Rituximab Treatment of Low Platelet Counts in a Patient with Chronic Graft-versus-Host Disease

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What is the problem and what is known about it so far?

Persons with leukemia are sometimes treated by replacing cancerous blood cells with early forms of normal blood cells taken from healthy persons. This process is called stem-cell transplantation. Chronic graft-versus-host disease (GVHD) is a common complication of stem-cell transplantation in which the transplanted cells attack the patient's own tissues. Antibodies, which are made in a type of cell called B cells, are substances that normally help the body to fight off infection and get rid of foreign substances. When antibodies are directed against the body's own cells, they are called autoantibodies. Chronic GVHD is thought to interfere with the regulation of the transplanted B cells so that they start to make the autoantibodies that damage body tissues. One possible consequence of chronic GVHD is a low platelet count (thrombocytopenia). Platelets are tiny cells in the bloodstream that are important in blood clotting, and patients with thrombocytopenia are therefore at risk for bleeding. Rituximab is a drug used to treat patients with B-cell cancer (B-cell lymphoma). Because rituximab works by destroying B cells, it might reduce the risk for bleeding in chronic GVHD by reducing the production of autoantibodies that destroy platelets.

Why did the researchers do this particular study?

To evaluate the effectiveness of rituximab in treating the thrombocytopenia that develops after stem-cell transplantation for leukemia.

Who was studied?

A 32-year-old woman with leukemia whose platelet counts decreased to extremely low levels after receiving a stem-cell transplant. The patient's thrombocytopenia had not responded to other therapies before she was treated with rituximab.

How was the study done?

The authors describe in detail the patient's clinical course before and after they treated her with rituximab.

What did the researchers find?

About 1 month after transplantation, the patient developed a rash, which was diagnosed as chronic GVHD. About 200 days after transplantation, her platelet counts began to go down and continued to decrease despite treatment with several other drugs used to treat this condition (prednisone, immunoglobulins, mycophenolate, vincristine, and cyclophosphamide). At day 404 after transplantation, the doctors gave the patient rituximab; she was given subsequent doses on day 411, day 418, and day 425. Her platelet count began to increase after two doses of rituximab. The patient was then followed for 11 months, during which time her platelet count was normal.

What were the limitations of the study?

This report describes only one patient. It therefore does not prove that rituximab is an effective treatment for all patients with low platelet counts due to chronic GVHD.

What are the implications of the study?

Rituximab may be an effective treatment for patients who develop thrombocytopenia as a result of chronic GVHD.

Article and Author Information

  • The summary below is from the full report titled “Anti-CD20 Chimeric Monoclonal Antibody Treatment of Refractory Immune-Mediated Thrombocytopenia in a Patient with Chronic Graft-versus-Host Disease.” It is in the 15 August 2000 issue of Annals of Internal Medicine (volume 133, pages 275-279). The authors are V. Ratanatharathorn, E. Carson, C. Reynolds, L.J. Ayash, J. Levine, G. Yanik, S.M. Silver, J.L.M. Ferrara, and J.P. Uberti.

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