Greig, C.A., Johns, N., Gray, C., MacDonald, A., Stephens, N.A., Skipworth, R.J., . . . Fearon, K.C. (2014). Phase I/II trial of formoterol fumarate combined with megestrol acetate in cachectic patients with advanced malignancy. Supportive Care in Cancer, 22, 1269–1275.
To test the safety, tolerance, and efficacy of an appetite stimulant and an anabolic beta 2 agonist in patients with cancer cachexia
Patients were asked to take formoterol 40 mcg and megestrol 320 mg each morning and 40 mcg formoterol and 160 mg megestrol each evening for eight weeks. Self-reported intake and tablet counts were used to determine patient adherence. Patients were admitted to a clinical research facility on day 1 and discharged after the first dose of study drugs. Patients were contacted by phone after 24 hours for assessment. Additional assessments were done at weeks 2, 4, 6, 8, and 12.
Single arm observational
Patients showed an increase in muscle strength and muscle size. Mean body weight increased by 2.6%. Physical activity increased in three of six patients who responded to treatment. Appetite improved (p = .005). Adverse events were tremor (n = 7), peripheral edema (n = 3), tachycardia (n = 2), and dyspepsia (n = 2).
The combination of megestrol and formoterol may have benefit for patients with cachexia. The sample size of this study was too small to draw conclusions.
The combination of medications tested here may be associated with improved muscle function and appetite in patients with advanced cancer; however, due to the limitations of this study, efficacy and tolerance is not clear. Further well-designed research with these medications is needed to determine efficacy, safety, and tolerance.