University College London, Royal Free Campus, Rowland Hill Street, London, United Kingdom.
Blood. 2010 Nov 4;116(18):3409-17. doi: 10.1182/blood-2010-01-242750. Epub 2010 Jul 23.
The Philadelphia chromosome is present in approximately 20% to 30% of adults with acute lymphoblastic leukemia (ALL). The poor prognosis of this relatively uncommon acute leukemia has led to the rapid adoption of treatment strategies such as unrelated donor hematopoietic stem cell transplant and tyrosine kinase inhibitors into clinical practice, despite a relative paucity of randomized clinical trials. Recently, there has been a surge of interest in the underlying biology of ALL. In combination with an accumulation of more mature clinical study data in Philadelphia-positive ALL, it is increasingly possible to make more rational and informed treatment choices for patients of all ages. In this article, I review available data and indicate how I personally interpret current evidence to make pragmatic treatment choices with my patients, outside of clinical trials. My strongest recommendation is that all physicians who are treating this rare disease actively seek appropriate clinical trials for their patients wherever possible.
费城染色体约存在于 20%至 30%的成人急性淋巴细胞白血病(ALL)患者中。这种相对罕见的急性白血病预后不良,导致即使随机临床试验相对较少,也迅速采用了治疗策略,如无关供体造血干细胞移植和酪氨酸激酶抑制剂。最近,人们对 ALL 的基础生物学产生了浓厚的兴趣。结合费城阳性 ALL 中更成熟的临床研究数据的积累,现在越来越有可能为所有年龄段的患者做出更合理、更明智的治疗选择。在本文中,我回顾了现有数据,并说明我个人如何解释当前的证据,以便在临床试验之外为我的患者做出实际的治疗选择。我最强烈的建议是,所有治疗这种罕见疾病的医生都应尽可能为患者积极寻找合适的临床试验。