目的 探讨CCLG-ALL2008方案治疗具有不同分子生物学特征的初诊儿童急性淋巴细胞白血病（ALL）的长期疗效。方法 选取按照CCLG-ALL2008方案治疗的940例初诊ALL患儿为研究对象，针对不同分子生物学特征ALL的长期疗效进行回顾性分析。结果 940例ALL患儿中，男570例，女370例，中位年龄5（1~15）岁，中位随访时间65（3~123）个月。完全缓解（CR）率为96.7%，预期10年总体生存（OS）率为（76.5±1.5）%，无事件生存（EFS）率为（62.6±3.0）%。患儿经治疗达CR后，总复发率为21.9%，其中ETV6-RUNX1阳性患儿复发率最低，且易于晚期复发；MLL重排阳性患儿复发率最高，且易于早期复发。ETV6-RUNX1阳性患儿的预期10年OS率明显高于伴有TCF3-PBX1阳性、BCR-ABL阳性、MLL重排及无分子生物学特征患儿（P < 0.05）。ETV6-RUNX1阳性患儿的预期10年EFS率明显高于伴有BCR-ABL阳性和MLL重排患儿（P < 0.05）。结论 分子生物学特征是影响ALL患儿长期预后的指标，MLL重排、BCR-ABL融合基因阳性是预后的不良指标，ETV6-RUNX1融合基因阳性患儿长期生存率较高。
Abstract
Objective To study the long-term clinical effect of the CCLG-ALL2008 regimen in the treatment of children newly diagnosed with acute lymphoblastic leukemia (ALL) with different molecular biological features. Methods A total of 940 children who were newly diagnosed with ALL were enrolled in this study. The children were treated with the CCLG-ALL2008 regimen. A retrospective analysis was performed for the long-term outcome of ALL children with different molecular biological features. Results Among the 940 children with ALL, there were 570 boys and 370 girls, with a median age of onset of 5 years (range 1-15 years) and a median follow-up time of 65 months (range 3-123 months). The complete response (CR) rate was 96.7%, the predicted 10-year overall survival (OS) rate was 76.5%±1.5%, and the event-free survival (EFS) rate was 62.6%±3.0%. After CR was achieved after treatment, the overall recurrence rate was 21.9%. The children with positive ETV6-RUNX1 had the lowest recurrence rate and were prone to late recurrence, and those with positive MLL rearrangement had the highest recurrence rate and were prone to early recurrence. The children with positive ETV6-RUNX1 had a significantly higher predicted 10-year OS rate than those with positive TCF3-PBX1, BCR-ABL, or MLL rearrangement and those without molecular biological features (P < 0.05). The children with positive ETV6-RUNX1 had a significantly higher predicted 10-year EFS rate than those with positive BCR-ABL or MLL rearrangement (P < 0.05). Conclusions Molecular biological features may affect the long-term prognosis of children with ALL, and positive MLL rearrangement and BCR-ABL fusion gene are indicators of poor prognosis. Children with positive ETV6-RUNX1 fusion gene have the highest long-term survival rate.
关键词
急性淋巴细胞白血病 /
生存分析 /
分子生物学特征 /
儿童
Key words
Acute lymphoblastic leukemia /
Survival analysis /
Molecular biological feature /
Child
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