Patricia Jane Giardina Professor of Clinical Pediatrics

Email
Phone
  • (212) 746-3415 (Clinical)

Positions

Current Research Projects

1. Transfusion Related Complications in Thalassemia: Currently, Dr. Giardina is the Principal Investigator of the CDC sponsored study of the Prevention of Complications in Thalassemia which is monitoring the national blood supply by prospectively testing blood specimens of Thalassemia patients for emerging pathogens among subjects from seven Thalassemia Treatment Centers (TTC) in the United States. Recently, she identified transfusion transmitted Babesisosis as an emerging pathogen in the Thalassemia patients at NYPH/WCMC. The efficacy of Babesia screening is being reviewed with the NYPH Blood Bank. In addition, the prevalence of alloimmunization and autoantibody development is being analyzed in over 400 Thalassemia Major and Intermedia subjects in the CDC's study.

2. The Thalassemia Longitudinal Cohort Study: This NIH sponsored research project involves the longitudinal study of the natural history of Thalassemia Major and Intermedia patients previously participating in the North American Thalassemia Clinical Research Network (TCRN) Study. Dr. Giardina is the Principal Investigator of the New York Region TCRN.

3. The Study of Bone Disease in Thalassemia: This study involves the prospective investigation of low bone mass in thalassemia and treatment options to prevent osteoporosis. Our previous cross sectional observational study of osteoporosis in Thalassemia Major and Intermedia had demonstrated profound bone loss in Thalassemia adolescents and adults beginning in childhood and associated with increase fracture rates and endocrinopathies. These studies involve annual screening for low bone mass and the identification of factors contributing to increased fracture risk, the causes of low bone mass, and the treatments to improve bone strength. Current pilot project is being conducted to identify ideal vitamin D levels to optimize bone strength.

4. Longitudinal Monitoring of Cardiac Siderosis in Thalassemia Major on Various Chelation Regimens: Patients with transfusional siderosis develop cardiac arrhythmias and biventricular myocardopathies leading to premature death. The impact of two new oral iron chelators FDA approved in the past few years and reliable non-invasive MRI studies which can now monitor iron excess in organ tissues is being studied. Annual MRI monitoring of liver iron concentration and cardiac iron are being studied in iron overloaded Thalassemia patients on various iron chelation regimens to assess the safety and efficacy of iron chelation regimens including Deferoxamine, Deferiprone, and Deferasirox alone and in combination.

5. Generation of Induced Pluri Potent Stem Cells(iPS) for the Cure of Beta Thalassemia: Studies are being conducted in collaboration with Stefano Rivella, PhD and Michel Sadelain, MD, PhD to develop iPS cells derived from Thalassemia patients' fibroblasts to generate stem cells and subsequently study the efficacy of correcting their own beta globin gene. This novel approach to generate patients'' corrected iPS cells allows scientists to investigate technologies to perfect beta globin gene transfer.

6. Gene Transfer for the Cure of Beta Thalassemia: In collaboration with Stefano Rivella's laboratory, this clinical study investigates the analysis of beta globin lentiviral vector transfer of the healthy beta globin gene to Thalassemia progenitor erythroid cells to determine the relationship between the number of copies of the lentiviral vector integrated and the total amount of hemoglobin produced.

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Background

Primary Affiliation

  • Weill Cornell Medical College, Cornell University