Treatment and prognostic impact of transient leukemia in neonates with Down syndrome
Treatment and prognostic impact of transient leukemia in neonates with Down syndrome
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DOI：
10.1182/blood-2007-10-118810
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发表时间：
2008-03-15
期刊：
影响因子：
20.3
通讯作者：
Reinhardt, Dirk
中科院分区：
文献类型：
--
作者：
Klusmann, Jan-Henning;Creutzig, Ursula;Reinhardt, Dirk
大约 10% 的唐氏综合症 (DS) 新生儿表现出独特的短暂性白血病 (TL)。尽管大多数患者 TL 会自行消退，但可能会发生早期死亡和发展为髓系白血病 (ML-DS)。预后因素以及治疗指征目前尚不确定。为了解决这个问题，我们前瞻性地收集了 146 名 TL 患者的临床、生物学和治疗数据。 5年总生存率（OS）和无事件生存率（EFS）分别为85%±3%和63%±4%。多变量分析揭示了高白细胞（WBC）计数、腹水、早产、出血素质、自发缓解失败和早期死亡之间的相关性。对 28 名白细胞计数高、血小板减少或肝功能障碍的患者进行阿糖胞苷 (0.5-1.5 mg/kg) 治疗。该疗法对具有早期死亡危险因素的儿童的结局产生有益影响（5 年 EFS，52% +/- 12% vs 28% +/- 11% [无治疗]；P = .02）。多变量分析证明了其有利的预后影响。共有 29 名 (23%) TL 患者随后发展为 ML-DS。有 TL 病史的 ML-DS 患者的 5 年 EFS (91% +/- 5%) 明显优于没有 TL 记录的患者 (70% +/- 4%)，这主要是因为复发率较低。因此，TL 病史可定义风险较低的 ML-DS 亚组。
Approximately 10% of the neonates with Down syndrome (DS) exhibit a unique transient leukemia (TL). Though TL resolves spontaneously in most patients, early death and development of myeloid leukemia (ML-DS) may occur. Prognostic factors as well as treatment indication are currently uncertain. To resolve that issue, we prospectively collected clinical, biologic, and treatment data of 146 patients with TL. The 5-year overall survival (OS) and event-free survival (EFS) were 85% plus or minus 3% and 63% plus or minus 4%, respectively. Multivariate analysis re-vealed a correlation between high white blood cell (WBC) count, ascites, preterm delivery, bleeding diatheses, failure of spontaneous remission, and the occurrence of early death. Treatment with cytarabine (0.5-1.5 mg/kg) was administered to 28 patients with high WBC count, thrombocytopenia, or liver dysfunction. The therapy had a beneficial effect on the outcome of those children with risk factors for early death (5-year EFS, 52% +/- 12% vs 28% +/- 11% [no treatment]; P = .02). Multivariate analysis demonstrated its favorable prognostic impact. A total of 29 (23%) patients with TL subsequently developed ML-DS. Patients with ML-DS with a history of TL had a significantly better 5-year EFS (91% +/- 5%) than those without documented TL (70% +/- 4%), primarily due to a lower relapse rate. A history of TL may therefore define a lower-risk ML-DS subgroup.