人体测量指标与儿童血压的相关性研究

目的调查分析人体测量指标对儿童血压的影响。方法随机抽样检查郑州地区6 790名6~13岁儿童,测量体质指数(BMI)、腰围(WC)、臀围(HC)、血压,计算腰围/臀围比(WHR)及腰围/身高比(WHtR),采用SPSS16.0软件进行统计分析。结果儿童高血压检出率为5.57%。控制年龄因素后采用偏相关分析发现,男、女童的BMI、WC、HC、WHtR与收缩压和舒张压均呈显著正相关(P均0.05)。无论男女,高血压组的BMI、WC、HC、WHR和WHtR的水平均高于正常血压组,差异均有统计学意义(P均0.05)。共检出肥胖儿童280名(4.12%),超重622名(9.16%)。肥胖、超重及正常体质量组的高血压比例的差异有统计学意义(P0.01),肥胖组高血压比例高于超重及正常体质量组。肥胖、超重组的收缩压、舒张压水平均高于正常体质量组,差异均有统计学意义(P0.05)。结论郑州地区6~13岁儿童高血压患病率处于同年龄段儿童的中低等水平。BMI、WC、HC、WHtR与男、女童血压具有显著相关性,尤以HC较为显著。     

儿童至成年体质指数变化对成年血压水平的影响

目的 探讨儿童到成年体质指数(BMI)变化对成年血压的影响.方法 对1987年"北京儿童血压研究"队列人群于18年后(2005年)追访,进行身高、体重、血压测量和高血压相关器官功能检测等.结果 ①受试对象儿童期超重肥胖检出率8.7%,有86.1%的超重肥胖儿童成年后仍然超重肥胖;BMI正常的儿童中,成年后超重的比例为25.8%,成年后肥胖的比例为8.8%.②控制年龄、性别,儿童、成年两时期BMI均正常组和儿童期超重或肥胖成年BMI正常组的收缩压(SBP)/舒张压(DBP)均值水平基本一致,明显低于成年超重或肥胖组;儿童至成年持续超重肥胖组成年SBP/DBP均值水平最高,其次为儿童期BMI正常成年肥胖组和儿童期BMI正常成年超重组.③调整儿童时期血压对成年血压的影响,无论儿童期BMI是否正常或超重,只要成年BMI正常后,高血压患病率均明显低于成年超重或肥胖组,其中儿童期BMI正常,成年肥胖者和儿童至成年持续超重肥胖者,成年高血压患病率明显增加.④Logistic同归分析,儿童至成年持续超重或肥胖,成年罹患高血压的风险最强,是两时期BMI都正常者的31.5倍(95%CI=7.5～132.8);儿童期BMI正常,成年肥胖或成年超重,高血压患病风险办明显增强,分别为是两时期BMI都正常者的22.9倍(95%CI=6.0～86.6)和6.0倍(95%CI=1.8～19.9);儿童期曾经超重肥胖,成年BMI转为正常者成年高血压患病风险与两时期BMI都正常者比较为零.结论 儿童期曾经超重肥胖,成年BMI恢复正常后,血压仍可恢复正常;儿童到成年期持续超重肥胖以及BMI的过快增长均可导致成年血压水甲以及高血压患病率增加,因此,防治儿童期超重、肥胖,尤其是预防儿童期超重肥胖向成年延续以及由儿童至成年BMI的过快增长是预防成人高血压的重要措施和有效手段.     

北京市7～17岁儿童青少年血压与肥胖状态的关系分析

目的探讨北京市7～17岁儿童青少年血压与肥胖状态[依据不同划分标准:体重指数(BMI)、腰围(WC)、体脂百分比(FMP)]的关系。方法选取2004年4月至10月进行的"北京市儿童青少年代谢综合征研究"(BCAMS)中19488名7～17岁儿童青少年(男9824名,女9664名)为研究对象,按照BMI、WC、FMP标准分别划分儿童青少年超重和肥胖状态,分析其血压与BMI、WC、FMP关系,高血压状态的变化及趋势。结果各种划分标准中血压按正常、超重和肥胖组顺序依次升高;控制年龄和性别后,BMI、WC、FMP与收缩压(SBP)和舒张压(DBP)成独立正相关关系(P0.001);BMI、WC、FMP超重组和肥胖组的高血压发生率显著高于正常组,差异有统计学意义(P0.001),肥胖组患高血压的相对危险度是正常组的3～7倍;BMI分组分别与FMP、WC分组组合,BMI肥胖,FMP肥胖组及BMI肥胖,腹型肥胖组患高血压风险分别是正常组的7.3和6.8倍。结论儿童青少年BMI、WC和FMP与SBP和DBP密切相关,用BMI指标预测儿童青少年高血压风险较WC及FMP更敏感;儿童青少年超重和肥胖增加高血压的发生风险,其高血压发生率随着肥胖程度增加呈现成倍上升趋势。     

