英国一项最新研究说，对患有急性淋巴细胞白血病的儿童而言，治疗后疾病复发是导致死亡的重要原因，而一种名为米托蒽醌的药物可提高疾病复发儿童的存活率，其效果明显优于过去使用的药物。
英国曼彻斯特大学等机构研究人员12月4日在医学刊物The Lancet 上报告说，200多名患有急性淋巴细胞白血病并且疾病复发的儿童参与了试验，其中约一半人服用传统的治疗药物伊达比星，而另一半人服用米托蒽醌，结果在3年后，前一组儿童的存活率为45%，而后一组儿童的存活率达到69%。
白血病又称血癌，急性淋巴细胞白血病是发生在儿童身上的主要白血病类型。据资助本次研究的英国癌症研究会介绍，对这种疾病的治疗近些年取得进展，患病儿童的存活率已经从50%上升到80%以上，但疾病复发仍然是个大问题，疾病复发儿童的存活率只在50%左右，近些年一直未能取得进展。
进行研究的瓦斯卡萨哈教授说，采用米托蒽醌治疗急性淋巴细胞白血病将为患者和他们的家庭带来福音。
推荐原文出处：
The Lancet doi:10.1016/S0140-6736(10)62002-8
Effect of mitoxantrone on outcome of children with first relapse of acute lymphoblastic leukaemia (ALL R3): an open-label randomised trial
Catriona Parker PhD a, Rachel Waters MSc c, Carly Leighton BNurs a b *?, Jeremy Hancock PhD d, Rosemary Sutton PhD e, Anthony V Moorman PhD f, Philip Ancliff FRCPath g, Mary Morgan MRCPath h, Ashish Masurekar MD a, Nicholas Goulden FRCPath g, Nina Green j, Tamas Révész FRACP i, Philip Darbyshire FRCPath j, Sharon Love BSc c, Prof Vaskar Saha FRCPCH a k
Summary
Background
Although survival of children with acute lymphoblastic leukaemia has improved greatly in the past two decades, the outcome of those who relapse has remained static. We investigated the outcome of children with acute lymphoblastic leukaemia who relapsed on present therapeutic regimens.
Methods
This open-label randomised trial was undertaken in 22 centres in the UK and Ireland and nine in Australia and New Zealand. Patients aged 1―18 years with first relapse of acute lymphoblastic leukaemia were stratified into high-risk, intermediate-risk, and standard-risk groups on the basis of duration of first complete remission, site of relapse, and immunophenotype. All patients were allocated to receive either idarubicin or mitoxantrone in induction by stratified concealed randomisation. Neither patients nor those giving interventions were masked. After three blocks of therapy, all high-risk group patients and those from the intermediate group with postinduction high minimal residual disease (≥10?4 cells) received an allogenic stem-cell transplant. Standard-risk and intermediate-risk patients with postinduction low minimal residual disease (&10?4 cells) continued chemotherapy. The primary outcome was progression-free survival and the method of analysis was intention-to-treat. Randomisation was stopped in December, 2007 because of differences in progression-free and overall survival between the two groups. This trial is registered, reference number ISCRTN45724312.
Findings
Of 239 registered patients, 216 were randomly assigned to either idarubicin (109 analysed) or mitoxantrone (103 analysed). Estimated 3-year progression-free survival was 359% (95% CI 259―459) in the idarubicin group versus 646% (542―732) in the mitoxantrone group (p=00004), and 3-year overall survival was 452% (345―553) versus 690% (585―773; p=0004). Differences in progression-free survival between groups were mainly related to a decrease in disease events (progression, second relapse, disease-related deaths; HR 056, 034―092, p=0007) rather than an increase in adverse treatment effects (treatment death, second malignancy; HR 052, 024―111, p=011).
Interpretation
As compared with idarubicin, mitoxantrone conferred a significant benefit in progression-free and overall survival in children with relapsed acute lymphobastic leukaemia, a potentially useful clinical finding that warrants further investigation.
Funding
Cancer Research UK, Leukaemia and Lymphoma Research, Cancer Council NSW, and Sporting Chance Cancer Foundation.
