State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, 300020, Tianjin, China.
Tianjin Institutes of Health Science, 301600, Tianjin, China.
Br J Haematol. 2023 Jun;201(6):1179-1191. doi: 10.1111/bjh.18779. Epub 2023 Mar 30.
To validate the efficacy and safety of haematopoietic stem cell transplantation (HSCT) in hepatitis-associated aplastic anaemia (HAAA) patients, we reviewed 260 patients who underwent HSCT for acquired aplastic anaemia and eventually included 30 HAAA patients and 90 non-HAAA patients using propensity score matching. In the HAAA group, the estimated 5-year overall survival rate (75.8% vs. 86.5%, p = 0.409), failure-free survival (FFS) rate (74.0% vs. 83.2%, p = 0.485), graft-versus-host disease (GVHD)-free FFS rate (61.2% vs. 67.6%, p = 0.669) after HSCT were slightly lower but not statistically significant than those in the non-HAAA group. Both groups did not significantly differ in engraftment, post-transplant severe infection, cytomegalovirus (CMV) or Epstein-Barr virus viraemia, or GVHD incidences. The patterns of immune reconstitution were broadly consistent between the two groups. When stratifying HAAA patients according to donor type, no significant differences in survival, transplant-related mortality, or GVHD cumulative incidences were observed. CMV viraemia (68.7% vs 8.3%, p = 0.009) occurred more commonly in haploidentical donor (HID) transplants than in matched sibling donor transplants. However, early CMV disease incidence (5.6% vs. 0.0%, p = 1.000) was low. Overall, the post-transplant outcomes of HAAA patients were comparable to those of non-HAAA patients after balancing potential confounders, and HID-HSCT can offer an alternative curative option for HAAA.
为了验证造血干细胞移植（HSCT）在乙型肝炎相关性再生障碍性贫血（HAAA）患者中的疗效和安全性，我们回顾了 260 例接受 HSCT 治疗获得性再生障碍性贫血的患者，最终纳入了 30 例 HAAA 患者和 90 例非 HAAA 患者，采用倾向评分匹配法进行匹配。在 HAAA 组中，HSCT 后 5 年总生存率（75.8%比 86.5%，p=0.409）、无失败生存率（74.0%比 83.2%，p=0.485）和移植物抗宿主病（GVHD）-无失败生存率（61.2%比 67.6%，p=0.669）略低，但无统计学意义。两组在植入、移植后严重感染、巨细胞病毒（CMV）或 EBV 血症或 GVHD 发生率方面无显著差异。两组的免疫重建模式基本一致。当根据供体类型对 HAAA 患者进行分层时，在生存、移植相关死亡率或 GVHD 累积发生率方面均无显著差异。与同胞供体 HSCT 相比，HID 移植中更常见 CMV 血症（68.7%比 8.3%，p=0.009）。然而，早期 CMV 疾病发生率（5.6%比 0.0%，p=1.000）较低。总体而言，在平衡潜在混杂因素后，HAAA 患者的移植后结局与非 HAAA 患者相当，HID-HSCT 可为 HAAA 提供一种替代的治疗选择。
