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Summary: A new steroid pill could help tame one form of graft-versus-host disease (GVHD), a complication of the stem cell transplants some cancer patients need. In a recent study, patients who got the pill had fewer flare-ups of GVHD in their stomach and intestine and had a lower risk of dying one year after their transplant.

Why it's important: About 60% of people who get stem cells transplanted from a donor (called an allogeneic transplant) develop GVHD in their stomach or small intestine, a condition known as gastrointestinal GVHD. This side effect can cause nausea, vomiting, inability to eat, and diarrhea. When it is severe it can cause fever, changes in bile production and flow, serious intestinal problems, and even death. Despite the dangers of this side effect, stem cell transplants are often the best treatment option for people with blood cancers like leukemia, or for people whose cancer treatment has destroyed their ability to produce normal blood cells. Because many patients need stem cell transplants, doctors are looking for new ways to prevent these severe complications.

What's already known: People who develop gastrointestinal GVHD are usually treated with the steroid prednisone. It works by suppressing the donor cells to control the GVHD effects; however, this action also suppresses the immune system, making the patients more susceptible to infections, some of which can be fatal. Other side effects of prednisone include weakness, high blood sugar, high blood pressure, and bone changes.

How this study was done: The drug used in this study, beclomethasone dipropionate (BDP), is a steroid that is usually used as a cream. The researchers, from the Fred Hutchinson Cancer Research Center, made it into a pill that delivered the steroid directly to the stomach and small intestine (the areas affected) without going into the bloodstream like prednisone does. The study included 129 patients with different types of leukemia, lymphoma, and other blood disorders who developed gastrointestinal GVHD after a stem cell transplant from a donor. About half of the participants were randomly assigned to take BPD pills, while the rest took a placebo (pills with no medicine in them). In addition to the BPD/placebo, patients in both groups took 10 days of standard prednisone treatments. If GVHD had improved in that time, the prednisone was scaled back over the next week and then stopped. All patients continued the BPD or placebo for a total of 50 days. The findings were published in the journal Blood.

What was found: Adding BPD to standard prednisone treatment allowed patients to stop prednisone sooner while maintaining control of the GVHD. After 50 days, just 18 patients in the BPD group had failed to respond to treatment, compared to 30 in the placebo group. The BPD also appeared to help patients survive longer after transplant. One year out,18 patients in the BPD group and 28 in the placebo group had died. The most common causes of death were cancer relapse and infection.

BPD also caused far fewer bad side effects than prednisone.

The bottom line: Treatment that controls GVHD without causing immune suppression is likely to lead to fewer life-threatening infections in transplant patients. The BPD pills offer patients a "more tailored" therapy that helps them avoid many of the side effects of prednisone, says David M. Hockenbery, MD, one of the doctors involved in the study.

"All of this improves the chances that patients will have a successful outcome," he says.

In July the FDA is expected to decide whether to approve BPD for oral use. If it is approved it will be only the second FDA-approved drug for treatment of GVHD in stem cell transplant patients.

Citation: "New drug therapy to combat GVHD in stem-cell patients shows significant reduction in deaths." Published in the Jan. 23, 2007, online edition of Blood. First author: David M. Hockenbery, MD, Fred Hutchinson Cancer Research Center, Seattle.