本文選題：特發(fā)性矮小癥　切入點：重組人生長激素　出處：《浙江大學》2017年碩士論文

【摘要】：目的特發(fā)性矮小癥ISS(idiopathic short stature)是一種以目前的醫(yī)療手段尚未找到明確病因,生長不足或身材矮小的疾病,占矮小患者的比例約為40%,是兒童矮小最常見的類型。矮小身材給特發(fā)性矮小癥(ISS)兒童帶來一系列社會和心理等方面的不良影響,改善特發(fā)性矮小癥患兒的成年終身高以及緩解由于身材矮小所造成的心理壓力,提高患兒的生活質量是治療ISS的最終目的。目前臨床上對特發(fā)性矮小的治療方式主要有西醫(yī)治療和中醫(yī)治療。西醫(yī)運用重組人生長激素(recombinant human growth hormone rhGH)進行治療;中醫(yī)治療主要是采用基于脾虛,腎虛,脾腎兩虛證型的健脾益腎中藥方進行治療。相比中醫(yī)治療,生長激素治療在正確選擇適應癥的范圍內效果顯著,尤其體現(xiàn)在在生長激素治療第一年。本研究探討特發(fā)性矮小癥(ISS)患兒經(jīng)重組人生長激素(rhGH)結合綜合干預治療后生長發(fā)育的情況和各項生化指標的變化來評估其促進生長的療效以及監(jiān)測其安全性并為調整用藥劑量提供依據(jù);并與中醫(yī)治療的效果進行比較,探尋有效的,安全的,個性化的,經(jīng)濟的治療方案。方法采用隨機數(shù)字表法將63例確診為特發(fā)性矮小癥(ISS)患兒分為三組:將2011年7月到2012年10月在某附屬體育醫(yī)院生長發(fā)育門診確診但未采取治療措施的18例ISS患兒分為未治療組,采用皮下注射重組人生長激素治療的25例ISS患兒為治療I組,使用中藥治療的20例ISS患兒為治療Ⅱ組,兩治療組結合綜合干預。三組患兒觀察時間均為12個月。記錄治療前患兒一般資料與治療6個月及12個月后生長發(fā)育情況即身高,體重,骨齡以及各項生化指標的變化,生化指標包括:甲狀腺功能(T3、T4、F T3、FT4、TSH),肝腎功能(ALT、AST、GGT、CRE、UREA),空腹血糖(FBG),胰島素樣生長因子1(IGF-1),胰島素樣生長因子結合蛋3(IGF-BP3)。結果三組特發(fā)性矮小癥(ISS)患兒一般資料(性別、年齡、骨齡、體重、身高,基礎生長激素)比較無統(tǒng)計學差異(P0.05)。兩治療組經(jīng)治療6個月和12個月后身高與未治療組比較差異有統(tǒng)計學意義(P0.05);治療12個月后,治療I組年生長速率顯著大于治療Ⅱ組,兩治療組均大于未治療組(P0.05)。兩治療組治療12個月后年骨齡變化量未見有統(tǒng)計學意義(P0.05),未見骨齡提前。兩治療組體重增加均大于未治療組,兩治療組之間比較無顯著性差異(P0.05),體重指數(shù)BMI均在正常范圍內。兩治療組IGF-1、IGF-BP3濃度較治療前顯著增加(P0.05),未見超過2SD。甲狀腺功能(T3、T4、F T3、FT4、TSH),肝腎功能(ALT、AST、GGT、CRE、UREA)空腹血糖(FBG)較治療前的差異無統(tǒng)計學意義(P0.05)。結論一年內在治療ISS患兒方法中,重組人生長激素治療結合綜合干預促生長療效顯著并優(yōu)于中藥治療組,IGF-1、IGF-BP3濃度較治療前顯著增加但未超過2SD,說明了治療的有效性以及治療劑量的合理性;骨齡未見提前,甲狀腺功能和空腹血糖未見異常,證明了藥物使用的安全性。
[Abstract]:Objective: idiopathic short stature ISS (idiopathic short stature) is a kind of current medical treatment has not yet found a clear cause, growth deficiency or short stature disease accounted for about 40% of the patients with short stature, is the most common type of children with short stature. Short stature to idiopathic short stature syndrome (ISS) adverse the influence of a series of social and psychological aspects of children, improvement in children with idiopathic short stature of the final adult height and alleviating the short stature caused by psychological pressure, improve the quality of life of children is the ultimate goal of treatment of ISS. The clinical treatment of idiopathic short stature are the main treatment of Western medicine and traditional Chinese medicine. Western medicine using recombinant human growth hormone (recombinant human growth hormone rhGH) treatment; Chinese medicine treatment is mainly based on the spleen, kidney, spleen and kidney deficiency type two prescription of invigorating spleen and tonifying kidney in the treatment phase. Compared with traditional Chinese medicine treatment, growth hormone treatment in the correct selection of indications within the scope of the effect is significant, especially in the first year of growth hormone therapy. The study of idiopathic short stature (ISS) in children with recombinant human growth hormone (rhGH) changes development combined with comprehensive intervention treatment and epigenetic biochemical indexes to to evaluate the curative effect and promote the growth of the safety monitoring and provide the basis for the adjustment of the dose; and compared with the traditional Chinese medicine treatment effect, explore effective, safe and personalized treatment plan, economy. Methods 63 patients diagnosed with idiopathic short stature with the random number table method (ISS) in children divided into three groups: July 2011 to October 2012 growth in an affiliated sports hospital development clinic diagnosed but did not take treatment measures in 18 cases with ISS were divided into untreated group, by subcutaneous injection of recombinant human growth hormone therapy 鐨，

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