4岁儿童体重状态与早期血压、心脏结构功能改变的研究

目的探讨4岁儿童体重状态与早期血压、心脏结构功能改变的相关性。方法在队列研究平台上, 通过横断面分析儿童体重状态和心血管指标的关系, 于2017至2020年在由上海新华医院牵头的"上海优生儿童队列"平台中, 对1 477名4岁儿童进行身高、体重、血压测量和心脏超声检查。根据不同体质指数(BMI)进行分类, 将研究对象分为5组(消瘦、体重过轻、正常、超重及肥胖), 利用单因素方差分析比较不同组间血压水平及心脏结构功能指标, 通过多因素线性回归模型分析儿童血压及心脏结构功能与体重状态之间的关联, 并采用多因素Logistic回归模型分析不同体重状态是否为儿童早期血压升高和左心室肥厚的独立危险因素。结果纳入的1 477名儿童中男772名、女705名。男童超重及肥胖115例(14.9%), 女童超重及肥胖68例(9.6%)。正常体重儿童916名(62.0%), 消瘦75例(5.1%), 体重过轻303例(20.5%), 超重130例(8.8%), 肥胖53例(3.6%)。各组对比发现, BMI越高, 4岁儿童的收缩压、舒张压、左心室质量指数、左心室后壁收缩期厚度、左心室后壁舒张期厚度、左心室收...     

上海地区7 326名在校学生高血压分布趋势及相关因素分析

目的了解上海城区中小学生血压现况、高血压检出率，为预防儿童高血压提供依据。方法测量上海城区11所中小学7326名6-18岁在校学生身高、体重、血压。结果在校学生高血压总检出率为6．9％（502／7326例），高血压检出率在体质指数（BMI）正常学生中仅为4．8％（303／6053例），在超重和肥胖学生分别为8．8％（84／946例）、17．9％（113／630例）；超重和肥胖学生高血压检出率显著高于BMI正常者（χ^2=98．9、78、7，P均〈0．01）；上海地区6—18岁儿童青少年血压与BMI独立正相关。结论在校儿童青少年超重和肥胖者高血压的危险性明显增加，上海地区6—18岁在校学生血压与BMI独立相关。防治儿童青少年超重和肥胖是预防高血压的重要措施。     

不同参考标准下重庆城区儿童超重／肥胖检出率比较

目的比较3种筛查标准下重庆城区儿童超重/肥胖现况,寻求适宜的儿童肥胖筛查标准。方法按照整群抽样原则抽取本市3个城区的12534名2～18岁儿童作为调查对象,测量其身高及体质量。并分别采用WHO的身高标准体质量法标准(标准1)、国际肥胖工作组(IOTF)的超重/肥胖体质量指数(BMI)标准(标准2)和国立卫生统计中心/疾病预防控制中心(NCHS/CDC)的BMI标准(标准3)评价儿童的超重/肥胖。结果按照标准1、2和3的规定,重庆城区儿童的超重检出率分别为13.0%、9.8%、9.8%;肥胖检出率分别为9.8%、2.9%、6.0%,均有显著性差异(Pa<0.001),且男童超重/肥胖检出率均高于女童。3种标准间,健康儿童、超重儿童或肥胖儿童的BMI均值均有显著性差异(Pa<0.001)。采用标准1和2得到的超重检出率,或采用标准1和3得到的超重检出率均在6岁前和14岁后2个年龄段存在统计学差异(Pa<0.01)。在2～6岁各年龄组,采用标准1和3得到的儿童肥胖检出率比较无显著性差异;在6～18岁各年龄组,采用标准1得到的肥胖检出率均显著高于采用标准2和3得到的肥胖检出率(Pa<0.01);在3～6岁、8～10岁、13岁组采用标准2和3得到的儿童肥胖检出率比较有显著性差异。结论儿童肥胖已经成为重庆城区重要的健康问题之一。采用3种标准筛查的儿童超重/肥胖检出率在2～18岁各年龄组均有所不同,应谨慎使用。     

