Off-label use of aprepitant for scleroderma-associated nausea and vomiting: A case report

Scleroderma is a disease characterized by excessive deposition of collagen and extracellular matrix proteins in affected organ systems, which results in tissue fibrosis and organ dysfunction. It is estimated that 90% of patients with systemic sclerosis have gastrointestinal involvement, with approximately 50% being symptomatic. Clinical manifestations of gastrointestinal scleroderma manifestations relate to impaired motility and absorption and ultimately malnutrition. Treatments for these symptoms often fail and are limited. The objective of this case report is to highlight the potential use of a substance P antagonist in the treatment of scleroderma associated nausea.

A 56-year-old woman presented with GI complications of her underlying systemic sclerosis. The majority of her stay was marked by severe nausea and vomiting, resistant to numerous therapies. Obstructive causes for her symptoms were ruled out with a gastroscopy. A motility study revealed completely absent peristalsis in her esophagus, likely due to complete fibrosis of smooth muscle fibres. For the first time, off-label use of aprepitant 80 mg once daily was trialed with success in hospital. With this medication, she was able to maintain an adequate oral intake and ultimately achieve discharge from hospital.

Despite the wide array of medications for gastrointestinal scleroderma, their effect is very limited. In this article, we report to the best of our knowledge the first successful use of the substance P antagonist aprepitant to treat refractory nausea and vomiting in a patient with gastrointestinal scleroderma. Not only does this medication carry promise in treating the GI symptoms of scleroderma, but it may also prove cost effective as compared to PEG insertions or TPN. This case study is congruent with recent evidence of the utility of aprepitant in other infiltrative disease conditions. This may spur interest randomized control trials for expanding the role of this medication.