Two-Day Drug Regimen Enough to Control Severe Immune Disease After Some Bone Marrow Transplants
Johns Hopkins and other cancer researchers report that a very short course of a chemotherapy drug, called cyclophosphamide, not only can prevent a life-threatening immune response in some bone marrow transplant recipients, but also can eliminate such patients’ need for the usual six months of immune suppression medicines commonly prescribed to prevent severe forms of this immune response. Patients receive cyclophosphamide for two days after their bone marrow transplant, in addition to two other chemotherapy drugs given before the transplant.
FAST FACTS:
• A new multi-center study confirms that the post-transplant cyclophosphamide is safe and effective for people who have received fully-matched bone marrow transplants.
• Bookended pre-and post-transplant treatments, which have been tested separately in other studies, already had promising track records in controlling cancer and preventing severe GVHD. Those successes led researchers from three hospitals, including Johns Hopkins, to combine the two therapies.
• The next step will be to test the short course therapy in a phase III randomized clinical trial that would directly compare results in patients who receive the cyclophosphamide treatment with those who receive either a separate experimental approach to prevent GVHD or the more traditional six-month immunosuppressive therapy.
Johns Hopkins Kimmel Cancer Center scientists first used cyclophosphamide to prevent severe graft-versus-host disease (GVHD) after bone marrow transplant involving haploidentical or “half-matched” transplants, a treatment first used in 2000 at the Cancer Center to treat leukemias and other blood cancers. The scientists began to use post-transplant cyclophosphamide in clinical trials of fully matched bone marrow transplants in 2004.
Now, the new multi-center study confirms that the post-transplant cyclophosphamide is safe and effective for people who have received fully-matched bone marrow transplants.
The shortened regimen, described online Sept. 29 in the Journal of Clinical Oncology, begins with intravenous busulfan and fludarabine, two chemotherapeutic drugs that wipe out a patient’s immune system and prepare his or her body to receive donated marrow. After the transplant, patients receive two days of cyclophosphamide to prevent GVHD and rejection of the new bone marrow. Conventionally, most transplant patients get six months of immunosuppressive treatment for that purpose.
The bookended pre-and post-transplant treatments, which have been tested separately in other studies, already had promising track records in controlling cancer and preventing severe GVHD. Those successes led researchers from three hospitals, including Johns Hopkins, to combine the two therapies, says Leo Luznik, M.D., an associate professor of oncology at the Johns Hopkins University School of Medicine and leader of the study.
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