Soliman Ranin, Heneghan Carl, Bolous Nancy S, Sidhom Iman, Ahmed Sonia, Roberts Nia, Oke Jason, Elhaddad Alaa
Department for Continuing Education, University of Oxford, Oxford, UK.
Health Economics and Value Unit, Children's Cancer Hospital, Cairo, Egypt.
Expert Rev Hematol. 2022 Apr;15(4):345-357. doi: 10.1080/17474086.2022.2069096. Epub 2022 May 4.
Survival outcomes of children with relapsed/refractory (r/r) acute leukemia remain poor. Novel expensive treatments have been developed to improve their outcomes, yet, limited evidence exists about cost-effectiveness of alternative treatment strategies.
A systematic review was conducted to summarize health-economic evidence about costs/cost-effectiveness of treating r/r acute leukemia in children/young adults. We searched Medline, Embase, and Cochrane databases until August 13th, 2021. Eligible articles included peer-reviewed original studies addressing r/r pediatric/young-adult acute lymphoblastic leukemia (ALL), and acute myeloid leukemia (AML). Quality assessment was conducted using Consolidated Health Economics Evaluation Reporting Standards (CHEERS) checklist.
The majority of papers focused on CAR-T cell therapy, which is still a novel treatment for r/r ALL, and was found to be cost-effective, yet, there remain concerns over its long-term effectiveness, affordability, and equity in access. The next best treatment option is Blinatumomab, followed by Clofarabine therapy, whereas FLA-IDA salvage chemotherapy provides least value for money. The quality of evidence is moderate to high, with limited generalizability of findings due to high variability in outcomes obtained from modeling studies. Limited studies evaluated r/r AML. We provide recommendations to deliver cost-effective treatments in real-world contexts, with implications for healthcare policy and practice.
复发/难治性（r/r）急性白血病患儿的生存结局仍然很差。已开发出新型昂贵的治疗方法来改善其结局，然而，关于替代治疗策略的成本效益的证据有限。
进行了一项系统综述，以总结有关治疗儿童/青年r/r急性白血病的成本/成本效益的卫生经济证据。我们检索了Medline、Embase和Cochrane数据库，直至2021年8月13日。符合条件的文章包括同行评审的原创研究，涉及r/r儿童/青年急性淋巴细胞白血病（ALL）和急性髓细胞白血病（AML）。使用综合卫生经济评估报告标准（CHEERS）清单进行质量评估。
大多数论文关注嵌合抗原受体T细胞（CAR-T）疗法，这仍是一种用于r/r ALL的新型治疗方法，被发现具有成本效益，然而，人们仍对其长期有效性、可负担性和获得的公平性存在担忧。次优治疗选择是博纳吐单抗，其次是氯法拉滨疗法，而氟达拉滨-去甲氧柔红霉素挽救化疗的性价比最低。证据质量为中等至高，由于模型研究获得的结果差异很大，研究结果的可推广性有限。评估r/r AML的研究有限。我们提供了在现实环境中提供具有成本效益的治疗的建议，对医疗政策和实践具有启示意义。
