The current project will develop and maintain systems for the management of patients with thalassemia at the Southern California Comprehensive Thalassemia Center (SCCTC). This will be accomplished by 1) providing comprehensive healthcare services to prevent complications through assessment, surveillance, outreach, education, consultation, and management; (2) participating in blood safety monitoring and surveillance efforts; (3) maintaining a prevention evaluation monitoring system to assess the efficacy of prevention services; (4) collaborating with lay organizations to deliver consistent prevention messages aimed at preventing complications; and (5) develop research ideas and methods to determine, measure, and modify treatment and follow-up protocols to maximize the achievement of optimal medical-driven outcomes. The Southern California Comprehensive Thalassemia Center will address these goals by 1) working closely with the other CDC thalassemia centers to participate in blood safety monitoring and research projects at a national level, 2) continuing active participation in the Thalassemia Clinical Research Network, 3) developing outreach and education programs at this center and by working collaboratively with the Northern California Thalassemia center, and 4) maintaining and developing new research initiatives based on data from our local multidisciplinary comprehensive care model. The thalassemia research initiatives from the Southern California Comprehensive Thalassemia Center in general are related to the assessment and management of transfusional iron overload and measurement of tissue iron by magnetic resonance imaging. Specific projects based on data collected from the CDC comprehensive care model but funded externally to the CDC program include 1) the relationship between cardiac iron and cardiac function, 2) the utility of MRI for the measurement of liver iron, 3) the efficacy of the deferasirox for the removal of cardiac iron, and 4) the relation between pancreatic iron and pancreatic function. These studies leverage the unique expertise of the Southern California Comprehensive Thalassemia Center of the Center co-director, Dr. Wood, who is a cardiologist and biomedical engineer. The Southern California Comprehensive Thalassemia Center will continue the model of using the analysis of data collected in the context of providing multidisciplinary comprehensive clinical management of patients with thalassemia to modify approaches to treatment and to generate hypotheses for more formal study of treatments and management approaches that will reduce the complications of patients with thalassemia and improve their health. Project Narrative To implement a blood safety monitoring surveillance system at Childrens Hospital Los Angeles to benefit persons with thalassemia. Information from this system will be used by the Hematologic Diseases Branch of CDC to assess the safety of the blood supply and to develop and monitor the effectiveness of interventions designed to reduce or prevent the complications of thalassemia. ? ? ? ?

Agency
National Institute of Health (NIH)
Institute
Centers for Disease Control and Prevention (NCBDD)
Type
Research Project--Cooperative Agreements (U01)
Project #
1U01DD000309-01
Application #
7429915
Study Section
Special Emphasis Panel (ZCD1-ZDQ (11))
Program Officer
Taylor, Marcia
Project Start
2007-09-30
Project End
2012-09-29
Budget Start
2007-09-30
Budget End
2008-09-29
Support Year
1
Fiscal Year
2007
Total Cost
$206,250
Indirect Cost
Name
Children's Hospital of Los Angeles
Department
Type
DUNS #
052277936
City
Los Angeles
State
CA
Country
United States
Zip Code
90027
Meloni, Antonella; Rienhoff Jr, Hugh Young; Jones, Amber et al. (2014) Cardiac R2* values are independent of the image analysis approach employed. Magn Reson Med 72:485-91
Vichinsky, Elliott; Neumayr, Lynne; Trimble, Sean et al. (2014) Transfusion complications in thalassemia patients: a report from the Centers for Disease Control and Prevention (CME). Transfusion 54:972-81; quiz 971
Meloni, Antonella; Rienhoff Jr, Hugh Y; Jones, Amber et al. (2013) The use of appropriate calibration curves corrects for systematic differences in liver R2* values measured using different software packages. Br J Haematol 161:888-91
Fung, Ellen B; Harmatz, Paul R; Milet, Meredith et al. (2008) Fracture prevalence and relationship to endocrinopathy in iron overloaded patients with sickle cell disease and thalassemia. Bone 43:162-8