Department of Bone Marrow Transplantation and Haematology, St Vincent's Hospital, Darlinghurst, New South Wales, Australia.
School of Clinical Medicine, UNSW Medicine & Health, Kensington, New South Wales, Australia.
Am J Hematol. 2022 Nov;97(11):1464-1477. doi: 10.1002/ajh.26654. Epub 2022 Jul 19.
Myelofibrosis (MF) is a clonal myeloproliferative neoplasm characterized by inflammation, marrow fibrosis, and an inherent risk of blastic transformation. Hematopoietic allogeneic stem cell transplant is the only potentially curative therapy for this disease, however, survival gains observed for other transplant indications over the past two decades have not been realized for MF. The role of transplantation may also evolve with the use of novel targeted agents. The chronic inflammatory state associated with MF necessitates pretransplantation assessment of end-organ function. Applying the transplant methodology employed for other myeloid disorders to patients with MF fails to acknowledge differences in the underlying disease pathophysiology. Limited understanding of the causes of poor transplant outcomes in this cohort has prevented refinement of transplant eligibility criteria in MF. There is increasing evidence of heterogeneity in molecular disease grade, beyond the clinical manifestations which have traditionally guided transplant timing. Exploring the physiological consequences of disease chronicity unique to MF, acknowledging the heterogeneity in disease grade, and using advanced prognostic models, molecular diagnostics and other organ function diagnostic tools, we present an innovative review of strategies with the potential to improve transplant outcomes in this disease. Larger, prospective studies which consider the impact of molecular-based disease grade are needed for MF transplantation.
骨髓纤维化（MF）是一种克隆性骨髓增生性肿瘤，其特征为炎症、骨髓纤维化和固有向白血病转化的风险。同种异体造血干细胞移植是该疾病唯一潜在的治愈性治疗方法，然而，在过去二十年中，其他移植适应证的生存获益并未在 MF 中实现。随着新型靶向药物的使用，移植的作用也可能发生演变。MF 相关的慢性炎症状态需要在移植前评估终末器官功能。将用于其他髓系疾病的移植方法应用于 MF 患者，无法承认潜在疾病病理生理学的差异。由于对该队列中移植不良结果的原因了解有限，因此无法改进 MF 中的移植资格标准。越来越多的证据表明，在传统指导移植时机的临床表现之外，分子疾病分级存在异质性。探索 MF 特有的疾病慢性的生理后果，承认疾病分级的异质性，并使用先进的预后模型、分子诊断和其他器官功能诊断工具，我们提出了一种具有改善该疾病移植结果潜力的创新策略综述。MF 移植需要更大的、前瞻性的研究，这些研究需要考虑基于分子的疾病分级的影响。
