Division of Internal Medicine, Icahn School of Medicine at Mount Sinai, New York, NY, 10029, USA.
Division of Hematology and Medical Oncology, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, One Gustave L Levy Place, Box 1079, New York, NY, 10029, USA.
Ann Hematol. 2022 May;101(5):935-951. doi: 10.1007/s00277-022-04826-7. Epub 2022 Mar 28.
Thrombotic events are a distinctive feature of the myeloproliferative neoplasms (MPNs) polycythemia vera (PV) and essential thrombocythemia (ET). Patients with these MPNs may also experience a poor quality of life secondary to symptom burden, as well as progression of disease to acute leukemia or myelofibrosis. Over the years, various risk stratification methods have evolved in order to attempt to predict thrombotic risk, which is the largest contributor of morbidity and mortality in these patients. More than half of PV and ET patients are low- or intermediate-risk disease status at the time of diagnosis. While therapeutic development is presently focused on high-risk patients, there is a paucity of therapies, outside of aspirin and therapeutic phlebotomy, which can reduce the thrombotic risk or delay disease progression in low-risk patients. In this review, we first describe the various complications that patients with PV and ET experience, and then detail our evolving understanding of risk stratification in these diseases. We then highlight the available evidence on the management of low-risk PV and ET and include a description of novel therapies currently under investigation in this space. We conclude with recommendations for future directions to advance our understanding and improve the treatment of low-risk PV and ET.
血栓事件是骨髓增生性肿瘤（MPN）真性红细胞增多症（PV）和特发性血小板增多症（ET）的一个显著特征。这些 MPN 患者可能由于症状负担，以及疾病进展为急性白血病或骨髓纤维化，导致生活质量下降。多年来，已经开发出各种风险分层方法，以尝试预测血栓形成风险，这是这些患者发病率和死亡率的最大贡献因素。超过一半的 PV 和 ET 患者在诊断时处于低危或中危疾病状态。虽然目前的治疗重点是高危患者，但除了阿司匹林和治疗性放血外，几乎没有其他治疗方法可以降低低危患者的血栓形成风险或延缓疾病进展。在这篇综述中，我们首先描述了 PV 和 ET 患者经历的各种并发症，然后详细介绍了我们对这些疾病风险分层的不断发展的认识。然后，我们重点介绍了低危 PV 和 ET 的现有管理证据，并描述了该领域目前正在研究的新型治疗方法。最后，我们为未来的研究方向提出了建议，以提高我们对低危 PV 和 ET 的认识并改善其治疗效果。
