Pituitary Center, Department of Medicine, Cedars-Sinai Medical Center, 110 George Burns Rd, Los Angeles, CA, 90048, USA.
Pituitary. 2011 Sep;14(3):284-94. doi: 10.1007/s11102-011-0310-7.
Although acromegaly is a rare disease, the clinical, economic and health-related quality of life (HRQoL) burden is considerable due to the broad spectrum of comorbidities as well as the need for lifelong management. We performed a comprehensive literature review of the past 12 years (1998-2010) to determine the benefit of disease control (defined as a growth hormone [GH] concentration <2.5 μg/l and insulin-like growth factor [IGF]-1 normal for age) on clinical, HRQoL, and economic outcomes. Increased GH and IGF-1 levels and low frequency of somatostatin analogue use directly predicted increased mortality risk. Clinical outcome measures that may improve with disease control include joint articular cartilage thickness, vertebral fractures, left ventricular function, exercise capacity and endurance, lipid profile, and obstructive apnea events. Some evidence suggests an association between controlled disease and improved HRQoL. Total direct treatment costs were higher for patients with uncontrolled compared to controlled disease. Costs incurred for management of comorbidities, and indirect cost could further add to treatment costs. Optimizing disease control in patients with acromegaly appears to improve outcomes. Future studies need to evaluate clinical outcomes, as well as HRQoL and comprehensive economic outcomes achieved with controlled disease.
尽管肢端肥大症是一种罕见疾病,但由于存在广泛的合并症以及需要终身管理,其临床、经济和健康相关生活质量(HRQoL)负担相当大。我们对过去 12 年(1998-2010 年)的文献进行了全面回顾,以确定疾病控制(定义为生长激素[GH]浓度<2.5μg/l 和 IGF-1 年龄正常)对临床、HRQoL 和经济结果的益处。GH 和 IGF-1 水平升高以及生长抑素类似物使用频率低直接预测死亡率增加。可能随着疾病控制而改善的临床结果衡量指标包括关节软骨厚度、椎骨骨折、左心室功能、运动能力和耐力、血脂谱和阻塞性呼吸暂停事件。一些证据表明,控制疾病与改善 HRQoL 之间存在关联。与控制疾病相比,未控制疾病患者的总直接治疗成本更高。管理合并症和间接成本的费用可能会进一步增加治疗成本。优化肢端肥大症患者的疾病控制似乎可以改善结局。未来的研究需要评估控制疾病实现的临床结局以及 HRQoL 和全面的经济结局。