Department of Haematology, Great Ormond Street Hospital for Children, London, UK.
Clinical Trial Service Unit, University of Oxford, Oxford, UK.
Arch Dis Child. 2016 May;101(5):449-54. doi: 10.1136/archdischild-2015-309617. Epub 2016 Feb 10.
Minimal residual disease (MRD) is defined as the presence of sub-microscopic levels of leukaemia. Measurement of MRD from bone marrow at the end of induction chemotherapy (day 28) for childhood acute lymphoblastic leukaemia (ALL) can highlight a large group of patients (>40%) with an excellent (>90%) short-term event-free survival (EFS). However, follow-up in recent published trials is relatively short, raising concerns about using this result to infer the safety of further therapy reduction in the future.
We examined MRD data on 225 patients treated on one of three UKALL trials between 1997 and 2003 to assess the long-term (>10 years follow-up) outcome of those patients who had low-risk MRD (<0.01%) at day 28.
Our pilot data define a cohort of 53% of children with MRD <0.01% at day 28 who have an EFS of 91% and long-term overall survival of 97%. Of 120 patients with day-28 MRD <0.01% and extended follow-up, there was one death due to treatment-related toxicity, one infectious death while in complete remission, and four relapse deaths.
The excellent outcome for childhood ALL in patients with MRD <0.01% after induction chemotherapy is sustained for more than 10 years from diagnosis. This supports the potential exploration of further reduction of therapy in this group, in an attempt to reduce treatment-related mortality and late effects.
微小残留病（MRD）定义为亚微观水平的白血病存在。在儿童急性淋巴细胞白血病（ALL）诱导化疗结束时（第 28 天）从骨髓中测量 MRD，可以突出一大群患者（>40%）具有极好的（>90%）短期无事件生存（EFS）。然而，最近发表的试验随访时间相对较短，这引发了对使用该结果推断未来进一步减少治疗安全性的担忧。
我们检查了 1997 年至 2003 年期间在三项英国 ALL 试验之一中治疗的 225 名患者的 MRD 数据，以评估那些在第 28 天具有低风险 MRD（<0.01%）的患者的长期（>10 年随访）结局。
我们的初步数据定义了一个队列，其中 53%的第 28 天 MRD<0.01%的儿童 EFS 为 91%，长期总生存率为 97%。在 120 名第 28 天 MRD<0.01%且随访时间延长的患者中，有 1 例因治疗相关毒性而死亡，1 例在完全缓解时因感染性死亡，4 例因复发而死亡。
在诱导化疗后 MRD<0.01%的儿童 ALL 患者的优异结局在诊断后超过 10 年持续存在。这支持了在该组中进一步减少治疗的潜在探索，以试图降低治疗相关死亡率和晚期效应。
