Shah Ankit, Barrientos Jacqueline C
Division of Hematology-Oncology, Department of Medicine at Rutgers New Jersey Medical School, Newark, NJ, USA.
CLL Research and Treatment Center, Division of Hematology-Oncology, Department of Medicine at Zucker School of Medicine at Hofstra/Northwell, Hempstead, NY, USA.
Onco Targets Ther. 2021 Mar 25;14:2109-2119. doi: 10.2147/OTT.S189032. eCollection 2021.
The development of highly effective targeted therapies has led to a new treatment paradigm in patients with chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL). Despite these advances, many patients will eventually require alternative treatment strategies due to the emergence of tolerability issues or resistance to these novel agents. Duvelisib is a first-in-class, potent oral agent with dual inhibitor activity against the δ and γ isoforms of phosphoinositide 3-kinase (PI3Kδ and PI3Kγ), which are specific to the hematopoietic system. Dysregulation of the PI3K/PTEN/AKT/mTOR pathway has been implicated in cancer cell growth, survival and metabolism and has been the subject of cancer drug development in recent years. Duvelisib demonstrated activity in CLL/SLL in early trials, leading to further evaluation in the Phase 3 DUO trial that compared duvelisib against ofatumumab in patients with relapsed/refractory CLL/SLL. This trial led to the Food and Drug Administration (FDA) approval for the treatment of adult patients with CLL/SLL after at least two prior lines of therapy. The major reason for therapy discontinuation is the development of serious adverse events, which include severe infections and diarrhea/colitis, precluding its widespread use. Ongoing clinical trials are evaluating duvelisib in combination strategies and with alternate dosing schedules in patients with CLL/SLL. With close monitoring, duvelisib can be a promising drug for the treatment of patients with relapsed or refractory CLL/SLL. This review summarizes the relevant clinical data from recent clinical advances in CLL and aims to interpret the duvelisib trials while exploring strategies to improve its use and adverse event management in the era of novel targeted agents.
高效靶向疗法的发展为慢性淋巴细胞白血病或小淋巴细胞淋巴瘤(CLL/SLL)患者带来了新的治疗模式。尽管取得了这些进展,但由于耐受性问题的出现或对这些新型药物产生耐药性,许多患者最终仍需要 alternative treatment strategies。度维利塞是一种一流的强效口服药物,对造血系统特有的磷酸肌醇3激酶(PI3Kδ和PI3Kγ)的δ和γ同工型具有双重抑制活性。PI3K/PTEN/AKT/mTOR通路的失调与癌细胞的生长、存活和代谢有关,并且是近年来癌症药物开发的主题。度维利塞在早期试验中显示出对CLL/SLL的活性,从而在3期DUO试验中进行了进一步评估,该试验将度维利塞与奥法木单抗用于复发/难治性CLL/SLL患者进行比较。该试验导致美国食品药品监督管理局(FDA)批准度维利塞用于治疗至少经过两线先前治疗的成年CLL/SLL患者。治疗中断的主要原因是严重不良事件的发生,包括严重感染和腹泻/结肠炎,这妨碍了其广泛使用。正在进行的临床试验正在评估度维利塞在CLL/SLL患者中的联合治疗策略和交替给药方案。通过密切监测,度维利塞可能成为治疗复发或难治性CLL/SLL患者的有前景的药物。本综述总结了CLL近期临床进展中的相关临床数据,旨在解读度维利塞试验,同时探索在新型靶向药物时代改善其使用和不良事件管理的策略。