肢端肥大症治疗的成本效果分析初探及其生存质量的横断面调查

【作者】 黄敏强；

【导师】 邓洁英； 顾锋；

【作者基本信息】 北京协和医学院 ， 临床医学， 2009， 博士

【摘要】 背景：目前肢端肥大症的治疗方案仍以手术为首选,放疗或药物作为术后辅助治疗的手段。但是手术治疗的成功率不高,尤其对大腺瘤及侵袭性大腺瘤的患者,手术常常并不能完全地切除肿瘤。生长抑素类似物(SSA)治疗能够降低GH水平及缩小肿瘤体积,而术前GH水平以及肿瘤大小是手术效果的预测因子,而因此在术前短期应用SSA治疗在理论上可以提高手术的效果。目的：利用卫生经济学的成本效果分析手段来评价先短期使用药物(主要是指长效生长抑素类似物)治疗后再行手术治疗(术前用药)和以手术为初始治疗方法(初治手术)这两种治疗方案在治疗肢端肥大症中的治疗效果和成本。方法：回顾分析了168份符合入选标准的病历,其中20份为术前用药,148份为初治手术,得出两种治疗方案的治疗成功率、手术并发症等数据；并利用马尔科夫(Markov)模型模拟肢端肥大症术后在实际临床中可能出现的三种情况：术后未愈者进行二次手术+放疗、放疗+药物过渡治疗及长期用药(分别简称为二次手术、放疗、长期用药),进而按这三种情况分别评价两种方案的治疗效果及成本。结果：在治疗微腺瘤方面,两种治疗方案的治疗成功率无统计学的显著差异。而在治疗大腺瘤方面,术前用药的治疗成功率要高于初治手术,术前用药患者的预期寿命和质量调整生命年均高于初治手术治疗,而成本方面,在二次手术组,术前用药高于初治手术,而在放疗组及长期用药组,术前用药低于初治手术。结论：术前用药这种方案在治疗肢端肥大症大腺瘤方面较初治手术治疗方案具有成本效果优势。目的：了解肢端肥大症患者的生存质量情况及其影响因素。方法：横断面调查,利用肢端肥大症生存质量调查量表(ACROQoL)评价患者的生存质量情况。结果：生化控制组在心理精神的子维度外貌评分高于未控制组(P=0.044),而在其它维度并无显著性差异。而在按有无并发症及放疗划分的亚组中,仅在控制组内,无并发症亚组在人际关系评分高于有并发症亚组。结论：肢大对患者的生存质量有负面的影响,GH水平影响肢大患者的心理精神子维度外貌评分。更多还原

【Abstract】 Background Many studies have showed that GH levels at the time of diagnosis and tumor size are predictive of surgical outcomes in patients with acromegly, and therefore, any therapy that decrease GH levels and tumor size before surgery would have a theoretical advantage. We estimated the cost effectiveness of two therapeutic schemes: preoperative medical therapy followed by surgery and surgery as primary therapy.Methods Retrospective analysis of168cases. We estimated the cost per year of life gained and the cost per quality-adjusted years of life gained with the two therapeutic schemes. To estimate the cost and the effects, Makov models were used.Results In the patients with macroadenoma, the remission rate of the therapeutic scheme of preoperative medical therapy followed by surgery is higher than that of surgery as primary therapy. We modeled three different treatments after surgery using Makov model, and we found that as for macroadenoma, the cost per year of life gained and the cost per quality-adjusted years of life gained with the therapeutic scheme of preoperative medical therapy followed by surgery are higher than that of the therapeutic scheme of surgery as primary therapy in the subgroup of secondary surgery. However, the cost per year of life gained and the cost per quality-adjusted years of life gained with the therapeutic scheme of preoperative medical therapy followed by surgery are lower than that of the therapeutic scheme of surgery as primary therapy in the subgroup of radiotherapy and drug therapyConclusions In acromegalic patients with macroadenoma, the therapeutic scheme of preoperative medical therapy with long acting SSA followed by surgery is cost effective. Objective:The aim of this study was to evaluate the quality of life(QOL) in patients with treated acromegaly and its influential factors.Design:cross-sectional study.Metods:We used the Acromegaly Quality of Life Questionnaire(ACROQoL). to evaluate QOL in patients with treated acromgaly.61patients wre divided into controlled and uncontrolled groups based on the remission criteria and further subdivided into eight groups according to with/without complications or radiotherapy. Comparisons between groups were analyzed.Results:In comparison with the uncontrolled groups, the controlled groups showed better ACROQoL scores in psychological subscale "appearance"(P=0.044). And in subgroups, only the subgroup of with complications in controlled group was found to have higher scores in psychological subscale "personal relationship" than the subgroup of without complications.Conclusions:Acromegaly has an important impact on quality of life. The level of GH is negatively associated with psychological subscale "appearance" in patients with acromegaly.更多还原