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Graft-Versus-Host Disease

Safety and potential efficacy of low-dose methotrexate for treatment of chronic graft-versus-host disease

Summary:

Low-dose methotrexate (MTX) is widely used in autoimmune diseases because of its anti-inflammatory activity. We report here the results of a retrospective study to review the outcomes of low-dose MTX used for treatment of refractory chronic graft-versus-host disease GVHD, with the goal of reducing the amount of prednisone needed to control the disease. In all, 14 patients with refractory chronic GVHD received MTX at a dose of 7.5 mg/m2/weekly for 3–50 weeks. Also, 11 patients had skin involvement, often with scleroderma or fasciitis. The median duration of chronic GVHD at the start of MTX was 38 (range 1–135) months. In this retrospective review, we found no grade 3–4 toxicities, and none of the patients needed blood transfusion or growth factors. In 10 patients (71%), GVHD could be adequately controlled with prednisone at doses below 1 mg/kg every other day without the addition of other agents. Four patients decreased the amount of concomitant immunosuppressive treatment, five continued with the same regimen, four required an increase in immunosuppressive treatment, and one decided to discontinue all treatment. From this preliminary analysis, MTX appears to be a well-tolerated, inexpensive and possibly steroid-sparing agent that is worthy of further evaluation in prospective trials for treatment of chronic GVHD.

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Acknowledgements

We thank Judy Campbell, RN and Colleen McKinnon, RN for outstanding assistance in the care of patients with chronic GVHD, and Chris Davis and the Long-Term Follow-Up staff at the Fred Hutchinson Cancer Research Center for assistance in data collection. This work was supported in part by PHS grants HL36444, CA18221, CA78902, CA15704 and CA18029 from the National Institutes of Health, Department of Health and Human Services, and Associazione Italiana Ricerca Cancro (AIRC), CNR, San Paolo Foundation.

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Correspondence to M E D Flowers.

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Giaccone, L., Martin, P., Carpenter, P. et al. Safety and potential efficacy of low-dose methotrexate for treatment of chronic graft-versus-host disease. Bone Marrow Transplant 36, 337–341 (2005). https://doi.org/10.1038/sj.bmt.1705022

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