Battaglia Giulia, Lazzarotto Davide, Tanasi Ilaria, Gurrieri Carmela, Forlani Laura, Mauro Endri, Capraro Francesca, Ciotti Giulia, De Bellis Eleonora, Callegari Chiara, Tosoni Luca, Fanin Matteo, Morelli Gian Luca, Simio Claudia, Skert Cristina, Gottardi Michele, Zaja Francesco, Toffoletti Eleonora, Damiani Daniela, Fanin Renato, Tiribelli Mario
Division of Hematology and BMT, Azienda Sanitaria Universitaria Friuli Centrale, 33100 Udine, Italy.
Hematology Unit, Department of Engineering for Innovation Medicine, Azienda Ospedaliera Universitaria Integrata di Verona, 37126 Verona, Italy.
J Clin Med. 2025 Jan 22;14(3):700. doi: 10.3390/jcm14030700.
: Nucleophosmin-1 () mutation accounts for 30% of acute myeloid leukemia (AML) cases and defines either low- or intermediate-risk AML, depending on -ITD mutation. New combination regimens (NCRs), adding midostaurin and gemtuzumab ozogamicin (GO) to the 3 + 7 scheme, are commonly used, though there are no data that compare NCRs with intensive induction chemotherapy. : To evaluate the efficacy and safety of NCRs and FLAI in + AML, we retrospectively analyzed 125 patients treated with FLAI ( = 53) or NCRs ( = 72) at seven Italian Centers. : The median age was 61 years and 51/125 (41%) were -ITD+. The complete remission (CR) rate was 77%, slightly better with NCRs (83% vs. 68%; = 0.054). NCRs yielded a superior median overall survival (OS) (not reached (NR) vs. 27.3 months; = 0.002), though the median event-free survival (EFS) was similar (NR vs. 20.5 months; = 0.07). In low-risk AML, CR was higher in NCRs (94% vs. 72%, = 0.02), as were median OS (NR vs. 41.6 months; = 0.0002) and EFS (NR vs. 17.8 months; = 0.0085). In intermediate-risk AML (-ITD+), there were no differences in CR (60% vs. 71%; = 0.5), OS ( = 0.27), or EFS ( = 0.86); only allogeneic transplantation improved OS (NR vs. 13.4 months; = 0.005), regardless of induction regimen. The safety profile was similar, except for delayed platelet recovery with FLAI (22 vs. 18 days; = 0.0024) and higher-grade II-IV gastrointestinal toxicity with NCRs (43% vs. 18.8%; = 0.0066). Our data suggest the superiority of NCRs over FLAI in low-risk patients, while all outcomes were comparable in intermediate-risk patients, a setting in which only transplants positively impacted on survival.
核磷蛋白-1（）突变占急性髓系白血病（AML）病例的30%，并根据-ITD突变定义低危或中危AML。新的联合方案（NCRs），即在3 + 7方案中加入米哚妥林和吉妥珠单抗奥唑米星（GO），虽尚无将NCRs与强化诱导化疗进行比较的数据，但仍普遍使用。为评估NCRs和FLAI在+ AML中的疗效和安全性，我们回顾性分析了意大利七个中心接受FLAI（ = 53）或NCRs（ = 72）治疗的125例患者。中位年龄为61岁，51/125（41%）为-ITD+。完全缓解（CR）率为77%，NCRs组略高（83%对68%； = 0.054）。NCRs组的中位总生存期（OS）更长（未达到（NR）对27.3个月； = 0.002），尽管中位无事件生存期（EFS）相似（NR对20.5个月； = 0.07）。在低危AML中，NCRs组的CR更高（94%对72%， = 0.02），中位OS（NR对41.6个月； = 0.0002）和EFS（NR对17.8个月； = 0.0085）也是如此。在中危AML（-ITD+）中，CR（60%对71%； = 0.5）、OS（ = 0.27）或EFS（ = 0.86）无差异；只有异基因移植改善了OS（NR对13.4个月； = 0.005），与诱导方案无关。安全性方面相似，除了FLAI组血小板恢复延迟（22天对18天； = 0.0024）以及NCRs组有更高比例的II-IV级胃肠道毒性（43%对18.8%； = 0.0066）。我们的数据表明，在低危患者中NCRs优于FLAI，而在中危患者中所有结果相当，在这种情况下只有移植对生存有积极影响。
