国内造血干细胞移植的经济分析及对策

通过分析我国造血干细胞移植患者所需的经费结构、经费的来源现状,结合我国现阶段的宏观经济走势和社会保障机制,探讨解决造血干细胞移植费用的措施.     

造血干细胞移植进展

造血干细胞移植（ＳＣＴ）仍然在国内外蓬勃发展。四年前全国报告能进行异基因ＳＣＴ（ＡｌｌｏＳＣＴ）的为２２个单位，而至１９９９年，仅浙江省一个省份能进行ＡｌｌｏＳＣＴ的单位已超过１０个。全国除西藏外，各省自治区都有大小不同的ＳＣＴ单位。到１９９８年底的不完全统计，全国已作异基因ＳＣＴ７２０例。其中北京医科大学血液病研究所为３２６例。而ＩＢＭＴＲ（国际骨髓移植登记组）的报告，１９９７年ＡｌｌｏＢＭＴ的全球总数约１７０００例。ＡｌｌｏＳＣＴ的造血干细胞来源主要为骨髓。较少数为外周血，脐带血更…     

造血干细胞移植后并发感染121例的临床分析

目的探讨造血干细胞移植(HSCT)后并发感染的临床病原学特点和相关危险因素。方法共有121例造血系统疾病患者接受HSCT,其中51例行异基因外周造血干细胞移植,40例行异基因骨髓移植,4例行异基因外周血造血干细胞和骨髓混合移植,5例行脐带血移植,16例行自体造血干细胞移植,5例行自体CD34~+细胞移植。患者均采用化疗进行预处理。采用短程甲氨蝶呤联合环孢素A预防移植物抗宿主病(GVHD),部分病例加用达利珠单抗或抗淋巴细胞球蛋白。结果121例患者中,116例患者获得造血重建,3例移植失败,2例在移植物植活前因严重感染死亡。移植后60d内,83例(68．6%)共发生感染97次,64例(77．1%)发生感染时中性粒细胞＜0．5×10~9/L,患者均有不同程度的发热。口咽黏膜和呼吸道(上、下呼吸道)是最常见的感染部位,占64．9%。21例患者的23次感染中,共分离出20株细菌和7株真菌,65%为革兰氏阴性杆菌。10例患者并发巨细胞病毒感染。因感染死亡的患者共8例,均为异基因造血干细胞移植患者。发生Ⅱ～Ⅳ度急性GVHD患者的感染发生率(88．9%,24/27)高于0～Ⅰ度急性GVHD患者(60．3%,44/73,P＜0．01)。结论造血干细胞移植后60d内的感染多发生于粒细胞缺乏期；口咽黏膜炎和呼吸道是常见感染部位,下呼吸道感染者的死亡率高,侵袭性真菌感染者的预后不佳；发生Ⅱ～Ⅳ度急性GVHD者有较高的感染发生率。     

异基因造血干细胞移植后严重的出血性膀胱炎多因素分析

目的 探讨异基因造血干细胞移植（allo-HSCT）后严重的（≥Ⅱ度）出血性膀胱炎（HC）的危险因素。方法 对1997年4月至2004年12月期间的114例allo-HSCT患者的资料进行回顾性分析。以预处理实施之日为观察起点，至移植后＋180 d随访中止。选择11个临床参数，即：年龄、性别、疾病类型、供者类型、预处理方案、移植时疾病状态、急性移植物抗宿主病（aGVHD）、aGVHD的预防、预处理方案中抗胸腺细胞球蛋白（ATG）的应用、中性粒细胞及血小板植活时间做Cox单因素分析。将在单因素分析中P＜0.1作为有统计学意义的因素进行Cox多因素回归分析。移植后180 d内HC累计发生率的计算应用Kaplan-Meier法。结果 （1）114例患者中有29例发生HC，＋180 d内HC的累计发生率为26％，其中Ⅱ级12例，Ⅲ级11例，Ⅳ级6例。（2）单因素分析表明，以下因素与HC的发生密切相关；男性（RR=2.885，P=0.021）、年龄≤25岁（RR=3.265，P=0.002）、Ⅲ～Ⅳ度aGVHD（RR=4.039，P=0.002）、非血缘供者（RR=4．347，P=0.000）、加强的GVHD预防方案（RR=2.218，P=0.045）、疾病进展期（RR=2.668，P=0.009）。（3）对上述有统计学意义的因素进行Cox多因素分析，只有男性（RR=2.993，95％CI 1.218～7.358；P=0.017）和非血缘供者（RR=4.478，95％CI 2.049～9.786；P=0.000）为HC的独立危险因素。结论 男性受者和非血缘供者的造血干细胞移植后发生HC的危险性显著增加。     

造血干细胞移植

20世纪50年代初期造血干细胞移植兴起,随着医学科技发展,大剂量化疗（预处理）＋造血干细胞移植为各种晚期肿瘤和癌症治疗提供了有力的支持,目前对造血干细胞的来源、种类、采集、活性测定,异基因造血干细胞的配型、动员剂的效果、骨髓中癌细胞污染的处理、移植后并发症的诊治、疾病观察指标、临床适应证和治疗效果的评估等等都逐渐规范化,且有很多治疗成功病例的报告。     

造血干细胞移植治疗系统性红斑狼疮

本文综述了近10余年造血干细胞移植(haemopoietic stem cell transplantation,HSCT)治疗系统性红斑狼疮(systemic lupus erythematosus,SLE)的相关进展.     

