唐氏综合征患儿的髓性白血病：1990 年至 2003 年法国基于登记的经验报告。

Myeloid leukaemia in children with Down syndrome: report of the registry-based French experience between 1990 and 2003.

机构信息

Bordeaux University and Bordeaux University Hospital, Pediatric Oncology and Hematology Unit, 33076 Bordeaux, France.

出版信息

Pediatr Blood Cancer. 2010 Jul 1;54(7):927-33. doi: 10.1002/pbc.22515.

DOI:10.1002/pbc.22515

PMID:20405513

Abstract

AIM

To determine the epidemiology of myeloid leukaemia (ML) in children with Down syndrome (DS) and the efficacy of two approaches, low-dose cytarabine-based regimen (LDC) and standard-dose intensive chemotherapy (SD).

PROCEDURE

All children with Down syndrome aged from 2 months to 15 years with ML/myelodysplasia registered in the French registry between January 1990 and December 2003 were included.

RESULTS

Forty-four patients were included. The median age was 1.75 years. The French-America-British subtypes were as follows: M7: 24, M0: 6, M2: 5, M6: 2. Forty-three patients were treated with curative prospect, 20 patients with LDC regimen and 22 according to SD protocols, 1 was given the LDC regimen plus autologous stem-cell transplantation. The event-free survival (EFS) and overall survival (OS) at 5 years were 64.4% and 76.8%. At 5 years, OS in LDC and SD groups were 65% and 85.9% (P = 0.08). EFS were 45% and 80.3% respectively (P < 0.01).

CONCLUSION

Children with DS can adequately tolerate SD chemotherapy with a significant superiority of EFS relative to LDC. We suggest that higher levels of cure can be obtained in DS-ML with SD chemotherapy including cytarabine and anthracyclines.

摘要

目的

确定唐氏综合征（DS）患儿中髓性白血病（ML）的流行病学，以及两种方法（低剂量阿糖胞苷为基础的方案[LDC]和标准剂量强化化疗[SD]）的疗效。

方法

所有年龄在 2 个月至 15 岁之间，在法国注册处登记的患有 ML/骨髓发育不良的唐氏综合征患儿均纳入本研究。

结果

共纳入 44 例患儿。中位年龄为 1.75 岁。法国-美国-英国（FAB）分型如下：M7：24 例，M0：6 例，M2：5 例，M6：2 例。43 例患儿接受了治愈性治疗，20 例患儿采用 LDC 方案，22 例患儿采用 SD 方案，1 例患儿采用 LDC 方案联合自体干细胞移植。5 年时无事件生存（EFS）和总生存（OS）率分别为 64.4%和 76.8%。5 年时，LDC 和 SD 组的 OS 分别为 65%和 85.9%（P = 0.08）。EFS 分别为 45%和 80.3%（P < 0.01）。