不同的造血干细胞移植方式对重型再生障碍性贫血治疗效果的影响

目的:比较异基因外周造血干细胞移植(Allo-PBSCT)、异基因骨髓移植(Allo-BMT)及Allo-PBSCT和Allo-BMT混合移植3种不同的移植方式治疗重型再生障碍性贫血(SAA)的临床效果。方法:37例SAA患者接受了同胞人类白细胞抗原(HLA)全相合异基因干细胞移植,其中Allo-PBSCT组11例,Allo-BMT组14例,混合移植组12例。观察所有患者移植近期中性粒细胞(Neu)和血小板的造血时间、短小片段重复序列(STR)植入状况、粒细胞缺乏(粒缺)合并感染的发生率、急慢性移植物抗宿主病(GvHD)发生率,以及远期3年植入失败率、病死率和无病生存率。结果:Allo-PBSCT组、Allo-BMT组、混合移植组的Neu造血时间分别为(13.5±2.3)d、(21.2±3.5)d和(14.7±2.4)d,血小板造血时间分别为(24±4)d、(22±5)d和(23±5)d,Allo-PBSCT组和混合移植组的Neu造血时间均短于Allo-BMT组(P均<0.05),而3组间血小板平均造血时间比较差异无统计学意义。3组所有患者在移植后第28日时检测STR均证实为完全供者嵌合体。Allo-PBSCT组和混合移植组粒缺合并感染发生率分别为9%和17%,均明显低于Allo-BMT组的57%(P均<0.05);Allo-PBSCT组GvHD总发生率(包括急性和慢性)为36%,与均无GvHD发生的Allo-BMT组及混合移植组比较差异具统计学意义(P均<0.05);Allo-PBSCT组3年的植入失败率、病死率和无病生存率分别为36%、9%和64%,混合移植组和Allo-BMT组所有患者3年内均无病生存、STR均表现为稳定植入,Allo-PBSCT组3年植入失败率高于混合移植组和Allo-BMT组(P均<0.05),3年无病生存率低于混合移植组和Allo-BMT组(P均<0.05)。结论:在上述3种不同的移植方式中,混合移植同时具有Neu重建快、粒缺合并感染发生率低、GvHD发生率和植入失败发生率低、无病生存率高等优点,临床上可以优先考虑采用此方式进行治疗。     

Effect of postremission high dose cytarabine-based consolidation chemotherapy before allogenic stem cell transplantation in outcomes of acute myeloid leukemia patients

Background and Aim

This is a retrospective study aiming to investigate the effect of the number of high dose cytarabine-based chemotherapy (HiDAC) courses in patients with acute myeloid leukemia before allogenic stem cell transplantation (ASCT).

ABO血型不合的同胞异基因外周血干细胞移植

目的探讨HLA配型相合、ABO血型不合的同胞异基因外周血干细胞移植(alloPBSCT)的疗效。方法对27名HLA配型相合、ABO血型不合的血液恶性肿瘤患者作同胞alloPBSCT(实验组,供、受者ABO血型主侧不合的有15例,次侧不合的有10例,主次侧均不合的有2例),其中急性髓细胞白血病(AML)6例、急性淋巴细胞白血病(ALL)8例、慢性粒细胞性白血病(CMLLP)10例、骨髓增生异常综合征(MDSRAEBT)2例、非霍奇金氏淋巴瘤(ⅣB)1例;并选用同期的35名ABO血型相合的移植患者作比较(对照组)。移植物抗宿主病(GVHD)的预防采用霉酚酸酯(MMF)、环孢菌素A(CSA)和短程甲氨喋呤(MTX)三联预防方案。结果62例全部造血重建。实验组:27名alloPBSCT患者均未出现急性溶血反应,主侧不合者红系造血明显延迟,供/受者血型为A/O的患者中有3例(3/7)发生纯红细胞再生障碍性贫血(PRCA),27名患者于移植后25～153d血型成功转变为供者型;实验组GVHD发生率、VOD发生率、CMV感染、HC发生率及疾病复发率、死亡率与对照组相比差异无统计学意义(P>0.05)。结论ABO血型不合可以进行alloPBSCT,并且不影响干细胞移植的植活、GVHD及其它移植相关并发症的发生和预后。供/受者血型为A/O是主侧ABO血型不合患者alloPBSCT后PRCA发生的高危因素。     

