重组人生长激素替代治疗对体脂含量的影响研究

目的分析重组人生长激素(rhGH)治疗生长激素缺乏症(GHD)后患儿体质量变化的规律及原因;寻找rhGH替代治疗后便捷、灵敏地监测脂代谢变化的指标,了解机体脂代谢变化与rhGH疗效的相关性。方法随机选择完全性生长激素缺乏症(GHD)患儿15例,给予rhGH0.033mg/(kg·d)治疗,疗程6个月。观察用药前后身高(Ht)、生长速度(GV)、身高标准差积分(HtSDS)的变化,评价rhGH的促生长作用;测量治疗前、治疗3个月后、治疗6个月后的体质量(Wt)、体脂含量(Fat%)、体质指数(BMI)、腰臀比(WHR),检测治疗前、治疗6个月后的血脂水平,评价GHD患儿rhGH治疗前、后脂代谢状况。分析rhGH治疗后机体脂代谢变化与rhGH疗效的相关性。结果治疗后患儿Ht、GV、HtSDS改善显著(P<0.01),rhGH促生长作用肯定;治疗后患儿Fat%、血HDL、LDL水平较治疗前显著下降(P<0.05),机体脂代谢得到改善;rhGH治疗3个月与治疗6个月的ΔFat%与ΔGV存在负相关性(r=-0.625,P=0.0096)。结论rhGH治疗GHD后的体质量增长,为非脂肪的增长;体脂含量监测便捷无创,较血脂...     

重组人生长激素治疗生长激素缺乏症疗效观察

目的 观察基因重组人生长激素(rhGH)对生长激素缺乏症(GHD)患儿的疗效。方法 对15例GHD患儿应用rhGH治疗,每晚睡前皮下注射0.1 IU/kg,疗程6个月。结果 患儿身高由治疗前109.3±9.9cm增加到115.5±11.3 cm;年身高生长速度由治疗前2.8±0.6cm/年增加到11.6±3.5cm/年。治疗期间除少数患儿出现亚临床甲状腺功能低下,注射部位有轻度反应外,未发现明显副作用。结论 皮下注射rhGH是治疗儿童GHD的一种安全有效的方法。     

内分泌系统疾病

071372重组人生长激素治疗生长激素缺乏症患儿影响生长速度疗效的因素/潘思年…∥中华儿科杂志.-2006,44(7).-544~54594例生长激素缺乏症(GHD)患儿治疗前年生长速度(GV)(4.4±1.6)cm,用重组人生长激素(rhGH)后,生长速度明显加快(P<0.01),以头6个月的GV最快,第2~5年GV渐降,但无统计学意义。相关分析提示:头6个月生长速度(GV1)与骨龄(BA0)、类胰岛素样生长因子(IGF-1)SDS、身高Z分值(HtSDS)和激发试验GH峰(SPGH)呈负相关。逐步回归分析显示:BA0和SPGH是影响GV1的独立因素。认为用rhGH治疗至少在头4年内能使GHD患儿呈现有效…     

骨龄和遗传身高对重组人生长激素治疗生长激素缺乏症患儿疗效的影响

目的 观察重组人生长激素(rhGH)治疗原发性生长激素缺乏症(GHD)患儿的疗效,分析开始治疗时骨龄和遗传身高(FPH)对GHD治疗效果的影响.方法 对GHD患者应用国产rhGH治疗,选取其中36例完全性GHD男性青春期前患者,按骨龄不同将36例患者分成骨龄<9岁组(19例)和骨龄≥9岁组(17例);按FPH不同将36例患者分成FPH<170.79 cm组(13例),170.79 cm≤FPH<174.69 cm组(14例)和FPH≥174.69组(9例),分别进行治疗后0.5 a平均生长速率(GV)监测.结果 治疗时骨龄<9岁组GV值为(12.9±1.8)cm/a,明显大于骨龄≥9岁组[(9.2±2.1)cm/a](t=2.31 P<0.05);FPH≥174.69组GV值为(12.4±2.1)cm/a,明显大于FPH<170.79组[(10.1±2.0)cm/a](t=2.26 P<0.05).结论 开始治疗时骨龄和遗传身高对GHD患儿的促生长疗效存在显著影响.     

不同剂量生长激素治疗生长激素缺乏症患儿的疗效

目的探讨不同剂量生长激素治疗生长激素缺乏症(GHD)的疗效。方法GHD患儿35例分为2个治疗组。A组16例,rhGH每周的总剂量为0.5IU/kg,分5d皮下注射,每次注射剂量为0.1IU/kg;B组19例,rhGH总剂量为0.7IU/(kg.周),分7d皮下注射,每次注射剂量为0.1IU/kg。患儿均连续使用rhGH皮下注射最少6个月。观察指标为治疗前后身高增长速度、治疗前后实际年龄的身高均值标准差计分、胰岛素样生长因子-Ⅰ(IGF-Ⅰ)和胰岛素样生长因子结合蛋白-3(IGFBP-3)、治疗前后骨成熟情况(骨龄/实际年龄的变化)。结果治疗期间二组患儿身高均明显增加,生长速度均明显增快,身高均值标准差记分均明显升高,同一组患儿治疗前后差异有统计学意义。二组患儿治疗前后各指标比较无统计学差异。结论GHD儿童应用rhGH每周0.5IU/kg,与每周0.7IU/kg比较疗效相同。每周0.5IU/kg,可节省药物剂量,延长患儿用药时间,也减轻了患儿注射的痛苦。     

