新生儿单纯肠穿孔临床分析

目的分析新生儿单纯肠穿孔的发病机制、临床特点、外科治疗以及预后。方法对1990年1月至2005年12月本院新生儿外科收治的18例单纯肠穿孔(IIP)以及16例坏死性小肠结肠炎(NEC)肠穿孔进行回顾性对比研究。结果与NEC穿孔相比,IIP常发生于胎龄较大、出生体重较高的新生儿。IIP平均发病年龄为(20·0±17·8)d,晚于NEC组[(7·8±2·6)d,P<0·05],发病形式较缓,从发病至肠穿孔平均时间为(5·0±4·1)d[NEC组(2·6±2·0)d,P<0·05]。50%病例穿孔前合并腹泻、肠炎或其他炎性疾病的病史,穿孔以回盲部(60%)及横结肠脾曲(22%)为多见。其病死率为16%(NEC组56%,P<0·05),预后明显好于NEC穿孔组。结论新生儿单纯肠穿孔为区别于NEC穿孔的独立疾病,有不同发病机制和治疗预后。     

新生儿肠穿孔101例临床分析

目的分析新生儿肠穿孔的临床特点,为改善新生儿肠穿孔的预后提供理论依据。方法回顾性分析2000年1月至2014年6月入住新生儿重症监护病房的101例新生儿肠穿孔患儿的临床资料。结果新生儿肠穿孔的主要病因是新生儿坏死性小肠结肠炎(NEC,41例,40.6%),其次为特发性肠穿孔(17例,16.8%)、先天性巨结肠(10例,9.9%)。特发性肠穿孔组患儿平均出生体重和平均胎龄明显高于NEC组(P0.05);NEC组致病菌以肠球菌为主,特发性肠穿孔组以革兰阴性菌为主,两组病原菌分布不同(P0.05)。Logistic多元回归分析显示,酸中毒、多部位肠穿孔、穿孔至手术时间较长是新生儿死亡的独立危险因素。结论新生儿肠穿孔病因多样,以NEC为主;NEC所致肠穿孔与特发性肠穿孔具有不同的致病菌,两者可能是相互独立的疾病;早期诊断、尽早手术是挽救新生儿肠穿孔患儿生命的主要措施。     

新生儿胃肠穿孔80例临床特点及预后分析

目的分析新生儿胃肠穿孔的病因、临床特征、治疗及预后。方法回顾性分析2004年1月至2015年12月复旦大学附属儿科医院新生儿科收治的80例新生儿胃肠穿孔患儿的临床资料,根据出生胎龄分为早产儿组与足月儿组,比较两组间的病因、临床表现、治疗及预后。结果 80例新生儿胃肠穿孔中,早产儿62例,足月儿18例。两组病因均以坏死性小肠结肠炎(necrotizing enterocolitis,NEC)为主,临床均以腹胀为主要表现,早产儿发生反应差、休克、弥散性血管内凝血明显多于足月儿(P0.05)。早产儿组平均发病日龄9(1.75,20)d,足月儿组平均发病日龄4.5(1,7.75)d。62例手术治疗,其中胃穿孔8例,肠穿孔54例,18例因未手术穿孔部位不明确。死亡32例,病死率40%,早产儿死亡26例(病死率41.9%),足月儿死亡6例(病死率33.3%)。结论新生儿胃肠穿孔是新生儿期的严重疾病,病死率高。早期诊断、积极治疗和尽早外科干预可能提高患儿的存活率,显著改善患儿的预后。     

新生儿坏死性小肠结肠炎与自发性肠穿孔临床对比分析

目的分析新生儿坏死性小肠结肠炎(NEC)与自发性肠穿孔(SIP)的临床特征。方法回顾分析1996年5月至2016年8月收治的NEC发生肠道穿孔以及同期收治的SIP患儿临床资料,比较两组患儿围生期相关指标,主要合并症或并发症,治疗结局等。结果共纳入101例肠穿孔患儿,NEC组70例、SIP组31例。两组患儿的性别比、生产方式、胎龄、出生体质量、窒息、宫内窘迫、早产和低体质量比例,以及发病日龄、住院天数方面的差异均无统计学意义(P0.05)。NEC组患儿病死率高于SIP组,合并或并发低蛋白血症以及败血症的比例均高于SIP组,差异均有统计学意义(P0.05)。NEC组最常见穿孔部位为大肠,其次为小肠;SIP组最常见穿孔部位为小肠,其次为大肠;NEC组与SIP组患儿穿孔部位及穿孔数量比较,差异均无统计学意义(P0.05)。结论与SIP患儿相比,NEC患儿更容易罹患败血症及低蛋白血症,病死率也更高。     

