Department of Therapeutic Center of Anemia, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Science & Peking Union Medical College (CAMS & PUMC), Tianjin, China.
Eur J Haematol. 2020 Jun;104(6):562-570. doi: 10.1111/ejh.13398. Epub 2020 Mar 5.
To assess the outcomes of children with acquired aplastic anemia (AA) treated in China with first-line porcine anti-lymphocyte immunoglobulin (p-ALG)/rabbit anti-thymocyte immunoglobulin (r-ATG) combined with cyclosporine A (CSA).
We performed a single-center, non-randomized, retrospective cohort study to assess the outcomes of 189 children with AA treated in China with first-line p-ALG/r-ATG combined with CSA between 2014 and 2018.
No significant differences were observed in the overall response rates at 3, 6, 12, or 24 months (3 months: 61.9% vs 67.4%, P = .5; 6 months: 70.9% vs 73.9%, P = .69; 12 months: 77.3% vs 73.3%, P = .58; 24 months: 81.6% vs 78.6%, P = .59) after either p-ALG- or r-ATG-based immunosuppressive therapy. No significant differences were observed in overall survival or failure-free survival between the p-ALG group and the r-ATG group.
Our results reveal that the therapeutic efficacy and safety of p-ALG combined with CSA did not differ significantly from those of r-ATG combined with CSA as first-line therapy for pediatric patients with AA. Moreover, p-ALG has the advantage of significantly lower cost compared with r-ATG.
评估中国采用一线猪抗淋巴细胞免疫球蛋白（p-ALG）/兔抗胸腺细胞免疫球蛋白（r-ATG）联合环孢素 A（CSA）治疗获得性再生障碍性贫血（AA）患儿的结局。
我们进行了一项单中心、非随机、回顾性队列研究，评估了 2014 年至 2018 年期间中国采用一线 p-ALG/r-ATG 联合 CSA 治疗的 189 例 AA 患儿的结局。
在第 3、6、12 和 24 个月时，整体反应率无显著差异（3 个月：61.9% vs 67.4%，P =.5；6 个月：70.9% vs 73.9%，P =.69；12 个月：77.3% vs 73.3%，P =.58；24 个月：81.6% vs 78.6%，P =.59），接受 p-ALG 或 r-ATG 为基础的免疫抑制治疗后。p-ALG 组和 r-ATG 组在总生存率或无失败生存率方面无显著差异。
我们的结果表明，p-ALG 联合 CSA 的治疗效果和安全性与 r-ATG 联合 CSA 作为儿科 AA 患者的一线治疗方法没有显著差异。此外，p-ALG 的优势在于与 r-ATG 相比，成本显著降低。
