Kobbe Guido, Schroeder Thomas, Haas Rainer, Germing Ulrich
a Medical Faculty, Department of Hematology, Oncology and Clinical Immunology , Heinrich Heine University, University Hospital Düsseldorf , Düsseldorf , Germany.
Expert Rev Hematol. 2018 May;11(5):411-422. doi: 10.1080/17474086.2018.1452611. Epub 2018 Mar 22.
Allogeneic blood stem cell transplantation (aBSCT) still is the only curative therapy for patients with myelodysplastic syndromes. While it carries the hope for cure for some patients, it may result in severe toxicity and death from complications or recurrent disease in others. Recent developments have improved patient and donor selection as well as technical aspects of the transplant procedure and post-transplant care, including early detection and treatment of relapse. Areas covered: This review will discuss current stratification tools to identify suitable patients, donors and transplant techniques. In addition, it will cover the prognostic and predictive value of cytogenetics and somatic mutations and elucidate strategies for minimal residual disease detection as well as prophylactic and preemptive treatment of recurrent disease. Expert commentary: aBSCT will continue to be the most powerful curative treatment option for patients with MDS. Advances in the understanding of disease biology will allow to incorporate molecular alterations in current prognostic scores, tracking of specific mutations during therapy and the use of novel, targeted therapies to augment the graft versus leukemia effect.
异基因造血干细胞移植（aBSCT）仍然是骨髓增生异常综合征患者唯一的治愈性疗法。虽然它为一些患者带来了治愈的希望，但对另一些患者而言，它可能导致严重的毒性反应以及因并发症或疾病复发而死亡。近期的进展改善了患者和供体的选择以及移植程序和移植后护理的技术层面，包括复发的早期检测和治疗。涵盖领域：本综述将讨论当前用于识别合适患者、供体和移植技术的分层工具。此外，它将涵盖细胞遗传学和体细胞突变的预后和预测价值，并阐明微小残留病检测策略以及复发性疾病的预防性和抢先治疗策略。专家评论：aBSCT将继续是骨髓增生异常综合征患者最有效的治愈性治疗选择。对疾病生物学理解的进展将允许把分子改变纳入当前的预后评分中，在治疗期间追踪特定突变，并使用新型靶向疗法增强移植物抗白血病效应。
