葡萄牙对所有符合条件的大 B 细胞淋巴瘤患者使用 axicabtagene ciloleucel 治疗对健康结局的影响——基于模型的方法

A Population Level Health Impact model was developed to obtain the weighted average results for 2L and 3L + models. Both underlying models used a partitioned survival model with extrapolations performed using mixture cure assumption. At model entry, patients were treated with axi-cel or hSoC. Each cohort of "year of entry/line of entry" was followed for 5 years. Outcomes were life years (LY), progression-free survival years (PFSY), quality-adjusted life-years (QALY), and absolute number and proportion of patients alive at 5 years and potentially cured (not progressed) at 5 years. Model inputs were based on publicly available literature, a modified Delphi panel, and real-world data. Utilities and mortality data were Portuguese-specific.

Of 284 axi-cel-eligible patients in 2024, 23 (8%) received axi-cel. Over 5 years, the Estimated Opportunity Lost by not treating all eligible patients with axi-cel was 203 LY, 45 deaths, and 36 patients that could have been potentially cured. The Potential Future Gains from treating all eligible patients with axi-cel are an estimated 527 more PFSY, 582 more LY, 119 more patients alive, and 85 more potentially cured.

The model suggests that limited axi-cel uptake in Portugal resulted in lost health benefits for axi-cel-eligible patients. When considering the next 3 years, substantial health benefits may be gained if all eligible patients receive axi-cel treatment.