Pituitary Center, Departments of Medicine and Neurological Surgery, Oregon Health and Science University, Portland, OR, United States.
Division of Endocrinology, Metabolism and Molecular Medicine, Northwestern University Feinberg School of Medicine, Chicago, IL, United States.
Front Endocrinol (Lausanne). 2021 Mar 15;12:627711. doi: 10.3389/fendo.2021.627711. eCollection 2021.
Medical treatment for acromegaly commonly involves receiving intramuscular or deep subcutaneous injections of somatostatin receptor ligands (SRLs) in most patients. In addition to side effects of treatment, acromegaly patients often still experience disease symptoms even when therapy is successful in controlling GH and IGF-1 levels. Symptoms and side effects can negatively impact patients' health-related quality of life. In this study, we examine the disease- and treatment-related burden associated with SRL injections as reported through the use of the Acromegaly Treatment Satisfaction Questionnaire (Acro-TSQ ) and clinician-reported symptom severity through the Acromegaly Index of Severity (AIS). Patients included in this analysis were enrolled in a randomized phase 3 study, were biochemically-controlled (an IGF-1 < 1.3 × the upper limit of normal [ULN] and average GH < 2.5 ng/ml) and receiving SRL injections for ≥6 months with a stable dose of either long-acting octreotide or lanreotide monotherapy for ≥4 months. The sample (N = 91) was 65% female, 91% Caucasian, with a mean [standard deviation (SD)] age of 53 (1) years. Two-thirds of patients reported that they still experience acromegaly symptoms; 82% of these said they experience symptoms all of the time. Three-fourths experienced gastrointestinal (GI) side effects after injections, and 77% experienced treatment-related injection site reactions (ISRs). Patients commonly reported that these interfered with their daily life, leisure, and work activities. Those with higher symptom severity, as measured by the AIS, scored significantly worse on several Acro-TSQ domains: Symptom Interference, GI Interference, Treatment Satisfaction, and Emotional Reaction. Despite being biochemically controlled with injectable SRLs, most patients reported experiencing acromegaly symptoms that interfere with daily life, leisure, and work. GI side effects and ISRs were also common. This study highlights the significant disease burden that still persists for patients with acromegaly that have achieved biochemical control with the use of injectable SRLs.
大多数患者在接受肢端肥大症治疗时，通常需要接受肌肉内或皮下注射生长抑素受体配体（SRL）。除了治疗的副作用外，即使生长激素和 IGF-1 水平得到成功控制，肢端肥大症患者仍经常经历疾病症状。症状和副作用会对患者的健康相关生活质量产生负面影响。在这项研究中，我们通过使用肢端肥大症治疗满意度问卷（Acro-TSQ）和临床医生报告的症状严重程度通过肢端肥大症严重程度指数（AIS）来检查与 SRL 注射相关的疾病和治疗相关负担。这项分析中包括的患者参加了一项随机 3 期研究，生化控制（IGF-1 <1.3×正常上限[ULN]和平均 GH <2.5ng/ml）并接受 SRL 注射至少 6 个月，并且至少 4 个月使用长效奥曲肽或兰瑞肽单药治疗剂量稳定。该样本（N=91）为 65%女性，91%白种人，平均[标准差（SD）]年龄为 53（1）岁。三分之二的患者报告仍有肢端肥大症症状；其中 82%的患者表示他们一直有症状。四分之三的患者在注射后出现胃肠道（GI）副作用，77%的患者出现与治疗相关的注射部位反应（ISR）。患者普遍表示这些会干扰他们的日常生活、休闲和工作活动。根据 AIS 测量的症状严重程度较高的患者，在几个 Acro-TSQ 领域的评分明显较差：症状干扰、GI 干扰、治疗满意度和情绪反应。尽管使用可注射 SRL 进行生化控制，但大多数患者报告称，肢端肥大症症状会干扰日常生活、休闲和工作。GI 副作用和 ISR 也很常见。这项研究强调了即使使用可注射 SRL 控制生化，肢端肥大症患者仍存在严重的疾病负担。
