骨髓增生异常综合征38例分析

骨髓增生异常综合征 (ＭＤＳ)是一组恶性克隆性干 祖细胞性疾病 ,其主要特征为无效造血所致的难治性血细胞减少和极易发展为急性白血病。我院自1982年至今诊治 38例现介绍如下。1　临床资料1 1　年龄　 38例中男 2 3例 ,女 15例 ,男∶女为 1.53∶1;发病年龄 3～ 81岁 ,平均 37.4岁 ,其中 3～ 17岁 2例( 5% ) ,18～ 50岁 2 9例 ( 76% ) ,51～ 81岁 7例 ( 19% )。1 2　诊断标准　根据 1982年ＦＡＢ提出的关于骨髓增生异常综合征分型及诊断标准的建议和 1986年 9月天津学术会议提出的新的分型标准的建议 ,对我院 38例病例进行了重新的复查与分…     

骨髓增生异常综合征核型分析

<正>骨髓增生异常综合征(MDS,myelodysplastic syndrome)是一组源于造血干细胞的恶性克隆性疾病,具有发展成急性白血病的高危倾向。目前MDS的诊断主要是按照FAB诊断分型标准[1]。但是,MDS具有高度的异质性,表现复杂,不典型的MDS用FAB形态学分型常难以作出准确诊断。有研究表明[2],染色体核型异常是MDS的遗传学特     

骨髓增生异常综合征24例报告

骨髓增生异常综合征(MDS)是一种源于不同阶段造血祖细胞增殖、分化功能异常的获得性干细胞疾病。其特点为不同程度的红细胞、白细胞和血小板减少,骨髓增生亢进,并有三系血细胞或任两系血细胞的病态造血,如可有巨大血小板、有核红细胞及粒系核浆发育不平衡等,在疾病过程中可以转化为白血病。现将我院诊治的24例MDS报告如下。 临床资料 1.一般资料24例MDS均系我院自1982年至1996年的住院患者,其中男、女各12例,男:女为1:1。年龄15～79岁,平均47.6岁,其中40岁以上者居多共17例(占70.8％)。诊断依据按全国第一     

低增生性骨髓增生异常综合征在临床实验中的鉴别诊断

低增生性骨髓增生异常综合征（hypoplasia myelodysplastic syndrome，HMDS）是骨髓增生异常综合征中的一种特殊类型，以低骨髓细胞容积和病态造血为特征，临床上与再生障碍性贫血（aplastic anemic，AA）较难区别。本文就HMDS与AA实验室检查的异同点进行了探讨，现报告如下：     

19例骨髓增生异常综合征血象骨髓象分析

骨髓增生异常综合征(MDS)，是因骨髓干细胞分化障碍而致的一种克隆性恶性血液病。发病率有逐年增加的趋势。为提高诊断率，现将作者收集的19例MDS血象骨髓象分析如下。     

66例成人骨髓增生异常综合征的诊断分析

目的进一步认识骨髓增生异常综合征（MDS）的临床和实验室诊断特点,提高诊断率。方法回顾性分析了本院收治的66例成人MDS患者的临床和实验室检查资料。结果66例患者中,3系均减少者36例、2系减少者21例,1系减少者9例。其中WBC增加者4例,PLT增加者6例,WBC和PLT均增加者1例。骨髓细胞学检查示增生活跃为51例,均有不同程度的病态造血表现,增生减低型MDS骨髓病理学较骨髓细胞学检测诊断率高。染色体核型异常检出4例（25％）,以RAEB居多。结论完善细胞学、骨髓涂片、病理学及遗传学的检查,对MDS的诊断、治疗方案和预后评估均具有重要的作用。     

细胞形态学观察在骨髓增生异常综合征诊断中价值

目的探讨骨髓增生异常综合征（MDS）细胞形态学病态造血特点,以寻求诊断MDS的价值。方法以WHO MDS分类标准为诊断金标准,收集2007年3月1日至2012年3月6日期间,诊断的MDS患者165例。非克隆性疾病患者165例作为对照组。分析骨髓与血片细胞学检查中病态造血特征在克隆性和非克隆性疾病中的诊断价值。结果 MDS病态造血形态学诊断的主要依据：粒系Auer小体、核出芽、微核;红系核出芽;外周血片中出现巨核细胞、原粒细胞或早幼红细胞。结论细胞形态学是诊断MDS的基础,但也存在一定的局限性,尤其早期MDS细胞形态学改变不典型时,恶性克隆处于非显性状态,表达特征亦不明显,需要结合其他检测手段,以便早期诊断和治疗。     

