JAMA：干细胞治疗或可改善自身免疫性及心血管疾病

据2月27日《美国医学协会期刊》（JAMA）上的一则研究显示，对以往发表的研究进行审阅后发现，从成人血液或骨髓中获得的干细胞可能对某些罹患自身免疫性疾病及心血管疾病的患者具有治疗性裨益。

就宽泛意义上说，干细胞分两类：胚胎干细胞与成人干细胞。人类胚胎干细胞是从受精后4-5天的胚泡（这是一种胚胎发育的早期形式）中分离出来的。成人干细胞则存在于身体各处的组织之中，其功能是作为替换损伤或老化细胞的储库。作者在文章中写道：“与传统想法相反，用来自血液（即来自外周血液或脐带血）和骨髓的干细胞进行治疗的临床指针正在迅速增加，而这些干细胞可以轻易安全地获取。”

美国西北大学医学院的Richard K. Burt及其同僚，对以往发表的应用来自血液和骨髓的干细胞临床指针及治疗效果的文章进行了审阅。

对自身免疫性疾病来说，在26篇报告的854名病患中，与治疗有关的非骨髓清除性（不引起骨髓抑制）干细胞移植的死亡率不到1%（2/220病人），药物诱导的骨髓清除性干细胞移植的死亡率不到2%（3/197），而强力骨髓清除性干细胞移植疗法的死亡率为13%（13/100），即那些使用了包括全身放射疗法或高剂量busulfan（这是一种用来治疗某些类型慢性白血病的药物）治疗的病人。

在那些与心血管疾病有关的报道中，共有17篇报告的1002名罹患心肌梗塞的病患，其中有16篇报告中报道了493名患有慢性冠心病的患者及3项荟萃分析。这些研究的证据提示，对这些冠心病患者施行干细胞移植可能与其心功能的轻度改善有关。（来源：EurekAlert!中文版）

生物谷推荐原始出处：

JAMA

Vol. 299 No. 8, February 27, 2008

Clinical Applications of Blood-Derived and Marrow-Derived Stem Cells for Nonmalignant Diseases

Richard K. Burt, MD; Yvonne Loh, MD; William Pearce, MD; Nirat Beohar, MD; Walter G. Barr, MD; Robert Craig, MD; Yanting Wen, MD; Jonathan A. Rapp, MD; John Kessler, MD

JAMA. 2008;299(8):925-936.

Context Stem cell therapy is rapidly developing and has generated excitement and promise as well as confusion and at times contradictory results in the lay and scientific literature. Many types of stem cells show great promise, but clinical application has lagged due to ethical concerns or difficulties in harvesting or safely and efficiently expanding sufficient quantities. In contrast, clinical indications for blood-derived (from peripheral or umbilical cord blood) and bone marrow–derived stem cells, which can be easily and safely harvested, are rapidly increasing.

Objective To summarize new, nonmalignant, nonhematologic clinical indications for use of blood- and bone marrow–derived stem cells.

Evidence Acquisition Search of multiple electronic databases (MEDLINE, EMBASE, Science Citation Index), US Food and Drug Administration [FDA] Drug Site, and National Institutes of Health Web site to identify studies published from January 1997 to December 2007 on use of hematopoietic stem cells (HSCs) in autoimmune, cardiac, or vascular diseases. The search was augmented by hand searching of reference lists in clinical trials, review articles, proceedings booklets, FDA reports, and contact with study authors and device and pharmaceutical companies.

Evidence Synthesis Of 926 reports identified, 323 were examined for feasibility and toxicity, including those with small numbers of patients, interim or substudy reports, and reports on multiple diseases, treatment of relapse, toxicity, mechanism of action, or stem cell mobilization. Another 69 were evaluated for outcomes. For autoimmune diseases, 26 reports representing 854 patients reported treatment-related mortality of less than 1% (2/220 patients) for nonmyeloablative, less than 2% (3/197) for dose-reduced myeloablative, and 13% (13/100) for intense myeloablative regimens, ie, those including total body irradiation or high-dose busulfan. While all trials performed during the inflammatory stage of autoimmune disease suggested that transplantation of HSCs may have a potent disease-remitting effect, remission duration remains unclear, and no randomized trials have been published. For reports involving cardiovascular diseases, including 17 reports involving 1002 patients with acute myocardial infarction, 16 involving 493 patients with chronic coronary artery disease, and 3 meta-analyses, the evidence suggests that stem cell transplantation performed in patients with coronary artery disease may contribute to modest improvement in cardiac function.

Conclusions Stem cells harvested from blood or marrow, whether administered as purified HSCs or mesenchymal stem cells or as an unmanipulated or unpurified product can, under appropriate conditions in select patients, provide disease-ameliorating effects in some autoimmune diseases and cardiovascular disorders. Clinical trials are needed to determine the most appropriate cell type, dose, method, timing of delivery, and adverse effects of adult HSCs for these and other nonmalignant disorders.

Author Affiliations: Divisions of Immunotherapy (Drs Burt, Craig, and Wen), Cardiology (Drs Beohar and Rapp), Rheumatology (Dr Barr), and Gastroenterology (Dr Craig), Department of Medicine; Division of Vascular Surgery, Department of Surgery (Dr Pearce); and Department of Neurology (Dr Kessler), Northwestern University Feinberg School of Medicine, Chicago, Illinois; and Department of Hematology, Singapore General Hospital, Singapore (Dr Loh).