Butt Ayesha, Allen Cecily, Purcell Adriana, Ito Satoko, Goshua George
Department of Internal Medicine, Yale School of Medicine, New Haven, CT 06510, USA.
Division of Hematology, Department of Medicine, Johns Hopkins University, Baltimore, MD 21218, USA.
J Clin Med. 2023 Jul 25;12(15):4887. doi: 10.3390/jcm12154887.
In this review, we examine the current landscape of health resource utilization and cost-effectiveness data in the care of patient populations with immune thrombotic thrombocytopenic purpura. We focus on the therapeutic (therapeutic plasma exchange, glucocorticoids, rituximab, caplacizumab) and diagnostic (ADAMTS13 assay) health technologies employed in the care of patients with this rare disease. Health resource utilization and cost-effectiveness data are limited to the high-income country context. Measurement of TTP-specific utility weights in the high-income country context and collection of health resource utilization data in the low- and middle-income country settings would enable an evaluation of country-specific quality-adjusted life expectancy and cost-effectiveness of these therapeutic and diagnostic health technologies. This quantification of value is one way to mitigate cost concerns where they exist.
在本综述中，我们审视了免疫性血栓性血小板减少性紫癜患者护理中卫生资源利用和成本效益数据的现状。我们重点关注用于治疗这种罕见疾病患者的治疗性（治疗性血浆置换、糖皮质激素、利妥昔单抗、卡泊单抗）和诊断性（ADAMTS13检测）卫生技术。卫生资源利用和成本效益数据仅限于高收入国家背景。在高收入国家背景下测量TTP特异性效用权重，并在低收入和中等收入国家环境中收集卫生资源利用数据，将有助于评估这些治疗和诊断卫生技术在各国的质量调整生命预期和成本效益。这种价值量化是在存在成本担忧的情况下减轻成本顾虑的一种方式。
