Division of Cancer Medicine, University of Texas, MD Anderson Cancer Center, Houston, Texas, USA.
Department of Leukemia, University of Texas, MD Anderson Cancer Center, Houston, Texas, USA.
Br J Haematol. 2024 Jul;205(1):48-60. doi: 10.1111/bjh.19557. Epub 2024 Jun 10.
Myeloproliferative neoplasms (MPN) are characterized by a clonal proliferation of myeloid lineage cells within the bone marrow. The classical BCR-ABL negative MPNs are comprised of polycythaemia vera, essential thrombocythaemia and primary myelofibrosis. Historically, the majority of MPNs are diagnosed in adults older than 60 years of age; however, in recent years, there has been recognition of MPNs in the adolescent and young adult (AYA) population. AYAs with MPN, typically defined as between the ages of 15 and 39 years old, may comprise up to 20% of patients diagnosed with MPN. They demonstrate unique patterns of driver mutations and thrombotic events and remain at risk for progression to more aggressive disease states. Given the likely long length of time they will live with their disease, there is a significant unmet need in identifying well-tolerated and effective treatment options for these patients, particularly with the advent of disease modification. In this review, we provide a comprehensive overview of the clinical features, disease course and management of AYA patients with MPN and, in doing so, highlight key characteristics that distinguish them from their older counterparts.
骨髓增殖性肿瘤(MPN)的特征是骨髓中髓系细胞的克隆性增殖。经典的 BCR-ABL 阴性 MPN 包括真性红细胞增多症、原发性血小板增多症和原发性骨髓纤维化。历史上,大多数 MPN 发生于 60 岁以上的成年人;然而,近年来,青少年和年轻成人(AYA)中也发现了 MPN。AYA 中的 MPN 通常定义为年龄在 15 至 39 岁之间,可能占 MPN 患者的 20%。他们表现出独特的驱动突变和血栓形成事件模式,并且仍然存在向更具侵袭性疾病状态发展的风险。鉴于他们可能长期患有疾病,因此对于这些患者来说,确定耐受良好和有效的治疗方案具有重要意义,尤其是在疾病修饰治疗出现之后。在这篇综述中,我们全面概述了 AYA 中 MPN 患者的临床特征、疾病过程和管理,并在其中强调了使他们与老年患者区分开来的关键特征。