Children Hospital, Hematology Unit, Faculty of Medicine, Assiut University, Assiut, Egypt.
Ann Hematol. 2023 Nov;102(11):2997-3006. doi: 10.1007/s00277-023-05440-x. Epub 2023 Sep 13.
This study compared the efficacy and safety of CsA monotherapy with eltrombopag (E-PAG) + CsA combined treatment in children with severe aplastic anemia (SAA). The study including 30 children had SAA. Ten were a retrospective cohort treated with CsA monotherapy. The other 20 were prospective cohort received E-PAG + CsA. All patients were evaluated for partial (PR) and complete (CR) hematological response at 3, 6, and 12 months. overall response (OR), overall survival rates (OS), and treatment safety. OR for the E-PAG patients was 40% after 3 months of therapy. At 6 months, this had increased to 75% with significantly higher CR rate (40%) than in the CsA group (p = 0.0001). After a year of treatment, the CR for the E-PAG + CsA regimen had increased to 50% and the OR to 85%, compared to 20% in the CsA group (p = 0.0001). The OS at 12 months was 100% in the E-PAG+ CsA group compared to 80% in the CsA cohort. At 24 months, the OS in the E-PAG + CsA group was 90%. In conclusion, E-PAG+ CsA was found to be a safe and effective alternative treatment for children with SAA particularly in countries with limited resources.
这项研究比较了环孢素单药治疗与艾曲波帕（E-PAG）+环孢素联合治疗儿童重型再生障碍性贫血（SAA）的疗效和安全性。该研究纳入了 30 例 SAA 患儿，其中 10 例为接受环孢素单药治疗的回顾性队列，另 20 例为接受 E-PAG+环孢素的前瞻性队列。所有患者均在 3、6 和 12 个月时评估部分（PR）和完全（CR）血液学反应、总体反应（OR）、总生存率（OS）和治疗安全性。E-PAG 组患者在治疗 3 个月后的 OR 为 40%，在 6 个月时增加到 75%，CR 率（40%）明显高于环孢素组（p=0.0001）。治疗 1 年后，E-PAG+环孢素方案的 CR 增加到 50%，OR 增加到 85%，而环孢素组为 20%（p=0.0001）。E-PAG+环孢素组 12 个月时的 OS 为 100%，而环孢素组为 80%。24 个月时，E-PAG+环孢素组的 OS 为 90%。总之，E-PAG+环孢素是治疗儿童 SAA 的一种安全有效的替代治疗方法，特别是在资源有限的国家。
