Department of Medicine V, University of Heidelberg, 69120 Heidelberg, Germany.
Blood. 2013 Apr 18;121(16):3284-8. doi: 10.1182/blood-2012-11-469627. Epub 2013 Feb 22.
The purpose of this analysis was to provide 6-year follow-up of the CLL3X trial, which studied reduced-intensity allogeneic hematopoietic stem cell transplantation (HSCT) in patients with poor-risk chronic lymphocytic leukemia (CLL), and to investigate the effect of TP53, SF3B1, and NOTCH1 mutations on HSCT outcome. For 90 allografted patients, 6-year overall survival (OS) was 58% and 6-year event-free survival (EFS) was 38%. TP53, SF3B1, and NOTCH1 mutations were found in 30%, 26%, and 14% of the trial population, respectively. By univariate and multivariate analyses, the mutational status of the TP53, SF3B1, and NOTCH1 genes had no significant effect on OS and EFS. Studies of minimal residual disease confirmed durability of CLL eradication in mutated patients. We conclude that HSCT can provide long-term disease control in patients with poor-risk CLL independent of the presence of TP53, SF3B1, and NOTCH1 mutations. The trial has been registered at the US National Cancer Institute as #EU-20554, NCT00281983.
本分析旨在提供 CLL3X 试验的 6 年随访结果，该试验研究了高危慢性淋巴细胞白血病 (CLL) 患者的低强度异基因造血干细胞移植 (HSCT)，并探讨了 TP53、SF3B1 和 NOTCH1 突变对 HSCT 结果的影响。在 90 例接受同种异体移植的患者中，6 年总生存率 (OS) 为 58%，6 年无事件生存率 (EFS) 为 38%。在该试验人群中，分别发现了 30%、26%和 14%的 TP53、SF3B1 和 NOTCH1 突变。通过单变量和多变量分析，TP53、SF3B1 和 NOTCH1 基因突变的状态对 OS 和 EFS 均无显著影响。微小残留病研究证实了突变患者 CLL 清除的持久性。我们得出结论，HSCT 可在不考虑 TP53、SF3B1 和 NOTCH1 突变的情况下为高危 CLL 患者提供长期疾病控制。该试验已在美国国立癌症研究所注册，编号为 #EU-20554，NCT00281983。
