文章：

一项针对所有危险组急性早幼粒细胞白血病的全反式维甲酸联合三氧化二砷无化疗有效治疗方案（APL15试验）

An effective and chemotherapy-free strategy of all-trans retinoic acid and arsenic trioxide for acute promyelocytic leukemia in all risk groups (APL15 trial)

原文发布日期：2022-11-21

DOI: 10.1038/s41408-022-00753-y

类型: Article

开放获取: 是

英文摘要：

Patients with chronic lymphocytic leukemia (CLL) with disease progression on ibrutinib have worse outcomes compared to patients stopping ibrutinib due to toxicity. A better understanding of expected outcomes in these patients is necessary to establish a benchmark for evaluating novel agents currently available and in development. We evaluated outcomes of 144 patients with CLL treated at Mayo Clinic with 2018 iwCLL disease progression on ibrutinib. The median overall survival (OS) for the entire cohort was 25.5 months; it was 29.8 months and 8.3 months among patients with CLL progression (n = 104) and Richter transformation (n = 38), respectively. Longer OS was observed among patients with CLL progression who had received ibrutinib in the frontline compared to relapsed/refractory setting (not reached versus 28.5 months; p = 0.04), but was similar amongst patients treated with 1, 2, or ≥3 prior lines (18.5, 30.9, and 26.0 months, respectively, p = 0.24). Among patients with CLL disease progression on ibrutinib, OS was significantly longer when next-line treatment was chimeric antigen receptor T-cell therapy (median not reached) or venetoclax-based treatment (median 29.8 months) compared to other approved treatments, such as chemoimmunotherapy, phosphoinositide 3’-kinase inhibitors, and anti-CD20 monoclonal antibodies (9.1 months; p = 0.03). These findings suggest an unmet need for this growing patient population.
 

摘要翻译： 

接受DNMTI治疗的骨髓增生异常综合征（MDS）根据2006年IWG疗效标准评估治疗反应。完全缓解（CR）与总生存期（OS）的相关性最强。近期，在急性髓系白血病（AML）中已对伴有部分血液学恢复的完全缓解（CRh；即原始细胞<5%、中性粒细胞绝对计数>500、血小板>50）进行了评估，但其在MDS中的意义尚不明确。我们纳入了接受DNMTIs治疗的成人MDS患者，根据IWG 2006标准评估最佳总体治疗反应，并从疾病稳定（SD）或骨髓完全缓解（mCR）亚组中筛选出符合CRh标准的患者。我们依据不同治疗反应评估了治疗持续时间和总生存期。共纳入2007年至2018年间接受治疗的311例MDS患者，治疗中位年龄69岁（范围23-91岁），中位随访时间60个月。根据IWG 2006标准，治疗反应包括：CR（43例，14%）、部分缓解（PR）（2例，1%）、mCR（57例，18%）、SD（149例，48%）和疾病进展（PD）（60例，19%）。79例患者（25%）达到血液学改善（HI）。共有62例患者（20%）符合CRh标准，导致原mCR（现为26例，8%）和SD（现为118例，38%）患者重新分类。获得CR的患者治疗持续时间（中位8.1个月）与CRh患者（中位6个月，HR 1.4，95% CI 0.9–2.0）相似，且长于其他治疗反应（p<0.001）。不同反应组的总生存期存在差异：CR组（中位23.3个月）与CRh组（中位25个月，HR 1.28 [0.79–2.08]）中位OS相近，且均长于mCR组（17.2个月，HR 1.71 [0.96–3.05]）、SD组（16.3个月，HR 1.61 [1.04–2.48]）和PD组（8.7个月，HR 3.04 [1.91–4.83]）（p<0.001）。在校正异基因移植因素的多变量分析中，CR/CRh与OS的相关性得到确认。达到CRh反应的MDS患者，其生存期和治疗持续时间与达到CR反应者相似，且优于其他IWG标准定义的反应。这些数据支持将CRh纳入未来疗效标准和临床试验中进行进一步评估。

原文链接：

An effective and chemotherapy-free strategy of all-trans retinoic acid and arsenic trioxide for acute promyelocytic leukemia in all risk groups (APL15 trial)