Zhao Ran, Ji Dexiang, Zhou Yulan, Qi Ling, Li Fei
Jiangxi Provincial Key Laboratory of Hematological Diseases, Department of Hematology, The First Affiliated Hospital, Jiangxi Medical College, Nanchang University, Nanchang, Jiangxi, 330006, People's Republic of China.
Jiangxi Clinical Research Center for Hematologic Disease, Nanchang, Jiangxi, People's Republic of China.
Int J Gen Med. 2024 Sep 13;17:4025-4036. doi: 10.2147/IJGM.S465184. eCollection 2024.
Immunosuppressive therapy (IST) with horse or rabbit anti-human thymocyte immunoglobulin (h-/r-ATG) and hematopoietic stem cell transplantation (HSCT) are two baseline treatments for severe aplastic anemia (SAA) and transfusion-dependent non-severe aplastic anemia (TD-NSAA) patients. Addition of thrombopoietin receptor agonists (TPO-RAs) to standard IST therapy (h-/r-ATG) has greatly improved the survival of SAA, whereas porcine anti-lymphocyte globulin (p-ALG) combined with TPO-RAs still had a matter of debate.
We retrospectively compared the data of 48 AA patients in our center between 2020 and 2022, 23 AA patients received with p-ALG ± TPO-RAs, 25 AA patients underwent matched sibling donor (MSD-) or haploidentical (haplo-) HSCT.
For patients in the HSCT group, the ORR was 90.9% which was significantly higher than that in the IST±TPO-RAs group (45.5%, = 0.001) at 3 months; moreover, patients who underwent HSCT achieved faster transfusion independence, better CR rate, shorter time of recovery normal blood routine, and the percentage of normal blood routine (all < 0.05) compared with IST±TPO-RAs group. However, the ORR were similary at 6 months in the two groups (95.5% vs 81.8% = 0.342), with a median follow up of 19.8 months (range, 0.3-38.2 months), the 2-year FFS and OS in the two cohorts has no different. Subgroup analysis further indicated that the 2-year FFS and OS were similar between IST+TPO-RAs and haplo-HSCT subgroups, as well as in IST+TPO-RAs and MSD-HSCT cohorts. Moreover, the first-time hospitalizations were much more expensive in the HSCT group than in the IST±TPO-RAs group (402 756 vs. 292 902 yuan, = 0.002).
P-ALG-based-IST±TPO-RAs is a good treatment option with similar FFS and OS compared to allo- HSCT for AA patients without the opportunity of HSCT.
使用马或兔抗人胸腺细胞免疫球蛋白（h-/r-ATG）的免疫抑制治疗（IST）和造血干细胞移植（HSCT）是重型再生障碍性贫血（SAA）和输血依赖型非重型再生障碍性贫血（TD-NSAA）患者的两种基础治疗方法。在标准IST治疗（h-/r-ATG）中添加血小板生成素受体激动剂（TPO-RAs）已显著提高了SAA患者的生存率，而猪抗淋巴细胞球蛋白（p-ALG）联合TPO-RAs仍存在争议。
我们回顾性比较了2020年至2022年期间本中心48例再生障碍性贫血患者的数据，23例再生障碍性贫血患者接受了p-ALG±TPO-RAs治疗，25例再生障碍性贫血患者接受了同胞全相合供者（MSD-）或单倍体相合（haplo-）HSCT。
对于HSCT组患者，3个月时的客观缓解率（ORR）为90.9%，显著高于IST±TPO-RAs组（45.5%，P = 0.001）；此外，与IST±TPO-RAs组相比，接受HSCT的患者实现输血独立更快、完全缓解率更高、恢复正常血常规的时间更短，且血常规正常的比例更高（均P < 0.05）。然而，两组在6个月时的ORR相似（95.5%对81.8%，P = 0.342），中位随访时间为19.8个月（范围0.3 - 38.2个月），两组的2年无进展生存率（FFS）和总生存率（OS）无差异。亚组分析进一步表明，IST + TPO-RAs组与单倍体HSCT亚组之间以及IST + TPO-RAs组与MSD-HSCT队列之间的2年FFS和OS相似。此外，HSCT组的首次住院费用比IST±TPO-RAs组高得多（402756元对292902元，P = 0.002）。
对于没有HSCT机会的再生障碍性贫血患者，基于p-ALG的IST±TPO-RAs是一种良好的治疗选择，其FFS和OS与异基因HSCT相似。
