Cheson B D
National Cancer Institute, Bethesda, Maryland.
Ann Intern Med. 1990 Jun 15;112(12):932-41. doi: 10.7326/0003-4819-112-12-932.
To review the current therapeutic options for patients with the myelodysplastic syndromes.
Studies reported between 1968 and September 1989 were identified through computer searches using MEDLINE and through extensive searching of bibliographies of identified articles.
All articles, abstracts, and reviews were evaluated, and those that contained therapeutic data were analyzed for response rates, survival, and toxicity.
Therapies for the myelodysplastic syndromes have included hormones, chemotherapy, bone marrow transplantation, and differentiating agents, including hematopoietic growth factors. With the exception of bone marrow transplantation, none is curative or increases survival. Hematopoietic growth factors are of interest for clinical trials, because they increase the number of neutrophils and, occasionally, the number of platelets in patients with the myelodysplastic syndromes. Nonetheless, hematopoietic growth factors are not without toxicity; most notably, they are associated with a risk for acceleration to acute myeloid leukemia, and their effect on survival remains unknown.
Standard therapy for the myelodysplastic syndromes is supportive care. When the disease progresses, patients with aggressive histologic or other poor-risk features should be considered for aggressive chemotherapy, with or without growth factors, or bone marrow transplantation. Patients with good prognostic features are candidates for therapy with growth factors or other potential differentiating agents. Carefully designed and conducted clinical trials that incorporate correlative laboratory science are essential to developing a more rational approach to therapy.
回顾目前针对骨髓增生异常综合征患者的治疗选择。
通过使用MEDLINE进行计算机检索以及广泛查阅已识别文章的参考文献，找出1968年至1989年9月间报道的研究。
对所有文章、摘要和综述进行评估，对包含治疗数据的文献分析其缓解率、生存率和毒性。
骨髓增生异常综合征的治疗方法包括激素、化疗、骨髓移植以及分化诱导剂，包括造血生长因子。除骨髓移植外，这些方法均无法治愈疾病或提高生存率。造血生长因子因可增加骨髓增生异常综合征患者的中性粒细胞数量，偶尔还能增加血小板数量，而受到临床试验的关注。尽管如此，造血生长因子并非没有毒性；最显著的是，它们与疾病进展为急性髓系白血病的风险相关，且其对生存率的影响尚不清楚。
骨髓增生异常综合征的标准治疗是支持性治疗。当疾病进展时，具有侵袭性组织学特征或其他高危特征的患者应考虑接受强化化疗，可联合或不联合生长因子，或进行骨髓移植。具有良好预后特征的患者可选用生长因子或其他潜在的分化诱导剂进行治疗。精心设计并实施的、纳入相关实验室研究的临床试验对于制定更合理的治疗方法至关重要。
