Assistance Publique-Hôpitaux de Paris, Hôpital Avicenne, Bobigny, France.
J Clin Oncol. 2012 May 10;30(14):1641-6. doi: 10.1200/JCO.2011.38.4560. Epub 2012 Apr 2.
Acute promyelocytic leukemia (APL) is rare in children. All-trans-retinoic acid (ATRA) combined with chemotherapy, the reference treatment of APL, is generally considered to produce similar results in children and adults. However, previously published childhood APL studies have generally analyzed all patients age < 18 years as a group, without further dividing according to age.
We compared disease characteristics and outcomes of children (age ≤ 12 years), adolescents (13 to 18 years), and adults (> 18 years) included in two multicenter APL clinical trials (APL 93 and 2000 trials).
Of the 833 patients age ≤ 60 years included in the two trials, 26 (3%), 58 (7%), and 749 (90%) were children, adolescents, and adults, respectively. Children had significantly higher baseline WBC counts (P < .001). The complete remission (CR) rate (92%, 100%, and 94.5%, respectively) and 5-year cumulative incidence of relapse (CIR; 28%, 20%, and 23%, respectively) did not differ between children, adolescents, and adults, whereas adolescents had significantly better overall survival (OS; 5-year OS, 93.6% v 80.4% in adults and 80.4% in children; P = .03). However, in children age ≤ 4 years, the 5-year CIR was 52%, compared with 17.6% in children age 5 to 12 years (P = .006), although most of the younger children who relapsed experienced durable salvage with autologous or allogeneic stem-cell transplantation.
Adolescents and children age > 4 years with APL treated with ATRA and chemotherapy have outcomes at least as favorable as those of adults. Younger children seem to experience more relapses and may require reinforcement of first-line treatment.
急性早幼粒细胞白血病（APL）在儿童中较为罕见。全反式维甲酸（ATRA）联合化疗是 APL 的标准治疗方法，一般认为在儿童和成人中的疗效相似。然而，之前发表的儿童 APL 研究通常将所有年龄<18 岁的患者作为一个整体进行分析，而没有进一步根据年龄进行细分。
我们比较了纳入两项多中心 APL 临床试验（APL93 和 APL2000 试验）的儿童（≤12 岁）、青少年（13 至 18 岁）和成年患者（>18 岁）的疾病特征和结局。
在这两项试验中，年龄≤60 岁的 833 例患者中，儿童、青少年和成年患者分别占 3%（26 例）、7%（58 例）和 90%（749 例）。儿童的基线白细胞计数明显较高（P<0.001）。完全缓解率（CR；分别为 92%、100%和 94.5%）和 5 年累积复发率（CIR；分别为 28%、20%和 23%）在儿童、青少年和成年患者之间无差异，而青少年的总生存率（OS；5 年 OS，成年患者为 80.4%，儿童为 93.6%和 80.4%；P=0.03）明显更好。然而，在≤4 岁的儿童中，5 年 CIR 为 52%，而 5 至 12 岁的儿童为 17.6%（P=0.006），尽管大多数复发的年龄较小的儿童通过自体或异基因干细胞移植进行了持久的挽救治疗。
接受 ATRA 和化疗治疗的 APL 青少年和>4 岁的儿童患者的结局与成年患者一样有利。年龄较小的儿童似乎复发更多，可能需要加强一线治疗。
