School of Pharmacy Hopes to Close Clinical Gap for Elderly Blood Disorder

The National Institutes of Health has awarded UMB researchers $777,000 grant to study treatments for blood disorders that affect more than 10,000 elderly patients each year.

By Steve Berberich
November 3, 2009

The National Institutes of Health has awarded a competitive grant of more than $777,000 to University of Maryland School of Pharmacy and Johns Hopkins University researchers to study the costs, risks, and benefits of treatments for blood disorders that affect more than 10,000 elderly patients each year.

The challenge grant targets a widely accepted practice by clinicians of controlling anemia of myelodysplastic syndromes (MDS), sometimes called pre-leukemias, with erythropoietin, a hormone controlling red blood cell production.

Drugs called erythropoietin stimulating agents, or ESA’s, are effective in treating anemia in MDS patients. However, little is known about the drugs’ longer-term health effects. Myelodysplastic syndromes are the most common hematologic malignancy in the elderly.

“In this study, we will use data from regional cancer registries that are linked to Medicare enrollment and insurance claims data,” said Amy J. Davidoff, PhD , research associate professor in the Department of Pharmaceutical Health Services Research (PSHR) at the School of Pharmacy. “We will examine the characteristics of Medicare beneficiaries with MDS, patterns of treatment, safety and effectiveness of ESA use, and costs of care.”

Doctors have treated MDS patients with ESAs to control anemia for about 15 years. This treatment has had a 20 to 40 percent response rate and median response duration of two years. However, there is a serious lack of large-scale clinical trial evidence supporting longer-term clinical benefits, according to Davidoff and co-grantees, Sheila Weiss, PhD, a professor in PHSR at the School of Pharmacy, Maria Baer, MD, professor and director of the Hematologic Malignancies Program at the University of Maryland Marlene and Stewart Greenebaum Cancer Center, and Steven Gore, MD, professor at Hopkins Sidney Kimmel Comprehensive Cancer Center.

In 2007, the Food and Drug Administration administered a warning concerning the use of ESAs in patients with cancer-related anemia due to apparent increase in thromboembolic events, as well as a possible decrease in relapse-free survival. The absence of large-scale randomized trials in MDS precludes definitive conclusions regarding the safety and efficacy of these agents in this population; clinical trials randomizing between ESAs and supportive care would be of limited feasibility in the US.

The challenge grant is one of only 840 such NIH grants awarded across the country from more than 20,000 applications.