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异基因造血干细胞移植治疗骨髓增生异常综合征和慢性粒-单核细胞白血病:国际专家小组的建议

Allogeneic hematopoietic stem cell transplantation for MDS and CMML: recommendations from an international expert panel.

作者信息

de Witte Theo, Bowen David, Robin Marie, Malcovati Luca, Niederwieser Dietger, Yakoub-Agha Ibrahim, Mufti Ghulam J, Fenaux Pierre, Sanz Guillermo, Martino Rodrigo, Alessandrino Emilio Paolo, Onida Francesco, Symeonidis Argiris, Passweg Jakob, Kobbe Guido, Ganser Arnold, Platzbecker Uwe, Finke Jürgen, van Gelder Michel, van de Loosdrecht Arjan A, Ljungman Per, Stauder Reinhard, Volin Liisa, Deeg H Joachim, Cutler Corey, Saber Wael, Champlin Richard, Giralt Sergio, Anasetti Claudio, Kröger Nicolaus

机构信息

Department of Tumor Immunology, Radboud Institute of Molecular Life Sciences, Radboud University Medical Center, Nijmegen, The Netherlands.

St. James's Institute of Oncology, Leeds, United Kingdom.

出版信息

Blood. 2017 Mar 30;129(13):1753-1762. doi: 10.1182/blood-2016-06-724500. Epub 2017 Jan 17.

Abstract

An international expert panel, active within the European Society for Blood and Marrow Transplantation, European LeukemiaNet, Blood and Marrow Transplant Clinical Trial Group, and the International Myelodysplastic Syndromes Foundation developed recommendations for allogeneic hematopoietic stem cell transplantation (HSCT) in myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML). Disease risks scored according to the revised International Prognostic Scoring System (IPSS-R) and presence of comorbidity graded according to the HCT Comorbidity Index (HCT-CI) were recognized as relevant clinical variables for HSCT eligibility. Fit patients with higher-risk IPSS-R and those with lower-risk IPSS-R with poor-risk genetic features, profound cytopenias, and high transfusion burden are candidates for HSCT. Patients with a very high MDS transplantation risk score, based on combination of advanced age, high HCT-CI, very poor-risk cytogenetic and molecular features, and high IPSS-R score have a low chance of cure with standard HSCT and consideration should be given to treating these patients in investigational studies. Cytoreductive therapy prior to HSCT is advised for patients with ≥10% bone marrow myeloblasts. Evidence from prospective randomized clinical trials does not provide support for specific recommendations on the optimal high intensity conditioning regimen. For patients with contraindications to high-intensity preparative regimens, reduced intensity conditioning should be considered. Optimal timing of HSCT requires careful evaluation of the available effective nontransplant strategies. Prophylactic donor lymphocyte infusion (DLI) strategies are recommended in patients at high risk of relapse after HSCT. Immune modulation by DLI strategies or second HSCT is advised if relapse occurs beyond 6 months after HSCT.

摘要

一个活跃于欧洲血液与骨髓移植学会、欧洲白血病网、血液与骨髓移植临床试验组以及国际骨髓增生异常综合征基金会的国际专家小组,制定了关于骨髓增生异常综合征(MDS)和慢性粒单核细胞白血病(CMML)异基因造血干细胞移植(HSCT)的建议。根据修订后的国际预后评分系统(IPSS-R)对疾病风险进行评分,以及根据造血干细胞移植合并症指数(HCT-CI)对合并症进行分级,被认为是HSCT资格的相关临床变量。符合条件的高危IPSS-R患者以及具有不良风险遗传特征、严重血细胞减少和高输血负担的低危IPSS-R患者是HSCT的候选者。基于高龄、高HCT-CI、极差风险的细胞遗传学和分子特征以及高IPSS-R评分组合的MDS移植风险非常高的患者,采用标准HSCT治愈的机会较低,应考虑在研究性研究中治疗这些患者。对于骨髓原始细胞≥10%的患者,建议在HSCT前进行细胞减灭治疗。前瞻性随机临床试验的证据不支持关于最佳高强度预处理方案的具体建议。对于高强度预处理方案有禁忌证的患者,应考虑采用降低强度的预处理。HSCT的最佳时机需要仔细评估可用的有效非移植策略。建议对HSCT后复发风险高的患者采用预防性供体淋巴细胞输注(DLI)策略。如果在HSCT后6个月以上发生复发,建议采用DLI策略或第二次HSCT进行免疫调节。

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