Bunin Nancy J, Davies Stella M, Aplenc Richard, Camitta Bruce M, DeSantes Kenneth B, Goyal Rakesh K, Kapoor Neena, Kernan Nancy A, Rosenthal Joseph, Smith Franklin O, Eapen Mary
Children's Hospital of Philadelphia, Philadelphia, USA.
J Clin Oncol. 2008 Sep 10;26(26):4326-32. doi: 10.1200/JCO.2008.16.4442.
Identify prognostic factors that influence outcome after unrelated donor bone marrow transplantation in children with acute myeloid leukemia (AML).
Included are 268 patients (age <or= 18 years) with AML in second complete remission (n = 142), relapse (n = 90), or primary induction failure (n = 36) at transplantation. All patients received bone marrow grafts from an unrelated donor and a myeloablative conditioning regimen. Cox regression models were constructed to identify risk factors that influence outcome after transplantation.
In this analysis, the only risk factor that predicted leukemia recurrence and overall and leukemia-free survival was disease status at transplantation. The 5-year probabilities of leukemia-free survival were 45%, 20%, and 12% for patients who underwent transplantation at second complete remission, relapse, and primary induction failure, respectively. As expected, risk of acute but not chronic graft-versus-host disease (GVHD) was lower with T-cell-depleted bone marrow grafts; T-cell-depleted grafts were not associated with higher risks of leukemia recurrence. We observed similar risks of leukemia relapse in patients with and without acute and chronic GVHD.
Children who underwent transplantation in remission had a superior outcome compared with children who underwent transplantation during relapse or persistent disease. Nevertheless, 20% of children who underwent transplantation in relapse are long-term survivors, suggesting that unrelated donor bone marrow transplantation is an effective therapy in a significant proportion of children with recurrent or primary refractory AML.
确定影响急性髓系白血病(AML)儿童接受非亲缘供者骨髓移植后预后的因素。
纳入268例年龄≤18岁的AML患者,这些患者在移植时处于第二次完全缓解期(n = 142)、复发期(n = 90)或初次诱导失败期(n = 36)。所有患者均接受了来自非亲缘供者的骨髓移植以及清髓性预处理方案。构建Cox回归模型以确定影响移植后预后的危险因素。
在该分析中,唯一预测白血病复发以及总生存和无白血病生存的危险因素是移植时的疾病状态。处于第二次完全缓解期、复发期和初次诱导失败期的患者,其5年无白血病生存率分别为45%、20%和12%。正如预期的那样,去除T细胞的骨髓移植降低了急性移植物抗宿主病(GVHD)而非慢性GVHD的风险;去除T细胞的移植物与白血病复发风险升高无关。我们观察到有和没有急性及慢性GVHD的患者白血病复发风险相似。
与在复发期或疾病持续期接受移植的儿童相比,处于缓解期接受移植的儿童预后更好。然而,20%复发期接受移植的儿童是长期存活者,这表明非亲缘供者骨髓移植对于相当一部分复发或原发难治性AML儿童是一种有效的治疗方法。