Scoleri-Longo Yolanda, Pechlivanoglou Petros, Gupta Sumit
Department of Paediatrics Post Graduate Medical Education The Hospital for Sick Children Toronto Ontario Canada.
The Hospital for Sick Children Research Institute Toronto Ontario Canada.
EJHaem. 2023 Nov 13;5(1):166-177. doi: 10.1002/jha2.814. eCollection 2024 Feb.
Survival rates for pediatric acute lymphoblastic leukemia (pALL) have improved dramatically; relapsed/refractory (r/r) acute lymphoblastic leukemia (ALL) remains challenging. Immunotherapies are rapidly evolving treatments for r/r ALL with limited cost-effectiveness data. This study identifies existing economic evaluations of immunotherapy in pALL and summarizes cost-effectiveness. Medline, Embase, and other databases were searched from inception to October 2022. Cost-effectiveness analyses evaluating immunotherapy in pALL were included. Costs reported in 2021 USD. Of 2960 studies, 11 met inclusion criteria. Tisagenlecleucel was compared to standard of care, clofarabine monotherapy, clofarabine combination therapy, or blinatumomab. No studies have evaluated blinatumomab or inotuzumab ozogamicin. Six studies found tisagenlecleucel to be cost-effective, five of which were supported by Novartis. Four found that it had the potential to be cost-effective, and one found that it was not cost-effective. The cost-effectiveness of tisagenlecleucel was highly dependent on list price and cure rates. This study can inform the use of tisagenlecleucel in pALL.
儿童急性淋巴细胞白血病（pALL）的生存率已显著提高；复发/难治性（r/r）急性淋巴细胞白血病（ALL）仍然具有挑战性。免疫疗法是r/r ALL快速发展的治疗方法，但成本效益数据有限。本研究确定了pALL中免疫疗法的现有经济评估，并总结了成本效益。检索了从数据库建立至2022年10月的Medline、Embase和其他数据库。纳入了评估pALL中免疫疗法的成本效益分析。成本以2021年美元报告。在2960项研究中，11项符合纳入标准。将替雷利珠单抗与护理标准、氯法拉滨单药治疗、氯法拉滨联合治疗或博纳吐单抗进行了比较。没有研究评估博纳吐单抗或奥加伊妥珠单抗。六项研究发现替雷利珠单抗具有成本效益，其中五项得到诺华公司的支持。四项研究发现它有可能具有成本效益，一项研究发现它不具有成本效益。替雷利珠单抗的成本效益高度依赖于标价和治愈率。本研究可为替雷利珠单抗在pALL中的应用提供参考。
