Fontana Diletta, Gambacorti-Passerini Carlo, Piazza Rocco
Department of Medicine and Surgery, University of Milano-Bicocca, Monza, Italy.
Hematology and Clinical Research Unit, San Gerardo Hospital, Monza, Italy.
Front Oncol. 2021 Nov 11;11:756348. doi: 10.3389/fonc.2021.756348. eCollection 2021.
Atypical chronic myeloid leukemia is a rare disease whose pathogenesis has long been debated. It currently belongs to the group of myelodysplastic/myeloproliferative disorders. In this review, an overview on the current knowledge about diagnosis, prognosis, and genetics is presented, with a major focus on the recent molecular findings. We describe here the molecular pathogenesis of the disease, focusing on the mechanisms of action of the main mutations as well as on gene expression profiling. We also present the treatment options focusing on emerging targeted therapies.
非典型慢性髓性白血病是一种罕见疾病,其发病机制长期以来一直存在争议。它目前属于骨髓增生异常/骨髓增殖性疾病组。在本综述中,我们概述了目前关于诊断、预后和遗传学的知识,主要侧重于最近的分子研究结果。我们在此描述该疾病的分子发病机制,重点关注主要突变的作用机制以及基因表达谱。我们还介绍了治疗选择,重点是新兴的靶向治疗。