Quality Care Cancer Alliance, Tacoma, Washington, USA.
Department of Public Health, Syracuse University, Syracuse, New York, USA.
Acta Haematol. 2024;147(3):333-343. doi: 10.1159/000533175. Epub 2023 Sep 29.
Chronic immune thrombocytopenia purpura (ITP) in adults is a serious autoimmune disease in which platelets are prematurely destroyed, leaving the patient vulnerable to bruising and bleeding. Initial treatment starts with corticosteroids. In patients who become resistant or intolerant to corticosteroids, the thrombopoietic agents (TPOs), consisting of romiplostim (ROM), eltrombopag (ELT), and avatrombopag (AVA), or the spleen tyrosine kinase inhibitor fostamatinib (FOS), are appropriate next lines of therapy. In this study, the comparative safety, effectiveness, and cost of care between fostamatinib and the TPOs were evaluated in a real-world setting.
A retrospective analysis of 17 community hematology practices across the USA was conducted to identify adult ITP patients who received one of the four agents. Data collection consisted of patient demographics, disease characteristics, as well as number and type of prior treatments. From the first day until the end of treatment, data were also collected on platelet (PLT) counts, adverse events, the use of rescue IVIG, platelet transfusions, and corticosteroids. Multivariable logistic regression analysis was used to compare PLT-related endpoints between agents.
A sample of 179 ITP patients who had received at least one of the four agents was identified. This resulted in a final sample of 51, 87, 127, and 44 patients who received FOS, ELT, ROM, or AVA, respectively. At month six, there were no significant differences between FOS and the TPOs in terms of the proportion of patients with the PLT count being ≥30 × 103/μL, ≥50 × 103/μL as well as the proportion of patients whose PLT levels doubled relative to baseline. The frequency of thromboembolic events (TEs) was 3.9% in FOS patients compared to 9.2%, 4.7%, and 11.4% in the ELT, ROM, and AVA groups. The mean cost per patient with FOS was $99,209 (95% CI: $59,595-$115,074), compared to $92,426 (95% CI: $68,331-$115,519), $108,482 (95% CI: $84,782-$132,182), and $131,050 (95% CI: $83,327-$179,897) for ELT, ROM, or AVA, respectively.
In this real-world analysis, FOS was comparable to the TPOs in maintaining PLTs at clinically beneficial levels. Given these findings, the choice of therapy should be based on overall patient safety, preexisting risk factors for TEs, and cost effectiveness.
成人慢性免疫性血小板减少性紫癜（ITP）是一种严重的自身免疫性疾病，其中血小板过早被破坏，使患者容易出现瘀伤和出血。初始治疗采用皮质类固醇。对于对皮质类固醇耐药或不耐受的患者，可使用促血小板生成素（TPO），包括罗米司亭（ROM）、艾曲波帕（ELT）和阿伐曲泊帕（AVA）或脾酪氨酸激酶抑制剂芦可替尼（FOS）作为下一线治疗方案。在这项研究中，在真实世界环境中评估了芦可替尼与 TPO 之间的安全性、有效性和治疗成本。
对美国 17 家社区血液病诊所进行了回顾性分析，以确定接受四种药物之一的成人 ITP 患者。数据收集包括患者人口统计学特征、疾病特征以及先前治疗的次数和类型。从治疗的第一天到结束，还收集了血小板（PLT）计数、不良事件、IVIG 抢救使用、血小板输注和皮质类固醇的数据。使用多变量逻辑回归分析比较药物之间的 PLT 相关终点。
确定了至少接受四种药物之一的 179 名 ITP 患者的样本，最终确定了 51、87、127 和 44 名分别接受 FOS、ELT、ROM 或 AVA 的患者。在第六个月，FOS 组与 TPO 组在 PLT 计数≥30×103/μL、≥50×103/μL的患者比例以及 PLT 水平相对于基线翻倍的患者比例方面没有显著差异。FOS 组血栓栓塞事件（TE）的发生率为 3.9%，ELT、ROM 和 AVA 组分别为 9.2%、4.7%和 11.4%。FOS 每位患者的平均治疗费用为 99209 美元（95%CI：59595-115074），ELT、ROM 和 AVA 分别为 92426 美元（95%CI：68331-115519）、108482 美元（95%CI：84782-132182）和 131050 美元（95%CI：83327-179897）。
在这项真实世界分析中，FOS 在维持血小板达到临床有益水平方面与 TPO 相当。鉴于这些发现，治疗选择应基于患者整体安全性、TE 的先前存在的风险因素和成本效益。
