Polverelli Nicola, Hernández-Boluda Juan Carlos, Gagelmann Nico, Gurnari Carmelo, Malagola Michele, Duarte Fernando Barroso, Funke Vaneuza A M, Zerbi Caterina, McLornan Donal P
Unit of Bone Marrow Transplantation and Cellular Therapies - Division of Hematology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.
Department of Hematology, Hospital Clínico Universitario, Valencia, Spain.
Bone Marrow Transplant. 2025 Jan;60(1):10-18. doi: 10.1038/s41409-024-02437-6. Epub 2024 Oct 14.
Significant efforts have been made to effectively select myelofibrosis (MF) patients who can benefit from allogeneic hematopoietic cell transplantation (allo-HCT), the only current cure for MF. The recent EBMT/ELN 2024 recommendations offer valuable guidance for hematologists and transplant physicians. However, several grey areas remain in day-to-day clinical practice regarding the feasibility and optimal preparation for transplantation in patients with this disease. Effective spleen size reduction, often achieved with JAK inhibitors, appears crucial for transplant success. For resistant cases, switching JAK inhibitors, splenectomy, or spleen irradiation may be considered, taking into account patient profiles, treatment availability and center preferences. Managing splanchnic vein thromboses, portal, and pulmonary hypertension is critical as these conditions may affect transplant outcomes. Cytopenias, particularly transfusion-dependent anemia and thrombocytopenia, complicate treatment and impact on outcomes, though new drugs show promise. Comorbidities play a significant role and tools like the Hematopoietic Cell Transplantation-Comorbidity Index (HCT-CI) and frailty assessments are useful for evaluating transplant risks while allowing the implementation of corrective measures. Especially in low- and medium-income countries where access to novel therapies may be challenging, allo-HCT still represents an attractive therapeutic option for MF. Future directions include integrating new therapeutics into the transplant algorithm and leveraging artificial intelligence for more informed risk assessment, highlighting the need for tailored approaches to improve allo-HCT outcomes in such a setting.
为有效筛选出能够从异基因造血细胞移植(allo-HCT)中获益的骨髓纤维化(MF)患者,人们付出了巨大努力,allo-HCT是目前唯一可治愈MF的方法。近期发布的欧洲血液与骨髓移植学会(EBMT)/欧洲白血病网(ELN)2024版建议为血液科医生和移植医生提供了宝贵指导。然而,在日常临床实践中,对于该疾病患者移植的可行性和最佳预处理方案仍存在一些不明确的地方。使用JAK抑制剂常常能有效缩小脾脏大小,这似乎对移植成功至关重要。对于耐药病例,可考虑更换JAK抑制剂、脾切除术或脾脏照射,同时要综合考虑患者情况、治疗可及性和中心偏好。处理内脏静脉血栓形成、门静脉高压和肺动脉高压至关重要,因为这些情况可能影响移植结果。血细胞减少,尤其是依赖输血的贫血和血小板减少,会使治疗复杂化并影响治疗结果,不过新药显示出了一定前景。合并症起着重要作用,造血细胞移植合并症指数(HCT-CI)和虚弱评估等工具有助于评估移植风险,同时便于采取纠正措施。尤其是在中低收入国家,获得新型疗法可能具有挑战性,但allo-HCT对MF患者而言仍是一种有吸引力的治疗选择。未来的方向包括将新疗法纳入移植方案,并利用人工智能进行更明智的风险评估,这凸显了在这种情况下需要采用个性化方法来改善allo-HCT的治疗效果。