研究动态
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成人急性髓系白血病中与WT1基因突变同时出现的克隆性突变框架:Alliance for Clinical Trials in Oncology研究。

Framework of clonal mutations concurrent with WT1 mutations in adults with acute myeloid leukemia: Alliance for Clinical Trials in Oncology study.

发表日期:2023 Aug 22
作者: Bhavana Bhatnagar, Jessica Kohlschmidt, Shelley J Orwick, Daelynn R Buelow, Sydney Fobare, Christopher C Oakes, Jonathan E Kolitz, Geoff Uy, Wendy Stock, Bayard L Powell, Deedra Nicolet, Erin K Hertlein, Krzysztof Mrózek, James S Blachly, Ann-Kathrin Eisfeld, Sharyn D Baker, John C Byrd
来源: Blood Advances

摘要:

反应不佳的多发性骨髓瘤患者(MM)的预后比原发反应者更差,在这个人群中缺乏有效的治疗方法。然而,现代治疗时代初次耐药疾病的预后尚未进行研究。我们回顾了在我院接受三联/四联疗法治疗的MM患者,以评估初次耐药疾病的发生率和拯救性治疗对此人群的影响。我们共鉴定了1127名患者,其中1086名患者在进行了4至6个疗程后进行了血液学反应评价。在这些患者中,93.3%(1013名)显示出治疗反应,而6.7%(73名)患者患有初次耐药疾病。初次耐药疾病的患者中,中位总生存期(OS)为51.3个月,单变量和多变量分析中,患者存活期缩短的风险增加(危险比[HR],3.5 [95%置信区间(CI),2.5-4.9],HR,4.3 [95% CI,2.6-6.9])。在初次耐药疾病患者的亚组分析中,接受二线自体干细胞移植(ASCT)的患者与未接受ASCT的患者相比,具有较长的二次无进展生存期(20.9 vs 8.1个月;P < .01)和二次OS(74.7 vs 31.3个月;P = .02)。我们得出结论,在当前时代,早期进展仍是影响较短OS的重要因素,并且对于这一人群,拯救性ASCT可能是最有益的选择。
Patients with multiple myeloma (MM) who do not respond to initial therapy have worse outcomes than primary responders, and effective treatments are lacking in this population. However, the outcomes of primary refractory disease in the modern treatment era have not yet been studied. We reviewed patients with MM treated with triplet/quadruplet therapy at our institution to assess the incidence of primary refractory disease and the impact of salvage therapies in this population. We identified 1127 patients, of whom 1086 were evaluated for hematologic responses after 4 to 6 cycles. Of these, 93.3% (1013) had evidence of response, whereas 6.7% (73) had primary refractory disease. With a median overall survival (OS) of 51.3 months, patients with primary refractory disease had an increased risk of shorter survival in univariable and multivariable analyses (hazard ratio [HR], 3.5 [95% confidence interval (CI), 2.5-4.9]; HR, 4.3 [95% CI, 2.6-6.9], respectively). In the subgroup analysis of patients with primary refractory disease, those who received second-line autologous stem cell transplantation (ASCT) had increased second progression-free survival (20.9 vs 8.1 months; P < .01) and second OS (74.7 vs 31.3 months; P = .02) compared with patients who did not. We conclude that early progression remains a significant factor for shorter OS in the current era, and salvage ASCT could be the most beneficial option for this population.