冷冻新鲜芦荟漱口液预防造血干细胞移植患者口腔黏膜炎的效果

目的：观察冷冻新鲜芦荟漱口液预防造血干细胞移植患者并发口腔黏膜炎的效果。方法选取2013年1月—2014年1月42例接受造血干细胞移植的恶性血液病患者作为研究对象。按照随机数字表法将患者分为研究组20例和对照组22例。对照组使用常规口腔干预方法,研究组在常规口腔干预方法的基础上增加冷冻新鲜芦荟漱口液漱口,取新鲜库拉索芦荟或中华芦荟约30～40 g洗净,去刺及表皮后,将果冻状部分放入100℃的250 ml开水中泡制,待溶液冷却后放入2～4℃的冰箱冷冻6～8h后用于患者漱口。比较两组患者口腔黏膜炎发生程度。结果研究组发生口腔黏膜炎0级11例,Ⅰ级6例,Ⅱ级2例,Ⅲ级1例,对照组分别为5,3,10,4例,两组比较差异有统计学意义（ U＝112．5,P＜0．01）。结论冷冻新鲜芦荟漱口液在预防造血干细胞移植患者并发口腔黏膜炎方面有一定疗效,且经济有效。     

Factors associated with peripheral blood stem cell yield in healthy pediatric donors

Background and Objectives

Although several studies have reported on the use of children as donors for peripheral blood stem cells (PBSC), data on the predictive factors of CD34+ stem cell yield in healthy pediatric donors are very limited.

Peripheral blood stem cell collection for allogeneic hematopoietic stem cell transplantation: Practical implications after 200 consequent transplants

Background

Proper stem cell mobilization is one of the most important steps in hematopoietic stem cell transplantation (HSCT). The aim of this paper is to share our 6 years’ experience and provide practical clinical approaches particularly for stem cell mobilization and collection within the series of more than 200 successive allogeneic HSCT at our transplant center.

异基因外周血造血干细胞移植预处理的护理

目的探讨异基因外周血造血干细胞移植（allo-PBSCT）预处理的相关毒性及护理特点，以提高护士时相关病情的观察及护理水平，使患者的移植手术顺利进行。方法对7例行allo-PB-SCT的患者行移植前的各项准备，并均行颈内静脉置管。预处理方案均采用不含全身放疗（TBI）的清髓性联合化疗方案：马利兰（BU）＋环磷酰氨（CY）。结果7例患者均顺利度过预处理期，并成功接受千细胞移植。结论在预处理中加强细致、全面的观察及护理，并及早发现问题，及时处理不良反应是保证患者安全，顺利进行allo-PBSCT的前提，是PBSCT成功很关键的一环。     

异基因造血干细胞移植治疗恶性淋巴瘤临床疗效

目的：评估淋巴瘤病人的异基因造血干细胞移植的疗效。方法：对14例淋巴瘤病人进行了异基因造血干细胞移植，5例为非清髓型移植，其预处理方案为氟达拉宾，抗胸腺细胞球蛋白及马法兰，9例清髓型移植病人选择了全身照射 环磷酰胺、全身照射 环磷酰胺 抗胸腺细胞球蛋白、罗氮芥、阿糖胞苷、环磷酰胺、足叶乙甙、抗胸腺细胞球蛋白和马利兰、马法兰 美罗华。移植物抗宿主病预防：非清髓型为环胞霉素 麦考酚酸脂；清髓型为环胞霉素 MTX。结果：5例非清髓型移植病人中，4例植活，1例失败；9例清髓型移植病人中，4例病人早期死亡。在随访的10例病人中，3例复发。结论：对于化疗失败，反复复发淋巴瘤患者，有适合HLA配型者应尽早行异基因造血干细胞移植。     

Comorbidity predicts survival in myelodysplastic syndromes or secondary acute myeloid leukaemia after allogeneic stem cell transplantation

