ACRO研究：469例患者的最新情况

ACROSTUDY: Status Update on 469 Patients.

作者信息

Brue Thierry

机构信息

Department of Endocrinology, Université de la Méditerranée and Centre de Référence des Maladies Rares d'Origine Hypophysaire, Hôpital de la Timone, Assistance Publique Hôpitaux de Marseille, Marseille, France.

出版信息

Horm Res. 2009 Jan;71 Suppl 1:34-8. doi: 10.1159/000178035. Epub 2009 Jan 21.

DOI:10.1159/000178035

PMID:19153503

Abstract

BACKGROUND

ACROSTUDY is an international noninterventional surveillance study open to all patients with acromegaly treated with the growth hormone antagonist pegvisomant. This web-based registry contains data reflecting actual clinical care of patients, and it is being used to monitor the long-term safety and efficacy of pegvisomant therapy for acromegaly. Since ACROSTUDY was launched in 2004, there has been an increase in cumulative patient recruitment from 116 patients in 2005 to over 500 by mid-2008. As of May 2008, over 300 centers in 10 countries have contributed data from 469 patients. At the time of inclusion, 84% of patients had already been treated with pegvisomant. Of the 469 patients, the majority had received somatostatin analogue treatment (63% had received octreotide and 34% had received lanreotide); 20% reported no prior medical treatment. Overall, 74% had prior transsphenoidal surgery, 3% had undergone craniotomy and 35% had received radiation therapy (including stereotactic radiosurgery). Interestingly, 67% were treated with pegvisomant alone at study start, 4% took pegvisomant with a dopamine agonist, 26% with a somatostatin analogue and 3% took pegvisomant with both types of analogues. The starting dose of pegvisomant was 10 mg/day for 65% of patients treated with subcutaneous daily injections, and the mean insulin-like growth factor I (IGF-I) concentration at baseline was 526 ng/ml. Annual assessments since study launch in 2004 found 62-78% of patients had normal IGF-I levels after 1-4 years of pegvisomant treatment at mean doses of 18.7-22 mg/day. Adverse events were reported for 13% of patients, including 6 serious adverse events considered possibly related to the drug. An increase in tumor size relative to the size at start of pegvisomant therapy was reported for 24 of the 469 patients (5.1%). Liver function test results more than twice the upper limit of normal were observed in 7.7%.

CONCLUSIONS

Further analyses of the ACROSTUDY database will provide a better understanding of pegvisomant treatment in clinical practice.

摘要

背景

ACROSTUDY是一项国际非干预性监测研究，面向所有接受生长激素拮抗剂培维索孟治疗的肢端肥大症患者开放。这个基于网络的注册数据库包含反映患者实际临床治疗情况的数据，正用于监测培维索孟治疗肢端肥大症的长期安全性和疗效。自2004年ACROSTUDY启动以来，累计招募患者数量有所增加，从2005年的116例增加到2008年年中时的500多例。截至2008年5月，10个国家的300多个中心提供了469例患者的数据。纳入研究时，84%的患者已经接受过培维索孟治疗。在这469例患者中，大多数曾接受过生长抑素类似物治疗（63%接受过奥曲肽治疗，34%接受过兰瑞肽治疗）；20%的患者报告此前未接受过任何治疗。总体而言，74%的患者曾接受经蝶窦手术，3%的患者接受过开颅手术，35%的患者接受过放射治疗（包括立体定向放射外科治疗）。有趣的是，67%的患者在研究开始时仅接受培维索孟治疗，4%的患者将培维索孟与多巴胺激动剂联用，26%的患者与生长抑素类似物联用，3%的患者将培维索孟与两种类型的类似物联用。65%接受皮下每日注射治疗的患者培维索孟起始剂量为10毫克/天，基线时胰岛素样生长因子I（IGF-I）的平均浓度为526纳克/毫升。自2004年研究启动以来的年度评估发现，在平均剂量为18.7 - 22毫克/天的培维索孟治疗1 - 4年后，62 - 78%的患者IGF-I水平正常。13%的患者报告了不良事件，其中包括6例被认为可能与药物相关的严重不良事件。469例患者中有24例（5.1%）报告相对于培维索孟治疗开始时肿瘤大小有所增加。7.7%的患者肝功能检查结果超过正常上限的两倍。