The objective of this protocol is to determine whether leucovorin treatment can normalize hematopoietic cell growth and differentiation in patients with 5q-syndrome which may lack the gene for dihydrofolate reductase (DHFR) enzyme. The patients will be treated with oral leucovorin for 3 months and will be monitored for improvements in their counts. In addition, bone marrow colony assays will be performed in the presence and absence of leucovorin. The DHFR gene will be sequenced in each patient to screen for DHFR mutations. FISH analysis for the DHFR will also be performed on the cytogenetic material to assess the presence and copy number of the DHFR gene.

Agency
National Institute of Health (NIH)
Institute
Clinical Center (CLC)
Type
Intramural Research (Z01)
Project #
1Z01CL010293-04
Application #
6546545
Study Section
Cognition and Perception Study Section (CP)
Project Start
Project End
Budget Start
Budget End
Support Year
4
Fiscal Year
2001
Total Cost
Indirect Cost
Name
Clinical Center
Department
Type
DUNS #
City
State
Country
United States
Zip Code