Fleseriu Maria, Barkan Ariel, Brue Thierry, Duquesne Edouard, Houchard Aude, Del Pilar Schneider Maria, Ribeiro-Oliveira Antonio, Melmed Shlomo
Pituitary Center, Departments of Medicine and Neurological Surgery, Oregon Health & Science University, Portland, OR 97239, USA.
A. Alfred Taubman Health Care Center, University of Michigan, Ann Arbor, MI 48109, USA.
J Endocr Soc. 2023 Aug 23;7(10):bvad104. doi: 10.1210/jendso/bvad104. eCollection 2023 Aug 28.
Treatment of acromegaly is multimodal for many patients, and medical treatments include somatostatin receptor ligands (SRLs), dopamine agonists (DAs), and growth hormone receptor antagonists (GHRAs). However, recent real-world evidence on treatment patterns for patients with acromegaly is limited.
This study evaluated medication usage, treatment changes, adherence, persistence, comorbidities, and health care resource utilization using deidentified data from MarketScan, a US claims database.
Eligible patients (n = 882) were those receiving monotherapy or combination therapy for ≥90 days without treatment gaps.
Mean age at diagnosis was 48.6 years; 50.1% of patients were female. Over half (59.4%) had 1 line of treatment (LOT); 23.1% had 2 LOTs; 17.5% had at least 3 LOTs. Most patients (94.6%) initiated treatment with monotherapies. The most common first-line monotherapy treatments were cabergoline (DA, 36.8%), octreotide long-acting release (first-generation SRL, 29.5%), and lanreotide depot (first-generation SRL, 22.5%). Adherence for first-line treatments (proportion of days covered) was higher for first-generation SRLs (lanreotide depot: 0.8) compared with DAs (0.7). Treatment persistence (time between the first treatment record and a change in LOT/censoring) in LOT 1 was higher for GHRAs (24.8 months) and first-generation SRLs (20.0 months) compared with DAs (14.4 months). Female patients and those diagnosed at a younger age were more likely to have shorter treatment persistence. The most prevalent comorbidities were hyperlipidemia, essential hypertension, and sleep apnea.
Patients with more comorbidities had more health care visits during the first year after diagnosis, suggesting increased disease burden. Real-world evidence on treatment patterns provides insights into recommendations for individualized therapy.
对于许多肢端肥大症患者而言，治疗是多模式的，药物治疗包括生长抑素受体配体（SRLs）、多巴胺激动剂（DAs）和生长激素受体拮抗剂（GHRAs）。然而，关于肢端肥大症患者治疗模式的近期真实世界证据有限。
本研究使用美国索赔数据库MarketScan的去识别数据，评估了药物使用、治疗变化、依从性、持续性、合并症以及医疗资源利用情况。
符合条件的患者（n = 882）为接受单药治疗或联合治疗≥90天且无治疗中断的患者。
诊断时的平均年龄为48.6岁；50.1%的患者为女性。超过一半（59.4%）的患者接受1线治疗（LOT）；23.1%的患者接受2线治疗；17.5%的患者接受至少3线治疗。大多数患者（94.6%）以单药治疗开始治疗。最常见的一线单药治疗是卡麦角林（多巴胺激动剂，36.8%）、长效奥曲肽（第一代生长抑素受体配体，29.5%）和长效兰瑞肽（第一代生长抑素受体配体，22.5%）。第一代生长抑素受体配体（长效兰瑞肽：0.8）的一线治疗依从性（覆盖天数比例）高于多巴胺激动剂（0.7）。与多巴胺激动剂（14.4个月）相比，生长激素受体拮抗剂（24.8个月）和第一代生长抑素受体配体（20.0个月）在第1线治疗中的治疗持续性（首次治疗记录与治疗线/审查变化之间的时间）更高。女性患者和诊断时年龄较小的患者治疗持续性更可能较短。最常见的合并症是高脂血症、原发性高血压和睡眠呼吸暂停。
合并症较多的患者在诊断后的第一年有更多的医疗就诊次数，表明疾病负担增加。治疗模式的真实世界证据为个体化治疗建议提供了见解。
