Department of Haematology, University College Hospital, London, UK.
Unit of Blood Diseases and Stem Cell Transplantation, University of Brescia, Brescia, Italy.
Br J Haematol. 2023 Oct;203(2):169-181. doi: 10.1111/bjh.19010. Epub 2023 Aug 1.
Management approaches for accelerated and blast phase myeloproliferative neoplasms remain challenging for clinicians and patients alike. Despite many therapeutic advances, outcomes for those patients who are not allogeneic haematopoietic cell transplant eligible remain, in general, very poor. Estimated survival rates for such blast phase patients is frequently reported as less than 6 months. No specific immunological, genomic or clinicopathological signature currently exists that accurately predicts the risk and timing of transformation, which frequently induces a high degree of anxiety among patients and clinicians alike. Within this review article, we provide an up-to-date summary of current understanding of the underlying pathogenesis of accelerated and blast phase disease and discuss current therapeutic approaches and realistic outcomes. Finally, we discuss how the horizon may look with the introduction of more novel agents into the clinical arena.
对于临床医生和患者来说，加速期和急变期骨髓增殖性肿瘤的治疗方法仍然具有挑战性。尽管有许多治疗进展，但对于不符合异基因造血细胞移植条件的患者，总体预后仍然非常差。此类急变期患者的估计生存率通常报道不足 6 个月。目前尚无能够准确预测转化风险和时间的特定免疫、基因组或临床病理特征，这常常会引起患者和临床医生的高度焦虑。在本文综述中，我们对加速期和急变期疾病的发病机制的现有认识进行了最新总结，并讨论了目前的治疗方法和现实结果。最后，我们讨论了随着更多新型药物进入临床领域，前景可能会如何。
