Stempel Jessica M, Wang Rong, Lee Alfred I, Zeidan Amer M, Ma Xiaomei, Podoltsev Nikolai A
Section of Medical Oncology and Hematology, Department of Internal Medicine, Yale Comprehensive Cancer Center, Yale University School of Medicine, 333 Cedar Street, WWW208, New Haven, CT, 06510, USA.
Cancer Outcomes, Public Policy, and Effectiveness Research (COPPER) Center, Yale University, New Haven, CT, USA.
Ann Hematol. 2025 Apr;104(4):2507-2515. doi: 10.1007/s00277-025-06307-z. Epub 2025 Mar 19.
Acquired aplastic anemia (AA) is an immune-mediated disorder leading to bone marrow failure characterized by pancytopenia, with infectious and bleeding complications. The disease course may be complicated by paroxysmal nocturnal hemoglobinuria (PNH), necessitating screening with flow cytometry (FC) at the time of AA diagnosis. Management strategies vary based on disease severity. Severe AA patients are usually heavily transfusion-dependent (HT-AA) and typically treated with antithymocyte globulin, calcineurin inhibitor (CNI) and eltrombopag (EPAG) as triple therapy, while allogeneic hematopoietic stem cell transplant (HSCT) is often reserved for younger patients with matched sibling donors. Moderate AA patients are less transfusion-dependent (LT-AA) and may be observed or treated with CNI or EPAG. We conducted a retrospective cohort study using Blue Cross Blue Shield Axis database, examining adult patients diagnosed with AA between 07/01/2016 and 06/30/2022. We evaluated their management within the first 6 months following the diagnosis. Of 793 identified individuals (542 LT-AA, 251 HT-AA), with a median age of 49 years, only 42.6% received AA-directed therapy. Triple therapy and HSCT were infrequently used for patients with HT-AA (4.4% and 18.7%, respectively), while the most common treatment was the combination of a CNI and EPAG (LT-AA 37.8%, HT-AA 51.7%). The median time from diagnosis to treatment was 22 days, with older patients (age ≥ 40 years) experiencing treatment initiation delays (p = 0.03). FC testing was underutilized with only 55.5% of patients undergoing evaluation. These findings highlight the need for better access to diagnostic evaluation and appropriate AA-directed therapy for patients with AA in real-world settings.
获得性再生障碍性贫血（AA）是一种免疫介导的疾病，可导致以全血细胞减少为特征的骨髓衰竭，并伴有感染和出血并发症。疾病进程可能并发阵发性夜间血红蛋白尿（PNH），因此在AA诊断时需要进行流式细胞术（FC）筛查。治疗策略因疾病严重程度而异。重度AA患者通常严重依赖输血（HT-AA），通常采用抗胸腺细胞球蛋白、钙调神经磷酸酶抑制剂（CNI）和艾曲泊帕（EPAG）进行三联治疗，而异基因造血干细胞移植（HSCT）通常仅用于有匹配同胞供体的年轻患者。中度AA患者对输血的依赖程度较低（LT-AA），可进行观察或采用CNI或EPAG治疗。我们使用蓝十字蓝盾轴心数据库进行了一项回顾性队列研究，研究对象为2016年7月1日至2022年6月30日期间确诊为AA的成年患者。我们评估了他们在诊断后的前6个月内的治疗情况。在793名确定的个体中（542名LT-AA，251名HT-AA），中位年龄为49岁，只有42.6%的患者接受了针对AA的治疗。HT-AA患者很少使用三联疗法和HSCT（分别为4.4%和18.7%），而最常见的治疗方法是CNI和EPAG联合使用（LT-AA为37.8%，HT-AA为51.7%）。从诊断到治疗的中位时间为22天，老年患者（年龄≥40岁）开始治疗的时间有所延迟（p = 0.03）。FC检测未得到充分利用，只有55.5%的患者接受了评估。这些发现凸显了在现实环境中为AA患者提供更好的诊断评估和适当的AA导向治疗的必要性。