广西南宁儿童青少年高血压现状及其与肥胖关系的研究

目的 了解广西南宁地区6~18 岁儿童青少年高血压现状及超重/ 肥胖与高血压发生的关系。方法 采用随机整群抽样方法对南宁地区7 893 名 6~18 岁儿童青少年进行问卷调查并测量血压、身高、体重等生长发育指标。结果 男女生收缩压（SBP）和舒张压（DBP）均随着年龄的增长呈递增趋势。高血压、高收缩压（HSBP）、高舒张压（HDBP）检出率分别为6.58%、4.02%、3.81%,其中男生高血压、HSBP 检出率明显高于女生（P<0.05）。正常组、超重组、肥胖组高血压检出率分别为3.87%、9.84%、19.23%。高血压、HSBP 及HDBP 检出率均表现为肥胖组> 超重组> 正常组 （P<0.05）。与正常组相比,超重组、肥胖组高血压发生的相对危险度（95%CI）分别为2.71（1.69~5.96）和5.91（3.46~7.63）。血压与年龄、身高、体重及体重指数均呈正相关（P<0.01）。结论 广西南宁6~18 岁儿童青少年血压的分布特征为：血压值与儿童青少年的性别、年龄、身高、体重和体重指数相关; 肥胖与高血压患病率密切相关,随着体重的增加,儿童青少年患高血压的危险亦增加。     

两种评价方法对3912例儿童青少年超重及肥胖检出率的比较

目的 评价身高标准体重法(weight-for-height,WFH)和中国儿童体质指数(body mass index,BMI)法对儿童青少年超重及肥胖检出率的吻合程度.方法 于2006年8-10月采用分层整群随机抽样方法抽取上海市徐汇区3所小学和2所中学7～15岁儿童、青少年3 912人,其中男性2 070人,女性1 842人,分别用上述两种方法检测超重及肥胖儿童的人数,计算检出率并进行比较.结果 采用WFH及BMI标准对肥胖及超重的检出率分别为27.7%和26.5%,差异无统计学意义(x2=1.3683,P>0.05).两种方法对儿童青少年超重及肥胖的检出一致性较好(Kappa=0.70,P=0.013);并且均显示男性超重及肥胖率高于女性(WFH男:女=31.82%:21.72%;BMI男:女=30.82%:21.99%),超重比例高于肥胖(WFH 超重:肥胖=14.9%:12.9%BMI超重:肥胖=14.4%:12.1%),青春前期高于青春发育期.结论 WFH和BMI检测儿童、青少年超重及肥胖具有较好的一致性,两者均可应用于对儿童及青少年营养状况的评价.     

瘦素和脂联素在儿童肥胖相关性高血压发病中的作用

目的探讨瘦素和脂联素在儿童肥胖相关性高血压发病中的作用。方法基于北京市儿童青少年代谢综合征研究项目的现况调查结果，非随机选择3 502名6~18岁学龄儿童（其中男1 784名，女1 718名）为研究对象，按照超重（包括肥胖）和高血压状态将研究对象分为4组，正常体重正常血压组（对照组，1 497名）、正常体重高血压组（HBP组，125名）、超重但血压正常组（OB组，1 349名）和超重合并高血压组（OB+HBP组，531名）。通过比较4组人群血清瘦素和脂联素水平，以及瘦素和脂联素与血压之间的相关回归分析，探讨其与肥胖和血压之间的关系。结果超重肥胖人群BMI、血压、胰岛素和瘦素水平显著升高，脂联素水平降低。HBP组与对照组BMI、瘦素、脂联素水平差异无统计学意义。OB组和OB+HBP组与对照组比较，BMI、SBP、DBP、胰岛素和瘦素水平升高，脂联素水平降低，与HBP组比较仍可见BMI、胰岛素和瘦素水平升高，脂联素水平降低。与OB组比较，OB+HBP组BMI和胰岛素水平及男性的瘦素水平明显升高。血压与年龄、BMI、胰岛素、瘦素均呈显著正相关（r=0.260~0.643,P<0.01），与脂联素呈显著负相关（r=-0.171~-0.332, P<0.01）。但在调整胰岛素或BMI后，瘦素、脂联素与血压的相关性减弱或消失。结论 超重人群血压、胰岛素及瘦素水平均高于对照人群，脂联素水平低于对照人群。瘦素、脂联素可能通过肥胖或胰岛素抵抗与血压相关。     

