从基线至第10天胎儿血红蛋白的增加是急性髓系白血病（AML）对地西他滨应答的生物标志物：EORTC白血病组、意大利成人血液病恶性肿瘤组（GIMEMA）和德国MDS研究组的AML21随机III期试验结果。
完整摘要 · 中文译文
对治疗无反应或反应延迟是DNA低甲基化药物（HMA）用于急性髓系白血病（AML）患者时的主要挑战。胎儿血红蛋白（HbF）是预测HMA治疗反应的潜在生物标志物，但仅在小型单中心研究中得到过探讨。我们在随机3期欧洲癌症研究与治疗组织（EORTC）-1301试验AML21中研究了HbF与治疗结局之间的关联，该试验纳入了606例适合治疗的老年AML患者，接受10天地西他滨或“3 + 7”化疗作为诱导治疗。在地西他滨组（n = 135）中HbF检测了两次，在3 + 7组（n = 97）中于基线检测。在地西他滨组中，HbF从基线至第10天的升高与达到完全缓解（CR）或血细胞计数未完全恢复的CR（CRi）的几率增加相关（比值比[OR] 2.40，95%置信区间[CI] 1.15-5.02，p = 0.020）。比较HbF升高患者与未升高患者的死亡风险比为0.83（95% CI 0.55-1.28，p = 0.40）。地西他滨组的基线HbF与CR/CRi无关（OR 0.89，95% CI 0.45-1.75，p = 0.73），与总生存期也无关联（HR 0.86，95% CI 0.58-1.28，p = 0.47）。总之，这项大型前瞻性研究进一步充实了证据，表明HbF是一种易于检测的早期生物标志物，可用于预测HMA治疗反应。
完整摘要 · 英文原文
Lack of response or delayed response to therapy are major challenges in the use of DNA-hypomethylating agents (HMA) in patients with acute myeloid leukaemia (AML). Fetal haemoglobin (HbF) is a potential biomarker for response to HMA treatment but has only been studied in small single-centre studies. We investigated the association between HbF and treatment outcome in the randomized phase 3 European Organisation for Research and Treatment of Cancer (EORTC)-1301 trial AML21, which included 606 fit, older AML patients who received 10-day decitabine or '3 + 7' chemotherapy as induction. HbF was measured twice in the decitabine arm (n = 135) and at baseline in the 3 + 7 arm (n = 97). In the decitabine arm, HbF increase from baseline to day 10 was associated with increased odds of reaching complete remission (CR) or CR with incomplete count recovery (CRi) (odds ratio [OR] 2.40, 95% confidence interval [CI] 1.15-5.02, p = 0.020). The hazard ratio (HR) for death comparing patients with HbF increase to those without was 0.83 (95% CI 0.55-1.28, p = 0.40). Baseline HbF in the decitabine arm was not associated with CR/CRi (OR 0.89, 95% CI 0.45-1.75, p = 0.73) or overall survival (HR 0.86, 95% CI 0.58-1.28, p = 0.47). In conclusion, this large prospective study adds to the body of evidence that HbF is an easy-to-measure early biomarker for response to HMA treatment.