Deyell Rebecca J, Shereck Evan B, Milner Ruth A, Schultz Kirk R
Division of Pediatric Hematology/Oncology/Bone Marrow Transplantation, British Columbia Children's Hospital, University of British Columbia, Vancouver, British Columbia, Canada.
Pediatr Hematol Oncol. 2011 Sep;28(6):469-78. doi: 10.3109/08880018.2011.568043. Epub 2011 Jun 27.
Immunosuppressive therapy (IST) is recommended for children with acquired aplastic anemia (AA) who lack a human leukocyte antigen (HLA)-matched sibling donor for hematopoietic cell transplantation (HCT). Hematopoietic growth factors have often been included in IST supportive care, but prolonged exposure may increase the risk of secondary clonal evolution. The authors evaluated response, survival, and the incidence of clonal evolution following cyclosporine-based IST without hematopoietic growth factor exposure in a population-based pediatric cohort, identified retrospectively. Forty-five patients with a median age of 7.3 years (range 1.2-17.0 years) were included. Partial (PR) and complete (CR) response was achieved in 82% and 64%, at a median of 55 days (range 11-414 days) and 7.6 months (range 2.8-82.2 months), respectively. Patients with associated seronegative hepatitis had an increased likelihood of PR and CR on multivariate analyses (PR: hazard ratio [HR] 3.15, 95% confidence interval [CI] 1.40, 7.11; CR: HR 2.99, 95% CI 1.35, 6.62), whereas older children were less likely to achieve IST response than children younger than 5 years at diagnosis. Five- and 10-year overall survival was 96% ± 4% and 90% ± 7%, respectively, and 5-year failure-free survival was 63% ± 8%. There was no infection-related mortality, although 16.4% of patients had at least 1 episode of documented bacteremia. The 5-year cumulative incidence of relapse was 12.9% and of clonal evolution was 3.2%. The authors conclude that children with AA who receive IST without hematopoietic growth factor support have excellent response and survival outcomes and a low incidence of clonal evolution.
对于缺乏人类白细胞抗原（HLA）匹配同胞供体进行造血细胞移植（HCT）的获得性再生障碍性贫血（AA）患儿，推荐采用免疫抑制治疗（IST）。造血生长因子常被纳入IST支持治疗中，但长期使用可能会增加继发克隆演变的风险。作者在一项回顾性确定的基于人群的儿科队列中，评估了在不使用造血生长因子的情况下，基于环孢素的IST治疗后的反应、生存率和克隆演变发生率。纳入了45例中位年龄为7.3岁（范围1.2 - 17.0岁）的患者。部分缓解（PR）和完全缓解（CR）分别在中位55天（范围11 - 414天）和7.6个月（范围2.8 - 82.2个月）时达到，比例分别为82%和64%。多因素分析显示，伴有血清阴性肝炎的患者达到PR和CR的可能性增加（PR：风险比[HR] 3.15，95%置信区间[CI] 1.40，7.11；CR：HR 2.99，95% CI 1.35，6.62），而年龄较大的儿童比诊断时年龄小于5岁的儿童达到IST反应的可能性更小。5年和10年总生存率分别为96%±4%和90%±7%，5年无失败生存率为63%±8%。尽管16.4%的患者至少有1次记录在案的菌血症发作，但无感染相关死亡。5年复发累积发生率为12.9%，克隆演变发生率为3.2%。作者得出结论，接受无造血生长因子支持的IST治疗的AA患儿反应和生存结果良好，克隆演变发生率低。
