Kröger Nicolaus, Rathje Kristin, Gagelmann Nico
Department of Stem Cell Transplantation, University Medical Center Hamburg, Hamburg, Germany.
Adv Exp Med Biol. 2025;1475:167-175. doi: 10.1007/978-3-031-84988-6_9.
Allogeneic hematopoietic stem cell transplantation (HSCT) is a potentially curative treatment option for patients with myelofibrosis. Because of inherent risk of the procedure, optimal timing in the course of the disease is crucial in order to balance risk and benefit. Besides optimal timing, reducing spleen size-a hallmark of the disease-is needed to ensure timely and sufficient engraftment after HSCT.Disease- and transplant-specific risk scores have been developed to predict the outcome of myelofibrosis with (MTSS) and without transplantation (DIPSS, MIPSS, and MYSEC), and current recommendation is to offer HSCT in primary or secondary myelofibrosis patients up to 70 years of age with intermediate-2 or high-risk DIPSS or MYSEC score or high risk according to MIPSS70 and a low or intermediate risk according to MTSS. Regarding the spleen size, the current European recommendation is for patients with splenomegaly greater than 5 cm below the lower costal margin or splenomegaly-related symptoms to receive a spleen-directed treatment, ideally with JAK-inhibitor and in case of resistance to use second-generation JAK inhibitor or spleen irradiation close to the transplant or splenectomy.
异基因造血干细胞移植（HSCT）是骨髓纤维化患者一种潜在的治愈性治疗选择。由于该手术存在固有风险，在疾病进程中选择最佳时机对于平衡风险和获益至关重要。除了最佳时机外，减小脾脏大小（该病的一个标志）对于确保HSCT后及时且充分的植入是必要的。已经开发了疾病特异性和移植特异性风险评分来预测接受移植（MTSS）和未接受移植（DIPSS、MIPSS和MYSEC）的骨髓纤维化患者的预后，目前的建议是为年龄在70岁及以下、DIPSS或MYSEC评分为中危2或高危、或根据MIPSS70为高危且根据MTSS为低危或中危的原发性或继发性骨髓纤维化患者提供HSCT。关于脾脏大小，目前欧洲的建议是，对于脾肿大超过肋缘下5 cm或有脾肿大相关症状的患者，应接受针对脾脏的治疗，理想情况下使用JAK抑制剂，若耐药则在移植前使用第二代JAK抑制剂或进行脾脏照射或脾切除术。
