匹配相关移植与免疫抑制治疗加艾曲波帕治疗重型再生障碍性贫血一线治疗：一项多中心前瞻性研究。
Matched related transplantation versus immunosuppressive therapy plus eltrombopag for first-line treatment of severe aplastic anemia: a multicenter, prospective study.
机构信息
National Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Institute of Blood and Marrow Transplantation of Soochow University, 188 Shizi Street, Suzhou, 215006, Jiangsu Province, China.
Department of Hematology, Haikou Municipal People's Hospital, Affiliated Haikou Hospital Xiangya School of Medicine Central South University, Haikou, Hainan Province, China.
出版信息
J Hematol Oncol. 2022 Aug 12;15(1):105. doi: 10.1186/s13045-022-01324-1.
This study prospectively compared the efficacy and safety between matched related donor-hematopoietic stem cell transplantation (MRD-HSCT) (n = 108) and immunosuppressive therapy (IST) plus eltrombopag (EPAG) (IST + EPAG) (n = 104) to determine whether MRD-HSCT was still superior as a front-line treatment for patients with severe aplastic anemia (SAA). Compared with IST + EPAG group, patients in the MRD-HSCT achieved faster transfusion independence, absolute neutrophil count ≥ 1.0 × 10/L (P < 0.05), as well as high percentage of normal blood routine at 6-month (86.5% vs. 23.7%, P < 0.001). In the MRD-HSCT and IST + EPAG groups, 3-year overall survival (OS) was 84.2 ± 3.5% and 89.7 ± 3.1% (P = 0.164), whereas 3-year failure-free survival (FFS) was 81.4 ± 4.0% and 59.1 ± 4.9% (P = 0.002), respectively. Subgroup analysis indicated that the FFS of the MRD-HSCT was superior to that of the IST + EPAG among patients aged < 40 years old (81.0 ± 4.6% vs. 63.7 ± 6.5%, P = 0.033), and among patients with vSAA (86.1 ± 5.9% vs. 54.9 ± 7.9%, P = 0.003), while the 3-year OS of the IST + EPAG was higher than that of the MRD-HSCT among the patient aged ≥ 40 years old (100.0 ± 0.0% vs. 77.8 ± 9.8%, P = 0.036). Multivariate analysis showed that first-line MRD-HSCT treatment was associated favorably with normal blood results at 6-month and FFS (P < 0.05). These outcomes suggest that MRD-HSCT remains the preferred first-line option for SAA patients aged < 40 years old or with vSAA even in the era of EPAG.
本研究前瞻性比较了匹配相关供者造血干细胞移植（MRD-HSCT）（n=108）与免疫抑制治疗（IST）加艾曲波帕（EPAG）（IST+EPAG）（n=104）在确定 MRD-HSCT 是否仍然作为重型再生障碍性贫血（SAA）患者一线治疗的疗效和安全性。与 IST+EPAG 组相比，MRD-HSCT 组患者更快实现输血独立性，绝对中性粒细胞计数≥1.0×10/L（P<0.05），且 6 个月时正常血常规比例更高（86.5% vs. 23.7%，P<0.001）。MRD-HSCT 和 IST+EPAG 组 3 年总生存率（OS）分别为 84.2±3.5%和 89.7±3.1%（P=0.164），而 3 年无失败生存率（FFS）分别为 81.4±4.0%和 59.1±4.9%（P=0.002）。亚组分析表明，年龄<40 岁（81.0±4.6% vs. 63.7±6.5%，P=0.033）和重型再障（vSAA）患者（86.1±5.9% vs. 54.9±7.9%，P=0.003）中，MRD-HSCT 的 FFS 优于 IST+EPAG，而年龄≥40 岁患者（100.0±0.0% vs. 77.8±9.8%，P=0.036）中 IST+EPAG 的 3 年 OS 高于 MRD-HSCT。多变量分析表明，一线 MRD-HSCT 治疗与 6 个月时正常血液结果和 FFS 呈正相关（P<0.05）。这些结果表明，即使在 EPAG 时代，MRD-HSCT 仍然是年龄<40 岁或 vSAA 的 SAA 患者的首选一线治疗选择。
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