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异基因造血干细胞移植在原发性骨髓纤维化中的应用

The Use of Allogeneic Hematopoietic Stem Cell Transplantation in Primary Myelofibrosis.

作者信息

Wolfe Heather R, Horwitz Mitchell E, Rein Lindsay A M

机构信息

Department of Medicine, Duke University Medical Center, Durham, NC 27707, USA.

Division of Hematologic Malignancies and Cellular Therapy, Department of Medicine, Duke University Medical Center, Durham, NC 27707, USA.

出版信息

J Pers Med. 2022 Apr 2;12(4):571. doi: 10.3390/jpm12040571.

Abstract

Primary myelofibrosis (PMF) is a BCR-ABL1 negative myeloproliferative neoplasm characterized by clonal proliferation of myeloid cells. This leads to reactive bone marrow fibrosis, ultimately resulting in progressive marrow failure, hepatosplenomegaly, and extramedullary hematopoiesis. PMF is considered the most aggressive of the BCR-ABL1 negative myeloproliferative neoplasms with the least favorable prognosis. Constitutional symptoms are common, which can impact an individual's quality of life and leukemic transformation remains an important cause of death in PMF patients. The development of the Janus kinase 2 (JAK2) inhibitors have provided a good option for management of PMF-related symptoms. Unfortunately, these agents have not been shown to improve overall survival or significantly alter the course of disease. Allogenic hematopoietic stem cell transplantation (allo-HSCT) remains the only curative treatment option in PMF. However, allo-HSCT is associated with significant treatment-related morbidity and mortality and has historically been reserved for younger, high-risk patients. This review examines patient, disease, and transplant-specific factors which may impact transplant-related outcomes in PMF. Through the vast improvements in donor selection, conditioning regimens, and post-transplant care, allo-HSCT may provide a safe and effective curative option for a broader range of PMF patients in the future.

摘要

原发性骨髓纤维化(PMF)是一种BCR-ABL1阴性骨髓增殖性肿瘤,其特征为髓系细胞的克隆性增殖。这会导致反应性骨髓纤维化,最终导致进行性骨髓衰竭、肝脾肿大和髓外造血。PMF被认为是BCR-ABL1阴性骨髓增殖性肿瘤中侵袭性最强、预后最差的类型。全身症状很常见,会影响个体的生活质量,白血病转化仍然是PMF患者的一个重要死亡原因。Janus激酶2(JAK2)抑制剂的出现为PMF相关症状的管理提供了一个很好的选择。不幸的是,这些药物尚未显示能改善总生存期或显著改变疾病进程。异基因造血干细胞移植(allo-HSCT)仍然是PMF唯一的治愈性治疗选择。然而,allo-HSCT与显著的治疗相关发病率和死亡率相关,并且在历史上一直仅用于年轻的高危患者。本综述探讨了可能影响PMF患者移植相关结局的患者、疾病和移植特异性因素。通过供体选择、预处理方案和移植后护理方面的巨大改进,allo-HSCT未来可能为更广泛的PMF患者提供一种安全有效的治愈选择。

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