同胞脐血移植治疗儿童急性白血病

脐血移植可以根治白血病、再生障碍性贫血、血红蛋白病及先天性免疫缺陷等疾病，尤其适合于儿童患者。临床资料表明同胞脐血移植的效果优于无关供者的脐血移植。对12例白血病或重症再障患儿进行了脐血HLA配型，结果配型完全相合4例，进行脐血移植3例，均成功植活，体重最大50kg。可以在胎儿出生时留取脐血配型，同时将脐血进行低温保存。提示剖宫产和自然分娩、体内采集脐血和体外采集脐血对脐血的采集体积无明显影响。单份同胞脐血可以重建几乎全部儿童患者的造血功能。对同胞脐血移植的病例，主张在分娩时留取脐血进行HLA配型，减少产前穿刺对孕妇及胎儿的损伤，同时脐血进行冷冻，若配型相合再进行脐血移植。     

脐血移植治疗难治性儿童白血病

目的：研究和ＨＬＡ不合多个脐血移植治疗难治性儿童白血病。方法：选择难治性白血病患儿，给予强烈化学治疗杀灭残存白血病细胞，再用多个脐血混合移植解救严重抑制状态的骨髓，从而缓解白血病，延长生命。结果：治疗６例，年龄２．５－１６岁，均为男性。用ＶＡＣ或ＡＶ方案化疗后，约１１天外周血颗粒细胞降至零。输注脐血后平均１３天外周血白细胞升至１．０×１０＾９／Ｌ以上。６０天内骨髓象正常，全部患者达到完成缓解。随诊     

脐血移植治疗难治性儿童白血病

目的：研究用HLA不合多个脐血移植治疗难治性儿童白血病。方法：选择难治性白血病患儿，给予强烈化学治疗杀灭残存白血病细胞，再用多个脐血混合移植解救严重抑制状态的骨髓，从而缓解白血病，延长生命。结果：治疗6例，年龄2．5一16岁，均为男性。用VAC或AV方案化疗后，约11天外周血颗粒细胞降至零。输注脐血后平均13天外周血白细胞升至l．0×109/L。60天内骨髓象正常，全部患者达到完成缓解。随诊I一4年，3例死亡，3例已分别存活36、44、ZO个月。结论：混合脐血移植对难治性白血病有较好疗效，其治疗作用机理值得进一步探讨。     

同胞脐血移植治疗儿童急性白血病二例报告

异基因骨髓移植是儿童高危白血病最有效的治疗方法。目前 ,由于家庭的缩小 ,同胞骨髓移植受到一定的限制 ,脐血中含有大量的造血干细胞 ,且具有来源广泛、采集方便、移植物抗宿主反应轻及污染率低等优点[1] ,越来越受到人们的重视。我们应用人类白细胞抗原 (ＨＬＡ)相合的同胞脐血移植治疗 1例急性单核细胞白血病及 1例恶性淋巴瘤Ⅳ期患儿 ,报道如下。病例和方法1 病例 :例 1男 ,9岁。 1997年 11月因发热、咽痛伴进行性苍白、乏力 10ｄ入院 ,经骨髓检查确诊为ＡＭＬ Ｍ5ｂ。住院后经ＤＡ(柔红霉素、阿糖胞苷 )方案化疗 1个疗程后缓解。脐血…     

