THE ROMANA STUDIES
Recent results from two international, randomized, double-blind phase III trials of anamorelin appear to have provided the first evidence of consistent, sustained improvement of anorexia/cachexia in people with advanced cancer. Data from the two studies, ROMANA 1 and ROMANA 2, suggest that anamorelin is well tolerated and increases LBM as well as body weight (LBM and fat mass), while addressing symptoms and concerns related to anorexia in patients with advanced NSCLC.16,17
Investigators randomly assigned 484 patients (ROMANA 1) and 495 patients (ROMANA 2) with unresectable stage III or stage IV NSCLC and cachexia (defined as 5% or greater loss of body weight within the prior 6 months or body mass index [BMI] less than 20 kg/m2 at screening) to receive an oral dose of either anamorelin (100 mg) or placebo once daily. Eligibility criteria for patients included a new line of chemotherapy or radiation initiated within 14 days of randomization, currently receiving maintenance chemotherapy, or had completed chemotherapy or radiation more than 14 days prior to randomization with no plan to initiate additional therapy during the study period16,17 (Figure 1).
Patients were excluded if they had known brain metastases, were receiving tube feeding or parenteral nutrition, or were taking a medication to increase appetite or treat weight loss. The co-primary end points were change in LBM (measured with dual energy x-ray absorptiometry) and handgrip strength (measured with hand-held dynamometer) over 12 weeks. Secondary end points included changes in body weight, anorexia/cachexia symptoms (measured with the 12-item Functional Assessment of Anorexia/Cachexia Treatment [FAACT] scale) and fatigue (measured with the 13-item Functional Assessment of Chronic Illness Therapy-Fatigue [FACIT-F] scale) over 12 weeks and overall survival (OS, pooled at 1 year from both studies). Exploratory analyses evaluated change in total body mass and fat mass from baseline to 12 weeks.16-18
Patients assigned to anamorelin experienced a statistically significant increase in LBM, compared with those receiving placebo (Figure 2). In the ROMANA 1 study, the median increase in LBM was 1.10 kg (95% confidence interval [CI]: 0.75, 1.42) in the anamorelin group (n=323), compared with a loss of 0.44 kg (95% CI: –0.88, 0.20) in the placebo group (n=161), yielding a treatment difference of approximately 1.5 kg (P<.001).
In the ROMANA 2 study, anamorelin-treated patients (n=330) achieved a median LBM increase of 0.75 kg (95% CI: 0.51, 1.00), whereas patients receiving placebo (n=165) experienced a median loss of 0.96 kg (95% CI: –1.27, –0.46), for a treatment difference of approximately 1.7 kg (P<.001).16,17