Kantarjian Hagop, Jain Nitin, Litzow Mark R, Luger Selina M, Papayannidis Cristina, Ribera Josep-Maria, Short Nicholas J, Chifotides Helen T, Jabbour Elias
Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Division of Hematology, Mayo Clinic, Rochester, Minnesota, USA.
Cancer. 2025 May 15;131(10):e35872. doi: 10.1002/cncr.35872.
The past decade has witnessed remarkable advances in deciphering the pathophysiology of acute lymphoblastic leukemia (ALL) and in developing novel targeted therapies. Basic research and genomic mapping have identified new prognostic biomarkers, targets, and ALL subtypes (e.g., Philadelphia-like ALL). The ongoing therapeutic revolution in ALL is driven by the addition to the treatment arsenal of therapies that target the ABL fusions, like the BCR::ABL1 tyrosine kinase inhibitors, as well as novel agents that target CD19 and CD22: the CD22 antibody-drug conjugate inotuzumab ozogamicin, the bispecific CD3/CD19 T-cell engager antibody blinatumomab, and CD19 chimeric antigen receptor T-cell therapies. These combinations have improved the long-term survival rates in B-cell ALL to 70%, and in Philadelphia chromosome-positive ALL to 80%-90%. The desired goals are to achieve cure rates comparable to those in pediatric ALL and to reduce or eliminate the need for prolonged intensive/maintenance chemotherapy and associated toxicities.
在过去十年中,急性淋巴细胞白血病(ALL)的病理生理学解读以及新型靶向治疗的开发都取得了显著进展。基础研究和基因组图谱已确定了新的预后生物标志物、靶点和ALL亚型(如费城样ALL)。ALL目前的治疗革命是由多种疗法加入治疗手段推动的,这些疗法包括靶向ABL融合蛋白的疗法(如BCR::ABL1酪氨酸激酶抑制剂),以及靶向CD19和CD22的新型药物:CD22抗体药物偶联物奥英妥珠单抗、双特异性CD3/CD19 T细胞衔接抗体贝林妥欧单抗,以及CD19嵌合抗原受体T细胞疗法。这些联合疗法已将B细胞ALL的长期生存率提高到70%,将费城染色体阳性ALL的长期生存率提高到80%-90%。理想目标是实现与儿童ALL相当的治愈率,并减少或消除长期强化/维持化疗及相关毒性的需求。