Prognostic factors for outcomes of pediatric patients with refractory or relapsed acute leukemia undergoing allogeneic progenitor cell transplantation.
Prognostic factors for outcomes of pediatric patients with refractory or relapsed acute leukemia undergoing allogeneic progenitor cell transplantation.
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DOI：
10.1016/j.bbmt.2010.07.019
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发表时间：
2011-04
期刊：
影响因子：
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通讯作者：
N. Watanabe;Yoshiyuki Takahashi;Kimikazu Matsumoto;A. Hama;H. Muramatsu;Sayoko Doisaki;K. Horibe;Koji Kato;S. Kojima
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文献类型：
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作者：
N. Watanabe;Yoshiyuki Takahashi;Kimikazu Matsumoto;A. Hama;H. Muramatsu;Sayoko Doisaki;K. Horibe;Koji Kato;S. Kojima
异基因干细胞移植(SCT)是治疗难治性或复发性急性白血病的唯一方法，但预后仍然很差。很少有报道描述儿童难治性急性白血病患者接受SCT的结果。为了确定这些患者的预后因素，我们回顾了1990至2008年间名古屋市3个移植单元的82名儿童晚期急性白血病患者的SCT结果。移植时的中位年龄为8岁(0.5-17岁)。首次难治性复发53例(64.6%)，第二次或以后复发16例(19.5%)，初次诱导失败13例(15.9%)。未经治疗的首次复发患者中仅有4例(4.9%)接受了移植，39例(47.6%)接受了无血缘关系的供体祖细胞移植。82例患者中61例死亡(77.9%)，中位生存期7.1个月(95%可信区间4.2~10.0个月)。中位无病生存期(DFS)为4.7个月(95%可信区间，2.6~6.9个月)。在多因素分析中，外周血原始细胞、脐带血移植、既往挽救化疗3个疗程以上是DFS的预测因素。这些结果支持异基因SCT对于大多数儿童难治性或复发性急性白血病患者只有很小的治愈机会的观点，并表明减轻白血病负担和早期最佳移植时机对于即使是难治性急性白血病患者的长期生存也是至关重要的。
Allogeneic stem cell transplantation (SCT) is the only curative therapy for patients with refractory or relapsed acute leukemia, although the prognosis remains poor. Few reports have described outcomes of SCT in pediatric patients with refractory acute leukemia. To identify prognostic factors for these patients, we retrospectively evaluated SCT outcomes for advanced acute leukemia in 82 pediatric patients from 3 transplant units in Nagoya City between 1990 and 2008. Median age at transplantation was 8 years (range, 0.5-17 years). Transplantation was performed in the first refractory relapse for 53 patients (64.6%), in the second or subsequent relapse for 16 patients (19.5%), and during primary induction failure for 13 patients (15.9%). Only 4 patients (4.9%) underwent transplantation in the untreated first relapse, and 39 patients (47.6%) received unrelated donor progenitor cells. Out of the 82 patients, 61 died (77.9%), with a median survival of 7.1 months (95% confidence interval [CI], 4.2-10.0 months). Median disease-free survival (DFS) was 4.7 months (95% CI, 2.6-6.9 months). In multivariate analysis, peripheral blood blasts, cord blood transplantation, and more than 3 courses of previous salvage chemotherapy were predictive of DFS. These results support the notion that allogeneic SCT offers only a small chance of cure for most pediatric patients with refractory or relapsed acute leukemia, and suggest that reduction of the leukemia burden and earlier optimal timing of transplantation are essential for long-term survival even in patients with refractory acute leukemia.