Different efficacy of mycophenolate mofetil as salvage treatment for acute and chronic GVHD after allogeneic stem cell transplant

Abstract

MMF would appear to be effective and safe for treating refractory chronic GVHD, yet not as effective for treating refractory acute gut GVHD. Accordingly, a prospective randomized clinical trial is warranted to assess the impact of MMF in the treatment of refractory GVHD.

Evaluation of mycophenolate mofetil for initial treatment of chronic graft-versus-host disease

“…First, clinical trials should use randomized designs whenever possible. Case-series reports and uncontrolled phase 2 studies suggested encouraging results with the use of MMF for treatment of steroid-refractory chronic GVHD, [6][7][8][9][10][11][12][13][14][15] but our results with the use of MMF for initial treatment of chronic GVHD offer no support for the use of MMF in the management of steroid-refractory chronic GVHD. Second, the field would benefit from a higher level of enthusiasm and support for enrolling patients in clinical trials.…”

“…Instead, the study was based on results of previous reports suggesting the efficacy of MMF for treatment of steroid-refractory chronic GVHD, [6][7][8][9][10][11][12][13][14][15] and the proposed effect size was based on the number of patients who could be enrolled in a multicenter study within a reasonable period of time. Even so, the negative results of this study did not simply reflect an overstringent primary endpoint or an unrealistic effect size because the much less stringent endpoint of complete response also did not show an advantage for the MMF arm.…”

“…[6][7][8][9][10][11][12][13][14][15] Moreover, the tolerability of MMF and ease of oral administration for prolonged periods of time made MMF an attractive alternative among other agents that could have been tested for efficacy in the initial treatment of chronic GVHD. Several hypothetical a priori explanations could be considered as possibly accounting for an (14) .07…”

“…Case-series reports and phase 2 studies have suggested that MMF might be effective for treatment of steroidrefractory chronic GVHD both in adults and in children. [6][7][8][9][10][11][12][13][14][15] In 2 large surveys, respondents endorsed MMF more frequently than other agents for treatment of high-risk or steroid-refractory chronic GVHD. 16,17 These results suggested that outcomes among patients with chronic GVHD might be improved by adding MMF to the initial systemic treatment regimen.…”

Mycophenolate mofetil for the management of steroid-refractory acute graft vs host disease

“…[31][32][33][36][37][38] Direct comparisons to other series examining MMF in refractory GVHD are difficult for several reasons. Basara et al reported an overall grade improvement in 65% of patients, where MMF was used as first-line therapy in addition to CSA and prednisolone on development of aGVHD or cGHVD.…”