Yedinak Chris G, Hopkins Sarah, Williams Jessica, Ibrahim Aly, Cetas Justin Schultz, Fleseriu Maria
Department of Medicine-Endocrinology, Diabetes, and Clinical Nutrition, OHSU Northwest Pituitary Center, Oregon Health & Science University, Portland, OR, USA; Department of Neurological Surgery, OHSU Northwest Pituitary Center, Oregon Health & Science University, Portland, OR, USA.
Front Endocrinol (Lausanne). 2017 Feb 27;8:35. doi: 10.3389/fendo.2017.00035. eCollection 2017.
Subcutaneous (SC) injection of pasireotide, a somatostatin analog, is approved for the treatment of adults with Cushing's disease (CD) for whom pituitary surgery was unsuccessful or is not an option. We highlight the symptomatic and biochemical improvement of six patients with recurrent CD treated with pasireotide SC at a single center for at least 1 year. Patients were treated either through commercial use ( = 5) or through the Phase 3 trial ( = 1; http://ClinicalTrials.gov identifier, NCT00434148; study number, B2305). Most patients ( = 5) were female, and the mean age at diagnosis was 35.8 years. All patients demonstrated biochemical control at 1 year of treatment. Three of the five real-world patients followed for more than 1 year remain on pasireotide SC and are controlled. Two patients discontinued pasireotide SC; one patient because of persistently elevated urinary-free cortisol levels and gallstones, and the other because of treatment for an unrelated brain tumor. Symptomatic improvement varied, but all patients demonstrated weight loss. Nausea and mild, transient injection-site reactions were the most frequently reported adverse events. Although glycated hemoglobin (HbA) increased after treatment initiation, four of five patients maintained HbA levels ≤7.0% while receiving pasireotide SC and concomitant individualized diabetes medication, if necessary. In patients who discontinued pasireotide SC, HbA levels decreased within 6 weeks. This report documents real-world use of pasireotide SC and indicates its effectiveness as a long-term treatment option for patients with CD. Although hyperglycemia was observed in most patients, it was managed with appropriate monitoring and treatment and was reversible upon discontinuation of pasireotide SC.
皮下注射帕西瑞肽（一种生长抑素类似物）已被批准用于治疗垂体手术失败或不适合垂体手术的成年库欣病（CD）患者。我们重点介绍了在单一中心接受帕西瑞肽皮下注射治疗至少1年的6例复发性CD患者的症状和生化指标改善情况。患者通过商业途径治疗（n = 5）或通过3期试验治疗（n = 1；http://ClinicalTrials.gov标识符，NCT00434148；研究编号，B2305）。大多数患者（n = 5）为女性，诊断时的平均年龄为35.8岁。所有患者在治疗1年时均实现了生化指标的控制。随访超过1年的5例真实世界患者中有3例仍在接受帕西瑞肽皮下注射治疗且病情得到控制。2例患者停用了帕西瑞肽皮下注射；1例患者是因为尿游离皮质醇水平持续升高和胆结石，另1例是因为接受了与帕西瑞肽无关的脑肿瘤治疗。症状改善情况各不相同，但所有患者均出现体重减轻。恶心和轻度、短暂的注射部位反应是最常报告的不良事件。虽然治疗开始后糖化血红蛋白（HbA）升高，但5例患者中有4例在接受帕西瑞肽皮下注射并在必要时联合个体化糖尿病药物治疗时，HbA水平维持在≤7.0%。停用帕西瑞肽皮下注射的患者，其HbA水平在6周内下降。本报告记录了帕西瑞肽皮下注射的真实世界应用情况，并表明其作为CD患者的长期治疗选择是有效的。虽然大多数患者出现了高血糖，但通过适当的监测和治疗进行了管理，并且在停用帕西瑞肽皮下注射后是可逆的。
