特定骨髓纤维化人群移植资格评估过程中的 "灰色地带 "和挑战:代表 EBMT 慢性恶性肿瘤工作组的观点。

IF 4.5 2区 医学 Q1 HEMATOLOGY Bone Marrow Transplantation Pub Date : 2024-10-14 DOI:10.1038/s41409-024-02437-6
Nicola Polverelli, Juan Carlos Hernández-Boluda, Nico Gagelmann, Carmelo Gurnari, Michele Malagola, Fernando Barroso Duarte, Vaneuza A M Funke, Caterina Zerbi, Donal P McLornan
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引用次数: 0

摘要

为有效选择骨髓纤维化(MF)患者,使其从异基因造血细胞移植(allo-HCT)中获益,我们付出了巨大的努力,异基因造血细胞移植是目前唯一能治愈骨髓纤维化的方法。最近的 EBMT/ELN 2024 建议为血液科医生和移植医生提供了宝贵的指导。然而,在日常临床实践中,关于该病患者移植的可行性和最佳准备工作仍存在一些灰色地带。有效缩小脾脏(通常使用 JAK 抑制剂)似乎是移植成功的关键。对于耐药病例,可考虑更换 JAK 抑制剂、脾脏切除术或脾脏照射,同时考虑到患者情况、治疗方法的可用性和中心的偏好。处理脾静脉血栓、门静脉高压和肺动脉高压至关重要,因为这些疾病可能会影响移植结果。细胞减少症,尤其是输血依赖性贫血和血小板减少症,会使治疗复杂化并影响疗效,尽管新药显示了前景。合并症起着重要作用,造血细胞移植合并症指数(HCT-CI)和虚弱评估等工具可用于评估移植风险,同时采取纠正措施。特别是在中低收入国家,获得新疗法可能具有挑战性,异体造血干细胞移植仍然是治疗中风的一种有吸引力的选择。未来的发展方向包括将新疗法整合到移植算法中,利用人工智能进行更明智的风险评估,强调在这种情况下需要量身定制的方法来改善allo-HCT的疗效。
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Navigating 'grey areas' and challenges during evaluation of transplant eligibility in specific myelofibrosis populations: a perspective on behalf of the Chronic Malignancies Working Party of the EBMT.

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.

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来源期刊
Bone Marrow Transplantation
Bone Marrow Transplantation 医学-免疫学
CiteScore
8.40
自引率
8.30%
发文量
337
审稿时长
6 months
期刊介绍: Bone Marrow Transplantation publishes high quality, peer reviewed original research that addresses all aspects of basic biology and clinical use of haemopoietic stem cell transplantation. The broad scope of the journal thus encompasses topics such as stem cell biology, e.g., kinetics and cytokine control, transplantation immunology e.g., HLA and matching techniques, translational research, and clinical results of specific transplant protocols. Bone Marrow Transplantation publishes 24 issues a year.
期刊最新文献
Utilization of hematopoietic cell transplantation and cellular therapy technology in Europe and associated Countries. Using the 2022 activity survey data to correlate with economic and demographic factors. A report from the EBMT. The BLIND study: blinatumomab and DLI approach for management of B-ALL relapse after allogeneic stem cell transplantation. A multicentric Italian experience. Measurable residual disease testing and allogeneic hematopoietic cell transplantation for AML: adapting Pre-MEASURE to clinical practice. The role of daratumumab in complications post-allogeneic hematopoietic stem cell transplantation: a single-center prospective study on PRCA and AIHA. High-dose chemotherapy with autologous haematopoietic stem cell transplantation in patients with isolated vitreoretinal lymphoma: a LOC network study.
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