新生儿破伤风应用静脉丙种球蛋白的免疫效果

探讨国产人血静脉丙种球蛋白(ⅣIG)辅助治疗新生儿破伤风的临床疗效及免疫功能.将40例新生儿破伤风患儿按病例编号分为常规组和ⅣIG组各20例.常规组采用破伤风抗毒素及安定类药物治疗;ⅣIG组在常规治疗组方案基础上加用ⅣIG(400mg/kg·d,静脉点滴,每日一次)共3天.并设20例健康新生儿作对照组.检测血清IgA、IgG、IgM和外周血T淋巴细胞亚群.比较实验前后的临床疗效和免疫功能变化.结果显示①新生儿破伤风患儿血清IgG、CD3、CD4水平明显低于健康对照组;②静脉注射ⅣIG后,可迅速提高新生儿破伤风患儿血清IgG水平(5.86±0.62vs 11.71±1.46)g/L,同时可提高CD4细胞数(32.55±2.51vs44.23±4.87)和CD4/CD8比值(1.30±0.19 vs 2.10±0.11),P均＜0.001;③ⅣIG组治愈率为85%,明显高于常规组55%,(x2=4.28,P＜0.05).而平均治愈天数由(14.55±2.78)d缩短为(10.50±2.54)d(t=4.13,P＜0.001).病死率由20%降至10%.结论免疫功能紊乱可能与新生儿破伤风有关.适宜的ⅣIG剂量辅助治疗新生儿破伤风,疗效肯定,副作用少,值得临床推广.     

影响新生儿坏死性小肠结肠炎预后的危险因素

目的 分析与新生儿坏死性小肠结肠炎(NEC)预后不良有关的因素,为改善NEC的预后提供理论依据.方法 对1995年4月至2006年4月我院收治的63例新生儿NEC患儿进行回顾性分析,根据转归不同分为治愈组和预后不良组,比较两组可能与预后相关因素的差异,寻找与预后不良有关的因素,再将单因素分析的结果代入Logistic回归方程进行多因素分析,找出与新生儿NEC预后有关的危险因素.结果 63例NEC患儿中根据Bell分期,Ⅰ期38例,Ⅱ期10例,Ⅲ期15例,治愈50例,死亡10例,病死率15.9%,死亡病例均为Ⅲ期患儿,加放弃3例,Ⅲ期预后不良率86.7%.按转归不同分为治愈组50例,预后不良组13例,将两组预后有关因素进行单因素分析发现,早产、呼吸暂停、腹膜炎、休克、DIC、硬肿症、高血糖症、低钠血症、代谢性酸中毒、血小板减少、白细胞异常、腹部x线明显异常、肠穿孔两组差异有统计学意义(P＜0.05),提示上述因素与新生儿NEC预后有关.把上述13个单因素进一步做多因素回归分析发现休克、代谢性酸中毒、硬肿症是与NEC预后不良关系最为密切的因素.经分析两组差异有统计学意义(P＜0.05).结论 新生儿NEC并发休克、硬肿症、代谢性酸中毒是影响预后的主要危险因素;积极防治NEC的合并症,有助于降低病死率,改善预后.     

新生儿坏死性小肠结肠炎肠穿孔与肠未穿孔患儿术后结局的对比研究CSCD

目的对比新生儿坏死性小肠结肠炎(necrotizing enterocolitis,NEC)肠穿孔与肠未穿孔患儿手术治疗后转归情况,为NEC手术时机的选择及术后治疗提供参考。方法回顾性分析2009年8月至2019年8月中国人民解放军总医院第七医学中心儿科医学部新生儿外科收治的237例经手术治疗的NEC患儿临床资料。按照是否发生肠穿孔分为肠未穿孔组(172例)与肠穿孔组(65例),收集两组患儿术中所见坏死肠管长度、手术后实施肠内及肠外营养时间、呼吸机使用时间、NICU入住时间、术后并发症以及预后情况。结果NEC肠未穿孔组172例中,治愈124例(124/172,72.1%),死亡48例(48/172,27.9%);肠穿孔组65例中,治愈48例(48/65,73.8%),死亡17例(17/65,26.2%)。两组术中所见坏死肠管长度以及术后肠外营养时间、肠内营养时间、呼吸机使用时间、NICU入住时间及术后并发症比较,差异均有统计学意义(P<0.05)。Bell分期为ⅢA与ⅢB期的患儿病死率比较,差异有统计学意义(χ^(2)=4.731,P=0.030)。结论NEC肠未穿孔的患儿可能存在更多肠管坏死,术后并发症多,康复时间长。建议对于NEC肠未穿孔患儿,可根据患儿临床实际情况探讨更合适的手术指征。     

