French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, Service d'Hématologie Greffe, Hôpital Saint-Louis, Paris, France.
Analysis Group, Inc., Boston, MA, USA.
Ann Hematol. 2020 Apr;99(4):743-752. doi: 10.1007/s00277-019-03809-5. Epub 2020 Feb 17.
This study assessed treatment patterns and healthcare resource utilization (HRU) of patients with severe aplastic anemia (SAA) with insufficient response to immunosuppressive therapy (IST). A retrospective chart review was conducted at Dana-Farber Cancer Institute (DFCI), United States, and Hôpital Saint-Louis (HSL), France. Eligible patients were ≥ 18 years old, diagnosed with acquired SAA between January 1, 2006, and July 31, 2016, had insufficient response to IST, and had ≥ 12 months of follow-up post-diagnosis. Overall survival (OS) was estimated using the Kaplan-Meier method. Among the 40 patients, mean age at diagnosis was 44 years and 53% were women. Median follow-up time after SAA diagnosis was 48.3 months. Ninety-five percent of patients received antithymocyte globulin (ATG) as primary therapy prior to hematopoietic stem cell transplant (HSCT). Most common secondary SAA therapies prior to HSCT were eltrombopag (28%) and androgens (15%). Seventy-five percent of patients received HSCT. Prior to HSCT, patients received an average of 2.7 red blood cell (RBC) and 3.3 platelet transfusions per month; patients had 0.9 hospitalizations, 0.4 emergency room visits, and 12.8 office visits per year. Five-year OS was 75%, with infection as the primary cause of death. Additionally, this study provides information on the subgroup of patients receiving eltrombopag which was the most common secondary therapy. This study quantified transfusion and HRU burden associated with SAA and demonstrated high 5-year survival in a recently treated cohort.
这项研究评估了免疫抑制治疗（IST）反应不足的重型再生障碍性贫血（SAA）患者的治疗模式和医疗资源利用（HRU）。在美国达纳-法伯癌症研究所（DFCI）和法国圣路易医院（HSL）进行了回顾性病历审查。合格患者年龄≥18 岁，在 2006 年 1 月 1 日至 2016 年 7 月 31 日期间被诊断为获得性 SAA，对 IST 反应不足，且在诊断后有≥12 个月的随访。采用 Kaplan-Meier 法估计总生存（OS）。在 40 名患者中，诊断时的平均年龄为 44 岁，53%为女性。SAA 诊断后中位随访时间为 48.3 个月。95%的患者在接受造血干细胞移植（HSCT）之前接受抗胸腺细胞球蛋白（ATG）作为一线治疗。在 HSCT 之前，最常见的继发性 SAA 治疗方法是血小板生成素受体激动剂（28%）和雄激素（15%）。75%的患者接受了 HSCT。在 HSCT 之前，患者每月平均接受 2.7 次红细胞（RBC）和 3.3 次血小板输注；每年有 0.9 次住院、0.4 次急诊就诊和 12.8 次就诊。5 年 OS 为 75%，感染是主要死亡原因。此外，本研究还提供了接受血小板生成素受体激动剂治疗的患者亚组信息，这是最常见的二线治疗方法。本研究量化了与 SAA 相关的输血和 HRU 负担，并在最近接受治疗的队列中展示了较高的 5 年生存率。
