Uderzo C
BMT Unit, Pediatric Hematologic Department, S. Gerardo Hospital, Monza, University of Milano Bicocca, Italy.
Haematologica. 2000 Nov;85(11 Suppl):9-11.
Acute lymphoblastic leukemia (ALL) accounts for approximately one third of all cancers in children and its outcome depends on risk factors at the time of diagnosis. While uniform chemotherapy adopted in multicenter studies provided a constant improvement in cure rates for standard risk patients, the results reached in very high risk patients have been disappointing. The objective of this review is to point out the role of allogenic bone marrow transplantation (alloBMT) in very high risk childhood ALL on the basis of results from the current clinical trials. EVIDENCE AND INFORMATION SOURCE: Data covered by Medline and produced by the authors involved in ongoing international studies cover a vast "scenario" of children with very high risk ALL who underwent allogeneic BMT.
The author outlines the crucial point of very high risk factors in childhood ALL in order to identify those children who are at risk of early relapse. The main reasons for pursuing alloBMT in this particular category of patients concern poor prognostic factors such as molecular biology markers, structural chromosomal abnormalities and biological factors (poor prednisone response) including resistance to initial induction chemotherapy. AlloBMT in childhood ALL in first complete remission seemed to lead to a promising disease-free survival in this patient population when compared with chemotherapy. The principal biases of the retrospective studies were the variable very high risk eligibility criteria, the different first-line therapies adopted before alloBMT and above all the waiting time to transplant which could have accounted for some advantage to alloBMT patients versus chemotherapy patients.
The author touches upon the preliminary results of an ongoing international prospective study as an example of reaching a consensus in the controversial treatment of childhood very high risk ALL. This attempt should provide more information regarding the role of alloBMT in this setting and should cover an area of particular interest.
急性淋巴细胞白血病(ALL)约占儿童所有癌症的三分之一,其预后取决于诊断时的危险因素。虽然多中心研究采用的统一化疗使标准风险患者的治愈率不断提高,但极高风险患者的治疗结果却令人失望。本综述的目的是根据当前临床试验结果,指出异基因骨髓移植(alloBMT)在儿童极高风险ALL中的作用。证据与信息来源:由参与正在进行的国际研究的作者提供的、被Medline收录的数据,涵盖了接受异基因BMT的极高风险ALL儿童的广阔“情况”。
作者概述了儿童ALL中极高风险因素的关键点,以便识别那些有早期复发风险的儿童。在这类特定患者中进行alloBMT的主要原因涉及预后不良因素,如分子生物学标志物、结构性染色体异常以及生物学因素(泼尼松反应差),包括对初始诱导化疗的耐药性。与化疗相比,儿童ALL首次完全缓解时进行alloBMT似乎能使该患者群体获得有希望的无病生存期。回顾性研究的主要偏差在于极高风险入选标准的差异、alloBMT前采用的不同一线治疗方法,尤其是移植等待时间,这可能是alloBMT患者相对于化疗患者具有某些优势的原因。
作者提及一项正在进行的国际前瞻性研究的初步结果,作为在儿童极高风险ALL的争议性治疗中达成共识的一个例子。这一尝试应能提供更多关于alloBMT在这种情况下作用的信息,并应涵盖一个特别受关注的领域。