Yokota Hirofumi, Miyao Kotaro, Sawa Masashi, Terakura Seitaro, Kurahashi Shingo, Ikoma Yoshikazu, Imahashi Nobuhiko, Morishita Takanobu, Okamoto Akinao, Kajiguchi Tomohiro, Ono Takaaki, Narita Tomoko, Kanemura Nobuhiro, Ozeki Kazutaka, Kojima Yumi, Naito Kensuke, Uchino Kaori, Tomita Akihiro, Iida Hiroatsu, Imoto Naoto, Kasahara Senji, Inagaki Yuichiro, Nishida Tetsuya, Murata Makoto
Department of Hematology and Oncology, Anjo Kosei Hospital, Anjo, Japan.
Department of Hematology and Oncology, Nagoya University Graduate School of Medicine, Nagoya, Japan.
J Hematol. 2024 Aug;13(4):142-149. doi: 10.14740/jh1289. Epub 2024 Aug 10.
To investigate whether the addition of eltrombopag (EPAG) to rabbit anti-thymocyte globulin (ATG)-based immunosuppressive therapy (IST) for newly diagnosed severe aplastic anemia (SAA) improves outcomes and affects the cumulative incidence of clonal evolution (CE), we conducted a multicenter retrospective analysis.
Data were collected from 101 patients, aged 15 - 65 years, undergoing initial IST.
No significant imbalance in age, sex, or severity was observed between the EPAG (n = 20) and non-EPAG (n = 81) groups. The median duration of EPAG administration in EPAG group was 16.1 months (range: 0.6 - 41.1 months). Six months after the initiation of IST, the complete response (CR) rate significantly improved in the EPAG group (P < 0.01). The cumulative incidence of allogeneic stem cell transplantation (allo-SCT) at 2 years and the 2-year overall survival (OS) were not significantly different between the two groups (allo-SCT, P = 0.31; OS, P = 0.64). Grade 3-4 adverse events in the EPAG group and the cumulative incidence of CE (P = 0.96) showed no increase.
In summary, IST showed significantly better initial efficacy in the EPAG group. Although the addition of EPAG did not reduce the need for allo-SCT, no increase was observed in the incidence of CE with long-term EPAG use.
为了研究在初诊的重型再生障碍性贫血（SAA）患者中，在基于兔抗胸腺细胞球蛋白（ATG）的免疫抑制治疗（IST）中添加艾曲泊帕（EPAG）是否能改善预后并影响克隆演变（CE）的累积发生率，我们进行了一项多中心回顾性分析。
收集了101例年龄在15 - 65岁之间接受初始IST治疗的患者的数据。
EPAG组（n = 20）和非EPAG组（n = 81）在年龄、性别或严重程度方面未观察到显著失衡。EPAG组中EPAG给药的中位持续时间为16.1个月（范围：0.6 - 41.1个月）。IST开始后6个月，EPAG组的完全缓解（CR）率显著提高（P < 0.01）。两组之间2年的异基因干细胞移植（allo - SCT）累积发生率和2年总生存率（OS）无显著差异（allo - SCT，P = 0.31；OS，P = 0.64）。EPAG组的3 - 4级不良事件以及CE的累积发生率（P = 0.96）均未增加。
总之，IST在EPAG组中显示出显著更好的初始疗效。虽然添加EPAG并未减少allo - SCT的需求，但长期使用EPAG并未观察到CE发生率增加。
