Jastaniah Wasil, Elimam Naglla, Abdalla Khalid, Felimban Sami, Abrar Mohammed Burhan
a Princess Noorah Oncology Center , King Saud Bin Abdulaziz University and King Abdulaziz Medical City , Jeddah , Saudi Arabia.
b Department of Pediatrics, Faculty of Medicine , Umm AlQura University , Makkah , Saudi Arabia.
Hematology. 2016 Apr;21(3):175-81. doi: 10.1080/10245332.2015.1101974. Epub 2016 Feb 24.
Treatment regimens tested in major clinical trials, conducted by cooperative groups, are often adapted as standard of care by cancer centers with the hope to replicate the treatment outcomes reported in these landmark studies. It is therefore postulated that applying clinical trial regimens in a non-clinical trial setting yield similar outcomes. The aim of the present study was to explore this hypothesis in the context of childhood acute lymphoblastic leukemia (ALL) in our institution.
We retrospectively evaluated 224 consecutive pediatric ALL cases treated between January 2001 and December 2007. Standard-risk (SR) patients were treated on CCG-1991 (regimen OD) while high-risk (HR) patients were treated on CCG-1961 (regimen D). Results were compared with those of the equivalent regimen in the original clinical trials. Statistical analysis was carried using chi-square or Fisher's exact test, Kaplan-Meier and log-rank tests.
Comparison of treatment outcomes revealed that SR patients had inferior 5-year overall survival (OS) of (89.0 ± 2.9 vs. 96.0% ± 0.9%); event-free survival of (82.3 ± 3.5 vs. 88.7% ± 1.4%); and relapse rate of (15.8 vs. 9.3% (P = 0.034)) compared to patients treated in the clinical trial. However, no statistically significant difference in treatment outcomes was observed between HR patients.
Despite using comparable regimens, suboptimal outcomes were noted in SR patients implying that similar treatments do not necessarily yield similar outcomes. This underscores the need to evaluate outcomes of adapted regimens to identify areas that need further improvement in centers not enrolling patients on prospective collaborative clinical trials.
由协作组开展的主要临床试验中所测试的治疗方案，常被癌症中心用作护理标准，以期复制这些具有里程碑意义的研究中所报告的治疗结果。因此据推测，在非临床试验环境中应用临床试验方案会产生相似的结果。本研究的目的是在我们机构中针对儿童急性淋巴细胞白血病（ALL）探讨这一假设。
我们回顾性评估了2001年1月至2007年12月期间连续治疗的224例儿科ALL病例。标准风险（SR）患者采用CCG - 1991（方案OD）治疗，而高风险（HR）患者采用CCG - 1961（方案D）治疗。将结果与原始临床试验中相应方案的结果进行比较。使用卡方检验或费舍尔精确检验、Kaplan - Meier检验和对数秩检验进行统计分析。
治疗结果比较显示，与临床试验中治疗的患者相比，SR患者的5年总生存率较低（分别为89.0 ± 2.9% 与96.0% ± 0.9%）；无事件生存率较低（分别为82.3 ± 3.5% 与88.7% ± 1.4%）；复发率较高（分别为15.8% 与9.3%，P = 0.034）。然而，HR患者的治疗结果未观察到统计学上的显著差异。
尽管使用了类似的方案，但SR患者的结果欠佳，这意味着相似的治疗不一定产生相似的结果。这凸显了在未招募患者参加前瞻性协作临床试验的中心评估适应性方案结果以确定需要进一步改进领域的必要性。