重庆市丰都县学龄儿童高血压发生与营养的关系

目的探讨重庆丰都县学龄儿童高血压的发生与营养的关系。方法采用整群随机抽样方法,选取重庆丰都县2所公立小学且户籍为该学校所在街道的8 033名儿童作为研究对象,进行身高、体重和血压测定,采用半定量食物频率问卷进行膳食调查,分析体重指数(BMI)、膳食营养素与儿童高血压发生的关系。结果最终纳入7 538名儿童进行分析,肥胖、超重、高血压检出率分别为9.11%、12.27%、11.83%;肥胖、超重儿童中高血压检出率分别为33.62%、17.84%,分别是正常儿童的4.02、2.13倍。Logistic多因素逐步回归分析显示,钙、钠摄入增加会增加高血压发生的风险(分别OR=1.003、1.002),而铁、单位体重钙摄入增加会降低高血压发生的风险(分别OR=0.979、0.926)。结论重庆丰都县学龄儿童高血压、肥胖流行趋势严峻,BMI和膳食营养素与儿童高血压发生密切相关,需积极控制儿童体重、调整饮食结构、限制钠摄入,以降低儿童高血压的发生。     

Resurgence of measles in Singapore: profile of hospital cases

OBJECTIVE: To ascertain the profile of cases of measles seen at a general hospital during a recent outbreak that occurred despite a measles vaccination program. METHODOLOGY: A retrospective study from January 1991 to March 1998. All patients with measles (ICD code 055. 9) seen at the emergency unit or as inpatients were included. RESULTS: There were 87 cases identified. The diagnosis was clinical in all and proven serologically in 71%. Eighty-five per cent of the cases occurred between January 1997 and March 1998. There was a bi-modal age distribution with peaks in the very young (相似文献   

Allergic factors associated with the development of asthma and the influence of cetirizine in a double-blind, randomised, placebo-controlled trial: First results of ETA®

There is a common progression known as the allergic march from atopic dermatitis to allergic asthma. Cetirizine has several antiallergic properties that suggest a potential effect on the development of airway inflammation and asthma in infants with atopic dermatitis. Methods. Over a two year period, 817 infants aged one to two years who suffered from atopic dermatitis and with a history of atopic disease in a parent or sibling were included in the ETAC^® (Early Treatment of the Atopic Child) trial, a multi-country, double-blind, randomised, placebo-controlled trial. The infants were treated for 18 months with either cetirizine (0.25mg/ kg b.i.d.) or placebo. The number of infants who developed asthma was compared between the two groups. Clinical and biological assessments including analysis of total and specific IgE antibodies were performed. Results. In the placebo group, the relative risk (RR) for developing asthma was elevated in patients with a raised level of total IgE (≥ 30 kU/I) or specific IgE (≥ 0.35 kUA/I) for grass pollen, house dust mite or cat dander (RR between 1.4 and 1.7). Compared to placebo, cetirizine significantly reduced the incidence of asthma for patients sensitised to grass pollen (RR = 0.5) or to house dust mite (RR = 0.6). However, in the population that included all infants with normal and elevated total or specific IgE (intention-to-treat - ITT), there was no difference between the numbers of infants developing asthma while receiving cetirizine or placebo. The adverse events profile was similar in the two treatment groups. Discussion. Raised total IgE level and raised specific IgE levels to grass pollen, house dust mite or cat dander were predictive of subsequent asthma. Cetirizine halved the number of patients developing asthma in the subgroups sensitised to grass pollen or house dust mite (i.e. 20% of the study population). In view of the proven safety of the drug, we propose this treatment as a primary pharmacological intervention strategy to prevent the development of asthma in specifically sensitised infants with atopic dermatitis.     

