脐血移植治疗儿童恶性血液病的临床研究

目的观察脐血移植(CBT)治疗儿童血液系统恶性疾病的植入率、移植相关并发症和生存情况。方法血液系统恶性疾病患儿19例,其中无关供者16例,同胞供者3例。脐血与患儿HLA6个位点全相合6例,1个位点错配10例,2个位点错配3例。移植时疾病状态,高危10例,标危9例。预处理主要采用Bu/Cy或Cy/TBI方案,同时应用抗胸腺球蛋白(ATG)。GVHD预防主要采用环孢菌素A(CsA)、骁悉(MMF)及甲基泼尼松龙(MP)三联方案,3例同胞供者移植则单用CsA预防GVHD。移植脐血的细胞数量是,有核细胞(TNC)5.96×107/kg(2.57～12.20),CD34+细胞2.20×105/kg(0.31～5.80)。结果18例成功重建粒系造血,中位重建时间17(11～35)天;15例血小板植入,中位重建时间38(25～112)天。发生急性GVHD9例,其中Ⅰ度～Ⅱ度7例,Ⅲ度～Ⅳ度aGVHD2例;慢性GVHD3例。移植后随访4～86个月,复发3例,死亡8例,其中移植相关死亡6例,移植相关死亡率(TRM)31.6%。因CMV感染导致间质性肺炎死亡4例,占移植相关死亡的66.7%(4/6)。2年总生存率(OS)和无事件生存率(EFS)分别为62.54%(±11.24%)、44.53%(±12.22%);标危移植组与高危移植组病人2年OS分别为88.89%(±10.48%)、40.00%(±15.49%)。结论本组儿童血液系统恶性疾病脐血移植的植入率、移植相关并发症和生存情况与国外文献报道相似,移植时疾病状态为标危时治疗效果较好,CMV感染所致间质性肺炎是移植相关死亡的主要原因。     

连续性血液净化治疗儿童暴发性心肌炎疗效观察

No abstract available

     

Cord blood transplant in childhood ALL

BACKGROUND: Optimal therapy for high risk and relapsed acute lymphoblastic leukemia (ALL) remains uncertain. Wider availability of cord blood from related and unrelated donors has prompted studies of its use for hematopoietic stem cell transplant (HSCT). PROCEDURE: We evaluated 26 consecutive cord blood transplants (CBT) for ALL performed at our center from 1996 to 2002 on studies using consistent conditioning therapy and graft-versus-host disease (GVHD) prophylaxis. Median patient age was 8.5 years (range, 0.5-24 year). Cord blood (CB) was from unrelated donors in 25/26 cases. Median CB nucleated cell dose was 3.26e7/kg (range, 0.8-12.9). RESULTS: With median follow-up of 548 days, 16/26 patients (62%) are event-free survivors. Acute GVHD developed in 14/24 evaluable patients, reaching grade III-IV in 7 patients. Chronic GVHD occurred in 10/22 evaluable patients. Multivariate analysis showed higher total nucleated cell dose per kilogram to be the strongest predictor of event-free survival. CONCLUSIONS: We conclude that CBT can effectively treat ALL in children with high risk features and following relapse.     

Defects in bile acid biosynthesis--diagnosis and treatment

Bile acid synthetic defects represent a specific category of metabolic liver disease. This article highlights the history and summarizes our analytical approach to the diagnosis and treatment of genetic defects in bile acid synthesis. By the application of mass spectrometry as a screening tool, it is possible to perform rapid diagnosis of potential inborn errors in bile acid synthesis from urinary bile acid analysis. Molecular techniques then afford the identification of specific mutations in genes encoding the enzymes responsible for bile acid synthesis. Using this approach, 6 of the 7 known genetic defects that are causes of progressive cholestatic liver disease, syndromes of fat-soluble vitamin malabsorption, or neurological disease, have been characterized. Bile-acid therapy using oral cholic acid has proven effective in most of these bile acid synthetic defects making early diagnosis crucial to optimum clinical prognosis.     

New blood pressure lowering drugs in childhood

The treatment by antihypertensive agents has recently been improved by the introduction of angiotensin converting-enzyme inhibitors, and of calcium antagonists, which have been applied successfully in children with acute and chronic hypertension. The converting enzyme inhibitor captopril is used increasingly for the treatment of severe forms of persistent hypertension in children. Side effects from this drug can largely be avoided by using low daily doses. The calcium-antagonist nifedipine can now be regarded as the drug of choice in the treatment of acute elevation of blood pressure in infants and children. For this indication it appears to be superior to other drugs because of its prompt and safe efficacy after sublingual administration.     

