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比较持续性非卧床腹膜透析中皮下注射促红细胞生成素三种给药方案的决策分析

A decision analysis comparing three dosage regimens of subcutaneous epoetin in continuous ambulatory peritoneal dialysis.

作者信息

Piccoli A, Puggia R M, Fusaro M, Favaro E, Pillon L

机构信息

Division of Nephrology, University of Padova, Italy.

出版信息

Pharmacoeconomics. 1995 May;7(5):444-56. doi: 10.2165/00019053-199507050-00008.

Abstract

Epoetin (recombinant human erythropoietin; EPO) therapy adds a significant cost to the management of end-stage renal disease, the majority of the extra expense being attributable to its acquisition cost. In a Japanese multicentre, randomised, prospective study, a significant dose-dependent response was documented with epoetin given subcutaneously (SC) once a week or once every 2 weeks to patients receiving continuous ambulatory peritoneal dialysis. Three different dosages were studied over 5 months in patients with a haematocrit (Hct) of 0.28 or less, namely 6000U (107 U/kg), 9000U (167 U/kg) and 12,000U (211 U/kg). Epoetin was given weekly for the first 2 months until the target Hct value of 0.33 was reached. The rates of response were 81, 85 and 100% with the 6000U, 9000U and 12,000U regimens, respectively. Subsequently, responders were maintained at the target Hct for an additional 3 months, with the administration frequency eventually being reduced to fortnightly or 4-weekly. Patients in the epoetin 6000U and 9000U groups who did not respond after 2 months' treatment underwent induction and maintenance with the 12,000U regimen. During the maintenance phase, patients receiving the epoetin 6000U and 9000U dosages required weekly (54 and 64%, respectively) or fortnightly (46 and 36%, respectively) injections. Patients receiving the 12,000U regimen were found to require weekly (9%), fortnightly (73%) or 4-weekly (18%) injections. Using these data, we performed a decision analysis that quantitatively incorporated the probability of attaining and maintaining target Hct levels in all patients (i.e. the effectiveness of epoetin), and direct costs as a function of both cumulative doses and injections required in all 3 strategies over 5 months. Decision analysis indicated that the most cost-effective SC epoetin strategy in patients undergoing peritoneal dialysis is epoetin 6000U weekly for 2 months, followed by maintaining the target Hct with weekly or 2-weekly epoetin 6000U for the next 3 months. Nonresponders should restart epoetin therapy using the 12,000U strategy. The 9000U and 12,000U strategies were associated with similar costs, because the economic advantages associated with the lower administration frequency of the 9000U regimen compared with the 6000U regimen were offset by its higher cumulative acquisition cost. In other words, decision analysis indicated that the most cost-effective strategy was to use the lowest effective dose, reserving the highest dosage for patients who do not respond after 2 months. The superiority of this strategy was confirmed by a sensitivity analysis performed on the cost of drug administration, which was varied from zero to $US60 per dose.(ABSTRACT TRUNCATED AT 400 WORDS)

摘要

促红细胞生成素(重组人促红细胞生成素;EPO)疗法给终末期肾病的治疗增加了显著成本,大部分额外费用归因于其购置成本。在一项日本多中心、随机、前瞻性研究中,对接受持续性非卧床腹膜透析的患者皮下注射(SC)促红细胞生成素,每周一次或每两周一次,记录到显著的剂量依赖性反应。对血细胞比容(Hct)为0.28或更低的患者在5个月内研究了三种不同剂量,即6000U(107U/kg)、9000U(167U/kg)和12000U(211U/kg)。促红细胞生成素在前2个月每周给药,直至达到目标Hct值0.33。6000U、9000U和12000U方案的反应率分别为81%、85%和100%。随后,反应者在目标Hct水平维持3个月,给药频率最终减至每两周一次或每四周一次。促红细胞生成素6000U组和9000U组中2个月治疗后无反应的患者采用12000U方案进行诱导和维持治疗。在维持阶段,接受促红细胞生成素6000U和9000U剂量的患者分别需要每周(分别为54%和64%)或每两周(分别为46%和36%)注射。接受12000U方案的患者需要每周(9%)、每两周(73%)或每四周(18%)注射。利用这些数据,我们进行了一项决策分析,定量纳入了所有患者达到并维持目标Hct水平的概率(即促红细胞生成素的有效性),以及作为所有三种策略在5个月内累积剂量和所需注射次数函数的直接成本。决策分析表明,腹膜透析患者中最具成本效益的皮下注射促红细胞生成素策略是促红细胞生成素6000U每周注射2个月,随后在接下来的3个月用促红细胞生成素6000U每周或每两周维持目标Hct水平。无反应者应采用12000U策略重新开始促红细胞生成素治疗。9000U和12000U策略的成本相似,因为9000U方案与6000U方案相比给药频率较低的经济优势被其较高的累积购置成本所抵消。换句话说,决策分析表明最具成本效益的策略是使用最低有效剂量,将最高剂量留给2个月后无反应的患者。对药物给药成本进行从0到每剂60美元变化的敏感性分析证实了该策略的优越性。(摘要截短至400字)

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