Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
Best Pract Res Clin Haematol. 2022 Jun;35(2):101370. doi: 10.1016/j.beha.2022.101370. Epub 2022 Aug 5.
Polycythemia vera (PV) is characterized by clonal proliferation of a hematopoietic stem cell leading to erythrocytosis. Patients with PV have significantly higher morbidity and mortality compared to the general population due to increased risk of thrombosis, hemorrhage, and well-characterized microvascular and constitutional symptoms. There is also a propensity to transform to myelofibrosis and to an aggressive form of acute leukemia, further increasing morbidity and mortality. Current management is aimed at reducing the risk of thromboembolic events and improving symptom burden; however, none of the existing therapies have proven the ability to deplete the underlying malignant clone, or definitively reduce the risk of disease, progression leaving a large area of unmet need. In this review, we highlight the pathophysiology of PV, current management and limitations therein. We propose highly debated clinical practices that require further investigation. We conclude by discussing therapies in development and how these may fill unmet needs and be incorporated into the future PV treatment paradigm.
真性红细胞增多症（PV）的特征是造血干细胞的克隆性增殖，导致红细胞增多。与普通人群相比，PV 患者由于血栓形成、出血以及特征性的微血管和全身症状的风险增加，发病率和死亡率显著更高。PV 还容易向骨髓纤维化和侵袭性急性白血病转化，进一步增加发病率和死亡率。目前的治疗方法旨在降低血栓栓塞事件的风险和改善症状负担；然而，现有的治疗方法都没有证明能够消除潜在的恶性克隆，或明确降低疾病进展的风险，因此存在大量未满足的需求。在这篇综述中，我们强调了 PV 的病理生理学、当前的治疗方法及其局限性。我们提出了一些存在争议的临床实践，这些实践需要进一步研究。最后，我们讨论了正在开发的治疗方法，以及这些方法如何填补未满足的需求，并纳入未来的 PV 治疗模式。
