文章：

采用儿科方案治疗后，青少年和年轻成人急性淋巴细胞白血病的疗效显著提高且毒性可耐受：日本成人白血病研究组II期研究

Markedly improved outcomes and acceptable toxicity in adolescents and young adults with acute lymphoblastic leukemia following treatment with a pediatric protocol: a phase II study by the Japan Adult Leukemia Study Group

原文发布日期：2014-10-17

DOI: 10.1038/bcj.2014.72

类型: Original Article

开放获取: 是

英文摘要：

The superiority of the pediatric protocol for adolescents with acute lymphoblastic leukemia (ALL) has already been demonstrated, however, its efficacy in young adults remains unclear. The ALL202-U protocol was conducted to examine the efficacy and feasibility of a pediatric protocol in adolescents and young adults (AYAs) with BCR–ABL-negative ALL. Patients aged 15–24 years (n=139) were treated with the same protocol used for pediatric B-ALL. The primary objective of this study was to assess the disease-free survival (DFS) rate and its secondary aims were to assess toxicity, the complete remission (CR) rate and the overall survival (OS) rate. The CR rate was 94%. The 5-year DFS and OS rates were 67% (95% confidence interval (CI) 58–75%) and 73% (95% CI 64–80%), respectively. Severe adverse events were observed at a frequency that was similar to or lower than that in children treated with the same protocol. Only insufficient maintenance therapy significantly worsened the DFS (hazard ratio 5.60, P<0.001). These results indicate that this protocol may be a feasible and highly effective treatment for AYA with BCR–ABL-negative ALL.

摘要翻译： 

儿童方案在青少年急性淋巴细胞白血病（ALL）治疗中的优越性已得到证实，但其对年轻成人的疗效仍不明确。ALL202-U方案旨在探讨儿科方案在BCR-ABL阴性青少年及年轻成人（AYA）ALL患者中的有效性和可行性。研究纳入了139名15-24岁患者，采用与儿童B-ALL相同的治疗方案。本研究主要目的是评估无病生存率（DFS），次要目标包括评估毒性反应、完全缓解率（CR）和总生存率（OS）。完全缓解率达到94%，5年无病生存率和总生存率分别为67%（95%置信区间58-75%）和73%（95%置信区间64-80%）。严重不良事件发生率与接受相同方案的儿童患者相当或更低。其中维持治疗不足显著降低无病生存率（风险比5.60，P<0.001）。研究结果表明，该方案可能是治疗BCR-ABL阴性青少年及年轻成人ALL患者可行且高效的治疗策略。

原文链接：

Markedly improved outcomes and acceptable toxicity in adolescents and young adults with acute lymphoblastic leukemia following treatment with a pediatric protocol: a phase II study by the Japan Adult Leukemia Study Group