Grain Audrey, Dourthe Marie-Emilie, Baruchel André
AP-HP, université de Paris, hôpital universitaire Robert-Debré, service d'hémato-immunologie pédiatrique, 48, boulevard Serurier, 75019 Paris, France.
AP-HP, université de Paris, hôpital universitaire Robert-Debré, service d'hémato-immunologie pédiatrique, 48, boulevard Serurier, 75019 Paris, France.
Bull Cancer. 2020 Feb;107(2):234-243. doi: 10.1016/j.bulcan.2020.01.009. Epub 2020 Feb 5.
The approval of tisagenlecleucel in B-lineage acute lymphoblastic leukemias in 2017 in the USA and in 2018 in Europe not only opened new hopes but forced to rethink the hospital organizations around this innovation. Indeed, if these treatments are very effective in the short term, the complex logistics required imply high quality inter-center and intra-center collaboration. Hematology, intensive care unit, apheresis, neurology, cell therapy and biology laboratories, and radiology services must therefore act in a coordinated manner. A specialized monitoring for the mid and long term must also be implemented. Many questions remain concerning the profile of eligible patients, the short and long-term safety, the longer-term efficacy, improving the persistence of CAR-T cells, controlling the risk of tumor escape, the use of allogenic CAR-T cells, or the application of this concept to T-cell ALL. The precise evaluation of the involved costs and the cost-effectiveness of these therapies will also be the subject of future studies.
2017年tisagenlecleucel在美国、2018年在欧洲被批准用于B系急性淋巴细胞白血病，这不仅带来了新希望，还促使人们围绕这一创新重新思考医院组织架构。的确，如果这些治疗在短期内非常有效，那么所需的复杂后勤工作意味着高质量的中心间和中心内协作。因此，血液科、重症监护室、血液分离术、神经科、细胞治疗和生物学实验室以及放射科服务必须协同行动。还必须实施中长期的专门监测。关于 eligible患者的特征、短期和长期安全性、长期疗效、提高CAR-T细胞的持久性、控制肿瘤逃逸风险、同种异体CAR-T细胞的使用，或将这一概念应用于T细胞急性淋巴细胞白血病等问题，仍有许多疑问。对这些疗法所涉及成本的精确评估以及成本效益也将是未来研究的主题。