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目的探讨重组人生长激素(recombinant human growth hormone,rhGH,简称GH)治疗生长激素缺乏症(growth hormone deficiency,GHD)患儿效果及影响因素,建立GH治疗效果预测模型。方法回顾性分析1996年8月至2010年9月首都儿科研究所生长发育门诊确诊为GHD和多垂体功能低下(multiple pituitary hormonedeficiency,MPHD)且接受规范GH治疗的矮身材患儿115例临床资料,采用2009年卫生部最新颁布的中国儿童体格发育标准对儿童身高、体重进行标化,标准差计算采用国际公认的LMS方法。以治疗过程中的身高标准差分值变化(delta in height SDS,ΔHtSDS)和生长速度(growth velocity,GV)为效果评价指标,进行疗效和影响因素分析。用多元回归方法以75例治疗满1年且随访较规律者为模型人群,建立治疗效果预测模型。同时前瞻性随访15例规范治疗的GHD患儿为模型验证对象,对模型进行验证。结果患儿治疗第1年身高平均增长(10.56±2.83)cm,ΔHtSDS升高0.93±0.52;治疗前3个月的ΔH...     

血清胰岛素样生长因子-1、胰岛素样生长因子结合蛋白-3在矮小症儿童诊断和疗效判断中的价值

目的探讨胰岛素样生长因子-1（IGF-1）及其结合蛋白-3（IGFBP-3）在矮小症儿童诊断及疗效判断中的价值。方法1．对124例青春发育前矮小症患儿用精氨酸激发试验和可乐定激发试验检测其血清生长激素（GH）水平，并根据患儿GH峰值分为生长激素缺乏组（GHD组，40例）、特发性矮小组（1SS组，84例）。选取20例健康儿童作为健康对照组。对所有儿童采用酶联免疫吸附法检测血清IGF—1和IGFBP-3。对GHD组、ISS组和健康对照组儿童血清IGF-1和IGFBP-3水平进行两两比较。2．对15例GHD和30例ISS患儿予国产重组人生长激素（rhGH）0．1IU／（kg&#183;d）治疗6个月，于治疗前及治疗6个月分别测定其身高、体质量、骨龄及血清IGF-1、IGFBP-3，并进行治疗前后的对照。结果1．GHD组和ISS组患儿血清IGF-1和IGFBP-3水平明显低于健康对照组（Pa〈0．01），GHD组与ISS组患儿血清IGF-1和IGFBP-3水平比较均有显著差异（Pa〈0．01），GHD组患儿治疗前后血清IGF-1、IGFBP-3比较有显著差异（Pa〈0．01）；诊断GHD，IGF-1的特异性为67．8％，敏感性为75％；IGFBP-3的特异性为88％，敏感性为85％。2．rhGH治疗后身高增长速度明显加快，血清IGF-1、IGFBP-3水平显著升高；治疗前血清IGF-1与治疗6个月生长速度呈显著负相关（r=-0．78P〈0．01）；治疗6个月后IGF-1的变化与治疗后生长速度呈显著正相关（r=0．82P〈0．01）。结论IGF-1、IGFBP-3可用于儿童矮小症的诊断及疗效评价。     

国产基因重组人生长激素治疗特发性矮小患儿的疗效

目的观察基因重组人生长激素(r-hGH)治疗特发性矮小(ISS)患儿促生长的疗效及对ISS患儿免疫功能影响。方法ISS患儿16例均采用r-hGH治疗,0.12U/(kg·d),每晚睡前0.5 h皮下注射,注射部位在脐周、大腿外侧及上臂外侧等, 总疗程6个月,治疗前后检测患儿身高、体质量、骨龄、骨龄预测身高、生长速率及血清免疫球蛋白改变。结果16例生长速率由(1.5±0.4)cm/6个月提高到(6.5±1.7)em/6个月,骨龄增长小于生活年龄增长,治疗前后通过骨龄预测身高由(150.0±11.7)cm提高到(156.0±8.9)cm,r-hCH治疗后患儿IgA显著提高(P<0.01)。结论国产r-hGH对ISS治疗安全、有效,不仅有促生长作用,同时能改善机体免疫功能。     

基因重组人生长激素治疗儿童生长激素缺乏症26例

目的　探讨基因重组人生长激素 (rhGH)对生长激素缺乏症 (GHD)患儿的疗效。方法　对 2 6例GHD给rhGH治疗 ,0 .1IU/ (kg·d) ,每晚皮下注射 ,疗程 0 .5年。结果　 2 6例身高平均增加 8.2± 0 .8cm/ 6个月 ,生长速率由治疗前 1.5± 0 .4cm/ 6个月增加至 6.5± 1.7cm/ 6个月 ,身高标准差由治疗前 4.5± 1.2减少至3 .2± 1.1,骨龄无明显增加 ,体重也略有增加。治疗期间第 1～ 3个月 75 %左右患儿出现甲状腺功能低下症状 ,但未影响体格线性增长。结论　rhGH是治疗GHD的一种有效、安全的促生长药物     