小巨核细胞在骨髓增生异常综合征中的诊断意义

目的:了解小巨核细胞(SMK)在骨髓增生异常综合征(MDS)中的诊断价值。方法:66例MDS患者的骨髓片,应用碱性磷酸酶-链霉卵白素组织化学染色法(SAP法)进行抗GPⅡbⅢa单抗(CD41a)染色,观察骨髓涂片的小巨核细胞的数量和形态变化。结果:SAP法染色的巨核细胞数量与瑞氏-吉姆萨染色比较差异有统计学意义(P<0.01),前者明显优于后者;MDS组、巨幼细胞贫血(MegA)组和对照组在巨核细胞类型和淋巴样SMK阳性率方面比较,差异有统计学意义(P<0.05)。结论:SAP法染色检查能够方便、快速地识别SMK;淋巴样SMK的检出和数量变化对MDS的鉴别诊断有重要意义,为早期诊断提供了可靠的依据。     

Arsenic trioxide for the treatment of myelodysplastic syndromes

The impressive activity of arsenic trioxide in acute promyelocytic leukaemia (APL) has renewed the interest in this old compound. Arsenic trioxide targets the sulfhydryl groups present in many proteins involved in oncogenesis and has a broad spectrum of biological activities. This article will review the mechanisms of action of the drug and their relevance to the treatment of myelodysplastic syndrome (MDS), a disease for which no standard treatment currently exists. The early clinical experience has confirmed the activity of arsenic trioxide in MDS. The preliminary results of ongoing Phase II studies conducted in patients with MDS suggest that arsenic trioxide produces haematological improvement including durable transfusion independence in ～ 30% of patients. The current data are presented and discussed in this review.     

The role of azacitidine in the treatment of myelodysplastic syndromes

Myelodysplastic syndromes (MDS) are a heterogeneous group of hematopoietic disorders characterized by ineffective hematopoiesis and potential transformation to acute myeloid leukemia. Supportive care including transfusions and growth factors remained the mainstay of treatment for decades; however, further understanding of the biology behind these diseases led to the investigation of novel agents. As hypermethylation of tumor suppressor genes, such as p15, was believed to play a key role in the pathogenesis of these diseases, hypomethylating agents were investigated. Azacitidine is one of two hypomethylating agents used in the treatment of MDS, and the first approved by US FDA. In preclinical studies, azacitidine demonstrated hypomethylating/differentiating activity with low concentration, whereas high concentration was associated with cytotoxic effects. In clinical trials, azacitidine not only improved the cytopenias associated with MDS but also delayed leukemic transformation, improved quality of life and improved overall survival in many patients so treated. Azacitidine was the first agent noted to change the natural history of the disease. Further studies are underway evaluating the role of azacitidine pre- and post-transplantation, in combination with other agents, as well as in treatment of acute myeloid leukemia patients who are not good candidates for intensive chemotherapy. Azacitidine is also likely to be studied in the treatment of other malignant conditions. Although both subcutaneous and intravenous administrations have been approved, oral azacitidine is presently under investigation.     

Pharmacotherapy of myelodysplastic syndromes

Importance of the field: Despite the remarkable progress in the treatment of patients with myelodysplastic syndromes (MDS) in the past decade, response to the hypomethylating agents azacitidine and decitabine in non-del(5q) MDS patients remains at ～ 50%, leaving half of patients needing treatment with essentially no options. As biologic insight into the molecular pathways that account for disease evolution and clinical heterogeneity is expanded, the arsenal of potential drugs that may elicit significant response is also increasing. One of the greatest challenges for the treating physician is to decide when to initiate therapy and which therapy (approved drug or newer agents still in clinical trial) is likely to be the most beneficial. While there is no single answer to these issues, there are several approaches that may be considered, and these are addressed in this review.

Areas covered in this review: This review examines the clinical outcomes of the FDA-approved drugs as well as of the promising new therapies that are in current clinical trials.

What the reader will gain: The clinician now has multiple treatment options for patients with MDS. It is important to consider multiple factors before initiating therapy with disease-modifying drugs. This review presents some of the decision-making approaches that are in practice at present.

Take home message: For the first time, various treatment options are available for patients with MDS. In light of the intense efforts now in progress, the next decade promises to be one of hope and excitement for both MDS patients and treating clinicians.     

骨髓增生异常综合征感染原因及治疗策略

骨髓增生异常综合征 （MDS） 是一组起源于造血干细胞的恶性克隆性肿瘤。由于患者外周血白细胞持续低下, 较高危患者还会接受去甲基化药物、 常规药物化疗、 造血干细胞移植等治疗, 且MDS多为老年患者, 感染的风险相对较高。MDS患者感染的主要原因是中性粒细胞减少, 此外, 淋巴细胞亚群平衡异常、 铁过载、 高龄、 合并症等也是易感染的相关因素。基于上述危险因素, 对患者的感染风险进行评估, 给予早期预防及制定合适的抗感染治疗策略, 可以提高患者治疗效果及延长生存时间。     

Current treatment options and strategies for myelodysplastic syndromes

Background: The myelodysplastic syndromes (MDS) are a group of heterogeneous disorders characterized by ineffective hematopoiesis. A number of scoring systems have been developed but they are of limited value in guiding individual patient treatment decisions. Objective: We have presented the benefits and limitations of various treatment approaches for MDS to help provide guidance in treatment decisions. Methods: An extensive literature search on PubMed from 2000 to 2008 was done using MESH keywords MDS, treatment, 5-azacytidine, decitabine, lenalidomide, antithymocyte globulin. Conclusion: In recent years there has been progress in supportive care and three agents have been approved for the treatment of MDS. With the exception of lenalidomide, which produced remarkable responses with 5q chromosomal deletion, these therapies benefit a minority of patients and the overall outcomes are still unsatisfactory. Allogeneic stem cell transplant remains the only curative option but the majority of MDS patients are not eligible because of older age and other medical problems. Immunosuppressive therapy is helpful in a fraction of patients. Several newer agents are being tested, offering promise for the future.     

