Anamorelin Increases Lean Body Mass in NSCLC Patients With Cachexia


Key Points

  • Anamorelin significantly increased lean body mass, as well as anorexia-cachexia symptom burden and body weight, in patients with advanced non–small cell lung cancer and cachexia.
  • No significant improvement in handgrip strength was observed.

Anamorelin (a ghrelin-receptor agonist) increased lean body mass but had no beneficial effect on handgrip strength vs placebo in patients with advanced non–small cell lung cancer (NSCLC) and cachexia, according to two international phase III trials (ROMANA 1 and 2) reported in The Lancet Oncology by Temel et al.

Study Details

In the identically designed double-blind trials, performed at 93 sites in 19 countries, 484 and 495 patients with inoperable stage III or IV disease, respectively, and cachexia (≥ 5% weight loss within 6 months or body mass index < 20 kg/m2) were randomized 2:1 to receive oral anamorelin at 100 mg (n = 323 and 330) or placebo (n = 161 and 165) for 12 weeks. The coprimary endpoints were change in lean body mass and handgrip strength on intent-to-treat analysis.


Lean body mass increased in anamorelin recipients over 12 weeks in both ROMANA 1 (median change  = +0.99 kg vs0.47 kg, P < .0001) and ROMANA 2 (+0.65 kg vs –0.98 kg, P < .0001). No difference in median change in handgrip strength was observed in ROMANA 1 (–1.10 kg vs –1.58 kg, P = .15) or ROMANA 2 (–1.49 kg vs –0.95 kg, P = .65).

Mean body weight increased with anamorelin in both trials (+2.20 kg vs +0.14 kg, P < .0001; +0.95 kg vs –0.57 kg, P < .0001), as did mean anorexia-cachexia score (P = .0004, P = .0016). A near-significant improvement in fatigue was observed in ROMANA 1 (P = .054), with no difference seen in ROMANA 2 (P = .86).

There were no differences in grade 3 or 4 treatment-related adverse events between the study groups; the most common was hyperglycemia, observed in one anamorelin patient (< 1%) in ROMANA 1 and four anamorelin patients (1%) in ROMANA 2.

The investigators concluded: “Anamorelin significantly increased lean body mass but not handgrip strength in patients with advanced non-small-cell lung cancer. Considering the unmet medical need for safe and effective treatments for cachexia, anamorelin might be a treatment option for patients with cancer anorexia and cachexia.”

The study was funded by Helsinn Therapeutics.

Jennifer S. Temel, MD, of Massachusetts General Hospital, is the corresponding author of The Lancet Oncology article.

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.