Transplant Medication Found Comparable to Cyclophosphamide in Inducing Remission

People with ANCA-associated vasculitis (AAV) who receive mycophenolate mofetil (MMF) as a remission induction therapy have similar remission rates as those given cyclophosphamide (CYC), an analysis of eight published studies has found. 

The study, “The Role of Mycophenolate Mofetil for the Induction of Remission in ANCA-Associated Vasculitis: A Meta-Analysis,” was published in Frontiers in Medicine. 

AAV is a diverse group of inflammatory diseases primarily affecting the small blood vessels of the kidneys. Cyclophosphamide is commonly used to induce remission in AAV patients, with remission rates ranging from 70%–90%, but can cause serious side effects such as infertility and cancer.

The immunosuppressant agent mycophenolate mofetil, which carries less risk of infertility or cancer and kidney toxicity than common treatments, has been suggested as an alternative to cyclophosphamide in some AAV patients. But more data is still needed before clinicians are comfortable with the use of mycophenolate as an induction therapy for AAV.

In the study, researchers in China sought to further analyze the efficacy of mycophenolate mofetil as an induction therapy in AAV patients, by reviewing studies published up to June 2020.

A total of eight studies, published from 2005 to 2019 and including 230 patients, were included in the meta-analysis. Among them, five studies evaluated mycophenolate mofetil as a first-line treatment and three evaluated patients who were treated with mycophenolate mofetil due to unresponsiveness or relapse with standard therapy.

In patients on mycophenolate mofetil, the remission rates in the eight studies ranged from 50% to 82%, and the pooled remission rate was 74%. However, nearly half of these patients (45%) relapsed during the study.

In the four randomized trials that compared the effects of mycophenolate mofetil (147 patients) and cyclophosphamide (143 patients), no statistical difference was observed between the two treatments in the rate of remission, infection, or leukopenia (a reduction in the number of white blood cells).

The main adverse events reported in patients on mycophenolate mofetil were infections (35.6%), gastrointestinal symptoms (14.8%), and leukopenia (14.8%). 

The findings suggest that mycophenolate could be used as an alternative to cyclophosphamide, but the investigators caution that mycophenolate is probably more effective in certain subgroups of patients, “and it is of great importance to identify these patients to minimize toxicity of CYC,” they wrote.

Thus, “when assessing the efficacy of new drugs in AAV or comparing the effects of different drugs in AAV, risk stratification (life-threatening or non-life-threatening) and subgrouping (such as different ANCA types and different organs involved) should be carried out first as AAV is a group of diseases with strong heterogeneity,” they concluded.