A profile of respiratory symptoms in urban and rural South Australian school children

This report describes the cross-sectional analyses of data from the first year of a longitudinal study using questionnaire and respiratory function data over a 5 year period from a sample of rural South Australian school children. The cumulative or lifetime prevalences of respiratory symptoms were estimated in 825 rural and 1261 urban school children aged between 5 and 15 years in order to determine if the prevalence rates differed between rural and urban school children. The study found the overall cumulative prevalence of asthma and/or wheezy breathing (AWB) to be 24.1% in the rural school children compared to 27.6% in the urban school children. Most children developed AWB symptoms before the age of 7 years, with 20% reporting moderately severe symptoms and 10% having more than one attack per fortnight. The cumulative prevalence of bronchitis, loose/rattly cough (BLRC) differed significantly between the rural school children (34.1%) and urban school children (47.9%). The BLRC symptoms preceded the development of AWB in many cases. Urban school children also reported a higher prevalence of atopic conditions.     

Hauttemperaturen verschiedener Körperoberflächenareale des Rumpfes bei Säuglingen

Summary In two groups of infants (3–53 weeks old) skin temperatures were controlled in different areas of the trunk—i.e.: regions of sternum, lungs, heart, liver, spleen, kidneys—at different room-temperatures (group I: 21–25°C; group II: 29–32°C). Rectal temperatures of some probands in both groups also had been controlled simultaneously. A definite change in the reaction to heat was proofed in different periods of the first year of life. In higher environmental temperatures the skin temperature was almost constant at every controll-point of the skin, even in older infants. In lower environmental temperatures the skin temperatures lowered continuously with age till 7. to 9. moth. From 10. to 12. month the lowering of skin temperature discontinued. The rectal temperatures were relatively constant in all infants. Only in infants from 7. to 12. month, whose skin temperatures were controlled in lower as well as in higher environmental temperatures, a tendency to higher rectal temperatures was proofed in warmer environmental temperatures.The significance of these results is discussed.

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Untersuchungen mit Unterstützung durch die Deutsche Forschungsgemeinschaft.     

Psychopathologie du syndrome d’Asperger et « aspérigisation » de la société contemporaine

The author has attempted here to point out, just for a start, the characteristics of Asperger syndrome from the point of view of psychopathology through a rereading of Hans Asperger's original paper (1944). This thesis merits reevaluation, if for no other reason than to fill the gaps in operational diagnostics based on the DSM. It is found by rereading that Asperger's view of the principal disturbances of autistic psychopathy include a “disturbance of natural evidence” or a “crisis of common sense”. This question of natural evidence that he evokes with regard to autistic psychopathy corresponds to W. Blankenburg's natural evidence, which constitutes a key concept for comprehending schizophrenia in the form poor-symptom (“symptomarme Schizophrenie”) that he observes in the speech of his patient Anne Rau. One can deduce from this that in terms of fundamental disturbances, Asperger syndrome and this “symptom-poor” schizophrenia overlap at the level of loss of natural evidence. It is moreover possible to classify Asperger syndrome among the disturbances of spacing in the sense meant by the evolutionary psychiatry of A. Stevens and J. Price. The author then develops our comprehension of Asperger syndrome from the point of view of the perspective proposed by the notion of resilience in people with Asperger syndrome and of the possibility for them, through these mechanisms of adaptation, to find in the organization of the personality of the “as if” type a position of relative equilibrium. They concur or overlap in the creation of crutches, of borrowed personalities secondarily legitimated by the reaction of the socius. This will end up in the production of inventions and œuvres (works). Clearly, one rarely encounters several cases that one could consider pertinently to be “successful” Asperger syndrome. Finally, the author notes that one can find a sort of isomorphism between Asperger syndrome and contemporary society when he proposes the term “asperigisation” to characterize our society, given that the equilibrium between emotion and logic is strongly disturbed in these patients, in whom logic undergoes hypertrophy while emotion is impoverished. From this perspective, the author hopes to suggest reasons for the increase in the number of cases of Asperger syndrome in the clinical setting and in society in general in our contemporary era.     

Bibliometric data: a disaster for many non-American biomedical journals

Bibliometric data published by the Institute of Scientific Information in Philadelphia (ISI), and which was previously discussed in Acta Paediatrica , has increasingly been used despite all the relevant and severe criticism that has been raised against this method of evaluating individual research results and grading scientific journals. It is obvious that the present trend regarding the use of bibliometric data as a basis for priorities and funding of research and for the promotion of individual scientists favours American-oriented research projects at the expense of those that are based on concepts of predominantly European relevance.

Conclusion: For the future of non-American research, it is important that no single super-power, i.e. the USA, should dominate scientific priorities. The condition for efficient European competition is that European Centres with high levels of competence for creative research and training of scientists from all over the world are established. In addition, it is important that the results of European research are published in prestigious European journals, as was the situation before World War II.     

