Hematology

Research Activities

Basic and Translational Research:

ZnPP-fluorometer: noninvasive field measurement of erythrocyte zinc protoporphyrin
The ultimate goal of this research is to develop a portable, battery-powered instrument for the noninvasive detection of iron deficiency, a device potentially similar to a digital oral thermometer in size and ease of operation. (Nestec Ltd. 4520328242)

Mechanisms of effect of iron status & interventions on malaria & other infections
This research uses mouse models to examine the mechanisms for effects of iron deficiency and iron interventions on infections with malaria and other microbial pathogens. (NIH: U01 HD064827)

Harmful effects of transfusion of older stored red cells: iron and inflammation
This research will test the hypothesis that transfusion of older stored red blood cells (RBC) produces a proinflammatory cytokine response. (NIH: R01 HL098014)

Oral iron chelators predicated on desferrithiocin
The project develops and evaluates desferrithiocin-based, orally active iron-chelating agents for the treatment of iron overload. (NIH: R37 DK049108)

TCD With Transfusions Changing to Hydroxyurea (TWiTCH)
The primary aim of this study is to compare standard therapy (red blood cell transfusions) with alternative therapy (hydroxyurea) for primary prevention of stroke in children with sickle cell disease with abnormal transcranial Doppler studies. (NIH: R01 HL095647)

Stroke With Transfusions Changing to Hydroxyurea (SWiTCH)
The major goal of the project is to compare an alternative treatment (hydroxyurea and phlebotomy) to standard treatment (chronic blood transfusions and iron chelation therapy) for secondary prevention of stroke in children with sickle cell disease and previous stroke. (NIH: U01 HL078787)

Neuropathology of severe malaria in Thailand: MRI studies
This exploratory research project is focused on the neuropathology of severe and cerebral malaria in Thailand. (NIH: R21 NS055348)

Malaria and the safety of iron interventions: absorption & NTBI
This research examines the effects of acute infection with Plasmodium falciparum on the absorption, pharmacokinetics and metabolism of iron from iron interventions. (NIH: U01 HD061233)

High-Tc susceptometer to monitor transfusional iron overload
This project is designed to validate our high-transition-temperature (high-Tc) superconducting magnetic susceptometer for monitoring iron overload in patients with chronic red blood cell transfusion. (FDA: R01 FD003702)

Vitamin D for sickle-cell respiratory complications: Phase 2
This Phase 2 clinical trial will determine if monthly oral vitamin D can reduce the risk of respiratory complications in children with sickle-cell disease. (FDA: R01 FD003894)

High Tc susceptometer for magnetic measure of liver iron
This Bioengineering Research Partnership is integrating bioengineering, basic science and clinical efforts in the design, development and clinical validation of a high-temperature (77°K; high-TC) super-conducting biosusceptometer for the direct, non-invasive measurement of hepatic iron stores in patients with iron overload from hereditary hemochromatosis, thalassemia major, sickle cell disease and other disorders. (NIH: NIAID - 5 R01 DK57209)

Magnetic resonance measurement of heart and liver iron
This research project combines radiological, clinical and laboratory efforts in the development and validation of quantitative magnetic resonance methods for the measurement of heart and liver iron. (NIH: NIDDK - 5 R01 DK066251)

Pathogenesis of severe malarial anemia in Thailand
This research project examines the pathogenesis of severe malarial anemia in Thailand to define those elements attributable to infection with Plasmodium falciparum. This mutlidisciplinary collaborative investigation is designed to determine the relative contributions of accelerated destruction and impaired production of erythrocytes in the pathogenesis of the severe anemia of falciparum malaria. (NIAID R01 AI1051310)

Oral chelators predicated on desferrithiocin
The major goals of this project are the development and evaluation of a new series of desferrithiocin-based, orally active iron-chelating agents for the treatment of iron overload. This research uses our animal model of iron-induced cardiomyopathy with quantitative magnetic resonance methods to provide serial, non-invasive assessment of cardiac dimensions and function simultaneously with estimates of cardiac and hepatic iron concentrations.(CU subcontract NIDDK R01 DK049108)

MRI method for in vivo quantification
This research project has the overall goal of developing a new method for measuring body iron stores and for separately determining the amounts of storage iron within ferritin and hemosiderin.(NIDDK R01 DK069373)

Clinical Research:

Stroke with Transfusions Changing to Hydroxyurea (SWiTCH)
A Phase III randomized multi-center clinical trial to compare standard therapy (erythrocyte transfusions with iron chelation) with alternative therapy (hydroxyurea with phlebotomy) for the prevention of secondary stroke and the management of iron overload in pediatric subjects with sickle cell anemia and previous stroke (Subcontract- St Jude Children’s Research Hospital; NIH/NHLBI)

A Longitudinal Cohort Study of Patients with Thalassemia in the Thalassemia Clinical Research Network (TCRN)
This is a prospective data collection study to estimate the incidence of thalassemia complications and to evaluate whether changes in patient monitoring, management, and treatment alter the clinical course. (Subcontract- New England Research Institute U01 HL065238)

Treatment of Pulmonary Hypertension and Sickle Cell Disease with Sildenafil (walk-PHaSST)
A randomized, double-blind, placebo-controlled phase II/III trial to determine the safety and efficacy of sildenafil for the treatment of patients with sickle cell disease and pulmonary hypertension (Subcontract -- in collaboration with AECOM -- NHLBI)

Medical Homes for Sickle Cell in Harlem and Northern Manhattan
Improve community-based outreach and follow-up for sickle disease and trait in Harlem and Northern Manhattan; establish standardized preventive care for children with sickle disease. (HRSA 1 H46MC09227-01)

Other Research Projects:

• Study of immunologic responses to red blood cell transfusions The overall goal of this study is to identify immune markers in patients with hemoglobinopathies that make them susceptible to RBC alloimmunization. We believe that this information will enable us to predict which transfused patients are likely to progress to alloimmunization which in turn would help in pre-selecting better matched units for such patients and reducing transfusion-associated morbidity.
• Pulmonary Hypertension in Children with Sickle Cell disease An observational study aimed to determine the prevalence and risk factors of pulmonary hypertension in pediatric sickle cell population
• Management of Acute Vaso-occlusive Pain in Pediatric Sickle Cell Disease A prospective study of the impact of introducing a clinical pathway for the management of sickle cell vaso-occlusive pain in the pediatric emergency department
• New technologies in newborn screening State of the art (Luminex) technologies applied to new and existing newborn screening, including metabolic, hematologic, immune, viral disorders
  Sub-contract with NY State Dept. of Health Wadsworth Center
• Consultation to the Advisory Committee on Heritable Disorders in Newborns and Children Provide expert input on content and process for evidence based review (Consultation to the Genetic Services Branch of HRSA)