无关血缘脐血移植治疗儿童恶性和非恶性疾病的临床研究

目的 探讨无关血缘脐血移植治疗儿童恶性和非恶性疾病的临床疗效.方法 24例进行非血缘脐血移植的患儿,包括急性淋巴细胞白血病(ALL)8例,急性髓系白血病(AML)4例,幼年慢性粒单细胞白血病(JMML)3例,慢性粒细胞白血病(CML)2例,骨髓增生异常综合征(MDS-RA)2例,急性混合型白血病、石骨症、X连锁肾上腺脑白质营养不良、Ⅵ型黏多糖和慢性肉芽肿各1例.HLA高分辨全相合6例,5个位点相合10例,4个位点相合5例,3个位点相合3例.预处理选用白消安(Bu)/环磷酰胺(CTX)/抗胸腺球蛋白(ATG)或全身放疗(TBI)/CTX/ATG为主方案.6例ALL采用TBI/CTX/ATG,其中2例加鬼臼乙叉苷(VP16),18例采用Bu/CTX/ATG,其中3例JMML加马法兰,1例ALL和1例AML加VP16,3例非恶性疾病加塞替哌.于0 d回输脐血有核细胞中位数为5.26(2.26～13.3)×107/kg,CD34细胞中位数为2.86(0.79～9.8)×105/kg.预防急性移植物抗宿主病(aGVHD)采用环孢素A和甲基泼尼松龙(MP)或骁悉,出现Ⅲ～Ⅳ度aGVHD者,加用CD25单抗.结果 脐血干细胞粒系植入率为91.7%(22/24),1例ALL未植入,1例CML于+130 d排斥;血小板植入率为83.3%(20/24),中性粒细胞≥0.5×109/L中位天数+15(+13～+28)d;血小板≥20×109/L中位天数+38(+25～+100)d.7例出现Ⅰ～Ⅱ度aGVHD;1例出现Ⅲ度aGVHD,给予MP均控制;2例出现Ⅳ度aGVHD,给予MP及CD25单抗后未控制.随访中位时间28(9～96)个月,均未发生慢性GVHD(cGVHD).现存活14例血型均转为供者型;死亡10例,其中原发病复发2例(急变期CML和婴儿型ALL各1例),Ⅳ度aGVHD合并感染2例,6例感染(CMV间质性肺炎4例,霉菌性肺炎2例).结论 无关血缘脐血是快速的造血干细胞来源之一,为恶性疾病患儿争取了治疗时间;因其cGVHD发生率低,对非恶性疾病患儿的治疗更具优势.     

HLA不全相合同胞脐血移植治疗儿童白血病一例

ＨＬＡ不全相合同胞脐血移植治疗儿童白血病一例朱为国钱新华程少杰邢献志刘纯霞官贵先黄志光脐血造血干细胞丰富，再生增殖能力强，是一种良好的造血干细胞来源。１９８９年世界首例脐血移植（ＣＢＴ）成功以来，儿童ＣＢＴ被认为是比骨髓移植更优越的治疗方法。国内目前...     

人类白细胞抗原1个位点不合无关供者脐血移植治疗急性髓性白血病一例

20 0 0年 5月 ,我们应用广州脐血库提供的一份人类白细胞抗原 (ＨＬＡ) 1个位点不合无关供者脐血进行移植 ,治疗 1例急性髓性白血病 (ＡＭＬ)患儿 ,现无病存活。报告如下。病例和方法1 病例 :患儿男 ,3岁 8个月。 1999年 1月被确诊为ＡＭＬ(Ｍ1) ,ＡＢＯ血型Ｂ型。经去甲氧柔红霉素 (ＩＤＡ) 阿糖胞苷 (Ａｒａ Ｃ)诱导治疗后达第 1次完全缓解 (ＣＲ1)。用依托泊甙、Ａｒａ Ｃ、表阿霉素、三尖杉酯碱、ＩＤＡ、米托蒽醌、6 巯基嘌呤、氨甲喋呤巩固维持治疗。 2 0 0 0年 2月在广州脐血库找到ＨＬＡ 1个位点 (Ａ位点 )不合无关供者脐血 ,ＡＢ…     

非血缘相关脐血移植治疗儿童高危白血病的临床观察

目的：非血缘脐血具有快速寻求、容易得到和HLA配型不严格的特点，该文进行了非血缘相关脐血移植（UD-UCBT）治疗儿童恶性白血病的研究并探讨其疗效问题。方法：对6例难治性白血病患儿，包括3例急性淋巴细胞白血病（2例高危CR1，1例标危CR2），2例幼年慢性粒单细胞白血病（1例缓解期，1例加速期）和1例急性髓系白血病（AML- M5，CR1）进行了非血缘相关脐血移植，HLA高分辨1例全相合，1例5个位点相合，1例4个位点相合，3例3个位点相合。预处理选用白消安/环磷酰胺/ATG或全身放疗/环磷酰胺/ATG为主方案。于 0 d 回输脐血，有核细胞中位数为8.51×107/kg，CD34+细胞中位数为1.81×105/kg。预防移植物抗宿主病（GVHD）采用环孢霉素A、甲基泼尼松龙和骁悉或CD25单抗。结果：中性粒细胞绝对值（ANC）≥0.5×109/L和PLT≥20×109/L的中位天数分别是+13 d、+30 d，移植证据均为供者型。4例出现Ⅰ~Ⅲ度GVHD，均控制。随访中位时间12个月，未发生慢性GVHD，现存活4例血型均转为供者型，无复发。结论：脐血提供快速有效的造血干细胞，为治疗儿童白血病提供良好时机，非血缘相关脐血移植能耐受HLA多个位点不相合。急性GVHD发生率也较高，存在移植物抗白血病作用。     

