Cosler Leon E, Eldar-Lissai Adi, Culakova Eva, Kuderer Nicole M, Dale David, Crawford Jeffrey, Lyman Gary H
Albany College of Pharmacy, Albany, New York, USA.
Pharmacoeconomics. 2007;25(4):343-51. doi: 10.2165/00019053-200725040-00006.
The prophylactic use of granulocyte colony-stimulating factors (G-CSFs) reduces the severity and duration of neutropenia and reduces the incidence of febrile neutropenia after cancer chemotherapy. However, the use of G-CSFs, particularly filgrastim, to treat established neutropenia remains controversial. A recent meta-analysis of randomised controlled trials (RCTs) evaluating G-CSF treatment for established febrile neutropenia demonstrated a reduction in prolonged hospitalisations. Because more than one-third of patients in the analysis were hospitalised for at least 10 days, this finding has broad pharmacoeconomic and clinical significance. This analysis presents the potential cost implications of G-CSF treatment for established neutropenia among hospitalised patients.
Direct medical costs ($US, year 2003 values) related to hospitalisation for established neutropenia were modelled using a hospital perspective and according to two treatment options: (i) no use of G-CSF during the neutropenic episode (control); and (ii) addition of daily G-CSF until neutrophil recovery. Within each option, we modelled the probability of a long stay (>or=10 days) and patient survival. The model used three data sets: discharge data from a consortium of academic medical institutions, drug cost data (filgrastim) from Federal payers, and estimates of G-CSF efficacy derived from a meta-analysis of RCTs of treatment in patients with established febrile neutropenia. The lowest expected total cost was predicted for both treatment options; sensitivity analyses and Monte Carlo simulations were used to evaluate the robustness of the model.
The G-CSF arm produced the lowest expected cost, and predicted net estimated savings of $US1046 per neutropenic episode compared with the control strategy. G-CSF was less expensive than the control for most reasonable estimates of cost per day and all lengths of stay (LOS) >or=10 days. G-CSF was the least costly strategy for 73.5% of 10,000 Monte Carlo iterations, while the no-G-CSF control strategy predicted savings in 26.5% of iterations.
This pharmacoeconomic model suggests that therapeutic use of G-CSF should be considered for patients with established neutropenia in order to reduce overall hospital cost. G-CSF treatment may offer substantial potential savings for hospitalised patients with established neutropenia over a wide range of model assumptions. Therapeutic G-CSF use among patients hospitalised for established neutropenia may complement the recommended prophylactic use of these agents for the prevention of neutropenic episodes.
预防性使用粒细胞集落刺激因子(G-CSF)可减轻中性粒细胞减少的严重程度和持续时间,并降低癌症化疗后发热性中性粒细胞减少的发生率。然而,使用G-CSF,尤其是非格司亭,来治疗已确诊的中性粒细胞减少症仍存在争议。最近一项对评估G-CSF治疗已确诊发热性中性粒细胞减少症的随机对照试验(RCT)的荟萃分析表明,可减少住院时间延长的情况。由于分析中超过三分之一的患者住院至少10天,这一发现具有广泛的药物经济学和临床意义。本分析呈现了G-CSF治疗已确诊住院患者中性粒细胞减少症的潜在成本影响。
从医院角度,根据两种治疗方案对与已确诊中性粒细胞减少症住院相关的直接医疗成本(2003年美元价值)进行建模:(i)中性粒细胞减少发作期间不使用G-CSF(对照);(ii)每日添加G-CSF直至中性粒细胞恢复。在每种方案中,我们对长时间住院(≥10天)和患者生存的概率进行建模。该模型使用了三个数据集:学术医疗机构联盟的出院数据、联邦支付者的药物成本数据(非格司亭)以及从已确诊发热性中性粒细胞减少症患者治疗的RCT荟萃分析得出的G-CSF疗效估计值。预测了两种治疗方案的最低预期总成本;采用敏感性分析和蒙特卡罗模拟来评估模型的稳健性。
G-CSF组产生了最低的预期成本,与对照策略相比,预计每例中性粒细胞减少发作可节省净估计成本1046美元。对于大多数合理的每日成本估计以及所有住院时间(LOS)≥10天的情况,G-CSF比对照便宜。在10000次蒙特卡罗迭代中,73.5%的情况G-CSF是成本最低的策略,而无G-CSF对照策略在26.5%的迭代中预计可节省成本。
这个药物经济学模型表明,对于已确诊中性粒细胞减少症的患者,应考虑使用G-CSF进行治疗,以降低总体医院成本。在广泛的模型假设下,G-CSF治疗可能为已确诊中性粒细胞减少症的住院患者带来可观的潜在成本节省。对于因已确诊中性粒细胞减少症住院的患者,治疗性使用G-CSF可能补充这些药物推荐的预防性使用,以预防中性粒细胞减少发作。