Mauro Francesca R, Caputo Maria D, Rosati Serena, Pepe Sara, De Benedittis Daniela, De Luca Maria L, Foà Robin
a Hematology, Department of Cellular Biotechnologies and Hematology , Sapienza University and Policlinico Umberto 1 , Rome , Italy.
Expert Rev Hematol. 2018 Aug;11(8):601-611. doi: 10.1080/17474086.2018.1495557. Epub 2018 Jul 18.
In recent years, innovative mechanism-based drugs have enriched the therapeutic armamentarium for patients with chronic lymphocytic leukemia (CLL) and are widely used in the clinical practice. These small molecules targeting the B-cell receptor signaling pathway and the BCL-2 anti-apoptotic protein offer new chemo-free options to both unfit patients and high-risk patients who show a poor response to chemoimmunotherapy. Nonetheless, treatment with ibrutinib, idelalisib and venetoclax is associated with unique side effects. Awareness, prevention and the appropriate management of these specific toxicities are of crucial importance for a successful treatment. Areas covered: The purpose of this review is to discuss the most relevant studies on small molecules in CLL, with particular attention to the emerging toxicity profile of these agents and to the factors that should be considered to address the most appropriate treatment approach for each patient. Expert opinion: The increased knowledge on the biology of CLL has translated into the development of targeted agents that are highly effective and produce deep responses. Toxicities potentially associated with these agents should be known for an optimal management of CLL patients.
近年来，基于创新机制的药物丰富了慢性淋巴细胞白血病（CLL）患者的治疗手段，并在临床实践中广泛应用。这些靶向B细胞受体信号通路和BCL-2抗凋亡蛋白的小分子药物为不适合化疗的患者以及对化疗免疫疗法反应不佳的高危患者提供了新的无化疗选择。尽管如此，使用伊布替尼、idelalisib和维奈托克进行治疗会产生独特的副作用。认识、预防和妥善处理这些特定毒性对于成功治疗至关重要。涵盖领域：本综述的目的是讨论关于CLL中小分子药物的最相关研究，特别关注这些药物新出现的毒性特征以及为每位患者确定最合适治疗方法时应考虑的因素。专家意见：对CLL生物学知识的不断了解已转化为开发出高效且能产生深度反应的靶向药物。为了对CLL患者进行最佳管理，应了解这些药物可能相关的毒性。