新生儿缺氧缺血性脑病中性粒细胞CD11b、CD18变化及临床意义

本文探讨CD11b、CD18在新生儿缺氧缺血性脑病中的意义.HIE新生儿30例,(轻度13例,中度10例,重度7例)及20例对照新生儿采用流式细胞术检测生后24～48h、7 dCD11b、CD18(免疫荧光强度MFI)值.HIE组CD11b24～48h(2929.35±216.04)、7 d(1921.58±251.21)较对照组(1222.68±139.09)均明显升高(P＜0.05),HIE组CD1824～48h(324.48±9.45)、7 d(216.33±5.89)较对照组(94.23±19.09)均明显升高(P＜0.05).24～48h、7 d存在动态变化,以24～48h较高(P＜0.05).HIE轻、中、重之间均有显著性差异(P均＜0.05).结果提示,新生儿脑缺氧缺血再灌注后24～48h内中性粒细胞炎症反应显著,病情越重,CD11b、CD18水平越高.     

坏死性小肠结肠炎新生儿血浆D-乳酸水平变化的意义

目的 探讨坏死性小肠结肠炎(NEC)新生儿血浆D-乳酸水平变化的意义.方法 选择诊断为NEC 50例新生儿为NEC组(其中NECⅡ期30例,NECⅢ期20例).选择同期非NEC新生儿50例为对照组.NEC组于NEC确诊24 h内,对照组于相应日龄取外周静脉血2 mL,采用酶联免疫吸附法检测血浆D-乳酸水平,采用受试者工作特性(ROC)曲线确定血浆D-乳酸阳性标准.根据NEC发生时血浆D-乳酸水平分为D-乳酸升高组和D-乳酸正常组,根据NEC患儿病情转归分为病死组和存活组,比较不同组间血浆D-乳酸水平、新生儿危重病例评分(NCIS)分值、并脓毒症的发生率、病死率的差异.结果 NECⅢ期组、NECⅡ期组和对照组血浆D-乳酸水平分别为(36.2±10.3) mg·L-1、(28.6±12.5) mg·L-1和(3.8±2.6)mg·L-1,3组比较差异有统计学意义(F=7.63,P＜0.05).ROC曲线分析显示,血浆D-乳酸阳性标准为≥7 mg·L-1,预测NEC的敏感性为80.0％,特异性为84.6％,假阴性率为15.4％,假阳性率为20.0％.D-乳酸升高组并脓毒症发生率、病死率较高,NCIS评分较低,与D-乳酸正常组比较差异均有统计学意义(Pa＜0.05);病死组血浆D-乳酸水平、并脓毒症发生率较高,NCIS评分较低,与存活组比较差异均有统计学意义(Pa＜0.05).结论 血浆D-乳酸水平能较敏感地反映NEC新生儿的病情,可作为预测NEC程度和预后的指标之一.     

早产儿坏死性小肠结肠炎合并血培养阳性败血症的病例对照研究

目的 研究合并血培养阳性败血症的早产儿坏死性小肠结肠炎(necrotizing enterocolitis,NEC)的临床资料及预后情况,为临床更好的诊断、治疗及减少病死率提供参考.方法 对深圳市宝安区妇幼保健院NICU 2007年1月至2015年12月确诊Ⅱa以上NEC合并血培养阳性败血症的25例患儿的临床资料进行回顾性分析,并按1∶1比例随机选取同期确诊为单纯Ⅱa以上的NEC患儿25例作为对照组.结果 (1)Ⅱa以上NEC合并血培养阳性败血症组与单纯Ⅱa以上NEC组患儿在胎龄、出生体重、起病年龄、住院时间、并发症及白细胞计数等方面比较差异均无统计学意义(P＞0.05).Ⅱa以上NEC合并血培养阳性败血症组与单纯Ⅱa以上NEC组患儿在超敏CRP＞80mg/L和血小板计数＜50×109/L方面比较差异有统计学意义(P＜0.05).Ⅱa以上NEC合并血培养阳性败血症组死亡或放弃的人数较单纯Ⅱa以上NEC组高,差异有统计学意义(P＜0.05).(2)Ⅱa以上NEC合并血培养阳性败血症组患儿血培养的主要病原菌有肺炎克雷白杆菌、大肠埃希菌、屎肠球菌及粪肠球菌等,其中肺炎克雷白杆菌和大肠埃希菌占50%以上.药敏结果显示,肺炎克雷白杆菌和大肠埃希菌等革兰阴性菌均对环丙沙星、左氧氟沙星及美罗培南敏感.(3)Logistic回归分析结果显示,与Ⅱa以上NEC合并血培养阳性败血症有显著相关性的变量为血小板计数＜50×109/L(OR=3.48,95%CI:1.35~8.68)和男性患儿(OR=2.09,95%CI:1.02~4.85).结论 Ⅱa以上NEC合并血培养阳性败血症患儿预后不良,对可疑患儿,尽早使用有效抗生素治疗,减少病死率.     

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.