Division of Gerontology and Geriatric Medicine, School of Medicine, University of Washington, Seattle, WA, USA.
Geriatric Research, Education and Clinical Center, Veterans Affairs Puget Sound Health Care System, Seattle, WA, USA.
J Cachexia Sarcopenia Muscle. 2023 Apr;14(2):835-846. doi: 10.1002/jcsm.13191. Epub 2023 Mar 1.
Cancer cachexia is associated with reduced body weight, appetite and quality of life (QOL) with no approved treatments. Growth hormone secretagogues like macimorelin have potential to mitigate these effects.
This pilot study assessed the safety and efficacy of macimorelin for 1 week. Efficacy was defined a priori as 1-week change in body weight (≥0.8 kg), plasma insulin-like growth factor (IGF)-1 (≥50 ng/mL) or QOL (≥15%). Secondary outcomes included food intake, appetite, functional performance, energy expenditure and safety laboratory parameters. Patients with cancer cachexia were randomized to 0.5 or 1.0 mg/kg macimorelin or placebo; outcomes were assessed non-parametrically.
Participants receiving at least one of either macimorelin dose were combined (N = 10; 100% male; median age = 65.50 ± 2.12) and compared with placebo (N = 5; 80% male; median age = 68.00 ± 6.19). Efficacy criteria achieved: body weight (macimorelin N = 2; placebo N = 0; P = 0.92); IGF-1 (macimorelin N = 0; placebo N = 0); QOL by Anderson Symptom Assessment Scale (macimorelin N = 4; placebo N = 1; P = 1.00) or Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F; macimorelin N = 3; placebo N = 0; P = 0.50). No related serious or non-serious adverse events were reported. In macimorelin recipients, change in FACIT-F was directly associated with change in body weight (r = 0.92, P = 0.001), IGF-1 (r = 0.80, P = 0.01), and caloric intake (r = 0.83, P = 0.005), and inversely associated with change in energy expenditure (r = -0.67, P = 0.05).
Daily oral macimorelin for 1 week was safe and numerically improved body weight and QOL in patients with cancer cachexia compared with placebo. Longer term administration should be evaluated for mitigation of cancer-induced reductions in body weight, appetite and QOL in larger studies.
癌症恶病质与体重减轻、食欲下降和生活质量（QOL）下降有关，目前尚无批准的治疗方法。生长激素促分泌素如麦角隐亭具有减轻这些影响的潜力。
这项初步研究评估了麦角隐亭治疗 1 周的安全性和疗效。疗效定义为体重（≥0.8kg）、血浆胰岛素样生长因子（IGF）-1（≥50ng/ml）或 QOL（≥15%）在 1 周内的变化。次要结局包括食物摄入量、食欲、功能表现、能量消耗和安全实验室参数。患有癌症恶病质的患者被随机分配至 0.5 或 1.0mg/kg 麦角隐亭或安慰剂；结果以非参数方式评估。
至少接受一种麦角隐亭剂量的参与者被合并（N=10；100%为男性；中位年龄=65.50±2.12），并与安慰剂（N=5；80%为男性；中位年龄=68.00±6.19）进行比较。达到疗效标准：体重（麦角隐亭 N=2；安慰剂 N=0；P=0.92）；IGF-1（麦角隐亭 N=0；安慰剂 N=0；P=0.92）；安德森症状评估量表（麦角隐亭 N=4；安慰剂 N=1；P=1.00）或慢性疾病治疗疲劳功能评估（FACIT-F；麦角隐亭 N=3；安慰剂 N=0；P=0.50）的 QOL。未报告与治疗相关的严重或非严重不良事件。在麦角隐亭治疗组中，FACIT-F 的变化与体重（r=0.92，P=0.001）、IGF-1（r=0.80，P=0.01）和热量摄入（r=0.83，P=0.005）的变化直接相关，与能量消耗（r=-0.67，P=0.05）的变化呈负相关。
与安慰剂相比，癌症恶病质患者每日口服麦角隐亭治疗 1 周安全且可改善体重和 QOL。应在更大规模的研究中评估长期给药对减轻癌症引起的体重、食欲和 QOL 下降的效果。
