Objective: To analyze the clinical characteristics and to evaluate immunosuppressive therapy (IST) response and survival in hepatitis-associated aplastic anemia (HAAA).
Methods: We retrospectively analyzed clinical characteristics, IST response, long-term survival and clonal evolution in 41 HAAA patients, and compared those with age and bone marrow failure matched idiopathic aplastic anemia (IAA) patients.
Results: The prevalence of HAAA among cases of SAA was 4.34% (41/944). The proportion of VSAA in HAAA cases was significantly higher than IAA (65.9% vs 39.4%, P=0.001). There was no significant difference in the prevalence of hemorrhage and infections between HAAA and IAA patients, but the duration of infection persistence in HAAA group was much longer than IAA group [21 (4-100) d vs 13 (3-139) d, P=0.048]. The absolute counts of CD3(+) T-cell, CD3(+)CD4(+)T-cell, CD3(+)CD8(+)T-cell and ratio of CD4(+) T-cell/CD8(+) T-cell in HAAA were significant lower than that in IAA patients. However, the percentage of CD3(+)CD8(+)T-cell in HAAA was significant higher than that in IAA (P <0.05). The total response in HAAA and IAA patients treated with IST were 34.1% vs 34.1% (P=1.000), 56.1% vs 53.7% (P=0.787), and 73.2% vs 68.3% (P=0.558) at 3, 6, 12 months after IST, respectively. There were no significant difference in 5-year overall survival and event-free survival between HAAA and IAA patients (90% vs 87.1%, P=0.700; 71.9% vs 62.4%, P=0.450).
Conclusion: HAAA was a rare distinct variant of aplastic anemia with more severe bone marrow failure and more severe imbalance of the T cell immune system than IAA. Treatment outcomes were comparable in patients with HAAA and IAA.
目的: 分析肝炎相关再生障性贫血（HAAA）临床特征，评价其免疫抑制治疗（IST）疗效及生存状况。
方法: 回顾性分析944例接受IST的重型／极重型AA（SAA/VSAA）患者，比较41例HAAA患者与年龄、造血衰竭程度相匹配的123例特发性AA（IAA）临床特征、血液学反应率、长期生存率及克隆性演变情况。
结果: 944例SAA/VSAA患者中HAAA 41例（4.34%），HAAA患者中VSAA所占比例明显高于IAA患者（65.9％对39.4%，P=0.001）。HAAA与匹配的IAA比较，患者感染发生率差异无统计学意义，但感染控制所需时间明显延长[21 (4~100) d对13 (3~139) d，P=0.048]。HAAA患者CD3+、CD3+CD4+、CD3+CD8+ T淋巴细胞绝对值及CD4+/CD8+细胞比值均明显低于IAA患者，而CD3+ CD8+ T淋巴细胞比例明显高于IAA患者，差异均有统计学意义。HAAA与IAA患者IST后3个月（34.1％对34.1%，P=1.000）、6个月（56.1％对53.7%，P=0.787）及12个月（73.2％对68.3%，P=0.558）血液学反应率差异无统计学意义，两组患者预期5年总生存（OS）率、无事件生存（EFS）率比较差异均无统计学意义（OS率：90.0％对87.1%，P=0.700；EFS率：71.9％对62.4%，P=0.450）。
结论: HAAA少见，造血衰竭更为严重，感染相对难以控制，采用标准IST方案治疗可获得与IAA患者相当的疗效。