[Outcomes of very severe aplastic anemia patients with different absolute neutrophil counts after frontline immnunosuppressive therapy]

Zhonghua Xue Ye Xue Za Zhi. 2016 Apr;37(4):329-33. doi: 10.3760/cma.j.issn.0253-2727.2016.04.016.
[Article in Chinese]

Abstract

Objective: To analyze early hematopoietic response and long-term survival of very severe aplastic anemia (VSAA) patients with different absolute neutrophil counts (ANC) after frontline immnunosuppressive therapy (IST).

Methods: Clinical data and outcome of 145 VSAA patients treated with rabbit antithymocyte globulin combined with cyclosporine were retrospectively analyzed. Hematopoietic responses to IST and long-term survival were statistically analyzed for VSAA patients in different ANC subgroups.

Results: Pre-IST ANC=0.05×10(9)/L acted as the best cutoff level to predict IST response at 3, 6 months. For 145 VSAA patients, early death rate was 13.4% (11/82) vs 1.6% (1/63), respectively, in the ANC≤0.05×10(9)/L group and ANC>0.05×10(9)/L group (P<0.05). Hematopoietic response rates to IST was 22.0% vs 54.0% (P=0.000) at 3 months, 34.1% vs 63.5% (P=0.000) at 6 months; the overall five-year survival rate was only (62.5±5.4) % vs (91.4±3.7) % (P=0.000) and five-year event-free survival rate was (42.3±5.5) % vs (63.1±6.5) % (P=0.003), respectively, in the ANC≤0.05×10(9)/L group and ANC>0.05×10(9)/L group.

Conclusion: VSAA patients with extremely low ANC (≤0.05×10(9)/L) had high early death rate and with very low response rate to frontline IST and poor survival, so it is urgent to seek for the alternative frontline therapy that will bring faster and better outcome for these patients.

目的: 分析不同中性粒细胞绝对计数(ANC)阈值下极重型再生障碍性贫血(VSAA)患者接受一线免疫抑制治疗(IST)的早期血液学反应和长期生存情况。

方法: 回顾性分析145例接受一线IST的VSAA患者临床资料,比较不同ANC阈值下VSAA患者的早期血液学反应、5年总生存(OS)及无病生存(EFS)率。

结果: 以ANC 0.05×109/L为界观察VSAA患者IST后3、6个月的血液学反应,145例患者中ANC≤0.05×109/L者为82例,ANC>0.05×109/L者为63例,早期病死率分别为13.4%(11/82)、1.6%(1/63)(P<0.05);IST后3个月血液学反应率分别为22.0%、54.0%(P=0.000),IST后6个月分别为34.1%、63.5%(P=0.000);5年OS率分别为(62.5±5.4)%、(91.4±3.7)%(P=0.000),5年EFS率分别为(42.3±5.5)%、(63.1±6.5)%(P=0.003);两组差异均有统计学意义。

结论: ANC≤0.05×109/L的VSAA患者接受一线IST早期病死率高、疗效差,应当考虑起效更快、疗效更高的治疗决策。

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Anemia, Aplastic / blood
  • Anemia, Aplastic / drug therapy*
  • Animals
  • Antilymphocyte Serum / therapeutic use*
  • Cyclosporine / therapeutic use*
  • Disease-Free Survival
  • Humans
  • Immunosuppressive Agents / therapeutic use*
  • Leukocyte Count
  • Neutrophils / cytology*
  • Rabbits
  • Retrospective Studies
  • Survival Rate
  • Treatment Outcome

Substances

  • Antilymphocyte Serum
  • Immunosuppressive Agents
  • Cyclosporine

Grants and funding

基金项目:卫生公益性行业科研专项(201202017);临床医学专业学位研究生临床研究能力培养模式(PUMC-GS-2012009);协和青年科研基金(33320140063)