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Promise and Progress - Shortened BMT Treatment Gets the Job Done

Promise & Progress - A Spectrum of Achievements

Shortened BMT Treatment Gets the Job Done

Date: January 15, 2015


Journal of Clinical Oncology, Sept. 29, 2014

A short course of treatment with the drug cyclophosphamide may be all that’s needed to prevent life-threatening immune complications of bone marrow transplant.

The new multi-institutional study led by Kimmel Cancer Center bone marrow transplant expert Leo Luznik, M.D., found that two days of treatment with the drug cyclophosphamide is enough to prevent severe graft-versus-host-disease (GVHD) in patients receiving bone marrow from a fully matched donor.  GVHD occurs when the donor marrow—and all of the immune cells within—recognize their new host as foreign and attack vital organs and tissue. The abbreviated treatment appears to work as well as the typically prescribed six-month course of immunosuppressive therapy.

In a study of 92 fully matched bone marrow transplant patients who received the shortened treatment, about half developed GVHD, which is about the same results obtained with the longer standard immunosuppressive treatment. However, the percentage of patients with the chronic form of GVHD were drastically lower than seen with the standard treatment.

Specifically the shortened regimen includes pre-transplant treatment with two chemotherapy drugs that wipe out patients’ immune systems and prepare their bodies to accept the donated bone marrow. After the transplant, patients receive the two-day course of cyclophosphamide.   “Reducing the post-transplantation treatment allows for the earlier integration of other treatments,” says Christopher Kanakry, M.D., who collaborated with Luznik on the study. “With immunosuppressive therapies stopped earlier, immune-based treatments to go after any remaining cancer could be started much sooner.”

This work builds upon earlier studies successfully using cyclophosphamide to stave off severe GVHD in patients receiving bone marrow from half-matched donors. Additional clinical studies are planned.

The research was funded by Otsuka Pharmaceutical. 

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