Long-Term Outcome of Eltrombopag With First-Line Immunosuppressive Therapy for Newly Diagnosed Severe Aplastic Anemia.

IF 1.3 Q4 HEMATOLOGY

Journal of hematology Pub Date : 2024-08-01 Epub Date: 2024-08-10 DOI:10.14740/jh1289

Hirofumi Yokota, Kotaro Miyao, Masashi Sawa, Seitaro Terakura, Shingo Kurahashi, Yoshikazu Ikoma, Nobuhiko Imahashi, Takanobu Morishita, Akinao Okamoto, Tomohiro Kajiguchi, Takaaki Ono, Tomoko Narita, Nobuhiro Kanemura, Kazutaka Ozeki, Yumi Kojima, Kensuke Naito, Kaori Uchino, Akihiro Tomita, Hiroatsu Iida, Naoto Imoto, Senji Kasahara, Yuichiro Inagaki, Tetsuya Nishida, Makoto Murata

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Abstract

Background: 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.

Methods: Data were collected from 101 patients, aged 15 - 65 years, undergoing initial IST.

Results: 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.

Conclusion: 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.

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艾曲波帕与一线免疫抑制疗法联合治疗新诊断的重型再生障碍性贫血的长期疗效

背景：为了研究在以兔抗胸腺细胞球蛋白（ATG）为基础的免疫抑制疗法（IST）治疗新诊断的重型再生障碍性贫血（SAA）时添加艾曲波帕（EPAG）是否能改善治疗效果并影响克隆进化（CE）的累积发生率，我们进行了一项多中心回顾性分析：方法：我们收集了101名首次接受IST治疗的患者的数据，他们的年龄在15-65岁之间：EPAG组（20人）和非EPAG组（81人）之间在年龄、性别或严重程度上没有发现明显的不平衡。EPAG 组的 EPAG 施用时间中位数为 16.1 个月（范围：0.6 - 41.1 个月）。开始 IST 治疗 6 个月后，EPAG 组的完全缓解率（CR）明显提高（P < 0.01）。两组患者2年后异基因干细胞移植（allo-SCT）的累积发生率和2年总生存率（OS）无明显差异（allo-SCT，P = 0.31；OS，P = 0.64）。EPAG组的3-4级不良事件和CE累积发生率（P = 0.96）没有增加：总之，EPAG 组的 IST 初始疗效明显更好。结论：总之，IST在EPAG组显示出明显更好的初始疗效，虽然EPAG的加入并没有减少对同种异体移植的需求，但长期使用EPAG并没有观察到CE发生率的增加。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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Journal of hematology HEMATOLOGY-

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