Department of Pediatrics, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, 288 Nanjing Road, Tianjin, 300020, People's Republic of China.
Int J Hematol. 2011 Feb;93(2):199-205. doi: 10.1007/s12185-011-0768-0. Epub 2011 Feb 3.
To explore the efficacy of treatment for childhood acute promyelocytic leukemia (APL) with a combination of all-trans-retinoic acid (ATRA) and arsenic trioxide (As(2)O(3)) for remission induction, we reviewed the clinical course and outcome of 37 children with APL from January 1999 to December 2003. Among the 37 children (≤14 years) with newly diagnosed APL, we applied treatments that consisted of ATRA alone or in combination with As(2)O(3) in induction followed by consolidation and maintenance treatment. Overall, 35 (94.6%) of 37 children achieved complete remission (CR). Two patients died of intracerebral hemorrhage on days 1 and 2. The 5-year estimates of event-free survival (EFS), disease-free survival (DFS), and overall survival (OS) rates for the 37 patients were 79.2, 83.7, and 91.5%, respectively. There were 27 patients with white blood cell (WBC) count lower than 10 × 10(9)/L. In 27 patients with a WBC count <10 × 10(9)/L, 17 patients (group-I) were treated with ATRA alone and 10 patients (group-II) were treated with ATRA which was switched to As(2)O(3) due to the side effects of ATRA. Although the 5-year estimate of DFS between group-I and group-II showed no significant difference (P = 0.108), the DFS rate improved by 25% in group-II. Our results suggest that the combination of As(2)O(3) and ATRA might decrease the relapse rate compared with ATRA alone in induction therapy for childhood APL, at least in those with a WBC count less than 10 × 10(9)/L.
为了探索全反式维甲酸（ATRA）联合三氧化二砷（As2O3）诱导缓解治疗儿童急性早幼粒细胞白血病（APL）的疗效，我们回顾了 1999 年 1 月至 2003 年 12 月期间 37 例 APL 患儿的临床过程和结果。在 37 例（≤14 岁）新诊断的 APL 患儿中，我们采用了 ATRA 单独或联合 As2O3 诱导缓解，随后进行巩固和维持治疗。总体而言，37 例患儿中 35 例（94.6%）获得完全缓解（CR）。2 例患儿分别于第 1 天和第 2 天死于颅内出血。37 例患儿的 5 年无事件生存（EFS）、无病生存（DFS）和总生存（OS）率分别为 79.2%、83.7%和 91.5%。有 27 例患儿白细胞计数（WBC）低于 10×10^9/L。在 27 例 WBC 计数<10×10^9/L 的患儿中，17 例（组 I）单独接受 ATRA 治疗，10 例（组 II）因 ATRA 副作用而换用 As2O3 治疗。虽然组 I 和组 II 的 5 年 DFS 估计值无显著差异（P=0.108），但组 II 的 DFS 率提高了 25%。我们的结果表明，与 ATRA 单独诱导缓解相比，As2O3 联合 ATRA 可能降低儿童 APL 诱导缓解治疗的复发率，至少在 WBC 计数<10×10^9/L 的患儿中如此。
