Surgical Neurology Branch, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, Maryland 20892, USA.
J Clin Endocrinol Metab. 2013 Mar;98(3):892-901. doi: 10.1210/jc.2012-3604. Epub 2013 Jan 31.
Factors influencing the outcome of surgical treatment of pediatric Cushing's disease (CD) have not been fully established.
The aim of this study was to examine features influencing the outcome of surgery for pediatric CD.
In this prospective observational study, the clinical, imaging, endocrinological, and operative outcomes were analyzed in consecutive patients treated at the National Institutes of Health (NIH) from 1982 through 2010.
The study was conducted in a tertiary referral center.
Two hundred CD patients (106 females, 94 males) were included. Mean age at symptom development was 10.6 ± 3.6 years (range, 4.0 to 19.0 y). Mean age at NIH operation was 13.7 ± 3.7 years. Twenty-seven patients (13%) had prior surgery at another institution. Magnetic resonance imaging identified adenomas in 97 patients (50%). When positive, magnetic resonance imaging accurately defined a discrete adenoma in 96 of the 97 patients (99%), which was more accurate than the use of ACTH ratios during inferior petrosal sinus sampling to determine adenoma lateralization (accurate in 72% of patients without prior surgery). A total of 195 of the 200 patients (98%) achieved remission after surgery (189 [97%] were hypocortisolemic; 6 [3%] were eucortisolemic postoperatively). Factors associated with initial remission (P < .05) included identification of an adenoma at surgery, immunohistochemical ACTH-producing adenoma, and noninvasive ACTH adenoma. Younger age, smaller adenoma, and absence of cavernous sinus wall or other dural invasion were associated with long-term remission (P < .05). A minimum morning serum cortisol of less than 1 μg/dl after surgery had a positive predictive value for lasting remission of 96%.
With rare disorders, such as pediatric CD, enhanced outcomes are obtained by evaluation and treatment at centers with substantial experience. Resection of pituitary adenomas in pediatric CD in that setting can be safe, effective, and durable. Early postoperative endocrine testing predicts lasting remission. Because lasting remission is associated with younger age at surgery, smaller adenomas, and lack of dural invasion, early diagnosis should improve surgical outcome.
影响小儿库欣病（CD）手术治疗结果的因素尚未完全确定。
本研究旨在探讨影响小儿 CD 手术结果的因素。
在这项前瞻性观察性研究中，分析了 1982 年至 2010 年期间在国立卫生研究院（NIH）连续治疗的患者的临床、影像学、内分泌和手术结果。
研究在一个三级转诊中心进行。
纳入 200 例 CD 患者（女性 106 例，男性 94 例）。症状发作时的平均年龄为 10.6 ± 3.6 岁（范围，4.0 至 19.0 岁）。在 NIH 手术时的平均年龄为 13.7 ± 3.7 岁。27 例（13%）曾在其他机构接受过手术。磁共振成像（MRI）在 97 例患者（50%）中发现了腺瘤。当 MRI 阳性时，97 例患者中的 96 例（99%）准确地定义了离散腺瘤，这比使用 ACTH 在下腔静脉窦取样期间的比值来确定腺瘤侧化更准确（无手术史的患者中有 72%准确）。200 例患者中有 195 例（98%）术后达到缓解（189 例[97%]皮质醇水平降低；6 例[3%]术后皮质醇水平正常）。与初始缓解相关的因素（P <.05）包括手术时发现腺瘤、免疫组织化学 ACTH 分泌腺瘤和非侵袭性 ACTH 腺瘤。年轻、腺瘤较小、海绵窦壁或其他硬脑膜侵犯缺失与长期缓解相关（P <.05）。术后清晨血清皮质醇水平低于 1μg/dl 具有持续缓解的阳性预测值 96%。
对于小儿库欣病等罕见疾病，在具有丰富经验的中心进行评估和治疗可获得更好的效果。在该环境下，小儿 CD 垂体腺瘤的切除术可以是安全、有效和持久的。术后早期内分泌检测可预测长期缓解。由于长期缓解与手术时年龄较小、腺瘤较小以及无硬脑膜侵犯有关，因此早期诊断应改善手术结果。