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现将2006年我国儿童血液系统疾病的临床进展简述如下。1白血病白血病是高度异质性疾病,系列交叉抗原表达、抗原表达不同步、抗原过度表达、抗原表达缺乏和(或)异位抗原表达较为常见。帖立军等[1]对伴有髓系抗原表达的急性淋巴细胞白血病(my+ALL)和伴有淋系抗原表达的急性髓系白血病(Ly+AML)两组患儿进行临床、生物学特征及治疗相关因素分析,结果提示,174例ALL患儿中,my+ALL与my-ALL患儿除在B-ALL组达到完全缓解(CR)时间差异有显著性外(P<0.05),其他临床、生物学特征及治疗反应差异均无显著性(P>0.05),两组患儿5年无事件生存(EFS)…     

The acute crisis of blood pressure and its treatment in childhood (authors transl)]

Hypertension emergencies in childhood occur mainly in end-stage renal disease. In the management the intravenous administration of diazoxide (3--5 mg/kg) is of special importance. Diazoxide results in an immediate and prolonged effect in lowering the blood pressure in 15 children. Serious side effects were not observed. The experiences with the use of diazoxide in the treatment of hypertensive emergencies is discussed.     

Current state of cord blood transplantation in childhood

In 1989 E. Gluckman reported the successful cord blood transplantation in a boy with Fanconi anemia. Since then more than 1500 allogeneic cord blood transplantations have been performed worldwide. This has been possible because non-profit cord blood banks have been established that provide cryopreserved cord blood products from unrelated donors. However, cord blood transplantation is associated with specific risks that have sofar limited its more widespread use. Its main problem is the limited stem cell dose that is associated with a long aplasia and a high rate of engraftment failure. Therefore, cord blood is used as the stem cell source in only about 1 - 2 % of stem cell transplantations in childhood. The main advantage of cord blood transplantation lies in its low risk for graft-versus-host-disease (GvHD), one of the major causes for posttransplant morbidity and mortality particularly with unrelated stem cell donors. The low risk for GvHD is attributed to the low number of transplanted T cells and their functional immaturity. Another advantage of cord blood transplantation lies in the immediate availability of the cord blood units. Based on the experiences with allogeneic cord blood transplantation the indications for cord blood donation will be discussed.     

血液净化技术在儿童肾脏疾病中的应用

血液净化技术是从单纯超滤发展而来的一种血液净化疗法,其应用远远超出了肾脏病的范畴,已被用于各种原因引起的机体内环境紊乱的治疗.文章主要介绍儿童血液净化技术包括建立良好的血管通路、滤器的选择、何时需要进行体外循环预充、抗凝的方法、血液净化的参数凋节及血液净化压力监测,同时阐述血液净化技术在儿童肾脏疾病中的治疗现状及进展.     

Weight gain in childhood and blood lipids in adolescence

Aim: To assess the effect of weight gain in childhood on blood lipid levels in adolescence.
Methods: A population-based birth cohort carried out in Pelotas, Southern Brazil. All newborns in the city's hospitals were enrolled in 1982. The subjects have been followed up for several times in childhood. At age 18, 79% of all males were followed, and 2083 blood samples were available. Adjusted analyses controlled for household assets index, family income, parental schooling at birth, maternal smoking during pregnancy and breastfeeding duration.
Results: Birth weight for gestational age and weight gain in the first 20 months was not associated with blood lipid levels in adolescence. On the other hand, those subjects whose weight gain from 20 to 42 months of age was faster than that predicted from birth weight and weight-for-age z-score at the mean age of 20 months had lower high-density lipoprotein cholesterol (HDL) cholesterol [−0.78 (95% confidence interval: −1.28; −0.29)] and higher very low-density lipoprotein cholesterol (VLDL) and low-density lipoprotein cholesterol (LDL)/HDL ratio in adolescence. After controlling for current body mass index (BMI), the regression coefficient for HDL cholesterol decreased from −0.78 mg/dL to −0.29 mg/dL (95% confidence interval: −1.00 to 0.05).
Conclusion: Weight gain from 2 to 4 years is related to an atherogenic lipid profile in adolescence and this association is mediated by current BMI.