Chessells J M, Richards S M, Bailey C C, Lilleyman J S, Eden O B
Department of Haematology and Oncology, Hospital for Sick Children, London.
Br J Haematol. 1995 Feb;89(2):364-72. doi: 10.1111/j.1365-2141.1995.tb03313.x.
We have examined the factors influencing prognosis in over 4000 children with acute lymphoblastic leukaemia (ALL) aged 1-14 who have been treated on consecutive MRC UKALL trials from 1972 to 1990. During this time the results of treatment have improved steadily but are consistently superior in girls when compared with boys; the 5-year event-free survival in girls improving from 51% to 71% and in boys from 31% to 57%. These results were independent of age and presenting leucocyte count. Boys not only had a testicular relapse rate of 10% but an excess of bone marrow relapse, particularly evident after 2 years from diagnosis. Other prognostic factors included organomegaly and the morphology of leukaemic blast cells; immunophenotype of the leukaemia, however, had no independent significance after allowance for age, sex and leucocyte count. The influence of sex on prognosis was reaffirmed when we examined various methods of identifying children at highest risk of treatment failure for whom alternative therapy such as bone marrow transplantation might be justified. In MRC UKALL X children had been deemed 'high risk' on the basis of leucocyte count alone, but with further follow-up it has become apparent that girls with an initial leucocyte count of > 100 x 10(9)/l have a similar prognosis to boys with a lower count. We therefore derived a risk score based on sex, age and count which has given better discrimination between standard risk (66% 5-year survival) and poor risk (39%) survival than other methods. This group of worse-risk children includes 16% of boys but only 3% of all girls. Gender remains an important prognostic factor in UKALL trials and there are very few girls who are at highest risk of treatment failure. The reasons for this remain unclear, but the pattern of relapses suggests that boys more often get inadequate systemic therapy. We postulate that the reasons for treatment failure may relate to sensitivity to continuing (maintenance) chemotherapy.
我们研究了1972年至1990年期间在英国医学研究理事会（MRC）的连续ALL试验中接受治疗的4000多名1至14岁急性淋巴细胞白血病（ALL）儿童的预后影响因素。在此期间，治疗结果稳步改善，但与男孩相比，女孩的治疗结果始终更优；女孩的5年无事件生存率从51%提高到71%，男孩从31%提高到57%。这些结果与年龄和初诊时的白细胞计数无关。男孩不仅睾丸复发率为10%，而且骨髓复发率过高，在诊断后2年尤为明显。其他预后因素包括器官肿大和白血病原始细胞的形态；然而，在考虑年龄、性别和白细胞计数后，白血病的免疫表型没有独立的意义。当我们研究各种识别治疗失败风险最高的儿童的方法时，性别对预后的影响再次得到证实，对于这些儿童，可能有理由采用替代疗法，如骨髓移植。在MRC UKALL X试验中，儿童仅根据白细胞计数被视为“高危”，但随着进一步随访，很明显，初始白细胞计数>100×10⁹/L的女孩与白细胞计数较低的男孩预后相似。因此，我们根据性别、年龄和计数得出了一个风险评分，与其他方法相比，该评分在标准风险（5年生存率66%）和不良风险（39%）生存率之间的区分度更好。这组风险较高的儿童包括16%的男孩，但仅占所有女孩的3%。在UKALL试验中，性别仍然是一个重要的预后因素，很少有女孩处于治疗失败的最高风险。其原因尚不清楚，但复发模式表明男孩更常接受不足的全身治疗。我们推测治疗失败的原因可能与对持续（维持）化疗的敏感性有关。
