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Bone Marrow Transplantation Platform to Investigate the Role of Dendritic Cells in Graft-versus-Host Disease
Published on: March 17, 2020
Therapy with mycophenolate mofetil for refractory acute and chronic GVHD
T Furlong1, P Martin, M E D Flowers
1Division of Clinical Research, Fred Hutchinson Cancer Research Center, University of Washington School of Medicine, 1100 Fairview Avenue N., Seattle, WA 98109, USA. tfurlong@fhcrc.org
Bone Marrow Transplantation
|April 21, 2009
Summary
Oral mycophenolate mofetil (MMF) shows efficacy in treating graft-versus-host disease (GVHD). While responses varied, MMF offers a treatment option for refractory GVHD, though toxicity requires monitoring.
Area of Science:
- Hematology
- Immunology
- Pharmacology
Background:
- Graft-versus-host disease (GVHD) remains a significant complication following allogeneic stem cell transplantation.
- Refractory GVHD necessitates exploration of alternative therapeutic agents.
- Mycophenolate mofetil (MMF) is an immunosuppressive drug with potential application in GVHD management.
Purpose of the Study:
- To evaluate the pharmacokinetics and clinical efficacy of oral mycophenolate mofetil (MMF) in patients with refractory acute and chronic graft-versus-host disease (GVHD).
- To assess treatment response rates, survival outcomes, and the incidence of disease resolution and treatment withdrawal.
- To investigate potential correlations between mycophenolic acid plasma concentrations and treatment outcomes.
Main Methods:
- Prospective and retrospective studies were conducted.
- Patients with acute and chronic GVHD received oral MMF.
- Pharmacokinetic parameters (area under the curve of mycophenolic acid plasma concentrations) and clinical outcomes (response rates, survival, disease resolution, need for additional immunosuppression, MMF discontinuation due to toxicity) were assessed.
Main Results:
- In prospective acute GVHD studies, 47% of patients responded to MMF, with 6- and 12-month survival rates of 37% and 16%, respectively.
- In a retrospective acute GVHD study, 48% of patients responded, with 6- and 12-month survival rates of 55% and 52%, respectively.
- For chronic GVHD, the cumulative incidence of disease resolution was 9%, 17%, and 26% at 12, 24, and 36 months, respectively. 59% required additional immunosuppression, and 21% discontinued MMF due to toxicity. Suboptimal mycophenolic acid levels were observed in acute GVHD.
Conclusions:
- Oral mycophenolate mofetil (MMF) demonstrates effectiveness in the treatment of both acute and chronic graft-versus-host disease (GVHD).
- MMF can be a valuable therapeutic option for refractory GVHD, although careful monitoring for toxicity and pharmacokinetic parameters is warranted.
- Further research may optimize MMF dosing strategies to improve outcomes in specific GVHD populations.
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