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     

国产重组人生长激素治疗生长激素缺乏症的临床疗效

目的为评价国产重组人生长激素(r-hGH)治疗生长激素缺乏症(GHD)的有效性和安全性.方法用国产r-hGH对62例GHD患者进行了为期6个月的临床治疗.结果患儿平均身高从治疗前116.23±15.54 cm增加至122.53±15.30 crn,平均6个月净增高数为6.3±1.79 C1m,有显著性差异(P<0.01),生长速率从2.46±0.87 cm/a提高至12.61±3.58 cm/a,净生长速率为10.15±3.31 cm/a,较治疗前明显增高(P<0.01),提示r-hGH治疗GHD的促身高增长效果显著.完全性GH缺乏者治疗6个月身高净增高了6.58±1.68 cm,较部分性GH缺乏者治疗6个月身高净增高了5.28±1.91 Cm,有显著性差异,说明r-hGH治疗完全性GH缺乏者较部分性GH缺乏者更为有效.结论 r-hGH治疗GHD的疗效确切,有明显的促身高增长的作用,而对骨龄成熟无明显加速影响,该药使用安全,无明显副作用.     

生长激素在性早熟治疗中的应用

目前中枢性性早熟 (CPP)治疗首选促性腺激素释放激素类似物 (GnRHa) ,通过抑制垂体—性腺轴 ,使性早熟的症状得到控制 ;同时防止骨骺过早愈合 ,以改善成人身高。但在治疗过程中 ,由于GnRHa的作用 ,使性激素水平降低 ,部分患者生长速率也随之下降 ,甚至低于正常个体的生长速率 ,使其最终身高仍得不到改善 [1 ,2]。因此 ,针对这部分患者我们采用GnRHa联合应用r_hGH治疗 ,取得了良好的效果 ,现报告如下。资料与方法一、对象9例均为女孩 ,入选标准 :①8岁以前出现第二性征 ;②LHRH激发试验呈青春期反应(LH峰值>10mIU/ml,LH峰值/FSH峰…     

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

自1993年10月我们开始采用基因重组人生长激素（rhGH）治疗生长激素缺乏症（GHD）患儿,部分患儿疗程达7年半。现将治疗半年以上的患儿生长发育情况总结如下。     

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。     

生长与生长激素治疗还有新进展吗?

矮身材十分常见,而就诊者中由内分泌原因所致者不到5％。随时更新的、按不同种族制订的生长曲线图是评价儿童生长状况最重要的工具。矮身材的传统定义为身高低于正常生长曲线的第3百分位数,但实际上在此范围内大部分儿童是完全正常的。若采用0．4百分位数作为身材矮小的临界值,则其中1／3～1／2的儿童确有生长障碍。     

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。     

身材矮小与生长激素规范使用

身材矮小及身高生长不足已成为儿科内分泌及儿童保健科最常见的就诊原因,其最大的挑战是如何正确选择适宜的方法以鉴别正常变异与异常生长,明确矮小原因选择正确的干预手段。严格掌握生长激素治疗的适应证及规范化用药是保证安全性的根本措施。应限制生长激素对正常健康矮身材儿童的促生长治疗。     

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。     

Growth and Growth Hormone Therapy in Hypochondroplasia

ABSTRACT. The growth of 84 patients with hypochondroplasia (56 male, 28 female) was studied. A wide spectrum of severity was found from quite severe short limbed dwarfism to short apparently normal prepubertal children who manifested disproportion only at puberty when growth failed. The onset of puberty was at the normal time but the pubertal growth spurt appeared not to materialize and it is this lack which resulted in severely compromised adult heights of 145-165 cm in boys and 133–151 cm in girls. Twenty (12 M, 8 F) hypochondroplastic children aged between 4.3 and 12.8 years were recruited to a study of the effects of biosynthetic growth hormone. All had normal growth hormone responses (> 15 mU/I) to a pharmacological test of growth hormone secretion. Biosynthetic growth hormone in doses between 12-32 u/m²/week produced a significant acceleration in height velocity standard deviation score (SDS) for chronological age (CA) from a pretreatment mean of - 1.66 (SD 1.36) to + 1.62 (SD 1.52) ( p < 0.001). Significant increases were also observed in the height SDS for bone age (BA), sitting height (SH) SDS and subischial leg length (SILL) SDS. A longer period will be required to assess the effect of treatment on adult height prognosis.     

Synchronization of Growth Hormone Therapy

Based on the hypothesis of periodical refractoriness of the growing tissue to exogenous administration of GH, an experimental approach is presented in which the dosage of GH was adjusted to the natural occurrence of periodical changes of the lower leg growth velocity in five children with short stature. Two 6-month periods were compared, during which the children received an identical cumulative dose of GH of 224 IU/m². During the first period, the children received a constant GH dose of 14 IU/m²/week by daily subcutaneous injection, whereas a low/high dose alternation was administered at approximately 3-week intervals synchronously with the occurrence of mini growth spurts during the second 6-month period. In all the children, mean linear growth of the lower leg accelerated during the period of synchronization by 595% compared to the previous growth rate during constant GH administration.     

History of Growth Hormone Therapy

The first human to receive GH therapy was in 1956; it was of bovine origin and was given for 3 wk for metabolic balance studies revealing no effects. By 1958, three separate laboratories utilizing different extraction methods retrieved hGH from human pituitaries, purified it and used for clinical investigation. By 1959 presumed GHD patients were being given native hGH collected and extracted by various methods. Since 1 mg of hGH was needed to treat one patient per day, >360 human pituitaries were needed per patient per year. Thus, the availability of hGH was limited and was awarded on the basis of clinical research protocols approved by the National Pituitary Agency (NPA) established in 1961. hGH was dispensed and injected on a milligram weight basis with varied concentrations between batches from 0.5 units/mg to 2.0 units/mg of hGH. By 1977 a centralized laboratory was established to extract all human pituitaries in the US, this markedly improved the yield of hGH obtained and most remarkably, hGH of this laboratory was never associated with Creutzfeld-Jacob disease (CJD) resulting from the injection of apparently prior- contaminated hGH produced years earlier. However, widespread rhGH use was not possible even if a pituitary from each autopsy performed in the US was collected, this would only permit therapy for about 4,000 patients. Thus, the mass production of rhGH required the identification of the gene structure of the hormone, methodology that began in 1976 to make insulin by recombinant technology. Serendipity was manifest in 1985 when patients who had received hGH years previously were reported to have died of CJD. This led to the discontinuation of the distribution and use of hGH, at a time when a synthetic rhGH became available for clinical use. The creation of a synthetic rhGH was accompanied by unlimited supplies of hGH for investigation and therapy. However, the appropriate use and the potential abuse of this hormone are to be dealt with. The illegitimate use of rhGH, unequivocally the abuse by athletes is, and should be, of primary concern to society and should be halted. The abuse of prescribing rhGH in an attempt to retard the aging process also should receive attention.     

介入治疗在小儿腹部恶性肿瘤的应用

目的 对巨大腹部肿瘤采用介入治疗,为肿瘤完整切除创造有利条件。方法 对１２例腹部恶性肿瘤患儿进行介入治疗,其中男７例,女５例。年龄２个月￣７岁。肝母细胞瘤６例,神经母细胞瘤３例,肾母细胞瘤２例肾上腺皮质癌１例。采用Ｓｅｌｄｉｎｇｅｒ’ｓ技术进行动脉插管造影,确定肿瘤主要供血动脉,进行肿瘤供血动脉超选择性插管,局部注入化疗药物,然后用碘油、明胶海绵或白芨微球栓塞肿瘤的供血动脉。结果 １２例中７例手术