Bejar Rafael, Tiu Ramon V, Sekeres Mikkael A, Komrokji Rami S
From the Division of Hematology and Oncology, Moores Cancer Center, University of California, San Diego; Cleveland Clinic Taussig Cancer Institute, Cleveland, OH; H. Lee Moffitt Cancer Center & Research Institute, Tampa, FL.
Am Soc Clin Oncol Educ Book. 2013. doi: 10.1200/EdBook_AM.2013.33.e256.
Significant advances have been achieved in understanding and treating myelodysplastic syndromes (MDS) in the past decade. For the first time, three drugs were approved specifically for this disease. Novel sequencing techniques have expanded our understanding of the molecular basis of MDS. Several clinically significant recurrent gene mutations have been identified. The classification and risk stratification of MDS continues to evolve in light of such advances. However, treatment options remain limited and novel therapeutic strategies are needed. In this review we address key questions for management of MDS. How do we better classify and risk stratify MDS, tailoring treatment accordingly? How do we diagnose and manage the challenging group of patients with MDS/myeloproliferative neoplasms (MPN) overlap? And finally, what is on the horizon for novel therapies?
在过去十年中，骨髓增生异常综合征（MDS）的诊断和治疗取得了重大进展。首次有三种药物被专门批准用于治疗这种疾病。新型测序技术拓展了我们对MDS分子基础的认识。已鉴定出几种具有临床意义的复发性基因突变。鉴于这些进展，MDS的分类和风险分层仍在不断发展。然而，治疗选择仍然有限，需要新的治疗策略。在这篇综述中，我们探讨了MDS管理的关键问题。我们如何更好地对MDS进行分类和风险分层，并据此调整治疗方案？我们如何诊断和管理具有挑战性的MDS/骨髓增殖性肿瘤（MPN）重叠患者群体？最后，新疗法的前景如何？