重组人生长激素治疗颅咽管瘤术后生长激素缺乏症

目的探讨低剂量基因重组人生长激素(rhGH)治疗颅咽管瘤术后生长激素缺乏症(GHD)患儿的疗效和安全性。方法回顾性分析2008年4月-2011年4月在北京三博脑科医院内分泌门诊治疗的12例7～15岁术后病理确诊为颅咽管瘤且继发生长迟滞患儿的病例资料及随访资料。患儿均给予rhGH治疗(每晚睡前皮下注射0.1 IU.kg-1,每周5次注射),疗程3～36个月。定期检测肝功能、肾功能、激素水平等指标,并比较患儿治疗前后身高、体质量、生长速度、身高标准差计数、胰岛素样生长因子1(IGF-1)、骨龄等生长指标的改变。结果在rhGH治疗期间,12例患儿在治疗第1年生长速率增加显著,由(2.2±1.3)cm.a-1增加到(6.63±4.97)cm.a-1(P<0.01),身高标准差计数由治疗前-3.3±2.3增加到-3.2±2.8,血IGF-1治疗前为(38±64)μg.L-1,治疗后为(173±167)μg.L-1(患儿治疗后血清IGF-1水平达到正常范围),差异均有统计学意义(Pa<0.01)。治疗期间,患儿肝肾功能等均保持在正常值范围,骨龄无明显变化,随访时尚无患儿肿瘤复发。结论低剂量rhGH治疗儿童颅咽管瘤术后继发GHD是经济、有效的,在充分评估及严密监控下开展GH替代治疗是安全的。     

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.     

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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     

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.     

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.     

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   

Personality profiles and heart rate variability (vagal tone) in children with recurrent abdominal pain

The aim of the study was to explore psychological factors and autonomic activity in children with recurrent abdominal pain and to compare them with those in a control group of healthy children. The Personality Inventory for Children was used for assessment of developmental, emotional and psychosocial factors in 25 children with recurrent abdominal pain (age, 7-15 y). Parasympathetic and sympathetic functions in these children and in 23 healthy control subjects (age, 7-13 y) were also investigated, non-invasively using a computerized polygraph. Vagal tone (parasympathetic function) was indexed by calculation of respiratory sinus arrhythmia in beats/min. Skin conductance (sympathetic function) was recorded by the constant current method. On the Personality Inventory for Children, 16 patients had high scores on somatic concern. Several patients had scores in the clinical range for depression, withdrawal and anxiety, but the mean scores for these personality profile scales were well within the normal range of healthy children. Interestingly, there was a spike on the L (Lie)-scale for most of the patients and 15 patients had scores above or close to the clinical cut-off value. As compared with the scores in healthy children, vagal tone and sympathetic tone were normal. Conclusion: Many children with recurrent abdominal pain have scores in the clinical range for depression, withdrawal, anxiety and L-scale indicating coping problems, denial and a trend towards somatic concern that may contribute to the evolution of abdominal pain. Autonomic nerve activity was not disturbed in these children.     

Overcoming surfactant inhibition with polymers

Inhibition of the function of pulmonary surfactant in the alveolar space is an important element of the pathophysiology of many lung diseases, including meconium aspiration syndrome, pneumonia and acute respiratory distress syndrome. The known mechanisms by which surfactant dysfunction occurs are (a) competitive inhibition of phospholipid entry into the surface monolayer (e.g. by plasma proteins), and (b) infiltration and destabilization of the surface film by extraneous lipids (e.g. meconium-derived free fatty acids). Recent data suggest that addition of non-ionic polymers such as dextran and polyethylene glycol to surfactant mixtures may significantly improve resistance to inhibition. Polymers have been found to neutralize the effects of several different inhibitors, and can produce near-complete restoration of surfactant function. The anti-inhibitory properties of polymers, and their possible role as an adjunct to surfactant therapy, deserve further exploration.     

Understanding infant formula

The World Health organisation recommends breast feeding infants for the first six months of life. When this breast feeding does not occur either through parental choice or medical need, infant formulas will be required. There is a bewildering array of formulas on the UK market for many different requirements. When faced with an unsettled infant many parents (and healthcare professionals) will experiment with the infant formula available and then attend the paediatric clinic looking for help and advice. It is therefore essential that paediatricians understand what milks are available and what the key differences between different products are. This review attempts to provide a simple guide through many of the formulations currently available in the UK; and offers advice for the dietary management of the child with extra calorie requirements, infants with cow's milk protein allergy, gastro oesophageal reflux disease, apparent unresolved hunger and infantile colic. Whatever the underlying condition, there is likely to be an infant formula that is suitable in this generation of ever expanding formulations.