Mycophenolate mofetil (brand names CellCept, Myfortic) is an immunomodulatory agent (changing the function of the immune system) which inhibits the proliferation of lymphocytes. It is commonly used with other medications to help prevent rejection of organ transplantation and to help treat several autoimmune disorders.

Mycophenolate mofetil is taken orally twice a day on an empty stomach. The different brands available (Cellcept and Myfortic) are differently absorbed by the body and therefore cannot be substituted for each other.

Some of the side effects of mycophenolate mofetil include constipation, stomach pain or swelling, nausea, vomiting, difficulty falling asleep or staying asleep, pain (especially in the back, muscles, and joints), uncontrollable shaking in parts of the body, headache, and bloating.

Mycophenolate mofetil to treat PF

Apart from its uses in transplantation rejection prevention, mycophenolate mofetil recently emerged as an anti-inflammatory and an anti-proliferative agent because it acts on some growth factors, including TGF-beta, a type of cytokine that controls proliferation, cellular differentiation and other functions in cells. This finding made the drug an attractive candidate for the treatment of PF.

A small-scale retrospective study was carried out to test the safety and effectiveness of mycophenolate mofetil in PF. The study included 10 participants who received 2 grams of the drug per day for more than six months. Laboratory, functional, and radiological data demonstrated an acceptable safety profile for the drug with no severe adverse events. However, mycophenolate mofetil failed to show benefits regarding disease progression.

Another study identified 125 people treated with mycophenolate mofetil where a good tolerance and a low rate of discontinuation was shown. The use of the drug was also associated with stable or improved lung physiology during follow-up, appearing to be a promising therapy for pulmonary fibrosis.

There are also some studies that are currently recruiting participants. One is assessing the safety and effectiveness of rituximab together with mycophenolate mofetil (NCT02990286) in non-responders to a first-line treatment. Another is comparing the effectiveness of mycophenolate mofetil with that of a placebo in systemic sclerosis-related early interstitial lung disease (NCT02896205).

A third study is comparing the effect of bortezomib, mycophenolate mofetil, or a combination of both in treating scarring of the lung caused by pulmonary fibrosis of systemic sclerosis origin (NCT02370693). The primary objectives of this study are to assess the safety and tolerability of bortezomib with mycophenolate mofetil as measured by the incidence of serious adverse events.

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