CD+34CD+62L细胞输入量对儿童急性白血病无关脐血移植的影响

目的　研究CD 3 4 CD 62L细胞输入量在无关脐血移植治疗儿童急性白血病中对造血干细胞植入、中性粒细胞和血小板恢复时间的影响。方法　用流式细胞术分析复苏后的CD 3 4 CD 62L细胞数 ,并对 2 3例急性白血病儿童在无关脐血移植后的中性粒细胞和血小板恢复时间等临床资料进行测定。结果　 2 3例患儿中 ,2 1例被植入 ,其中性粒细胞 >5 0 0 / μl的时间为 11～ 2 3d(中位数为 17 5d) ,而在 18例患儿中 ,血小板 >2万 / μl的时间为 12～ 118d(中位数 4 1d)。当CD 3 4 CD 62L细胞输入量 >1 3× 10 5/kg时 ,有利于造血干细胞的植入 ,中性粒细胞的恢复时间与CD 3 4 CD 62L细胞输入量存在一定的相关性趋势 ,γ值为 - 0 32 4 ,0 0 5

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无关脐血移植治疗儿童噬血细胞综合征

目的探讨脐血移植对儿童噬血细胞综合征的治疗效果。方法一例16月龄确诊为噬血细胞综合征的幼儿,经HLH-2004推荐方案进行化疗及免疫治疗7个月后,病情未缓解,行HLA基因位点5/6相合无关脐血移植。预处理采用BU/CY+VP16方案:马利兰(BU)1mg/kg,第6h一次,用4d共16次(-8~-5d);足叶乙甙(VP16)30mg/kg,用1天(-4d);环磷酰胺(CY)60mg/kg,用2d(-3~-2d)。输入脐血有核细胞(NC)5·66×107/kg,CD3+4细胞1·21×105/kg。GVHD预防采用环胞菌素A(CsA)+骁悉(MMF)+抗胸腺细胞球蛋白(ATG)。移植后应用G-CSF加速造血重建。结果脐血造血干细胞未植入,自体造血恢复。移植治疗后,患儿病情逐渐好转。随访至移植后14个月,病情持续完全缓解。结论噬血细胞综合征经化疗和免疫治疗不能缓解者,应及时进行移植治疗;供体细胞植入失败的移植,也有可能暂时或长期缓解病情。     

Unrelated umbilical cord blood transplantation and unrelated bone marrow transplantation in children with hematological disease: A meta-analysis

Abstract:  UCB has been used as an alternative source of HSC. Both unrelated donor BM and UCB are available as potential options for transplantation. However, there have been limited comparisons of the outcomes of unrelated donor UCBT vs. UBMT in the unrelated setting. Our aim is to observe the therapeutic efficacy of UCBT and UBMT for treatment of pediatric hematological diseases. We electronically searched the Cochrane Central Register of Controlled Trials (CENTRAL), MEDLINE, and critically appraised all relevant articles (1989.1–2008.5). Comparative studies were carried out on clinical therapeutic effect of UCBT and UBMT with research on stem cells engraftment, complications, earlier mortality, and survival rate. We performed a meta-analysis using review manager 5.0 software (RevMan, The Nordic Cochrane Center, The Cochrane Collaboration) and adopted funnel plot regression to assess the publication bias. We obtained 324 records. Seven trials totaling 1453 patients have been assessed. Pooled comparisons of studies of UCBT and UBMT in children found that the incidence of engraftment failure and earlier transplantation-related mortality were higher with UCBT because of its delay of hematological recovery [OR = 4.96, 95% CI (3.25, 7.59), p < 0.00001 and OR = 2.36, 95% CI (1.79, 3.11), p < 0.00001 respectively], but CMV infection didn't increase obviously. There was no difference in long disease-free survival rate [OR = 0.85, 95% CI (0.65, 1.01), p = 0.06] between UCBT and UBMT due to the decrease of GVHD in UCBT [OR = 0.45, 95% CI (0.34, 0.60), p < 0.0001]. Our meta-analysis confirmed that UCBT in children is also an effective way to treat children with hematological disease and has equivalent survival outcomes compared with UBMT.     

