及用于患者护理管理的门诊就诊。计算接受羟基脲治疗和接受安慰剂治疗患者之间的年化差异成本。疼痛性危象住院治疗占研究两组成本的大部分，羟基脲组年均成本为12,160美元（95%CI：9,440美元，14,880美元），安慰剂组为17,290美元（95%CI：13,010美元，21,570美元）。均值差异为5,130美元（95%CI：60美元，10,200美元；P = 0.048）。胸部综合征是第二大成本，均值差异为830美元（95%CI： - 340美元，2,000美元；P = 0.16）。羟基脲组在急诊科就诊、输血及使用阿片类镇痛药方面的成本也较低。总体而言，接受羟基脲治疗的患者年均成本为16,810美元（95%CI：13,350美元，20,270美元），接受安慰剂治疗的患者年均成本为22,020美元（95%CI：17,340美元，26,710美元）。均值差异为5,210美元（95%CI： - 610美元，11,030美元；P = 0.21）。使用该药物时所需更强化监测的羟基脲成本，似乎因疼痛性危象医疗护理及镇痛药使用成本的降低而得到更多抵消。尽管总成本差异在统计学上不显著，但这些结果表明，在成年镰状细胞贫血患者的管理中，与安慰剂相比，羟基脲治疗具有成本效益。如果羟基脲能够预防慢性器官损伤的发生，长期节省可能会更大。