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孤独症谱系障碍(autistic-spectrum disorders,ASDs)近年来患病率逐年攀升至1%左右,其症状往往伴随终生,成为严重威胁儿童健康和发展的神经发育性疾患;注意缺陷多动障碍(attention deficit hyperactivity disorder,ADHD)是儿童期最常见的精神障碍,国内报道患病率为4.13%～5.83%,其症状可延续至青少年期,甚至到成年期[1]。这两类精神障碍在成年期的临床表现、共患病、治疗策略和预后与儿童期有哪些不同呢?本文通过回顾相     

Infiltrations of Epstein-Barr virus-carrying cells in granular lymphocyte-proliferative disorders corresponding to chronic active Epstein-Barr virus infection

A 21-year-old man with granular lymphocyte-proliferative disorders (GLPD) associated with chronic active Epstein-Barr virus (EBV) infection is described. Chromosomal analyses revealed several clonal abnormalities and two of them were mainly repetitious. High copy numbers of monoclonal EBV genome were also detected in the proliferative large granular lymphocytes (LGLs), indicating the monoclonal expansion of EBV-infected LGLs. The patient had an indolent course for several years, and there was no evidence of infiltrations of his bone marrow until the end stage. At autopsy, microscopic studies revealed marked infiltrations of LGL in the liver and spleen, and the infiltrating cells were NK-cell immunophenotype. The infiltrated LGLs showed latency I.     

LUTEINIZING HORMONE RECEPTOR MUTATIONS IN DISORDERS OF SEXUAL DEVELOPMENT AND CANCER

Human male sexual development is regulated by chorionic gonadotropin (CG) and luteinizing hormone (LH). Aberrant sexual development caused by both activating and inactivating mutations of the human luteinizing hormone receptor (LHR) have been described. All known activating mutations of the LHR are missense mutations caused by single base substitution. The most common activating mutation is the replacement of Asp-578 by Gly due to the substitution of A by G at nucleotide position 1733. All activating mutations are present in exon 11 which encodes the transmembrane domain of the receptor. Constitutive activity of the LHR causes LH releasing hormone-independent precocious puberty in boys and the autosomal dominant disorder familial male-limited precocious puberty (FMPP). Both germline and somatic activating mutations of the LHR have been found in patients with testicular tumors. Activating mutations have no effect on females. The molecular genetics of the inactivating mutations of the LHR are more variable and include single base substitution, partial gene deletion, and insertion. These mutations are not localized and are present in both the extracellular and transmembrane domain of the receptor. Inactivation of the LHR gives rise to the autosomal recessive disorder Leydig cell hypoplasia (LCH) and male hypogonadism or male pseudohermaphroditism. Severity of the clinical phenotype in LCH patients correlates with the amount of residual activity of the mutated receptor. Females are less affected by inactivating mutation of the LHR. Symptoms caused by homozygous inactivating mutation of the LHR include polycystic ovaries and primary amenorrhea.     

EXCITING NEW TREATMENT APPROACHES FOR PATHYPHYSIOLOGIC MECHANISMS OF SICKLE CELL DISEASE

During the past several decades, our understanding of the complex pathophysiology of vasoocclusion associated with sickle cell disease has improved greatly. Interaction of genes, hemoglobin molecules, red cell membrane and metabolic changes, cell-cell interactions and cell-plasma interactions, red cell adhesion to vascular endothelium, activation of coagulation, and vascular reactivity play a role in vaso occlusion. Penicillin prophylaxis of pneumococcal infections and appropriate use of blood transfusions and other supportive measures improved survival of sickle cell patients. Hydroxyurea made a major impact on sickle cell therapy when it was shown to decrease acute painful episodes, acute chest syndrome, and the need for blood transfusion in adults. Significant experience in the use of hydroxyurea has been accumulated in older children. The benefits and risks of hydroxyurea for younger children and long-term risks in all patients will be evaluated in future investigations. Other promising therapies include butyrate compounds, clotrimazole, magnesium supplementation, poloxamer 188, antiadhesion agents, anticoagulant approaches, and nitric oxide. Hemopoietic transplantation remains the only curative therapy. However, several transgenic mouse models are available for studies of gene therapy or other treatment approaches on biochemical, cellular, and pathologic effects of mutant genes.