Department of Neurosurgery, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China.
Key Laboratory of Endocrinology of Ministry of Health, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China.
Front Endocrinol (Lausanne). 2020 Dec 1;11:610519. doi: 10.3389/fendo.2020.610519. eCollection 2020.
Acromegaly is a rare, intractable endocrine disease. We aimed to describe the patient characteristics, diagnostic delays, treatment patterns, treatment outcomes, comorbidities and treatment costs of acromegaly in China.
This is a nationwide cross-sectional study. Patients diagnosed with and treated for acromegaly between 1996 and 2019 across China were surveyed via the Chinese Association of Patients with Acromegaly platform.
In total, 473 patients (58.8% females, mean age at diagnosis: 39.4±9.5 years) were included. The median disease duration was 3 years. The most common symptoms were extremity enlargement (91.8%) and facial changes (90.1%). Overall, 63.0% of patients experienced diagnostic delays within healthcare systems; 63.8% of the delays were <1 year. The most common first-line therapy was surgery with a transsphenoidal (76.1%) or transcranial approach (3.2%). Somatostatin analogues or dopamine agonists were administered in 20.5% of the patients as first-line therapies and in 41.7% as adjuvant therapies. Radiotherapy was performed in 32.1% of patients, 99.3% of whom received radiotherapy as an adjuvant therapy. After a median 5-year follow-up, 46.2% achieved biochemical control. Comorbidities were reported in 88.2% of the patients at follow-up; memory deterioration and thyroid nodules were the most common. Controlled patients had greater improvements in symptoms and comorbidities during follow-up than uncontrolled patients. The annual per-capita cost-of-treatment was $11013 in 2018, with medical treatments being the largest contributor (67%). Medical insurance covered 47.2% of all treatment costs.
This study provides the first comprehensive description of real-world acromegaly data in China, serving as a basis for future population-based studies.
肢端肥大症是一种罕见的、难以治愈的内分泌疾病。本研究旨在描述中国肢端肥大症患者的特征、诊断延迟、治疗模式、治疗结果、合并症和治疗费用。
这是一项全国性的横断面研究。通过中国肢端肥大症患者协会平台，对 1996 年至 2019 年期间在中国被诊断和治疗的肢端肥大症患者进行了调查。
共纳入 473 例患者（女性占 58.8%，诊断时的平均年龄为 39.4±9.5 岁）。疾病的中位病程为 3 年。最常见的症状是四肢增大（91.8%）和面部改变（90.1%）。总体而言，63.0%的患者在医疗系统内存在诊断延迟，其中 63.8%的延迟时间<1 年。最常见的一线治疗方法是经蝶窦（76.1%）或经颅（3.2%）手术。生长抑素类似物或多巴胺激动剂作为一线治疗药物在 20.5%的患者中使用，作为辅助治疗药物在 41.7%的患者中使用。32.1%的患者接受了放射治疗，其中 99.3%的患者接受了辅助放射治疗。中位随访 5 年后，46.2%的患者达到生化控制。在随访时，88.2%的患者报告有合并症；记忆力减退和甲状腺结节是最常见的。与未控制的患者相比，控制的患者在随访期间症状和合并症有更大的改善。2018 年的人均治疗费用为 11013 美元，其中医疗费用占比最大（67%）。医疗保险覆盖了所有治疗费用的 47.2%。
本研究首次全面描述了中国肢端肥大症的真实世界数据，为未来的基于人群的研究提供了依据。
