PO.ADV01 · 患者倡导
儿童CAR T细胞疗法的伦理与可及性
Ethics and access in pediatric CAR T-cell therapy
作者与单位 Authors & Affiliations
摘要 Abstract
中文摘要
先进的基因和细胞疗法，包括tisagenlecleucel（又称Kymriah）等CAR T细胞治疗，已显著改善了复发或难治性B细胞急性淋巴细胞白血病（ALL）儿童患者的预后。然而，以临床入选标准、机构能力和经济障碍为形式的可及性问题，持续阻碍着家庭和患者寻求这些治疗选择。当前的资格要求患者必须在常规治疗选择用尽后出现难治性疾病或多次复发，才有资格接受CAR T细胞疗法，这延误了获得已证实有效治疗的时机。尽管旨在挽救生命，但卫生政策的实施和相关规程限制了这些疗法所能产生的最大效益，同时可能加重患者的疾病进展并给家庭带来更高的经济负担。本海报采用生物伦理学视角审视当前儿童CAR T细胞疗法的可及性标准，探讨四项主要生物伦理原则：自主、有利、不伤害与公正。本海报从患者倡导的角度出发，重点指出了与以患者为中心的需求相契合的改进机会，涵盖先进基因细胞疗法的开发、实施和实践。
查看英文原文 English abstract
Advanced gene and cell therapies, including CAR T-celltreatments such as tisagenlecleucel (also known as Kymriah), have significantlyimproved outcomes for pediatric patients with relapsed or refractory B-cellAcute Lymphoblastic Leukemia (ALL). However, access, in the form of clinicaleligibility criteria, institutional capacity, and financial barriers, continuesto impede families and patients from pursuing these options. Currentqualifications require refractory disease or multiple relapses following theexhaustion of conventional treatment options before a patient becomes eligiblefor CAR T-cell therapy, delaying access to treatments with demonstratedefficacy. While designed to save lives, health policy implementations andprotocols limit the maximum impact of these therapies, while potentiallycontributing to disease progression for patients and higher financial burdenson families. This poster applies a bioethical approach to the currentaccessibility standards of pediatric CAR T-cell therapy, addressing the fourmain bioethical imperatives: autonomy, beneficence, non-maleficence, andjustice. From a patient advocacy perspective, this poster highlightsopportunities for improvement specifically aligned with patient-centered needsin advanced gene cell therapy development, implementation, and practice.