Aplastic anaemia (AA) is a disorder in which the bone marrow fails to produce enough blood cells [1]. For patients with severe aplastic anaemia (SAA) who are ineligible for haematopoietic stem cell transplantation (HSCT), intensive immunosuppressive therapy (IST) with antithymocyte globulin (ATG) and cyclosporin A (CsA) is recommended [2]. However, the efficacy of IST alone is higher in children than in adults. Recently, eltrombopag (EPAG) has been proven to enhance the haematologic response to AA treatment [3, 4]. Whether the difference between adults and children still exists during treatment with IST + EPAG remains unclear. To date, no direct comparison of adults and children has been conducted. The aim of this study was to evaluate the differences in treatment efficacy and survival between adults and children treated with different regimens.

The haematologic response was evaluated in each patient. Among the patients receiving IST alone, the complete response rate (CRR) of adults was lower than that of children at 12 months (31% vs. 48%, P = 0.048), but there was no difference in the overall response rate (ORR) between adults and children at 3, 6, or 12 months (52% vs. 59%, P = 0.426; 69% vs. 74%, P = 0.599; 76% vs. 75%, P = 1.000, respectively). Relatedly, there was no difference in the CRR at 3 and 6 months between the adults and children (3% vs. 11%, P = 0.113; 21% vs. 22%, P = 0.849, respectively). The time to response was 4.3 (IQR 2.9–6.3) months in adults and 3.2 (IQR 2.5–4.0) months in children (P = 0.243), and the time to CR was 8.5 (IQR 6.8–11.0) months in adults and 7.5 (IQR 6.0–10.5) months in children (P = 0.113). Among the patients with IST + EPAG, there was no difference in the ORR between adults and children at 3 or 6 months (67% vs. 64%, P = 0.868; 83% vs. 76%, P = 0.567, respectively), but adults had a higher ORR at 12 months (89% vs. 73%, P = 0.027). There was also no difference in the CRR at 3, 6 and 12 months between the two groups (13% vs. 21%, P = 0.229; 25% vs. 38%, P = 0.124; 54% vs. 50%, P = 0.614, respectively; Fig. 1). The time to response was 3.0 (IQR 2.8–3.3) months in adults and 2.5 (IQR 1.9–3.9) months in children (P = 0.361). Moreover, the time to CR was 6.0 (IQR 3.0–8.3) months for adults and 3.9 (IQR 2.5–5.7) months for children (P = 0.478).

再生障碍性贫血 （AA） 是一种骨髓无法产生足够血细胞的疾病 [1]。对于不适合造血干细胞移植 （HSCT） 的重度再生障碍性贫血 （SAA） 患者，建议使用抗胸腺细胞球蛋白 （ATG） 和环孢菌素 A （CsA） 进行强化免疫抑制治疗 （IST） [2]。然而，单独使用 IST 在儿童中的疗效高于成人。最近，艾曲波帕 （EPAG） 已被证明可增强对 AA 治疗的血液学反应 [3， 4]。在 IST + EPAG 治疗期间，成人和儿童之间的差异是否仍然存在尚不清楚。迄今为止，尚未对成人和儿童进行直接比较。本研究的目的是评估接受不同方案治疗的成人和儿童之间治疗效果和生存率的差异。

评估每位患者的血液学反应。在单独接受 IST 治疗的患者中，成人在 12 个月时的完全缓解率 （CRR） 低于儿童 （31% vs. 48%，P = 0.048），但成人和儿童在 3 、 6 或 12 个月时的总体缓解率 （ORR） 没有差异 （52% vs. 59%，P = 0.426;69% vs. 74%， P = 0.599;分别为 76% 和 75%，P = 1.000）。相关地，成人和儿童在 3 个月和 6 个月时的 CRR 没有差异 （分别为 3% vs. 11%，P = 0.113;21% vs. 22%，P = 0.849）。成人起效时间为 4.3 （IQR 2.9-6.3） 个月，儿童起效时间为 3.2 （IQR 2.5-4.0） 个月 （P = 0.243），成人 CR 时间为 8.5 （IQR 6.8-11.0） 个月，儿童为 7.5 （IQR 6.0-10.5） 个月 （P = 0.113）。在 IST + EPAG 患者中，成人和儿童在 3 或 6 个月时的 ORR 没有差异 （分别为 67% vs. 64%，P = 0.868;83% vs. 76%，P = 0.567），但成人在 12 个月时的 ORR 更高 （89% vs. 73%，P = 0.027）。两组在 3 、 6 和 12 个月的 CRR 也没有差异 （分别为 13% vs. 21%;P = 0.229;25% vs. 38%;P = 0.124;54% vs. 50%，P = 0.614;图 1）。成人的反应时间为 3.0 （IQR 2.8-3.3） 个月，儿童为 2.5 （IQR 1.9-3.9） 个月 （P = 0.361）。此外，成人的 CR 时间为 6.0 （IQR 3.0-8.3） 个月，儿童为 3.9 （IQR 2.5-5.7） 个月 （P = 0.478）。