脐血造血细胞含量与白血病脐血移植疗效分析

目的分析4711份库存脐血造血细胞含量及探讨脐血造血细胞含量与白血病脐血移植疗效的关系。方法分析4711例库存脐血总有核细胞数(TNC)和CD34+细胞数分布情况,探讨不同的造血细胞输入量、供受者HLA不相合数、受者性别、年龄、体重和疾病类型间植入率和生存率的差异。结果 4711例库存脐血TNC和CD34+细胞中位数分别为1.14×109/kg和4.06×106/kg,按3.7×107/kg有效TNC输入量计算,93.2%脐血可供体重50 kg以下受者移植。89例白血病患者移植后植入75例,植入率为84.3%。中性粒细胞绝对值≥0.5×109/L、血小板≥20×109/L和≥50×109/L的时间分别为移植后17、34和46 d。75例植入病例中,长期无病存活47例,死亡26例,2例复发;急性移植物抗宿主病(GVHD)Ⅰ～Ⅱ度、Ⅲ～Ⅳ度和慢性GVHD发生率分别为54.7%、20.0%、9.3%。影响移植植入率的因素包括受者年龄、TNC和CD34+细胞输入量;影响生存率的因素包括受者年龄、体重和输入CD34+细胞数。结论在无法找到HLA全相合骨髓供者时,可选择脐血作为替代骨髓的造血干细胞来源治疗儿童与成人白血病,TNC和CD34+细胞数仍是选择脐血移植物的参考指标。     

Unrelated cord blood transplantation in children with severe congenital neutropenia

Abstract:  SCN is an inherited hematological disorder with severe neutropenia and recurrent infections. Although there are some reports that recombinant rhG-CSF improves clinical outcome, allogeneic HSCT appears to be the only curative treatment for these patients. We report here two children with SCN successfully treated by CBT from unrelated donors. They were refractory to rhG-CSF treatment and have no identical family donor. Bu + CY were given as conditioning. Case 1 and Case 2 received 6/6 and 5/6 HLA-matched unrelated umbilical cord blood, respectively. The number of infused nucleated cells was 6, 18 × 10⁷/kg and CD34⁺  cell number was 3, 74 × 10⁵/kg in Case 1. Those cell numbers were 8, 8 × 10⁷/kg and 5, 34 × 10⁵/kg for Case 2, respectively. Neutrophil/platelet engraftments were 45/49 days in Case 1 and 24/36 days in Case 2. Grade II cutaneous acute GVHD was seen in Case 2 that was treated successfully with prednisolone. Both patients are well with normal hematological findings and full donor chimerism for post-transplant 20 and 24 months, respectively. We conclude that UCB can be considered as a safe source of stem cell in patients with SCN who need urgent HSCT.     

Unrelated donor cord blood transplantation for non‐malignant disorders in children and adolescents

This study analyzes the data reported to the Korean Cord Blood Registry between 1994 and 2008, involving children and adolescents with non‐malignant diseases. Sixty‐five patients were evaluated in this study: SAA (n = 24), iBMFS, (n = 16), and primary immune deficiency/inherited metabolic disorder (n = 25). The CI of neutrophil recovery was 73.3% on day 42. By day 100, the CI of acute grade II–IV graft‐versus‐host disease was 32.3%. At a median follow‐up of 71 months, five‐yr OS was 50.7%. The survival rate (37.5%) and CI of neutrophil engraftment (37.5%) were lowest in patients with iBMFS. Deaths were mainly due to infection, pulmonary complications, and hemorrhage. In a multivariate analysis, the presence of >3.91 × 10⁵/kg of infused CD34 +  cells was the only factor consistently identified as significantly associated with neutrophil engraftment (p = 0.04) and OS (p = 0.03). UCBT using optimal cell doses appears to be a feasible therapy for non‐malignant diseases in children and adolescents for whom there is no appropriate HLA‐matched related donor. Strategies to reduce transplant‐related toxicities would improve the outcomes of UCBT in non‐malignant diseases.     