Immunomodulatory drugs in myelodysplastic syndromes

This review summarises the mechanism of action of immunomodulatory analogues of thalidomide and their use in myelodysplastic syndromes. Thalidomide was found to have a response rate of ～ 20% in these patients. Lenalidomide – which is more potent and less toxic than thalidomide – has been used in three clinical trials and produced the best responses (60 – > 90%) in low- and intermediate-1-risk transfusion-dependent patients with del(5q). The responses are purely erythroid in nature, and are associated with major cytogenetic responses in > 50% of the del(5q) patients. Non-del(5q) low- and intermediate-1-risk transfusion-dependent patients also had a ～ 25% incidence of transfusion independence following therapy with lenalidomide. Median time to response is ～ 4 weeks and 90% of patients respond within 12 weeks. The precise mechanism of action remains unknown but anticytokine, antiangiogenic and immunomodulatory properties are thought to play a role.     

骨髓增生异常综合征治疗药物的研究进展

骨髓增生异常综合征是一组影响骨髓造血干细胞、祖细胞的克隆性疾病.近年来,随着对骨髓增生异常综合征发病机制的深入研究产生了许多新型药物.目前对骨髓增生异常综合征治疗的药物包括缺氧诱导因子脯氨酰羟化酶抑制剂、端粒酶抑制剂、剪切体抑制剂、异柠檬酸脱氢酶抑制剂、新型去甲基化剂、免疫检测点抑制剂、针对TP53突变药物、酪氨酸激酶...     

地西他滨联合HAG方案治疗骨髓增生异常综合征的临床疗效及其安全性评价

目的 探讨地西他滨联合HAG方案治疗骨髓增生异常综合征的临床疗效及其安全性评价。方法 选择2014年8月-2016年8月在南阳市第二人民医院治疗的骨髓增生异常综合征患者56例,随机分为两组,对照组26例,接受HAG化疗,观察组30例患者,接受地西他滨联合HAG化疗,比较两组患者的临床疗效以及不良反应的发生情况。结果 第1个疗程结束后,对照组患者的总缓解率为26.9%,观察组为30.0%,虽然观察组的缓解率较高,但是统计分析差异并不显著。两个疗程结束后,观察组的总缓解率为83.3%,显著高于对照组患者的57.7%,差异有统计学意义（P<0.05）。观察组发生25例次不良反应,显著低于对照组（43例次）,差异有统计学意义（P<0.05）。结论 地西他滨辅助治疗可以显著改善HAG化疗治疗骨髓增生异常综合征的临床疗效,降低化疗期间不良反应的发生率,提高患者的生活质量,值得临床推广应用。     

The clinical safety of lenalidomide in multiple myeloma and myelodysplastic syndromes

Thionamides, selective inhibitors of thyroid peroxidase-mediated iodination by tyrosine residues in thyroglobulin, have been effectively used in the treatment of hyperthyroidism. The choices for initial treatment of patients with Graves’ disease differ in various countries, and many physicians around the world prefer to administer thionamide drugs as the first choice of treatment for patients with hyperthyroidism. Although some thyroidologists more often consider radioiodine to be the treatment of choice because of its safety and ease of administration, thionamides remain the mainstay of treatment in thyrotoxic children and adolescents and in hyperthyroid women during pregnancy, postpartum period and lactation. A recent study with continuous thionamide treatment for patients with Graves’ disease shows its efficacy, safety and cost-benefit properties. Further studies of the effectiveness of continuous thionamide therapy in patients with thyrotoxicosis need to be designed and implemented to determine indications for such therapy in children, adolescents and adults with diffuse toxic goiter, in particular, in those who have had recurrence of hyperthyroidism after discontinuation of one complete course of treatment.     

骨髓增生异常综合征贫血原因及治疗策略

骨髓增生异常综合征 （MDS） 是一组高度异质性疾病, 贫血是MDS常见的临床表现, 也是影响MDS患者生存及生活质量的重要因素。MDS贫血的发病机制复杂, 患者的贫血可以是多种因素的结果。本文论述了MDS常见的贫血原因, 包括异常克隆的扩增、 免疫异常、 5q-的造血异常、 无效造血和铁过载等。针对不同原因的贫血, 可以采用促红细胞生成素、 免疫抑制剂、 祛铁治疗、 免疫调节治疗、 转化生长因子-β （TGF-β） 通路阻断剂、 去甲基化药物治疗和造血干细胞移植等策略。