Background Recent data suggest that, among other factors, comorbidity may be an important prognostic variable in patients with myelodysplastic syndromes (MDS) who are eligible for haematopoietic stem cell transplantation (SCT). Patients and methods We examined the overall survival (OS) and underlying risk factors in 45 adult patients with MDS (n = 38), chronic myelomonocytic leukaemia (n = 1), or secondary acute myeloid leukaemia (AML) arising from MDS (n = 6), who underwent allogeneic SCT at our Institution. Results With a median follow‐up of 37 months, OS for all patients was 23%, post‐transplant relapse occurred in 11 patients, and 10 patients died from treatment‐related complications. The overall outcome and survival was independent of cytogenetic abnormalities and International Prognostic Scoring System (IPSS). However, we identified comorbidity as defined by the haematopoietic cell transplantation specific comorbidity index (HCT‐CI), as a significant adverse prognostic variable in our MDS patients. Conclusions Based on these data and similar published data we recommend selecting patients with MDS or secondary AML for SCT according to the presence of comorbidities.     

Long-term results of allogeneic peripheral blood hematopoietic stem cell transplantation for severe aplastic anemia

ObjectiveAplastic anemia (AA) is a life-threatening disorder and may be associated with significant morbidity and mortality Currently, the first treatment option is allogeneic hematopoietic stem cell transplant (allo-HSCT) for patients younger than 40 years. Bone marrow is recommended as the stem cell source due to less graft versus host disease (GVHD) risk and better outcomes than peripheral blood (PB)-derived stem cell. The aim of this study is to share the data of AA patients who have underwent PB-derived allo-HSCT in our bone marrow transplantation center.MethodsTwenty-seven patients who underwent PB-derived allo-HSCT from human leukocyte antigen matched sibling donors were analyzed retrospectively.ResultsThe median follow-up time was 95.2 months (range, 4.8–235 months). The 10-year survival was 89 %. The median neutrophil and platelet engraftment time was 11 days (range, 9–16 days) and 13 days (range, 11–29 days), respectively. Primary platelet engraftment failure was observed in 1 patient (3.7 %). Acute and chronic GVHD observed in 2 (7.4 %) and 3 (11.1 %) patients, respectively. Neutropenic fever was observed in 13 (44.8 %) of patients until the engraftment after allo-HSCT. One patient died due to CMV infections, two died due to septic shock secondary to fungal infection.ConclusionAlthough there is no prospective data directly comparing BM with PB as stem cell source in AA, observational studies indicates better OS with BM. PB can be used in certain situations such as higher risk for graft failure and donor preference. This study demonstrated that PB-derived stem cell seems to be a reasonable alternative to BM.     

父母供者外周血单倍体干细胞移植治疗儿童复发难治急性白血病

背景：儿童复发难治急性白血病单纯化学治疗效果极差,异基因造血干细胞移植是治愈该类疾病的惟一有效方法。研究显示单倍体移植与同胞相合及非亲缘全相合造血干细胞移植的治疗效果接近,甚至优于后者,且父母作为供者单倍体造血干细胞移植依从性好,能够保证移植干细胞数量及预防原发病复发,明显提高了患者移植成功率及长期无白血病生存率。目的：回顾分析父母供者单倍体外周血造血干细胞移植治疗儿童复发难治急性白血病的疗效。方法：入选35例父母供者外周血单倍体造血干细胞移植治疗儿童复发难治急性白血病。均采用＂改良1,4-丁二醇二甲磺酸酯/环磷酰胺＋胸腺细胞免疫球蛋白＂预处理方案和环孢素、吗替麦考酚酯及甲氨蝶呤三联短程预防移植物抗宿主病。结果与结论：35例父母供者外周血单倍体造血干细胞移植治疗儿童复发难治急性白血病均植入成功。135例患儿回输单个核细胞中位数为5.82（3.23-8.45）×108/kg,其中CD34＋细胞中位数为4.52（2.37-11.51）×106/kg。2干细胞回输后100 d内,移植相关死亡率为14.3%。33-Ⅱ度急性移植物抗宿主病发生率为34.3%,Ⅲ-Ⅳ度急性移植物抗宿主病发生率为37.1%,慢性移植物抗宿主病总发生率为42.9%。42年无白血病生存率为42.9%,2年总生存率为51.4%,2年原发病复发率为34.3%,中位生存时间为24个月。提示对于无人类白细胞抗原相合同胞供者及不能及时寻找到非血缘人类白细胞抗原相合供者的儿童复发难治急性白血病,父母供者外周血单倍体造血干细胞移植是一种高效可行的治疗方法。     

