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Pulse cyclophosphamide for corticosteroid-refractory graft-versus-host disease
1Department of Internal Medicine--Hemato-oncology, University Hospital Brno, Brno, Czech Republic. jmayer@fnbrno.cz
Bone Marrow Transplantation
|February 8, 2005
Summary
Pulse cyclophosphamide (Cy) effectively treats skin, liver, and oral corticosteroid-resistant graft-versus-host disease (GVHD) with acceptable toxicity. Further investigation is warranted for this challenging condition.
Area of Science:
- Hematology
- Immunology
- Oncology
Background:
- Corticosteroid-resistant graft-versus-host disease (GVHD) presents significant management challenges, leading to high morbidity and mortality.
- Cyclophosphamide (Cy) is a widely used immunosuppressive and cytotoxic agent in pretransplant conditioning.
Purpose of the Study:
- To evaluate the efficacy and safety of pulse cyclophosphamide (Cy) in patients with corticosteroid-resistant GVHD.
Main Methods:
- Retrospective analysis of 15 patients with corticosteroid-resistant GVHD (acute, post-donor lymphocyte infusion, chronic).
- Treatment involved pulse Cy at a median dose of 1 g/m(2).
- Response rates, toxicity, and survival outcomes were assessed.
Main Results:
- Pulse Cy demonstrated high response rates for skin (100%) and oral cavity (100%) GVHD, and 70% for liver GVHD.
- Severe intestinal GVHD showed poor response.
- Toxicity was acceptable, with manageable myelosuppression; no increased risk of opportunistic infections, mixed chimerism, relapses, or PTLD was observed.
- Overall survival was 57%.
Conclusions:
- Pulse cyclophosphamide is a potentially effective and well-tolerated treatment option for specific manifestations of corticosteroid-resistant GVHD.
- The low cost of Cy is advantageous compared to newer therapies.
- Further prospective studies are needed to confirm these findings and establish optimal protocols for pulse Cy in refractory GVHD.