Jønsson V, Hansen M M, Ljungman P, Kaasa S
Department of Haematology, Finsen Center, Rigshospitalet, University of Copenhagen, Denmark.
Pharmacoeconomics. 1999 Feb;15(2):167-78. doi: 10.2165/00019053-199915020-00005.
Whereas individual cost-effectiveness analyses of new agents for acute leukaemia should be performed in target populations, any meaningful pharmacoeconomic evaluation of treatment options for this condition should include the many types of costs and outcomes in unselected, representative groups of patients. Both direct costs (e.g. costs for medication and hospitalisation) and indirect costs (e.g. lost productivity costs and reduced quality of life) are important parameters to assess, as are the costs of chronic adverse effects, research and development costs for new agents, and costs of procedure-related deaths. Complete remission, cure and survival are the 'success' response criteria for acute leukaemia treatments, in addition to prolonged life with acceptable quality of life for patients with incurable acute leukaemia. Death is 'failure', caused either by resistant disease (relapse and progressive disease) inspite of optimal chemotherapy or, sometimes, by insufficient treatment. All of these parameters should be taken into account when a pharmacoeconomic evaluation is performed (either for administrative or scientific purposes) in order to ensure a comprehensive and reliable background for the evaluation in question. Treatment of acute leukaemia is expensive with a total cost of about $US3000 per patient per day during the induction. Although 80% of children with acute leukaemia are cured, only less than 50% of adults are cured. Thus, a great cost is associated with death during treatment and only optimal medical treatment with full-scale combination chemotherapy and full supportive treatment can keep the number of deaths to a minimum.
虽然新型急性白血病治疗药物的个体成本效益分析应在目标人群中进行，但针对这种疾病的任何有意义的药物经济学治疗方案评估都应纳入未筛选的代表性患者群体中的多种成本和结果。直接成本（如药物和住院费用）和间接成本（如生产力损失成本和生活质量下降）都是重要的评估参数，慢性不良反应成本、新型药物的研发成本以及与治疗相关的死亡成本也同样重要。完全缓解、治愈和生存是急性白血病治疗的“成功”反应标准，对于无法治愈的急性白血病患者而言，延长生命并使其生活质量可接受也是标准之一。死亡即“失败”，其原因要么是尽管进行了最佳化疗但疾病仍具耐药性（复发和疾病进展），要么有时是治疗不足。在进行药物经济学评估（无论是出于管理目的还是科学目的）时，所有这些参数都应予以考虑，以确保所评估问题有全面且可靠的背景依据。急性白血病的治疗费用高昂，诱导治疗期间每位患者每天的总成本约为3000美元。虽然80%的急性白血病儿童可被治愈，但只有不到50%的成人能够治愈。因此，治疗期间的死亡会带来巨大成本，只有采用全面联合化疗和充分支持治疗的最佳医疗方案才能将死亡人数降至最低。