单倍体异基因造血干细胞移植治疗急性白血病疗效观察

目的观察单倍体异基因造血干细胞移植（Haplo—SCT）治疗急性白血病的疗效。方法对我科2006年6月至2007年12月5例急性白血病患者进行单倍体异基因造血干细胞移植,预处理方案：改良马利兰与环磷酰胺（BU／CY）方案。移植物抗宿主病（GVHD）预防：环孢素A、短程甲氨喋呤、霉酚酸酯和抗-CD25抗体。结果5例患者全部获得造血重建。1例因合并Ⅲ度肠道急性移植物抗宿主病死亡,其余4例随访20～30月,目前均无病存活。结论单倍体异基因造血干细胞移植造血重建稳定,严重并发症少,可作为造血干细胞来源。     

The experience of allogeneic hematopoietic stem cell transplantation in a patient with X-linked adrenoleukodystrophy

X-linked adrenoleukodystrophy (X-ALD), a progressive neurometabolic disorder that is caused by a defect in the gene ABCD1 (ATP-binding cassette, subfamily D, member 1), which encodes the peroxisomal ABC half-transporter ALD protein. Recently, allogeneic hematopoietic stem cell transplantation (alloHSCT) is the only therapy known to prevent disease progression. In this study, we would like to present our experience of alloHSCT for X-ALD from a HLA matched related sibling by the use of reduced intensity conditioning regimen composed of fludarabine, busulfan and ATG which allows us to reduce procedure-related toxicity and prevent mortality while achieving a curative effect.     

Peripheral blood stem cell grafts in allogeneic hematopoietic cell transplantation: It is not all about the CD34+ cell dose

Allogeneic Hematopoietic Cell Transplantation is a curative approach in various malignant and non-malignant disorders. The majority of adult transplants in the current era are performed using mobilized stem cells, harvested from the peripheral blood by leukapheresis. Peripheral blood stem cell (PBSC) collections are designed to target a dose of stem cells that will result in safe engraftment and hematopoietic recovery; however, 99 % of the cells contained in a PBSC graft are not stem cells and a growing number of studies attempt to characterize the associations between graft composition and transplant outcomes. A better understanding of the impact of the quantity and quality of various cell types in PBSC grafts may lead to development of novel collection strategies or improved donor selection algorithms. Here we review relevant findings from recent studies in this area.     

非清髓异基因外周血造血干细胞移植治疗血液病的临床观察

目的　探讨非清髓异基因外周血造血干细胞移植 (NASCT)在治疗血液病中的意义。方法　采用NASCT治疗 33例HLA相合的血液病患者。男 2 0例 ,女 13例 ,中位年龄 36岁 (18～ 5 9岁 )。33例中急性白血病第 1次完全缓解期 (CR1 ) 11例 ,CR2～ 34例 ,难治复发性急性白血病未缓解期 3例 ,重型再生障碍性贫血 (SAA) 4例 ,慢性粒细胞白血病慢性期 7例 ,骨髓增生异常综合征 (MDS) 2例 ,慢性淋巴细胞白血病和骨髓纤维化各 1例。非清髓预处理方案 :白血病患者采用环磷酰胺 (CTX)、阿糖胞苷及CD3单克隆抗体 ,6例患者在此基础上加用氟达拉宾。SAA和MDS患者采用CTX和抗胸腺细胞球蛋白。结果　 33例均顺利渡过造血抑制期。平均移植后第 10 .5天 (移植后第 8～ 2 1天 )中性粒细胞计数 >0 .5× 10 9 L ,第 15天 (移植后第 10～ 30天 )血小板计数 >30× 10 9 L。 33例中供者细胞完全植入2 4例 (其中 13例于移植后 1～ 6个月由供受者嵌合性植入转为完全植入 ) ,稳定混合嵌合体 4例 ,移植排斥 5例。 33例中发生急性和慢性移植物抗宿主病各 7例 (2 1.2 % )。随诊 2～ 36个月 2 5例 (75 8% )仍存活。结论　NASCT简便安全 ,并发症少 ,疗效较好 ,为治愈血液病提供了新手段。     

Hematopoietic stem cell transplantation in geriatric patients in Turkey

The incidence of most hematologic malignancies increases with age. Physicians increasingly refer older patients for hematopoietic stem cell transplantation (HSCT) due to more experience and improved supportive care in HSCT. This article discusses the available data regarding the feasibility, tolerability, toxicity, and effectiveness of autologous and allogeneic HSCT in older adults.     