Unrelated cord blood and bone marrow transplantation in pediatric leukemia

To investigate the role of cord blood as an alternative stem cell source for hematopoietic stem cell transplantation for pediatric acute leukemia, we retrospectively analyzed the outcomes of 35 unrelated cord blood transplantations (UCBT) and 56 unrelated bone marrow transplantations (UBMT) with myeloablative conditioning. The 5 year overall survival (OS) probability was 49.8% (95% confidence interval [95%CI]: 35.6–62.4%) for UBMT and 53.8% (95%CI: 34.0–70.1%) for UCBT (P = 0.92). The 5 year event‐free survival (EFS) probability was 47.3% (33.6–59.8%) for UBMT and 33.0% (15.9–51.2%) for UCBT (P = 0.38). OS and EFS were not significantly different between the groups. On multivariate analysis there was no significant difference between the groups. In conclusion, UCBT can have a role as important as that of UBMT in pediatric acute leukemia.     

Similar outcomes of allogeneic hematopoietic cell transplantation from unrelated donor and umbilical cord blood vs. sibling donor for pediatric acute myeloid leukemia: Multicenter experience in China

In a multicenter study, we have conducted a retrospective study on 73 pediatric AML patients who were primary refractory or in greater than CR1 and investigated MSD (or MMSD) (n = 20), URD (n = 23), and UCB (n = 30) HCT between January 1998 and October 2009. The median day to neutrophil engraftment was similar in all groups. The median day to platelet engraftment was longer in the UCB group. The number of HLA mismatch was higher in the UCB group (p = 0.034); however, the cumulative incidence of grade III–IV aGVHD was not different among all groups (p = 0.125); furthermore, cGVHD was lower in the UCB group (p = 0.078). The risk of relapse did not differ among all groups (RR = 1.28, p = 0.125), but the patients of MSD (or MMSD) grafts had a trend of higher risk recurrence. Sixty‐two patients survived with a median follow‐up of 58.2 months. Five‐yr LFS was 73.1%, 59.8%, and 59.6% for URD, UCB, and MSD (or MMDS), respectively (p = 0.426). Five‐yr LFS in CR1 was 68.9%, with a significantly better result compared to 41.7% in CR2 (p = 0.025). Our comparisons suggest that pediatric AML patients receiving UCB had a higher early TRM, a lower cGVHD rate, and a similar long‐term survival. The outcome of URD and UCB is comparable to that of a suitable sibling for pediatric AML.     

Unrelated donor cord blood transplantation for childhood severe aplastic anemia after a modified conditioning

Treatment of severe aplastic anemia (SAA) patients who lack human leukocyte antigen (HLA)-matched donors and failed immunosuppressive therapy (IST) is challenging. Recently, umbilical cord blood transplantation (CBT) after non-myeloablative therapy has been reported in adult but not in childhood SAA. However, most cases resulted in mixed donor chimerism and incomplete hematological recovery. We reported an 11-yr-old girl with recurred SAA 5 yr after IST who underwent unrelated donor CBT after a modified regimen. This patient had renal and cardiac dysfunction, and lacked suitable bone marrow donors. The 3.9 x 10(7)/kg CB cells from an HLA one-locus mismatched unrelated donor were infused after conditioning with total body irradiation (5 Gy), melphalan (120 mg/m(2)), and fludarabin (120 mg/m(2)). Hematological recovery was favorable in complete chimerism. A major complication was only skin graft-versus-host disease (grade I). CB could be an alternate stem cell source for childhood SAA after modified preparative regimen.     

Donor cell-derived acute myeloid leukemia after second allogenic cord blood transplantation in a patient with Fanconi anemia

DCL following hematopoietic stem cell transplantation has been reported in approximately 5% of all leukemic relapses. There have been several reports on DCL, mainly AML after umbilical cord blood transplantation. In this case study, we present a young boy diagnosed with Fanconi anemia who underwent an umbilical cord blood transplantation. Because of the graft failure, he was retransplanted one month later, also with a cord blood transplant. Two years after the second transplant, he developed AML, where 100% of the cells were of female donor origin. The donor, a now 14-yr-old female, was recently reported healthy.