Kidney Disease Following Hematopoietic Stem Cell Transplantation

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Skeletal Changes After Hematopoietic Stem Cell Transplantation in Osteopetrosis

Osteopetrosis is a rare skeletal dysplasia resulting from an osteoclast defect leading to increased bone mass and density. Hematopoietic stem cell transplantation can rescue the disease phenotype and prevent complications. However, little is known about the skeletal changes hematopoietic stem cell transplantation induces in patients with this disease. The purpose of this study was to describe the skeletal changes after hematopoietic stem cell transplantation in a retrospective cohort of patients diagnosed with osteopetrosis in one medical center over 13 years. For this purpose, all available epidemiological, hematological, biochemical, and radiographic data were collected and quantitatively analyzed. We found a significant early change in bone metabolism markers coinciding with hematopoietic recovery after stem cell transplantation. Hematopoietic stem cell transplantation induced a later significant improvement in both skeletal mineral distribution and morphology but did not lead to complete radiological normalization. Presumably, changes in bone metabolism, skeletal mineral distribution, and morphology were the result of renewed osteoclast function enabling bone remodeling. We propose that biochemical bone metabolism markers and radiological indices be routinely used to evaluate response to hematopoietic stem cell transplantation in patients with osteopetrosis. © 2020 American Society for Bone and Mineral Research.     

Reconstruction of the Hematopoietic System After Stem Cell Transplantation

The practice of hematopoietic stem cell transplantation to rescue patients from the myeloablative effects of chemo- or radiotherapy, or to replace defective hematopoiesis, is based on the assumption that hematopoietic stem cells in the graft have sufficient proliferative potential to supply mature blood cells for the remainder of the recipient’s lifespan. However, the mechanism(s) whereby this is achieved are not well understood. Here we address the reconstruction of the hematopoietic system by considering the effects of stem cell and progenitor cell renewal and differentiation. We conclude that stem cell self-renewal is necessary for hematological recovery and that infused committed progenitor cells (CFU-GM) may contribute to the neutrophil count in the early posttransplant period.     

单倍体相合造血干细胞移植联合间充质干细胞输注治疗异染性脑白质营养不良

目的探讨单倍体相合造血干细胞移植联合间充质干细胞输注治疗异染性脑白质营养不良的疗效。方法 7岁患儿,采用单倍体相合造血干细胞移植联合间充质干细胞输注的方法,经氟达拉滨、马利兰、环磷酰胺和抗人胸腺细胞球蛋白方案进行预处理,供者应用G-CSF动员和联合免疫抑制剂(包括环胞菌素A、氨甲喋呤、霉酚酸酯、CD25单抗)预防移植物抗宿主病。造血重建后,每周输注脐带来源的间充质干细胞1次,连续4周。结果患儿造血重建迅速,11 d时中性粒细胞0.5×109/L、血小板20×109/L;14 d时血白细胞芳基硫酸酯酶A水平明显上升;28 d时达正常水平。患者无急性GVHD发生。移植后1个月经植入证据检测,证实为完全供者造血,神经系统症状逐渐恢复。结论单倍体相合造血干细胞移植,联合间充质干细胞输注,治疗异染性脑白质营养不良,安全、有效,血白细胞芳基硫酸酯酶A水平回升迅速。这种新型疗法可能是治疗缺乏HLA全相合供者的异染性脑白质营养不良疾病患者的可靠选择。     

A Case Report of Lung Transplantation After Hematopoietic Stem Cell Transplantation and Literature Review

Pulmonary complications may occur after hematopoietic stem cell transplantation for hematologic malignancies. Lung transplantation is the only treatment option for end-stage lung failure. We presented a case of acute myeloid leukemia who received a hematopoietic stem cell transplantation and underwent bilateral lung transplantation with end-stage usual interstitial pneumonia and chronic obstructive lung disease. This case showed that lung transplantation could be successfully applied in properly selected hematologic malignancy patients with long disease-free survival, like lung transplantations performed for other indications.     

Liver Transplantation Followed by Allogeneic Hematopoietic Stem Cell Transplantation for Atypical Mevalonic Aciduria

Mevalonic aciduria because of mutations of the gene for mevalonate kinase causes limited synthesis of isoprenoids, the effects of which are widespread. The outcome for affected children is poor. A child with severe multisystem manifestations underwent orthotopic liver transplantation at age 50 months for the indication of end‐stage liver disease. This procedure corrected liver function and eliminated portal hypertension, and the patient showed substantial improvement in neurological function. However, autoinflammatory episodes continued unabated until hematopoietic stem cell transplantation was performed at 80 months. Through this complex therapy, the patient now enjoys a high quality of life without significant disability.     

脐血造血干细胞移植的现状与展望

自 198 9年Gluckman等成功地进行世界上第 1例脐血造血干细胞移植 (CB HSCT)以来 ,包括中国在内的世界各地相继开展了此项工作。伴随各地脐血库网络的建立及分离、保存、配型技术的不断完善 ,GB HSCT的例数迅速增长。据 2 0 0 2年资料的不全统计 ,全球共开展CB HSCT 15 0 0余例 ,其应用范围几乎函盖骨髓造血干细胞移植 (BM HSCT)的全部病种 ,已成为可替代BM HSCT的重要治疗手段。现有研究表明 :(1)脐血造血干细胞进入细胞周期的次数少、端粒长、端粒酶活性高 ,具有更强的增殖分化能力 ,其稳定植入的时间更长 ;(2 )CB HSCT能…