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骨髓增生异常综合征:诊断与治疗

The myelodysplastic syndromes: diagnosis and treatment.

作者信息

Steensma David P, Bennett John M

机构信息

Division of Hematology, Mayo Clinic College of Medicine, 200 First St SW, Rochester, MN 55905, USA.

出版信息

Mayo Clin Proc. 2006 Jan;81(1):104-30. doi: 10.4065/81.1.104.

Abstract

The myelodysplastic syndromes (MDSs) are common, acquired, clinically challenging hematologic conditions that are characterized by bone marrow failure and a risk of progression to acute leukemia. These disorders can arise de novo, especially in elderly patients or, less often, as a consequence of prior chemotherapy or radiotherapy for an unrelated disease. The MDS classification systems were revised recently and updated. These refined classification and prognostic schemes help stratify patients by their risk of leukemia progression and death; this knowledge can help clinicians select appropriate therapy. Although many treatments for MDS have been proposed and evaluated, at present, only hematopoietic stem cell transplantation offers any real hope for cure, and no available therapy beyond general supportive care offers benefit to more than a minority of patients. However, recent clinical trials enrolling patients with MDS have reported encouraging results with use of newer drugs, including lenalidomide, decitabine, and darbepoetin alfa. Other exciting treatment regimens are being tested. Here, we present a contemporary, practical clinical approach to the diagnosis and risk-stratified treatment of MDS. We review when to suspect MDS, detail how to evaluate patients who may have a form of the condition, explain key features of treatments that are currently available in the United States, and summarize a general, common-sense therapeutic approach to patients with MDS.

摘要

骨髓增生异常综合征(MDSs)是常见的、后天获得的、临床治疗颇具挑战的血液系统疾病,其特征为骨髓衰竭以及进展为急性白血病的风险。这些病症可原发出现,尤其在老年患者中,或较少见地,作为先前针对无关疾病进行化疗或放疗的结果。MDS分类系统最近进行了修订和更新。这些完善的分类和预后方案有助于根据患者白血病进展和死亡风险进行分层;这一知识可帮助临床医生选择合适的治疗方法。尽管已提出并评估了许多针对MDS的治疗方法,但目前只有造血干细胞移植提供了治愈的真正希望,除了一般支持性护理外,没有任何现有疗法能使超过少数患者受益。然而,最近纳入MDS患者的临床试验报告了使用包括来那度胺、地西他滨和阿法达贝泊汀在内的新药取得的令人鼓舞的结果。其他令人兴奋的治疗方案正在进行测试。在此,我们提出一种针对MDS诊断和风险分层治疗的当代实用临床方法。我们回顾何时怀疑MDS,详细说明如何评估可能患有某种形式该病症的患者,解释美国目前可用治疗方法的关键特征,并总结针对MDS患者的一般常识性治疗方法。

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