Geer Eliza B, Shafiq Ismat, Gordon Murray B, Bonert Vivien, Ayala Alejandro, Swerdloff Ronald S, Katznelson Laurence, Lalazar Yelena, Manuylova Ekaterina, Pulaski-Liebert Karen J, Carmichael John D, Hannoush Zeina, Surampudi Vijaya, Broder Michael S, Cherepanov Dasha, Eagan Marianne, Lee Jackie, Said Qayyim, Neary Maureen P, Biller Beverly M K
Endocr Pract. 2017 Aug;23(8):962-970. doi: 10.4158/EP171787.OR. Epub 2017 Jun 14.
Cushing disease (CD) results from excessive exposure to glucocorticoids caused by an adrenocorticotropic hormone-secreting pituitary tumor. Inadequately treated CD is associated with significant morbidity and elevated mortality. Multicenter data on CD patients treated in routine clinical practice are needed to assess treatment outcomes in this rare disorder. The study purpose was to describe the burden of illness and treatment outcomes for CD patients.
Eight pituitary centers in four U.S. regions participated in this multicenter retrospective chart review study. Subjects were CD patients diagnosed at ≥18 years of age within the past 20 years. Descriptive statistical analyses were conducted to examine presenting signs, symptoms, comorbidities, and treatment outcomes.
Of 230 patients, 79% were female (median age at diagnosis, 39 years; range, 18 to 78 years). Length of follow-up was 0 to 27.5 years (median, 1.9 years). Pituitary adenomas were 0 to 51 mm. The most common presenting comorbidities included hypertension (67.3%), polycystic ovary syndrome (43.5%), and hyperlipidemia (41.5%). Biochemical control was achieved with initial pituitary surgery in 41.4% patients (91 of 220), not achieved in 50.0% of patients (110 of 220), and undetermined in 8.6% of patients (19 of 220). At the end of follow-up, control had been achieved with a variety of treatment methods in 49.1% of patients (110 of 224), not achieved in 29.9% of patients (67 of 224), and undetermined in 21.0% of patients (47 of 224).
Despite multiple treatments, at the end of follow-up, biochemical control was still not achieved in up to 30% of patients. These multicenter data demonstrate that in routine clinical practice, initial and long-term control is not achieved in a substantial number of patients with CD.
BLA = bilateral adrenalectomy CD = Cushing disease CS = Cushing syndrome eCRF = electronic case report form MRI = magnetic resonance imaging PCOS = polycystic ovary syndrome.
库欣病（CD）是由分泌促肾上腺皮质激素的垂体瘤导致长期暴露于过量糖皮质激素引起的。未经充分治疗的CD与显著的发病率和死亡率升高相关。需要关于在常规临床实践中接受治疗的CD患者的多中心数据，以评估这种罕见疾病的治疗结果。本研究的目的是描述CD患者的疾病负担和治疗结果。
美国四个地区的八个垂体中心参与了这项多中心回顾性病历审查研究。研究对象为在过去20年内确诊、年龄≥18岁的CD患者。进行描述性统计分析以检查患者的症状、体征、合并症和治疗结果。
230例患者中，79%为女性（诊断时的中位年龄为39岁；范围为18至78岁）。随访时间为0至27.5年（中位时间为1.9年）。垂体腺瘤大小为0至51毫米。最常见的合并症包括高血压（67.3%）、多囊卵巢综合征（43.5%）和高脂血症（41.5%）。41.4%的患者（220例中的91例）通过初次垂体手术实现了生化控制，50.0%的患者（220例中的110例）未实现生化控制，8.6%的患者（220例中的19例）情况未明。在随访结束时，49.1%的患者（224例中的110例）通过多种治疗方法实现了控制，29.9%的患者（224例中的67例）未实现控制，21.0%的患者（224例中的47例）情况未明。
尽管进行了多种治疗，但在随访结束时，仍有高达30%的患者未实现生化控制。这些多中心数据表明，在常规临床实践中，相当一部分CD患者未实现初始和长期控制。
BLA = 双侧肾上腺切除术；CD = 库欣病；CS = 库欣综合征；eCRF = 电子病例报告表；MRI = 磁共振成像；PCOS = 多囊卵巢综合征
