The MDS Beacon™

Results of a recent study show that myelodysplastic syndromes patients who are in poor health and therefore ineligible for clinical trials may benefit from treatment with Vidaza and Zolinza.

The study authors found that the efficacy and safety of the combination therapy were similar to those of other treatment regimens commonly tested in clinical trials.

They concluded that their findings support treatment for patients who are ineligible for clinical trials, and they suggested that current eligibility criteria for Phase 1/2 clinical trials be reexamined.

Dr. Guillermo Garcia-Manero of the MD Anderson Cancer Center in Houston presented these findings at the 2011 American Society of Hematology (ASH) conference in San Diego earlier this month.

In order for a patient to be eligible to participate in most clinical trials for myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML), the patient must display acceptable overall health, kidney function, and liver function. Patients also cannot have any other co-existing diseases.

Researchers at the MD Anderson Cancer Center found that MDS and AML patients who are not eligible for clinical trials have a median survival of less than 60 days if they do not receive treatment.

In the present study, the researchers aimed to evaluate the influence of therapy on survival outcomes for MDS and AML patients who do not qualify to participate in clinical trials. All 30 patients in the study were treated with Vidaza (azacitidine) and Zolinza (vorinostat) for a median of 3.5 cycles. The researchers chose this combination therapy because they consider it to be safe and it has shown significant activity in both MDS and AML patients.

Vidaza is approved by the U.S. Food and Drug Administration (FDA) as a treatment for patients with MDS. Zolinza also is approved by the FDA, but as a treatment for specific kind of lymphoma. It can be prescribed, however, by a physician as a treatment for other conditions – such as MDS – if the physician feels there is sufficient evidence to justify such use of the drug.

The median patient age in the MD Anderson study of the Vidaza-Zolinza combination treatment was 74 years, and 53 percent of the patients had MDS. In addition, 53 percent had another type of cancer. The remaining patients had another co-existing disease, including chronic liver disease, liver failure, heart dysfunction, chronic bronchitis and emphysema, or poor overall health.

The overall response rate was 30 percent, with 27 percent achieving a complete response. Half of the patients who did not register a response to the treatment regimen nevertheless had stable disease for more than eight weeks.

The median survival was seven months, and 80 percent of patients survived longer than 60 days.

Thirty percent of the patients are still alive as of the date the trial data were analyzed in preparation for the ASH meeting. All of the deaths during the study were due to progression of the patient’s primary disease.

The researchers pointed out that the therapy was well tolerated since only one patient (3 percent) developed a serious, non-blood-related side effect (nausea and vomiting).

For more information, please see abstract 608 at the ASH 2011 meeting website.