Allogenic hematopoietic stem cell transplantation in sickle cell disease

Sickle cell disease (SCD) is one of the most common monogenic disorders worldwide and affects approximately 100,000 people in the United States alone. SCD can cause numerous complications, including anemia, pain, stroke, and organ failure, which can lead to death. Although there are a few disease-modifying treatments available to patients with SCD, the only current curative option is a hematopoietic stem cell transplant (HSCT). In this review, we will discuss the different approaches to allogeneic HSCT in the treatment of SCD and the outcomes of these approaches.     

Graft failure after allogeneic hematopoietic stem cell transplantation

Graft failure is a serious complication of allogeneic hematopoietic stem cell transplantation (allo-HSCT) defined as either lack of initial engraftment of donor cells (primary graft failure) or loss of donor cells after initial engraftment (secondary graft failure). Successful transplantation depends on the formation of engrafment, in which donor cells are integrated into the recipient’s cell population.In this paper, we distinguish two different entities, graft failure (GF) and poor graft function (PGF), and review the current comprehensions of the interactions between the immune and hematopoietic compartments in these conditions. Factors associated with graft failure include histocompatibility locus antigen (HLA)-mismatched grafts, underlying disease, type of conditioning regimen and stem cell source employed, low stem cell dose, ex vivo T-cell depletion, major ABO incompatibility, female donor grafts for male recipients, disease status at transplantation.Although several approaches have been developed which aimed to prevent graft rejection, establish successful engraftment and treat graft failure, GF remains a major obstacle to the success of allo-HSCT.Allogeneic hematopoietic stem cell transplantation (allo-HSCT) still remains to be the curative treatment option for various non-malignant and malignant hematopoietic diseases. The outcome of allo-HSCT primarily depends on the engraftment of the graft. Graft failure (GF), is a life-threatening complication which needs the preferential therapeutic manipulation. In this paper, we focused on the definitions of graft failure / poor graft function and also we reviewed the current understanding of the pathophysiology, risk factors and treatment approaches for these entities.     

氟达拉滨和非氟达拉滨预处理方案在非清髓异基因造血干细胞移植中的比较

目的　探讨氟达拉滨 (Flud)和非Flud预处理方案在非清髓造血干细胞移植 (NAST)中的作用和疗效。方法　用NAST治疗 36例HLA相合的血液病患者。预处理方案分为两组 :非Flud组 :阿糖胞苷、环磷酰胺、CD3 单克隆抗体 抗淋巴细胞球蛋白、环孢菌素A和霉酚酸酯 ;Flud组 :在非Flud组基础上加用Flud。结果　Flud组 1 6例患者中 1 4例 (87.5 % )形成供者细胞完全植入 (FDC) ;非Flud组2 0例患者中 1 6例 (80 .0 % )形成FDC ,Flud组早期FDC率明显高于非Flud组 (P <0 .0 1 ) ,但两组最终FDC率和移植排斥发生率无统计学差异 (P >0 .0 5)。Flud组中 6例 (37.5 % )发生Ⅰ～Ⅱ度急性移植物抗宿主病 (GVHD) ;非Flud组 4例 (2 0 .0 % )发生Ⅰ～Ⅳ度急性GVHD ,Flud组急性GVHD的发生率高于非Flud组 ,但无统计学差异 (P >0 .0 5)。Flud组 2例 (1 2 .5 % )、非Flud组 6例 (30 .0 % )发生慢性GVHD ,两组的慢性GVHD发生率无统计学差异 (P >0 .0 5)。Flud组 3例 (1 8.7% )、非Flud组 3例 (1 5 .0 % )发生慢性间质性肺炎 (IP) ,两组IP发生率无统计学差异 (P >0 .0 5)。两组患者的中性粒细胞、血小板的最低值和恢复时间均无显著性差异 (P >0 .0 5)。Flud组 1 6例患者中仅 1例急性髓系白血病M2 第 2次完全缓解 (CR2 )患者在移植后 4个